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Archive for the ‘Scientific & Biotech Conferences: Press Coverage’ Category

Highlights of LIVE Day 1: World Medical Innovation Forum – CARDIOVASCULAR • MAY 1-3, 2017  BOSTON, MA • UNITED STATES

Reporter: Aviva Lev-Ari, PhD, RN

 

Leaders in Pharmaceutical Business Intelligence (LPBI) Group will cover the event in

REAL TIME

Aviva Lev-Ari, PhD, RN will be streaming live from the floor of the Westin Hotel in Boston on May 1-3, 2017

@pharma_BI

@AVIVA1950

#WMIF17

Forthcoming SEVEN e-Books in 2017 AND Eight e-Books on Amazon.com

https://pharmaceuticalintelligence.com/2016/04/24/new-e-book-titles-forthcoming-on-amazon-com-in-2016-from-lpbi-groups-biomed-e-series-forthcoming-cover-pages/

 

Biggest Voices in Cardiovascular Care

2017 World Medical Innovation Forum: Cardiovascular, May 1-3, 2017, Partners HealthCare, Boston, at the Westin Hotel, Boston

https://worldmedicalinnovation.org/agenda/

 

Monday, May 1, 2017

7:00 am – 8:00 am
Lilly Foyer
8:00 am – 11:30 am
Pfizer Ballroom
First Look: The Next Wave of Cardiology Breakthroughs

Harvard Medical School investigators describe their most promising work in rapid fire presentations highlighting commercial opportunities in cardiovascular and cardiometabolic care. Nineteen rising stars from Brigham and Women’s Hospital and Massachusetts General Hospital will present in 10-minute sessions.

For a full look at speakers and presentations, please visit the Highlight’s page.

Early career Harvard Medical School investigators kick-off the World Medical Innovation Forum with rapid fire presentations of their high potential new technologies. Nineteen rising stars from Brigham Health and Massachusetts General Hospital will highlight in ten-minute presentations their discoveries and insights that will be the disruptive cardiovascular care of the future. This session is designed for investors, leaders, donors, entrepreneurs and investigators and others who share a passion for identifying emerging high-impact technologies. The top presenter each from BWH and MGH will be awarded the Austen-Braunwald Innovation Prize on Day 2 of the Forum. The prize carries a $10,000 award.

Novel Target Discovery Pipeline for Calcific Aortic Valve Disease
Elena Aikawa, MD, PhD
Director, Heart Valve Translational Research Program, Brigham and Women’s Hospital; Associate Professor of Medicine, Harvard Medical School

  • Aortic stenosis is a progress of Calcific Aortic Valve Disease (CAVD) – 80,000 patients – sole solution is surgery
  • Imaging methods for visualization of microcalcification formation: MRI, PET/CT, CAVD Gene network, proteomics heat mapNIRF imaging, microdissection, histology, Tissue layers cells
  • Serum Sortilin associated with aortic calcification and CVD risk
  • Discovery pipeline and CAVD Mapping

A zebrafish pipeline for cardiovascular precision medicine
Manu Beerens, PhD
Postdoctoral Research Fellow, Brigham and Women’s Hospital; Harvard Medical School

  • Cardiomyopathy – group of cardiac disorders: CHF, Atherosclerosis, metabolic syndrome, AF
  • Zebrafish at the forefront of CVD Precision Medicine
  • Luciferase activity vs ttn
  • high throughput screening to identify naxos modifiers
  • Endpoints: BNP levels Cardiac contractility

Using zebrafish to understand and harness cardiac regeneration

Caroline Burns, PhD
Associate Biologist, Massachusetts General Hospital; Associate Professor of Medicine, Harvard Medical School

  • Heart regeneration
  • Regenerative hearts vs Non-Regenerative hearts
  • How cardiomyocytes proliferation induced following injury
  • Uninjured, 1 day after, 7 days after,
  • Failure to regenerate is related to failure of myoocardial proliferation
  • Genetic factors required for myocardial proliferation
  • myocardial proliferation – by Chromatin – mononuclear >4c ploidy
  • Mononuclear in Human Heart – as research target
  • How to promote myocardial proliferation
  • Small molecule as enhanced to drive proliferation

Bioactive Lipid Profiling Can Identify Potential Targets for Altering Life Course Trajectories Toward Cardiometabolic Disease
Susan Cheng, MD
Associate Physician, Brigham and Women’s Hospital, Partners HealthCare; Assistant Professor, Harvard Medical School

  • Bioactive Lipids
  • Endogenous and exhaugenous factors
  • Biochemical intermediates
  • mechanisms
  • health and disease outcomes
  • Small lipid Mediators of Upstream: Eicosanoids and Incidence of Diabetes as Targets for Present and Future
  • cardiometabolic risk in future early vs late prediabetes, and DM
  • shared pathways
  • Statins favoral response
  • Stree or Injury
  • Linoleic Acid
  • Disease and Phynotyping specific investigations
  • dosing

 

Small Molecule Predictors of Outcome After Cardiac Interventions
Sammy Elmariah, MD
Assistant in Medicine, Massachusetts General Hospital; Assistant Professor of Medicine, Harvard Medical School

  • Valvular heart disease – elderly, Aortic valve stenosis leads to failure of compensatory ventricular activity of dysfunction
  • small molecule
  • fiadnostics biomarkers
  • Acute kidney injury due to metabolite, adenohomosestine
  • validate the model – after METABOLITE data is added to the risk classification
  • Personalizing the timing of Valve Intervention
  • Biomarkers in blood predicts systolic function, EF,
  • Metabolite-Driven clinical trial of Aortic stenosis
  • TAVR

Translational trials in microRNAs
Mark Feinberg, MD
Physician, Brigham and Women’s Hospital; Associate Professor of Medicine, Harvard Medical School

  • non-coding RNA Biologics – 98% of the genome
  • 2% are coding
  • MiRNA therapeutics for Atherosclerosis
  • MiRNA – Replacement Therapy- ApoE-?-NGL
  • Cholesterol, LDL
  • Vascular endothelium: Inflammation, HF, Diabetic wound healing – tissue thickness
  • Example: IncRNA, Example3: miR-26a – BMP-SMAD1-ID1-p21
  • 3 platforms for targeting non-coding RNAs in CVD:
  1. peptide-conjucated nc-RNA
  2. Ab-conjugasted nc-RNA

 

New approaches to controlling stem cell fate
Yick Fong, PhD
Research Scientist, Brigham and Women’s Hospital; Assistant Professor of Medicine, Harvard Medical School

  • controlling Stem cell fate by Transcription Factors
  • Pluripotent, fibroblasts – transformed into Bone, nerve, heart, pancreatic cells
  • This process is randon and inefficient
  • GOAL: Transplantation, drug/therapeutic screens
  • Cellular identity and function
  • In Vitro Reconstruction of cell-type specific Transcription
  • Identify Disease mechanism of Heart disease by mutation that cause disruptionCo-regulators disruption

 

Exercise Prescription to Improve Cardiovascular and Cancer Outcomes in Cancer Survivors
John Groarke, MD
Cardiologist, Brigham and Women’s Hospital; Instructor of Medicine, Harvard Medical

  • cancer survivers have risk for CVD
    Metabolic Equivalent ((METS)
  • METS is the highest to lower CVD in Cancer survivors
  • Onco-cardiac rehabilitation
  • Increase excercise performance vs physical de-conditioned state
  • Cardioprotection to mitigate CV Toxicities of cancer therapy

Personalizing Diabetic Management with Hemoglobin A1c
John Higgins, MD
Associate Pathologist, Massachusetts General Hospital; Associate Professor, Harvard Medical School

  • Non glucose factors that affect A1C
  1. RBC Age span – if circulate live longer accumulates more glucose
  2. AstraZeneca, Eli Lilly, Novo Nordics – ALL conduct clinical trials to lower A1C
  • Personalize DM Management
  • Using existing assays with the RBC Age adjustment — for achieving better future Outcomes
  • Device Manufacturers to adjust the device

Characterizing an Early HeartFailure pulmonary EF (HFpEF) Phenotype: Cardiometabolic Disease and Pulmonary Hypertension
Jennifer Ho, MD
Assistant Physician, Massachusetts General Hospital; Member of the Faculty of Medicine, Harvard Medical School

  • Ejection Frunction in Pulmonary Hypertension (PH)
  • Obesity >–>>> PH recapitulates human HP
  • PAECs – Molecular mechanisms – Pulmonary Artery #Endotheline Cells
  • P13K/Akt (Insulin) — AMPK  (Metformin) –>> eNOS — >> Vasodilation
  • Study ex-vivo PAEC from patients with HFpEF
  1. HFpEF subphenotypes
  2. drug therapy
  3. screening

Genetic Risk, Adherence to a Healthy Lifestyle, and Coronary Disease
Amit Khera, MD
Cardiologist, Massachusetts General Hospital; Instructor, Harvard Medical School

  • Integration of Genetic data and CVD data
  • GWAS –>> 60 variants associated with coronary risk
  • Polygenic genetic risk score
  • Risk for MI: Genetic risk Interpretation: Monogenic vs Polygenic
  • High risk comes from polygenic risk: Smoking, Obesity, Excercise, Health diet
  • Healthy lifestyle “corrects” genetic factor

Monogenic:

  • Hypercholesterolemia
  • Trycleceride
  • increased lipoproteins _ ASA Lp(a) inhibitors

Polygenic

  • life style, diet excercise
  • medicationS: Statins

A Novel Epigenetic Complex Implicated in Thoracic Aortic Aneurysm (TAA)
Mark Lindsay, MD, PhD
Physician, Massachusetts General Hospital; Assistant Professor, Harvard Medical School

  • Thoracic Aortic Aneurysm
  • Aortic dissection
  • VSCULAR SMOOOTH MUSCLE CELLS in the aorta’s leaves – neo-intima damage vs knockout MALAT1 – nepangiogenesis
  • GENETIC MUTATIONS as Biologic Probes
  • GENE: HDAC9 – BRG1, MALAT1 (RNA) – STRESS in aortic homeostasis
  • Aortic root and Ascending Aorta
  • Elastin and colagen

 

Atrial Fibrillation: Genetic Basis and Clinical Implications
Steven Lubitz, MD
Cardiac Electrophysiologist, Massachusetts General Hospital

  • 6 million in US 34 Million WOrldwide
  • Leading cause of stroke
  • AF is hard to diagnose
  • preventable with anticoagulation
  • AF is Familial and inheritable
  • Genetic Variation associated with AF – genetics stratified risk
  • AF Screening: as Stroke prevention – AliveCor
  • electronic health records are powerful repositories
  • AF genetic risk – as a Biomarker
  • Technologies for screening

Targeting Vascular Calcification to Prevent Cardiovascular Disease
Rajeev Malhotra, MD
Staff Cardiologist, Associate Medical Director of the Cardiac Intensive Care Unit, Associate Director of the Cardiopulmonary Exercise Laboratory, Massachusetts General Hospital; Instructor in Medicine, Harvard Medical School

  • Vascular Classification: Atherocalcification Vascula r disease
  • plaque destabilization
  • coronary and aortic
  • HUMAN GENETICS Studies: GWAS
  1. Genotype
  2. Aortic CT
  3. Identify Potential Genes, increase expression associated with classification
  4. Functional-Mechanical Studies: smooth muscle cell – more proliferative vs more contractile
  5. control vs inhibition
  6. Human model of Vascular classification vs Mouse Model
  7. Disease of Vascular classification: Calxiphylaxis – HEMODialysis patients – >50% mortality within 1 year of diagnosis
  8. Drug development and Clinical Trials

Stratifying Exercise Dysfunction
Bradley Maron, MD
Association Physician, Brigham and Women’s Hospital; Assistant Professor, Harvard Medical School

  • Exercise Dysfunction: Complex Pathophynotype
  • iCPET: O2, exercise capacity, Pulmonary function, Hemodynamics, Invesive cardiac performance,
  • NEW SYSTEM DESIGNED: Network-Based Clinical Risk Calculator by Four Clusters – Network determine cluster assignment
  • Point of care tool – integrate into iCPET
  • Provides insights into HTN, Valvular, Myocaritis, cardiomyopathy
  1. Pulmonary
  2. exercise capacity
  3. ventilation perf
  4. o2 transport
  5. Invasive cardiac

Novel Mouse Models of Remote Cardioprotection
Benjamin Olenchock, MD, PhD
Cardiovascular Medicine Specialist, Brigham and Women’s Hospital; Instructor in Medicine, Harvard Medical School

  • Ischemic Preconditing
  • Remote limb BP Cuff in pig prevented Ischemia in heart
  • Cell death is multi cell types and cell death
  • EGLN – succinate +CO2 – Transciptional Hypoxia responses
  • EGLN Inhibitor: Systemic Egln1 deletion vs Skeletal Muscle Egln1 Deletion – Study of Cardiac Protection
  • Cardioprotective Mediators: Tissue Hypoxia ++>> Altered hepatic Tryptophan Metabolism —>> Cardioprotection
  • Tumor xenograph

Harnessing Endogenous Mechanisms of Programmed Gene Expression for Therapeutic Benefit In Cardiometabolic Disorders
Jorge Plutzky, MD
Director, Preventive Cardiology, Cardiovascular Medicine, Brigham and Women’s Hospital

  • PPARs
  • RXR, RAR – Retinohyde –>> Retinoic Acid
  • Transcription factors: Physiology and Pathology
  • PPARalpha  <<<<—- Lipase, Lipid sustrate
  • Epigenetic Code
  • Histone Readers: Selective Inhibition of Tumor Oncogenes by DIsruption Enhancing ranked by BRD4 signal: Chemotaxis, adhesion, Migration, Thrombosis, Inflammation
  • Atherosclerosis: Knows protein, Unknown protein: Promoters, enhancers

 

Aging and the activin type II receptor pathway: a new target for heart failure therapy?
Jason Roh, MD
Assistant in Medicine, Massachusetts General Hospital; Instructor, Harvard Medical School

  • AGING and CVD – it is part of pathophysiology – CATABOLIC PROCESSES
  • Organ level

muscle waist, atropy –>> impaired function: Hand grip strength, walk speed —->> HF, systolic and diastolic Strain rate

  • Cell level: Isotype Abvs ActRII Ab

contractility, Seen in HF models and HFpEF

  • Activin-A decreases with AGE

Signaling and pulmonary vascular disease – PAH
Paul Yu, MD, PhD
Physician, Brigham and Women’s Hospital; Associate Professor of Medicine, Harvard Medical School

  • FIBROSIS: TGF
  • OSTEOGENESIS ANTI-APOPTOSIS
  • Activin/TGFBeta —>>> BMP9 (ALK1c)
  • Loss od signaling
  • Inflammation
  • high shear stress
  • PAH
  1. Idiopathic: Sporadic or heritable
  2. Associated PAH: Scleroderma or lupus
  • Dysregulated angiogenesis– Anti VEGF165 – Zr-bevacizumab – Using PET-CT
11:45 am – 1:05 pm
7th Floor
Discovery Café Breakout Sessions: Sharing Perspectives

Seven intensive workshops led by our top faculty will address cutting-edge cardiovascular topics. Seating is reserved at the point of registration. Lunch included.

Topics to be covered include:

  • Cardiac Replacement Therapy: The Next Ten Years
  • Heart Failure: Back in The Game through New Pathways
  • Payment Models: Provider’s Perspective
  • Molecular Imaging: New Biological Endpoints – Function Over Structure
  • Open Innovation in Medical Devices: What is it? What Are the Barriers?
  • Wearables for Cardiovascular Health:  How to Validate and Integrate in Care Paths?
  • Image Based Artificial Intelligence: Which Cardiac Disease Segments and Why?

For a full list of speakers, please visit the Highlight’s page.

Seven intensive workshops led by our top faculty will address cutting-edge cardiovascular topics. Seating is reserved at the point of registration. Lunch included.

Panels

Cardiac Replacement Therapy: The Next Ten Years- Great Republic | 7th Floor

  • Introducer: Seema Basu, PhD, Market Sector Leader, Partners HealthCare
  • Garrick Stewart, MD, Associate Physician, Medical Director, Mechanical Circulatory Support Unit, Brigham and Women’s Hospital; Instructor in Medicine, Harvard Medical School
  • Erin Coglianese, MD, Medical Director, Mechanical Cardiac Support Program, Massachusetts General Hospital

Heart Failure: Back in The Game through New Pathways- Essex North | 3rd Floor

  • Introducer: Dan Castro, Managing Director, Licensing, Partners HealthCare
  • Anju Nohria, MD, Director, Cardio-Oncology Program, Cardiovascular Medicine Specialist, Brigham and Women’s Hospital
  • Christopher Newton-Cheh, MD, Cardiologist, Heart Failure and Transplantation, Massachusetts General Hospital

Payment Models: Provider’s Perspective- North Star | 7th Floor

  • Introducer: Sepideh Hashemi, Market Sector Leader, Partners HealthCare
  • Thomas Gaziano, MD, Associate Physician, Cardiovascular Medicine, Brigham and Women’s Hospital; Assistant Professor, Harvard Medical School
  • Jason Wasfy, MD, Assistant Medical Director, Massachusetts General Hospital; Assistant Professor, Harvard Medical School

Molecular and Advanced Imaging: New Biological Endpoints – Function Over Structure- Baltic | 7th Floor

  • Introducer: Glenn Miller, PhD, Market Sector Leader, Partners HealthCare
  • Marcelo Di Carli, MD, Chief, Division of Nuclear Medicine and Molecular Imaging, Brigham and Women’s Hospital; Professor of Radiology and Medicine, Harvard Medical School
  • Farouc Jaffer, MD, PhD, Director, Coronary Intervention, Cardiac Catheterization Laboratory, Cardiology Division, Massachusetts General Hospital, Associate Professor of Medicine, Harvard Medical School
  • Sharmila Dorbala, MD, Director, Nuclear Cardiology, Brigham and Women’s Hospital; Associate Professor of Radiology, Harvard Medical School

Open Innovation in Medical Devices: What is it? What Are the Barriers?- Essex South | 3rd Floor

  • Introducer: Pat Fortune, PhD, Vice President for Market Sectors, Partners HealthCare
  • Elazer Edelman, MD, PhD, Senior Attending Physician, Brigham and Women’s Hospital; Professor of Medicine, Harvard Medical School; Thomas D. and Virginia W. Cabot Professor of Health Sciences and Technology, MIT
  1. Mortality of cardiovascular disease declines as a result of Medical Innovations as Devices
  2. Are innovations tappering off or New ones are coming??
  • Bruce Rosengard, MD, Chief Medical Science and Technology Officer, Johnson & Johnson Medical Devices Companies
  • Ronald Tompkins, MD, Director, Surgery, Innovation & Bioengineering, Massachusetts General Hospital; Sumner M. Redstone Professor of Surgery, Harvard Medical School

ALL

  1. Solution for Heart Failure – low hanging fruit was picked already – a workabke artificial heart more important than another Stent
  2. Large scale Programs better than multiple PI small grant applications, many are not innovating, conflict of interests, Academia and Industry relations
  3. 99% get better with a device but 1% is been harmed
  4. FDA – overworked Underfunded 52 applications reviewed per employee

 

Wearables for Cardiovascular Health:  How to Validate and Integrate in Care Paths?- Parliament/Adams | 7th Floor

  • Introducer: Thomas Aretz, MD, Vice President, Global Programs, Partners HealthCare
  • David Levine, MD, Home Hospital Director, Brigham and Women’s Hospital; Fellow in General Internal Medicine, Harvard Medical School
  • Kamal Jethwani, MD, Senior Director, Connected Health Innovation, Partners HealthCare; Assistant Professor, Dermatology, Harvard Medical School
  • Paolo Bonato, PhD, Director, Motion Analysis Laboratory, Spaulding Hospital; Associate Professor, Harvard Medical School

Image Based Artificial Intelligence: Which Cardiac Disease Segments and Why?  Empire | 7th Floor

  • Introducer: Trung Do, Vice President, Business Development, Partners HealthCare
  • George Washko, MD, Associate Physician, Brigham and Women’s Hospital; Associate Professor of Medicine, Harvard Medical School
  • Mark Michalski, MD, Director, CCDS, Brigham and Women’s Hospital, Massachusetts General Hospital
 
 
1:05 pm – 1:30 pm
1:30 pm – 1:35 pm
Boston Scientific Ballroom
Opening Remarks Christopher Coburn
Introduction by: Anne Klibanski, MD,
  • Chief Academic Officer, Partners HealthCare
  • Laurie Carrol Guthart Professor of Medicine, Academic Dean for Partners, Harvard Medical School
  • CEO, Partners HealthCare
1:35 pm – 1:55 pm
Boston Scientific Ballroom
Reinventing Cardiac Care

Two renowned clinical leaders provide an overview of the medical and economic challenges that cardiovascular and cardiometabolic disorders present.

They will highlight strategic direction in cardiac research and clinical care at Partners, and address how recent trends in investment, regulation, and policy may be dovetailed with efforts at Partners.

The experts also spotlight for attendees the various therapies, diagnostics, devices, and critical issues that will be discussed throughout the upcoming 2.5 days of the World Medical Innovation Forum.

  • Chief of Cardiovascular Medicine, Brigham and Women’s Hospital
  • Associate Professor of Medicine, Harvard Medical School
  1. New drugs: Molecular targets, Monoclonals, alternative to Statins
  2. devices implantable
  3. IT and EMR, BI
  4. Integration of Innovations, Clinical and Translational
  • Chief, Cardiology Division, Massachusetts General Hospital
  • Professor of Medicine, Harvard Medical School
  1. Aging and longevity of CVD Patients will increase the expense on CVD as disease
1:55 pm – 2:45 pm
Boston Scientific Ballroom
CEO Roundtable: Today’s Learning, Tomorrow’s Opportunities

Discussion on contribution of technology innovation to the treatment of cardiovascular disease reflecting on lessons and how they shape investment decisions.

Moderator: Benjamin Pless
  • Executive in Residence, Partners HealthCare Innovation
  1. Electrical physiology: implantable paceamker – first 1958, lead to RV, last 10years th enetire pacemaker implanted in the heart no leads. Surgical TAVR, Implantabke to Mitral valve
  • CEO, GE Healthcare
  1. Healthcare data, Analytics, data integration, machine learning, mapping efficiency,
  2. Data analytics is Present, Devices was the Past
  3. Shorten cycle of learning
  • CEO, Abiomed
  1. 20X of revenue growth since 2010
  2. HEART PUMP – only FDA approached for recover the heart, pump blood out of the heart
  3. Tracking ALL patients not samples – ALL Outcomes, all patients
  4. HF – 24×7 employees of Abiomed in labs in Hospitals, 5 years after heart attack, another attack 30% dies
  5. Protocol and standards
  • CEO, Edwards Lifesciences
  1. TAVR device no need for open heart surgery
  2. Selling services – heart-lung machine: Perfusion is a product and Service
  3. Structural heart disease – multiple innovations
  • CEO, Bard
  1. Oncology, Vascular, Urology, PDA
  2. Business model failure – clinical economic point of view
  3. Start up community and acquisitions – platforms for further investments
  • EVP, Clinical Advancement, UnitedHealth Group – Payer’s perspective
  1. Health Benefits
  2. Optum – Data to improve care,
  3. 30% of cost of care is WASTE, eliminate this cost item
  4. Heart transfer: Innvation cycle
  5. Collaboration with Pharma and with Devices: Data Analytics – fee for service vs Value Model – Total cost may be less
2:45 pm – 3:35 pm
Boston Scientific Ballroom
Tackling the AFib Epidemic

Evolving trends in diagnosis, prevention, and treatment of atrial fibrillation. Factors that will influence patient care over the next 5 years are considered, including risk stratification, procedure and technology options, and potential implications of CMS policies, such as bundling.

 

  • Associate Chief, Cardiology Division, Massachusetts General Hospital Heart Center
  • Professor of Medicine, Harvard Medical School
  1. HF treated by five drugs
  2. 3Million Patients in 2050 20Million Patients to experience HF
  3. Heart Rate – Rythm control in normal renge
  4. ablation surgery
  5. Prevention of Strokes
  6. AF: Chronic, persistent
  7. Risk is transferred to the providers
  8. Genetic profiling for early detection
  9. Going upstream for the Genetics and the Prevention
  • Director, Cardiac Arrhythmia Service, Massachusetts General Hospital
  • Associate Professor, Harvard Medical School
  1. AF in Men with Prostate Cancer and Women with Breast Cancer
  2. Pathological issues
  3. Cardiovergent or not
  4. poor definition of the AF underpinning
  5. NEW type of anticoagulation Safer that Warfrin
  6. 2/3 of AF are not on anticoagulation: Patient preference, doctor preference related to intolerance
  7. Screening AF with Genetics
  • VP, GM, AF Solutions, Medtronic
  1. AF 26Billion spend in the Hospitals
  2. Outcome improvement
  3. Lowering AF burden is very beneficial – endpoint of freedom of 30 seconds of AF for patient with comorbidities
  4. AF – multiple CV diseases
  5. Identify AF earlier – US not labeled product for AF Patient, outcome data needed
  6. drug management – which patient will benefit: help only patients that will benefit not any patient
  7. Wholistic view of AF
  • VP, Global Health Policy, Boston Scientific
  1. Payment reduction,
  2. Chronic condition with Acute episodes, post procedure
  3. life style modification
  4. Benefits last 3 years, then they come back
  5. Payers and MDs will ask for changes in life style for chance of success [weight reduction reached to qualify for knee replacement]
  6. Frint end and backend improvement – room to be optimistic
  • President, Cardiovascular and Neuromodulation, Abbott
  1. AF – inprovement of Patient quality of life
  2. extended patient life
  3. Innovation solutions : Improve Clinical outcomens and cost care reduction final Goal: CURE
  4. Each patient need the entire Tool Box creation of multiple tools
  5. Devices – how is the value determined? value delivered along a time span not insinq with cost
  6. Innovation in patient active participation in health management – tracking by Step,
  7. Incremental innovations
  • VP US Medical Affairs, CVMD TA, AstraZeneca
  1. Drugs for AF, biology of the disease little advancement, durg approach – understand the biology of the disease and streamline approach
  2. Disease progresses, remission,
  3. Is there a Biomarker as predictor for development of AF? delay onset of AF
  4. HTN is a potential cause of AF not in all cases
  5. Sleep Apnea, life style predictors of AF
  6. Drugs do not work have high toxicity for Arrhythmia 50% reduction in AF 25% placibo reduction – wrong target – Channels is not the right target
  7. Genomics and Biology — need to understand the disease better
3:35 pm – 4:05 pm
Boston Scientific Ballroom
1:1 Fireside Chat: Omar Ishrak, PhD, CEO, Medtronic
Moderator: Paul LaViolette
  • Managing Partner & COO, SV Life Sciences Advisers
  1. Technology and Value
  2. M&A
  3. Disease pathway
  • CEO, Medtronic
  1. Innovations are the essence of Medical Devices development as mission in technology
  2. Training Challenge in Surgical Robotic – patient comfort of minimal invasive therapy, cost lower
  3. Antibacterial sleeve saves cost of hospitalization, id infection occur Medtronic reimburses Hospital
  4. Respect of NOT INVENTED HERE – internal and external
  5. M&A 0 TAVR internal development THEN acquisition, HTN – acquisition did not work
  6. DIABETIC PUMP  – investment in R&D over 15 years
  7. Care management as Services – therapy and care management
  8. Technology company paid when it is delivered: understand cohorts,
  9. Strategy: Chronic Disease: AF, ablation is needed –
  10. Strategy: episodic care – success of intervention and the recovery from acuity, HF compensation in early stageCRT – hospitalization one year after the intervention is not acceptable
  11. 9 and 10 are measuring outcome differently
  12. Mitral Valve – platform for new generation of diagnostics
  13. ETERNAL: recover fast, improve outcome
4:05 pm – 4:55 pm
Boston Scientific Ballroom
Heart Failure’s Therapeutic Mandate

One million patients are hospitalized annually for HF—80% of total US cost of HF management. After discharge from HF hospitalization, 24% are rehospitalized within 30 days, greater than 50% within 6 months. Perspective on disease management, addressing the issues of hospital readmission and optimizing therapies.

 

Moderator: Akshay Desai, MD
  • Director, Heart Failure Disease Management, Brigham and Women’s Hospital
  • Associate Professor, Harvard Medical School
  1. How to leverage Big data
  2. Need for new therapies – collaboration of Academia and Pharma and Hospital
  • VP and Medical Director, Abbott
  1. HF – number of patient will double as Population grows and ages
  2. Pharmacogenetics will explain the pathophysiology
  3. Longterm management by specialist is important vs by PCP
  4. How do we randomize trials
  5. REIMBURSEMENT is key
  6. Glory of being persistant
  • VP, Global Translational Medicine Head (CVM), Novartis Institutes for BioMedical Research
  • Senior Lecturer, Harvard Medical School
  1. HF – lags behind
  2. heterogenous disease
  3. Preserve EF
  4. Genomics data collected on large population
  5. Big data is here, genotype, phynotypes,  – high quality data sets translation od data to therapeutics is coming
  6. Biomarkers important for endpoints
  7. Impresco – HF Drug with 20% improvement in EF
  • CMO, Myokardia
  1. HF – genetic and cellular level needed – in Oncology this is basic
  2. Precision medicine requires, devices to download CMR Cardiovascular magnetic resonance imaging (CMR), – data on fibrotics
  3. diagnosis etiology of HF is complex
  4. Preclinical trials are very important for early insights
  5. Younger patient with cardiomiopaty and older patient
  • SVP, CMO, Global Health Policy, Rhythm Management, Boston Scientific
  1. CHF is s clinical Syndrome,
  2. Telemeter physiologic information
  3. Physiology of HF – is well understood vs AF
  4. Translation of pathophysiology to therapeutics NOT YET accomplished
  5. Impending HF – device better that BP by MS
  6. Combination of therapies – what strategies yield best outcome, 20,000 participants in Clinical Trials is the wrong practice
  7. Translational science fails when clinical trials fail
  8. Need to be in the long haul, there is a model to be successful
4:55 pm – 6:00 pm
Boston Scientific Ballroom
CLINICAL HIGHLIGHT: A New Chapter of PAD

PAD is the most challenging atherosclerotic syndrome, largely due to the technological challenges of managing peripheral artery disease through minimally invasive strategies. Top physician, governmental, and industry leaders in the field discuss the potential for new breakthroughs including novel implantable devices, pharmacologic approaches, and reductions in associated cardiovascular morbidity and mortality.

The panel will also discuss, Below The Knee: The Persisting Unmet Need

 

Moderator: Michael Jaff, DO
  • President, Newton-Wellesley Hospital, Partners Healthcare
  • Professor of Medicine, Harvard Medical School
  1. Reimbursement strategy for PAD
  2. Congratulate CMS for covering PAD
  3. Clinical Trial design for devices for PAD – limitations as to what to propose to FDA and CMS
  • Director, Cardiology and Interventional Cardiology Fellowship Programs, Massachusetts General Hospital
  • Assistant Professor of Medicine, Harvard Medical School
  1. Nine million patient PAD
  2. systemic therapy, early access to care, arterial insufficiancy, better vascularization
  3. Modification diet for atherosclerotic disease – Mediterranean diet – conselling patients on a regular basis
  4. training operator – baloons is a game changer
  5. durable patency – no need to go back
  6. systemic burden od atherosclerosis
  7. Public recognition of PAD as an important complex disease
  • Director, Coverage and Analysis Group, CMS
  1. coverage incentives in the payment system
  2. design trials with FDA and CMS to ensure approval and reimbursement – know early
  3. Evidence-based tool, quality measure group
  4. CMS approach for site of treatment/service so to be reimbursed
  5. “Reasonable and Necessary” definition for PAD treatment – CMS continue to be innovative
  6. CMS needs data to cover technology
  7. CMS wishes to work with MDs
  • Chief, Peripheral Interventional Devices Branch, Food and Drug Administration
  1. heterogeneity, lack of data
  2. devices approval require consistent data, collaboration with NIH, CMS – design meaningful trials
  3. Match patients and match treatment
  4. mulriple companies work with FDA – FDA is willing to accept treatment, benefit and the labeling claims
  5. Early contacting FDA
  • CMO, Cardinal Health
  1. neuronal claudication vs vascular claudication
  2. continnum of care
  • Co-Director, Endovascular Surgery, Brigham and Women’s Hospital
  • Assistant Professor, Harvard Medical School
  • Critical Limb Ischemia (CLI)
  • 3D Printing – morphologic information on density and on patency
  • PAD is a tremendous challlenge PCI was solved PAD not yet
  • SVP and President, Peripheral Interventions, Boston Scientific
  1. ANTI peripheral therapies – above the knee and below the knee – different vessels
  2. No Consistency in wound care across institutions  – complexity on top of complexity
  3. Balance of rigorous science with adjustment by FDA
  4. Strategy – Category Leadership: not a singular technology: Bare stent, Stant and baloon and drug eluting stent
  5. PAD atherectomy
  6. Stem cells harvesting for clinical Trial – early stage research to assist patient early rather at late stage
  7. PAD Options is evolving
6:00 pm – 6:45 pm
Lilly Foyer

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LIVE – Real Time – 16th Annual Cancer Research Symposium, Koch Institute, Friday, June 16, 9AM – 5PM, Kresge Auditorium, MIT

 

REAL TIME PRESS COVERAGE & Reporter: Aviva Lev-Ari, PhD, RN

 

Summer Symposium 2017

https://ki.mit.edu/news/symposium/2017

@kochinstitute #KIsymposium @Pharma_BI @AVIVA1950

A leader in Convergence, MIT’s Koch Institute for Integrative Cancer Research will, on June 16, present its 16th annual Summer Symposium: the Convergence of Science and Engineering in Cancer Research. Convergence—the merging of historically distinct disciplines such as engineering, physics, computer science, chemistry, mathematics, and the life sciences—has created extraordinary opportunities in cancer research and care. Leaders in this emerging field will discuss innovative new approaches and technologies to better detect, monitor, treat, and prevent cancer. The symposium will also feature a panel of experts to discuss the impact of Convergence on the future of medical care.


INTRODUCTORY REMARKS

Tyler Jacks Tyler Jacks, PhD
Director, Koch Institute, MIT
David H. Koch Professor of Biology, MITLIVE – new Solutions for Cancer the mission og KI. Sponsors: Affiliates, Collaborators, Patrons, Friends, Vendors. Introduction to Prof. Sharp. 40th Anniversary of RNA related discovery leading to Nobel Prize of Prof. Sharp. 

Concluding the Symposium

  • continum
  • June 15, 2018 – NanoMedicine
  • Fall Symposium in September 2017
Phillip A. Sharp  

Phillip A. Sharp, PhD
Institute Professor, MIT
Koch Institute, MIT

LIVE – Convergence of Science and Engineering. Key note by a convergent personality – Eric Lander.

Convergence is a blueprint for innovation – NOW transformation of Life Science that follows the transformation of Physical sccience. Molecular biology – Delbruck, transmission of genetic phenotypes. Revolution of Biological Sciences in 20th Century

1st Revolution – DNA Structure, 1953

2nd Revolution – genomics Human Genome 2003

3rd Revolution – Integration of Science and the Environment in 21st Century

Such Small investment, 3.4% of NI Funding went to PI in BioEngineering 2000-2016

Introduction to Eric Lander – 1986 – to Whitehead Institute and MIT, now at Broad

KEYNOTE SPEAKER

Eric Lander

30 Years of Convergence

Eric Lander, PhD
President and Founding Director, Broad Institute of Harvard and MIT
Professor of Biology, Department of Biology, MIT
Koch Institute, MIT
Professor of Systems Biology, Harvard Medical School

LIVEPersonal Reflection on Convergence in BioMedicine

Process is important inconvergence — WHAT ARE WE CONVERGING TOWARD?

1985 — today — 2045

BioMedicine  – born from 4 Intellectual Revolutions:

  • Biochemistry – since 1890
  • Genetics – since 1900
  • Molecular Biology – since 1945, 1953, 2003
  • Recombinant DNA – since 1970

GENOMICS comes along: a View of

  • Completeness, Nature, 1986 – before the flood of data
  1. Biology is Finite – systematically perturbed
  2. Proteomics

Comprehensive views

Discovering Disease Genes – 1980s: Mendelian rare inherited, Common variation, Somatic

  • Human Genome – Finished, 2003
  • DNA, RNA, Proteins (Kinase, E3 Ligase) are finite and can be recognized from signatures

Human Genome Variations

Discovering Disease Genes – 2000s:

2017: 100 diseases are derived from Genome variation

  1. Disease Pathways discovered systematically: schesophrenia
  2. Cancer – Chromatin regulators and remodelers
  3. Regulatory elements: Conversation of species – finite and tractable
  • Evolutionary
  • signatures of cellular processes – connectivity map Dependency Map
  • 3D

Programmable Genome Targeting –

  1. CRISPR GPS
  2. Genome-wide CRISPR Screen

Human cells – unified coordinated to classify Cells and Tissues

  1. Combine single cells with CRISPR Screening to systematically discover cellularpathway

Comprehensive Tools

  1. Programmable Therapeutics –
  • DNA-directed Chemical synthesis
  • DNA-encoded Chemical Libraries
  • Programmable activation in cell type
  1. Healthcare systems –>> Learning systems

SPEAKERS

James Collins

Synthetic biology and next-generation diagnostics

James Collins, PhD
Termeer Professor of Medical Engineering and Science and Professor of Biological Engineering, MIT
Broad Institute of Harvard and MIT
Wyss Institute

LIVE

Synthetic Biology – Reprogramming life

  • Design & model network
  • Encode into DNA plasmid
  • Transfer to cell
  • monitor results

Potential for Synthetic Gene Biology

    • Paper-based synthetic Biology
    • distribution without refrigeration
    • RNA sensors with Colorimetric output
    • Rapid prototyping: Ebola sensors
    • Key features of Fieldable paper-based system
    • CRISPR-Cas9 Component for Strain Discrimination – NASBA-CRISPR Cleavage
    • Paper-based Diagnostics for the GUT microbiome
    • Paper-based Diagnostics for HPV: Rapid, Inexpensive
    • Sherlock – Nucleic Acid Detection with CRISPR-Cas13a
    • Human Genotyping Using SHERLOCK
    • Single-based gene usong Shrlock
Gad Getz

Cancer Genome and the Cloud

Gad A. Getz, PhD
Director, Cancer Genome Computational Analysis Group, Broad Institute of Harvard and MIT

LIVE –  Cancer is a disease of the Genome and epigenome – Life history of a Tumor

  • Drivers: Cancer phenotype
  • Smoking
  • Defect in DNA repair
  • Mutation type: C->T
  • Part 1: Finding Drivers Score genes by number and type of mutations: mutation tally and score of repair
  • 450 genes va 11 genes
  • 33 NEW cancer genes: 4,729 tumors, 21 tumor type, 254 significant genes
  • Gene Catalogue by Tumor type – 2,000 samples needed – to detect 90%
  1. Burden
  2. clustering
  3. Non-coding drivers: Promoter, Insulator
  4. 9 significant mutations in Breast cancer gene – promoter Hotspot in FOXA1 is activated through E2F – Estrogene receptor

Mutational Signatures

  1. Non-negative Matrix Factorization (NMF)
  2. posterior distribution of the signatures
  3. Homologuos recombination repair pathway

BRCA1/2: Signature analyzer: Breast Cancer mutation signatures

 

Mono-alleleic inactivation of BRCA

Germline mutation PALB2  PAthogenic mutations in genes

RAD51C – the third BRCA

Signature 3 is associated with BRCA

www.firecloud.org

 

Paula Hammond

Targeting Aggressive Cancers Nanolayers at a Time: A Platform Approach to Engineered Nanomedicine

Paula T. Hammond, PhD
David H. Koch Professor in Engineering, MIT
Head of the Department of Chemical Engineering, MIT
Koch Institute, MIT

LIVE

  • Layer-by-Layer (LBL) Assembly – The ultimate Nanofab Tool
  • Multilayered, multifunctional LbL Nanoparticles – chemotherapy at the core of a sphere
  • Engineering, Biology and Medicine — to understand activation
  • Incorporate siRNA – a modular design
  • pH-Dependent [6.0 – 7.4] Cell Uptake at Hypoxic Conditions
  • surface induce acid pH
  • avtive Tumor targeting (CD44) – Drug-loaded nanoparticle/PLL
  • Triple Targeting Threat: size, tumor hypoxia, cell receptor
  • A model target: MRP1 protein – Multidrug resistance
  • A tumor treatment study in TNBC subcutaneous model using synergisitc siRNA
  • Combo LbL NanoP’s work
  • KRAS inhibition: NSCLC: Kras siRNA + miRNA miR34a MicroRNA
  • cisplatin naoparticle – Orthotopic KP model: siRNA
  • Second Window NIR Imaging (NIR-II) – deep tissue of Ovarian Cancer
  • Nanoparticle Characterization
  • Ovarian Cancer imaging
  • Charged Assembly a Different way: Electrostatic Self-assembly of Oligopeptide Amphiphiles
  • Peptide Nanoparticles are suitable for Vivo – p53-Deficient Cells
  • Target: MK2 Mediates DNA Damage Repair
  • Small molecules: Not safe not specific

 

 

Robert Langer

New chemical engieering approaches to convergence

Robert S. Langer, ScD
David H. Koch Institute Professor, MIT
Koch Institute, MIT

LIVE

  • Lysozyme
  • soybean Trypsin inhibitor
  • Alkaline phosphatase
  • Catalase

Combinatorial lipid synthesis

Next generation LNPs: with novel lipids – potency improvement

Nano formulations for Entdothelium – combinatorial generation

Nano particle library

Current techniques:

Electroporation

  1. – insert inside the gene – CellSqueeze of the cytoplasm
  2. Full transcriptome microarray
  3. Applications to Personalized Medicine
  4. Injectable chip with combination drug therapy inserted into the Tumor, MIS,
  5. Confining region of tumor

Conversion : Clinicians and Engineers

Daniel Larson

Understanding transcription and splicing heterogeneity in cancer progression

Daniel Larson, PhD
NIH Stadtman Investigator, Center for Cancer Research
Head, Systems Biology of Gene Expression, National Cancer Institute

Daniel Larson, NCI – Transcription and Splicing in Heterogeneity in Cancer Progression

LIVE

  • TFF1
  • Transcription in living cells: Estrogene response
  • Transcription occures in bursts: seconds to several hours – regulated over multiple timescale
  • 4oth Anniversary of Splicing
  • high throughput approach for labelling thousands of genes at their endogenous loci
  • SLC2A1
  • Splicing times andd burst size are highly similar across genes.
  • Strong conservation in Eucaryote ccells
  • Splicing factor mutations emerged in almost all tumor types
  • 3″ SS recognition factor U2AF1 has a missense mutation in the zinc finger domain
  • DNA damage after X-Ray treatment: U2AF1 S34F cells show – cell survive high dose (20 Gy) irradiation, becomes senescent and live >1 month in culture abd secrete interleukin 8
  • Interleukin 8 Upregulated even before DNA damage – expression and secretion in rare cells
  • IL-8 induces Epithelial-Mesenchymal Transition i  primary mamary epithelial cells
  • Stochastic transcription and RNA splicing – IL-8 Secretion

 

 

Franziska Michor

Computational Models of Cancer

Franziska Michor, PhD
Professor of Computational Biology, Dana-Farber Cancer Institute
Harvard T.H. Chan School of Public Health

LIVE

  • Tumor evolve by natural selection
  • Tarceva – approval 2004 – NSCLC – patients with EGRF mutant
  • Effect of dosing:Number of sensitive cells in Two drug concentrations: Sensitive vs Resistant cells
  • Combination Treatment
  • Better parameter estimation: using single cell lineage tracing data
  • microenvironment determinants of Treatment response
  • Determining Optimum radiation schedules in GBM
  • mathematical modeling of treatment response
  • understanding the intratumor heterogeneity based on mouse modeling
  • optimal radiation schedule –>>
  • dose constraints – Ultra fractionated dose vs.
  • Practicality constraints
  • slower proliferation
  • Radiation plus temozolomide: Optimized; time post treatment/percent survival
  • Treatment modalities: Immunotherapy, chemo drugs

 

 

Chad A. Mirkin

Spherical Nucleic Acids as a Powerful New Platform for Cancer Therapy

Chad A. Mirkin, PhD
Director, International Institute for Nanotechnology
George B. Rathmann Professor of Chemistry, Department of Chemistry, Northwestern University

LIVE

Next wave of Pharmaceuticals

  1. Small molecules’
  2. biologics
  3. Nucleic Acids – limitations: Address disease in the Liver

 

  • The Promise of Therapeutic Oligonucleotides
  • Antisense DNA
  • Spherical Nucleic Acids (SNA)  – New way of thinking on DNA and RNA
  1. Hybridization Thermodynamics of SNAs
  2. SNAs enter cells rapidly and efficiently – over 60 different Cell Types
  3. Cy5-labeled SNA
  4. SNAs Come in many forms: Micellar SNA, Protein SNAs, Lipoprotein SNAs
  5. Late endosome nOT lysosomeow
  6. How does cell membrane recognition of SNAs and trigger exocytosis
  7. SNAs as Therapeutics: Skin (topical for Psoriasis), Brain (GBM)
  8. Immunostimulatory SNAs – activity of IS-SNAs in Vivo
  9. Oligonucleitides strands mimicking bacterial DNA or RNA
  10. SNA – Vaccines: Antigen presenting cells
  11. 3D Architecture of SNAs leads ti enhanced TLP9 Activation – B-cell NF-KB Activation
  12. SNAs Vaccines: -In Vivo Testing: retired Tumor growth vs control – inserted inside liposome
  13. Advantages SNAs Vaccine vs Standard Formulations vs adjuvant (antigen)
  14. SNA TLR-9 Activator Treatmeent vs Linear Oligo (EMT-6 Breast Cancer Model] – it potentiates activity of Anti-PD-1 Antibodies in PD-1 Resistant  tumor confers immunity

SNA Competitive advantage

Dissecting the tumor ecosystem with single cell genomics

Aviv Regev

 

Aviv Regev, PhD

Director, Klarman Cell Observatory and Cell Circuits Program, Broad Institute of Harvard and MIT
Professor of Biology, Department of Biology, MIT
Koch Institute, MIT

LIVE – Tumors: A complex cellular Ecosystem

  1. How to use Genomic sto study tumors?
  2. Option 1: Gene Atlas – single cell genomics can help dissect thie ecosystem – bulk cell
  3. Option 2: Tumor Cell Atlas – single cell
  4. Single cell RNA-Seq in Precision Medicine pipeline
  5. 19 metastasis melanomas: “non-design” design – 4600 cells
  6. Malignant or not? DNA or RNA-inferred
  7. Micronvironment of the cell – Diverse T cells, Naive,  – Tumar infiltrating T cells: Activation-independent variation in exhaustion program across cells
  8. cytotoxicity, exhausion, naive
  9. RNA-Seq associates T cell clones with their states

Cell-types and states in the melanoma ecosystem:

  • single cell signature used to cluster bulk tumors by their microenvironment composition
  • TCGA – Tumors – melanoma vs Cell type specific signature genes
  • inferring cell-cell interactions: Cell type A and Cell type B: Correlation of gene’s expression with inferred proportion of cell type B (bulk samples)
  • CAF expression of chemokines and complement associated with CD8 T cell infiltration
  • Primary Test Stronger control
  • The ecosystem of Malignant Melanoma – 19 malignant cells

Treatment naive vs Immunotherapy resistant (ITR) – CD8 cells from ITR sample – intratumor variation

How malignant melanocyte affect T-cells?

  • Infiltrated tumor vs exclusion tumor
  • ITR has prognostic value – predicts response to anti-PD1 in Pationts
  • ITR Signatures in malignant and CD8 T cells
Xiaowei Zhuang

Illuminating biology at the nanoscale and systems scale using single-molecule and super-resolution imaging

Xiaowei Zhuang, PhD
David B. Arnold Professor of Science, Harvard University

Session I: NEW VIEW

Xiaowei Zhuang, Harvard University — Single-Cell Transcriptome and Genome Imaging,

  • Science, 2015,
  • PNAS, 2016

LIVE

  • molecular specificity
  • moleculare-scale resolution
  • genome scale throughputspatially-resolved single-cell transcriptome
  1. subcellular organization of the transcriptome
  2. Spatial organization of transcriptome in tissue
  • Transcriptome Imaging
  1. single-molecule FISH (smFISH) [Image 1,2,3,4,, Decoded Image]
  2. Error-robust encoding: Hamming code

Modified Hamming distance 4 – multiplexed, hydridization

Applications:

  1. MERFISH imaging of Tissue
  2. Spacial organization of cells in Tissues
  3. High throughput image-based screening: Barcodes, gene activation/inhibition agents
  4. Tracing the 3D conformation of Chromatin
  5. Spatial organization of A-B compartments: Transient, Polarized, radial
  6. Super resolution of chromatin imaging with Transcriptome imaging

 

EXPERT PANEL: CONVERGENCE IN HEALTH

Cori Bargmann LIVE

Cori Bargmann, PhD
President of Science, Chan Zuckerberg Initiative
Torsten N. Wiesel Professor, The Rockefeller University

LIVE – translation of the needs in computational, biology, IT, mathematician, medicine, physics and engineering. BRAIN Initiative at NIH. How to motivate? by a Problem in need for solution

 

Marc N. Casper  

Marc N. Casper, MBA
President and CEO, Thermo Fisher Scientific

LIVE – convergence from a tool perspectiveLast 20 years – integration of Biology into the physical sciences,

SCREENING AND DIAGNOSTICS

Victor Dzau  

Victor Dzau, MD
President, National Academy of Medicine

LIVE

As cardiologist: Pace maker Convergence in Research.

Funding research does not encourage convergence

innovation is killing disease, need to introduce cost effectiveness – economics into the play. Recognition of cost in Precision Medicine: economic model as a tool to consider cost effectiveness,

Convergence to include SOcial Sciences and Economics

Diagnostics;

Tyler Jacks  

Tyler Jacks, PhD
Director, Koch Institute, MIT
David H. Koch Professor of Biology, MIT

LIVE

– Convergence at Koch

NIH created few CENTERS for research in Cancer, Brain, Nanotech, focus on Cancer did help the convergence

Next step of Convergence: common language, Learning by doing, mechanisms to encourage and continue the convergence

Better efficiency:

  • Which Drug to which Patient
  • Early detection and prevention
Nancy Simonian  

Nancy Simonian, MD
CEO, Syros Pharmaceuticals, Inc.

LIVE – convergence is required to understand the biology of a disease, genomics, computational biology and chemistry is a new approach

Cost of Medicines: reibbursement per value  vs no concern to value. Innovation t carry premium allowing cost reduction while effciency is been exploled.

Elias Zerhouni  

Elias Zerhouni, MD
President for Global Research and Development, Sanofi
Former Director, NIH

LIVE

Convergence of multidisciplines is a must for scientific solutions to emerge, horizontal integration

Patient Bill of Right

Defficiet every year in the US,

Susan Hockfield

Moderated by:

Susan Hockfield, PhD
President Emerita, MIT
Professor of Neuroscience, MIT
Koch Institute, MIT

 

 

 

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LIVE 11/17 1:45PM – 5PM – The 12th Annual Personalized Medicine Conference, HARVARD MEDICAL SCHOOL, Joseph B. Martin Conference Center, 77 Avenue Louis Pasteur, Boston

 

Leaders in Pharmaceutical Business intelligence (LPBI) Group

Covering in Real Time using Social Media this Event on

Personalized Medicine

Aviva Lev-Ari, PhD, RN, Founder LPBI Group & Editor-in-Chief

http://pharmaceuticalintelligence.com

Streaming LIVE @ HARVARD MEDICAL SCHOOL,

Joseph B. Martin Conference Center

@pharma_BI

@AVIVA1950

November 17

#PMConf

1:45 p.m. — Leadership in Personalized Medicine Award

  • Presenter: William S. Dalton, Ph.D., M.D., CEO, M2Gen, Chairman, Personalized Medicine Coalition

Science, Business and Patents: Millenium, Celgenics, and Medicine/Desease – Member of AAAS

co-Chair Cancer Consorcium

PM – 1990’s on. How Human Genome at Harvard will start a new center – reach out to the Global community, conference was born. PM as subject of a Global Conference, effirt started with Genzyme, Eric Launder, Broad, Collins at NIH – effort led to Obama Initiative in PM, Duke Medical System.

Challenge: Reimbursement for Genomics diagnosis

  • PM – P care – by sequencing of Genome – become available commercially inexpensivelly
  • Genetic component to become part and parcial of Medicine and Patient care

2:15 p.m. — Networking Break

2:45 p.m. — The Data Dilemma: Fulfilling Expectations of Big Data in the Future of Personalized Medicine

There is consensus that the massive amounts of genomic, clinical, claims and other types of data could yield important insights for research and clinical care. But for years, obstacles around technical standards, interoperability, privacy and confidentiality, data security, and consent have been held up as daunting challenges that inevitably slowed progress.  During this discussion, a panel of academic and industry experts will discuss their respective organizations’ strategies to obtain and analyze the data, including what has worked and what has not; the programs and processes that have led to the most productive data usage; examples of important knowledge that has been derived from data analysis; and the infrastructure they believe is needed to achieve fulfillment of the potential of big data in personalized medicine nationwide.

  • Moderator: Marcia A. Kean, M.B.A., Chairman, Strategic Initiatives, Feinstein Kean Healthcare
  1. How one works with 20 Partners at once?

 

  • Paul Bleicher, M.D., Ph.D., CEO, OptumLabs
  1. Data collaboration of 35 Partners – bring value to Medicine, Like Bell Labs
  2. Academics, Hospitals, Physician offices – Constellations – groups of projects
  3. DATA is KEY — Public and Private Partnerships
  • Christophe G. Lambert, Ph.D., Associate Professor, Center for Global Health, Division of Translational Informatics, Department of Internal Medicine, University of New Mexico
  1. VA Data, Co-Chair of Informatics, clinical , pharmaceutical stackholders
  2. Focus Groups Patient research Partners – How to automate data
  3. Centralization above nad decentralization, below COntrol mechanism govern all variables: Increase fitness of system vs Personal Control
  4. 1984-1998 Bi-Partisan support for Data in HealthCare
  5. Big Data for early detection, prevention, `
  6. AGING, Infectious and Pediatric disease – Investment in these areas
  • Adam Margolin, Ph.D., Director, Computational Biology, Oregon Health & Science University School of Medicine
  1. Project with Intel – across institutions
  2. consorsium – success ration
  3. data sharing #1 Priority at the National Level
  4. Add value by data sharing, strategic investment in the healh system
  • Edward J. Stepanski, Ph.D., Chief Operating Officer, Vector Oncology
  1. Propriatory real time reporting to Physicians – systematic – core asset, originally,
  2. Research Group use Warehouse doing Analytics, Tools development linked with clinical data with PRO and studies based on data integretion
  3. Success is more data – PRO data informing clinical data
  4. Defragmenting the care vs drive across town for care several units disaggregated geography vs all deaprtments in one location

 

3:45 p.m. — Keynote Speaker
“Medicine and the Targeted Marketing Problem”

We live in the golden age of cloud computing and machine learning.  The organizing conundrum for the “big data era,” however, is a surprising one — the “targeted marketing problem” (i.e., the ability to better match the right customers to targeted messages). This talk will explore overlaps and similarities between the targeted marketing problem and precision medicine, and how advances in data sciences can be leveraged to create a learning medical system that in turn points to the health care system of the future.

  • Introduction: Amy Abernethy, M.D., Ph.D., Chief Scientific Officer, Senior Vice President, Oncology, Flatiron Health

 

  • Anthony Philippakis, M.D., Ph.D., Cardiologist, BWH, Chief Data Officer, Broad Institute and Partner, GV (Venture Capital)

Learning from Users

Five causes for cardiac death:

  • MI,
  • a-Fib
  • Structural
  • PE
  • Aorta dissection

PreventionGenomic Sequencingvalue in Cardiology:

  • Estonia BioBank – mutation carrier
  • Familial Hypercholesterolemia – 4 genes involved,
  • Prediction sudden cardiac death – larger data sets
  • New Model for Human Subjects Research; DIrect-to-Participant: Potentia Advantages:
  • cost, scalability, facilitate re-contact, frequent collection,
  • My Research Legacy: Broad & AHA – Launched November 13, 2016 
  • Quantified Self –>> Quantigied Physical Exam: Face dysformia, Dysarthia, Ataxia,
  • Identify every patient in the World  with this disease

 

Data sharing: Inverting the Model ; ALL OF US  – 1 Million – Precision Medicine with IBM – Mandate to innovate – Diversity: People, Geography, Health Status

Innovation in Genomic data sharing – bring data to researchers

SIX types od data wil be collected: Participant-provided Info, mHealth Data, Consent EMR

 

DATA Research CoreVanderbuilt, Verily Broad

  1. pharmacogenomics

Launch start ups cost

  1. Open source
  2. Cloud
  3. developers start ups

PLATFORM

 

GATK – workhorse of genomic data – Launched 4/2016

Partnerships: Amazon, google genomics, microsoft, IBM, Watson, 

 

Announcing: BROAD + Intel Center for Advanced Genomic Data Engineering, Anthony Philippakis, M.D., Ph.D., Chief Data Officer, Broad Institute

Reference Architecture: Design: Single node, small cluster,

 

4:30 p.m. — Closing Remarks

  • Edward Abrahams, Ph.D., President, Personalized Medicine Coalition

 

– See more at: http://www.personalizedmedicinecoalition.org/Conference/November_17_Program#sthash.zpTNQYKd.dpuf

 

#PMConf

SOURCE

http://www.personalizedmedicinecoalition.org/Conference/November_17_Program

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LIVE 11/17 8AM – 1:45PM – The 12th Annual Personalized Medicine Conference, HARVARD MEDICAL SCHOOL, Joseph B. Martin Conference Center, 77 Avenue Louis Pasteur, Boston

Leaders in Pharmaceutical Business intelligence (LPBI) Group

Covering in Real Time using Social Media this Event on

Personalized Medicine

Aviva Lev-Ari, PhD, RN, Founder LPBI Group & Editor-in-Chief

http://pharmaceuticalintelligence.com

Streaming LIVE @ HARVARD MEDICAL SCHOOL,

Joseph B. Martin Conference Center

@pharma_BI

@AVIVA1950

November 17

#PMConf

 

Joseph B. Martin Conference Center
77 Avenue Louis Pasteur
Boston, MA 02115

7:00 a.m. — Registration and Continental Breakfast

8:00 a.m. — Opening Remarks

  • Edward Abrahams, Ph.D., President, Personalized Medicine Coalition

8:15 a.m. — Fireside Chat

  • Moderator: Meg Tirrell, Reporter, CNBC
  1. How did the the Economics changed
  • Daniel O’Day, CEO, Roche Pharmaceuticals – Joined Roche at 1989
  1. Roche  – 60% of investments goes to Cancer with embedded diagnostics, 20-30% of the market
  2. Hypothesis in the Lab starts an innovation – Phase I, Phase II is extension of Phase I – continue understanding of the Biology of the Disease
  3. Treat only patient that will benefit – PM – transformational benefit
  4. Early stage of discovery – protection of IP – work inside ONE company — less of an issur the protection of IP
  5. Diagnostics area – Roche collaborates with other Pharma
  6. Setting infrastructure for testing
  7. Diagnostics and Pharma are coming together – availability of big data – discover and develop with Foundation Medicine – Deep analysis of Molecular Medicine, decide o better hypothesis, do it in shorter time – 2015 – Commercialize the platform around the Globe, One standard for Clinical Trials – in China hard to move Clinical Trials Data out of China –
  8. Harnessing Global Data in Oncology – Clinical Trials
  9. Data accuracy
  10. Genentech, Foundation Medicine and Roche capabilities – FDA
  11. Comprehensive Genomics side – has needs yet to be developed for Payers to participate
  12. Foundation One – 30% more positive Lung Cancers found vs standard of testing
  13. Over simplification is dangerous, technology/diagnostocs/histology/genomics – sequencing ENHANCES not replaces
  14. Data of Phase III – robust genomics profiling: no diagnostics and wrong diagnostics
  15. In the next five years, Cancer immunotherapy, when and how resistance occur.
  16. Blood based assay – Patient journey  – fine tuning the Science tissue based sample
  17. Biomarkers: Tumor microenvironment
  18. Diagnostics is not rewarded appropriately, genetics, CMS,
  19. Outcome value for TX and Dx
  20. Mission of Roche will not Change with change of Gov’t, public Sector in the US, FDA – requires being faster a respondent,
  21. ” I am optimistic”

Questions from the audience

  • Democratizing access to sequencing data
  • accuracy of test results, Oncologist and PCP ordering genomics tests, value added
  • PM after medicine SOC (biopsy, tissue histology)
  • Reimbursement: Diagnostics vs drugs

8:45 a.m. — Coverage is King: Identifying the Evidence That Leads to Reimbursement

Many innovators in personalized medicine are unclear on the kinds of evidence that inform the coverage and payment decisions of payers. That lack of clarity can have negative financial consequences for personalized medicine companies with products and services that are on the market but not paid for. During this panel, payer representatives will help define the reimbursement landscape for the field by providing examples of the evidence they consider appropriate for coverage and payment. Confirmed panelists include:

  • Moderator:Amy M. Miller, Ph.D., Executive Vice President, Personalized Medicine Coalition
  1. What each of the companies does in PM
  2. Targeted therapeutics: 25% are targeted,
  3. Value of Diagnostics
  • Kristine Bordenave, M.D., Lead Medical Director, Humana
  1. Clinical perspective, how much it cost to patient, clinician, Pharma – both need to be paid,
  2. Population Health, 100% of GDP to go to HealthCare — can’t be
  3. Humana Perspective: How to cover – organized criming: Charges for Testin – several month doen in one day, sharing drugs, expired drugs, repackaged and sold, drugs resold
  4. Independent Research Department: MS, Pharmacists, Statisticians — Looking at the Value of Test in Population context
  5. Large Medicare, Small managed care company, Value-based contracts: working with Pharma – early on at Phase II stage
  6. Testing: genetics, radiographic — 60 gene panel – done by two labs,
  7. Duplication in ordering genetic testing: optometrists, Physical therapist, ordering genetic profiling
  • Matthew Fontana, M.D., Vice President and Chief Medical Officer, Pharmacy, Health Care Service Corporation
  1. WHY REFERE TO US AS PAYERS – BUSINESS MODEL: MULTIPLE CUSTOMERS MEDICAID, BIG PROVIDERS, MANAGED CARE INDUSTRY
  2. Cost and Revenue
  3. FDA is been pushed to ignore the science (DMD), lack of coordination in HealthCare
  4. Pay for diagnostics if not linked to Therapeutics – morbidity
  5. elaboration of Diagnostics
  6. Accuracy of testing – expensive  – misinterpretation
  • Elaine Jeter, M.D., MolDx Medical Director, Palmetto GBA
  1. Access of Patients – 25 of 50 States are participants in MOlDx — NOT New England States
  2. All molecular assays to register for code specificity – to be able to control appropriate coding – Panel matched to unit of service issue between Genome Profiling and assay
  3. Reimbursement – Lung Cancer – Clinical utility  – genomic profiling ONLY IF THE DATA IS IN A REGISTRY — IF PROVED UTILITY AND THE REGISTRY SHOULD BE IN PUBLIC DOMAIN
  4. Analytical minimal standard accepted
  5. Developed Assessment meetings – Labs come to receive guidance  – clinical utility information, as a contractor – Central Office allowed PM vs Lab developing tests – assist Lab – pay for service obtain end points
  6. Assays for Prostate Cancer – innovative – high disease demand, define endpoints
  7. Paying premium for FDA approved genetic testing – onlu 2% of molecular assays — 98% are not FDA approved
  8. 65,000 molecular tests in the market in the Registry only 10,000, every day 2-3 new molecular assay tests are introduced
  9. By statue, no screening covered by Medicare for Genetic testing – congress need to act upon that – change coverage of Medicare. Memogram and colonoscopy, lung X-ray – are by statue – covered by Medicare

Questions from the Audience

  • Why premium paid if FDA approved a test?
  • Screening and early detection

9:45 a.m. — Networking Break

10:15 a.m. — Harvard Business School Case Study Presentation

DNA-editing technologies have been hailed as revolutionary with the possibility to edit out mutations that cause disease.  Yet the CRISPR-Cas system is currently locked in a legal dispute between two great research institutions involving, as one journalist put it, “who owns molecular biology.”  The CRISPR technology in short raises the broader issue of whether these new techniques should be privately owned or placed in the public domain. The technology also raises serious ethical issues. The case study will serve as the point of departure for our discussion of these issues.

  • Leader:Richard Hamermesh, D.B.A., Senior Fellow and Former MBA Class of 1961, Professor of Management Practice, Harvard Business School
  1. Ethical issues in the case
  2. Stacks are very high

11:15 a.m. — Keynote Speaker

  • Introduction:William Chin, M.D., Chief Medical Officer, Executive Vice President, PhRMA
  • Keynote:Victor Dzau, M.D., President, National Academy of Medicine (ex-IOM) – part of NIH
  1. Global Landscape of PM: Integration into HealthCare — Cost effectiveness
  2. Evidence for PM
  3. better health and well being
  4. high value health care
  5. strong science & technology – 150 papers in JAMA ans NAS
  6. Precision Medicine: patient/public engagement, NGS: omics, biomarkers, collection of clinical & research data, integration of omics, EHR
  7. Challenges: Tests & Therapies
  8. efficacy of PM AFTER actually being used in clinical practice
  9. Evidence of PM efficacy for implementation in Practice
  10. Regulatory and Reimbursement for utility
  11. Reward Value vs cost
  12. Aligning Results: 10% incidence reduction vs 50% incidence reduction
  13. Evidence generation:
  14. Modeling could be used to assess the potential economic impact of PM approaches
  15. Final regulatory & Payment Pathwaysand payer approval
  16. Analytic Validity — clinical validity — economic impact analysis– Assess Clinical Utility
  17. Strength of evidence Low to High
  18. investigational experiment
  19. Assess cost effectiveness: initial experimentation — Economic analysis — provisional approval — validation — final approval
  20. Integration with clinical Practice: Clinician Educatuin, integration pathways
  21. Genomic Medicine:guideline and care pathways — Clinical DSS —
  22. Data infrastructure and sharing: EHR (DIGITize) – genomics – GA4GH
  23. PATIENT/PUBLIC ENGAGEMENT – concern of Privacy and Data Ownership – Fear of discrimination  — consent — education of Patients
  24. Future needs: data, National scale learning system, Global collaboration G2GH
  25. Support Data infrastructure
  26. PM – quality, access, cost, improve clinical outcomes, inequality mitigate
  27. PRECISION PUBLIC HEALTH – ZIKA, EBOLA,
  28. GENOMICS and Population Health Action Collaborative
  29. working Groups: Evidence generation

11:45 a.m. — Bag Lunch

12:45 p.m. — Personalizing Care: Strategies for Integrating Personalized Medicine into Health Care

Personalized medicine lacks sufficient literature on how health care providers can integrate personalized medicine into clinical care, which makes it difficult for providers to take advantage of the growing number of personalized medicine products and services now available to them. During this session, panelists who have spearheaded integration efforts will share the strategies they found most useful for speeding the pace of personalized medicine’s adoption in clinical settings. Confirmed panelists include:

  • Moderator:Howard McLeod, Pharm.D., Medical Director, DeBartolo Family Personalized Medicine Institute, Moffitt Cancer Center
  1. Awareness & education
  2. Patient empowerment
  3. value recognition
  4. IT and Information Management
  5. ensuring Access to Care: Case if Neuropathy than 15 more visits causing 8 other Patients to be pushed in the queue

 

  • Amy Abernethy, M.D., Ph.D., Chief Medical Officer, Chief Scientific Officer, Senior Vice President, Oncology, Flatiron Health
  1. Family and Care givers need to understand as well
  2. More Advocacy in Washington

 

  • Dax Kurbegov, M.D., Physician Vice President, National Oncology Service Line, Catholic Health Initiatives – CHI (103 Hospitals) and DIgnity ospitals – Community system
  1. Provide speed of service
  2. Permeate piece by piece by each institution, economics – problematic IT infrastructure for Genomics is expensive, centralized system needed
  3. broader beyond Oncology
  4. complex Patients with polypharmacy
  5. If physician needs to write a special note for service , patients are lost in the way for testing
  6. as NGS become accessible in labs — CHI provide infrastructure to LINK Patients with Experts and Labs outside the system
  • Lincoln Nadauld, M.D., Ph.D., Executive Director of Precision Genomics, Intermountain Healthcare, UT (22 Hospital 107 physicians – Molecular Tumor Board)
  1. Pilot Project approach implemented in 3 hospitals, built lab, implement by Molecular Tumor Board placed on the report
  2. Barriers: getting drug is difficult – mutation exists, drug exists — How to get the drug ordered, approved and shipped
  3. Patients want to know that their oncology is up to date the care is best, Patient advocate for themselves, Patient empowerment
  4. Barriers to PM – Physician compensated better for next line IV chemo vs Targeted Genomic-based therapy
  5. Tumor Board
  6. Get Genomics EARLY not late – it will max the course of treatment
  7. MOST PATIENT CAN’T TRAVEL FOR CARE because it is expensive
  8. Utility and Value asked by Payors, cost saving need be demonstrated not only efficacy vs SOC – reduce cost must be demonstrated

 

  • Peter H. O’Donnell, M.D., Assistant Professor of Medicine and Associate Director for Clinical Implementation, Center for Personalized Therapeutics, The University of Chicago
  1. Lab Testing, way to long, system for Physician to look at Genomic data,
  2. If Physician is buying in shared decisions with Patient easier
  3. all tests are bundled and results are presented to PCPs – they love that
  4. Drugs fail because Patients do not take them vs Pharmacogenomics – more likely to help Patients

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LIVE 11/16 3:15PM – 5:30PM – The 12th Annual Personalized Medicine Conference, HARVARD MEDICAL SCHOOL, Joseph B. Martin Conference Center, 77 Avenue Louis Pasteur, Boston

 

Leaders in Pharmaceutical Business intelligence (LPBI) Group

Covering in Real Time using Social Media this Event on

Personalized Medicine

Aviva Lev-Ari, PhD, RN, Founder LPBI Group & Editor-in-Chief

http://pharmaceuticalintelligence.com

Streaming LIVE @ HARVARD MEDICAL SCHOOL,

Joseph B. Martin Conference Center

@pharma_BI

@AVIVA1950

November 16

#PMConf

 

 

3:15 p.m. — Diagnostics Debate: Regulatory and Reimbursement Hurdles for Personalized Medicine Diagnostics

Nowhere are the regulatory and reimbursement challenges facing personalized medicine more evident than in the diagnostics industry, where the routes to market are often hampered by a lack of clarity regarding the possible changes to the regulatory pathway for laboratory-developed tests, ambiguity regarding the kinds of evidence that justify payment, and the need for large marketing budgets to sell low-cost procedures, all of which impede the development of sophisticated diagnostics with the power to transform medicine.

During this panel discussion, representatives from a diverse range of diagnostics companies, a payer and FDA will identify the most promising strategies to alter the landscape to encourage investment in personalized medicine diagnostic products, including the roles of other stakeholders such as the pharmaceutical industry and integrated health systems.

  • Moderator: Ronnie Andrews, Founder and Principal, The Bethesda Group

 

  • Suzanne Belinson, Ph.D., M.P.H., Executive Director, Center for Clinical Effectiveness, Blue Cross Blue Shield Association
  1. Centralized Evidence review – Payers making coverage decisions
  2. Blue Payers: Payers: can modify or use it as such
  3. Value proposition of Evidence Review: Medical Specialties, Payer, Providers, Manufecturer of Diagnostics, Clinical Research
  4. Where is the GAP, in evidence review
  5. engagement with Blue and non Blue, Community based Hospitals
  6. Test utilization – decision is made – withhold a treatment or modification of treatment — at the aggregate level – impact on outcome can be achieved
  7. reduce number of test is a health outcome,
  8. redude numbers of day in Hospital is a Health Outcome
  9. Curate information for Payers to use the information for policy
  10. Prime Therapeutics – PPM of 14 organization — are clients for Evidence Review by Center for Clinical Effectiveness, Blue Cross Blue Shield Association – 36 plans, IT issues, Patient is covered in IL, treatment is givrn in FL, different rules in FL.

 

  • Brad Gray, CEO, NanoString
  1. Seattle-based gene expression (emerged in academia), development of diagnosis
  2. IN VITRO DIAGNOSTIC KIT – PREDICTION OF RESPONSE TO BREAST CANCER
  3. Cengene – response to their druv
  4. Medidiation – prospate cancer
  5. Merck – response to Keytruda – multiple indications
  6. Universal asset development: Diagnostics several parameters in ONE test
  7. Policy change is not the focus bur the association of diagnostics with drugs

 

  • Alberto Perez, FDA

 

  • Michael Pellini, M.D., CEO, Foundation Medicine – had experience with Thermo Fisher Diagnostics
  • Molecular diagnostics space, information comapny not Lab or Diagnostics
  • Comprehensive tumor testing – ASSETS:
  1. EXTRACT MOLECULAR information from tissue vs multiple biopsies
  2. COMPEHENSIVE DB OF 1,000 PATIENTS – Genomic Profiling – Rows are cancer type Columns are Published studies providing evidence
  3. Decision Support: MD, Pathologists – Testing Platform submitted to FDA to get regulatory standards
  4. Biliary small tumor

 

4:30 p.m. — Visions of Value: Evaluating Evidence for Personalized Medicine

The fact that payers, providers, patients, industry representatives and regulators all define value differently makes it difficult for personalized medicine’s champions to contribute to and communicate about the body of evidence supporting the field. Participants in this panel discussion will bring the personalized medicine community closer to an accepted definition of value by identifying common elements in multiple stakeholders’ understanding of the concept.

  • Moderator: Susan Dentzer, President and CEO, Network for Excellence in Health Innovation
  1. Without Pricing adjustment Access can’t be accomplished
  2. drugs for HPC – patient is not sick enough to qualify for the drug
  • Donna Cryer, J.D., President and CEO, Global Liver Institute
  1. societal impact of a diagnosis
  2. Development of Value framework, development of he evidence that goes into Value frameworks, Outcomes desireable, Patient generated evidence
  3. Health high functioning citizens and a fibrant Health Care industry
  4. PM PARADIGM accepts Patients factors to be used in algorithm development
  5. Restrictive Formulary for non avarage patients – access at time of need

 

  • Michael Sherman, M.D., M.B.A., M.S., Senior Vice President, Chief Medical Officer, Harvard Pilgrim HealthCare
  1. Pay for Service is not connected to Value
  2. Charge the variable cost for testing
  3. DECISION re driven by costs: delivering drug lowering cholesterol to avoid facing expenses of trating CVD
  4. Higher price for success
  5. Care about the total cost of Care
  6. payment for innovations
  • Peter B. Bach, M.D., M.A.P.P., Director, Center for Health Policy and Outcomes, Memorial Sloan Kettering Cancer Center
  1. Oncology drugs and Priding to improve the decision making
  2. US and specialty drugs
  3. severe access problems for drugs to patient – colapse because of Pricing
  4. out performing in cost of treatment per patient
  5. Societal equation – drug prices
  6. AMA embrassed Value based Pricing on 11/16/2016
  7. Linking Price of Drug with what the drug does

 

  • Randy Barkholder, PhRMA
  1. consolidation Prices for Medicine and for Societal are different
  2. add value to the Healthcare system
  3. Performace and quality to measure value
  4. Drug companies representation and SOcietal values vs HealthCare system value

 

5:30 p.m. — Elements Café Cocktail Reception

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LIVE 11/16 1:15PM – 2:45PM – The 12th Annual Personalized Medicine Conference, HARVARD MEDICAL SCHOOL, Joseph B. Martin Conference Center, 77 Avenue Louis Pasteur, Boston

Reporter: Aviva Lev-Ari, PhD, RN

Leaders in Pharmaceutical Business intelligence (LPBI) Group

Covering in Real Time using Social Media this Event on

Personalized Medicine

Aviva Lev-Ari, PhD, RN, Founder LPBI Group & Editor-in-Chief

http://pharmaceuticalintelligence.com

Streaming LIVE @ HARVARD MEDICAL SCHOOL,

Joseph B. Martin Conference Center

@pharma_BI

@AVIVA1950

November 16

#PMConf

1:15 p.m. — Update: Kraft Precision Medicine Accelerator & Trials Challenge Award

An update on the activities of the Kraft Precision Medicine Accelerator and interviews with the winners of Harvard Business School’s “Precision Trials Challenge,” sponsored by the Kraft Precision Medicine Accelerator.

  • Presenter: Richard Hamermesh, D.B.A., Faculty Co-Chair, Kraft Precision Medicine Accelerator, Harvard Business School
  • Winner: MatchMiner
    • Team Lead: Ethan Cerami, Ph.D., Director, Knowledge Systems Group, Department of Biostatistics and Computational Biology, Dana-Farber Cancer Institute
  1. 17,000 patients now are genome sequenced
  2. Rational clinical trial design
  3. enroll patient n trial
  4. clinical decision support
  5. Trial-Centric Matching vs Patient Centric Matching
  6. Open Source PLATFORM
  7. Clinical Trial Markup Language (CTML)
  8. MatchMiner, Knowledge System, The Hyve

 

 

 

  • Runner Up: No Patients Left Behind
    • Team Lead: Gavin MacBeath, Ph.D., Co-Founder and Senior Vice President, Merrimack Pharmaceuticals
  1. Company brings Drug and biomarker assay match – for patient assignment to Trial
  2. Protein based not genomic based test

 

  • Runner Up: iCare for Cancer Patients
    • Team Lead: Leylah Drusbosky, Ph.D., Associate Professor of Medicine, University of Florida, Scientific Director, iCare for Cancer Patients
  1. iCare for Cancer Patients
  2. Protein Network Map: Drug interaction and Genomic aberrations
  3. Functional interactions
  4. Predictive SImulation Technology

Computational Biology Model: Proliferation, SUrvival apoptosis

VIrtual Cancer Clinical Trial Simulator – Bring new drugs to the RIGHT patient population: DIsease onhibition score

1:45 p.m. — Keynote Speaker
“Reforming Clinical Trials: How Alternative Trial Designs May Reshape Regulatory Review”

Traditional clinical trial designs are often too cumbersome and expensive to study the efficacy of personalized medicine products and services in sub-populations of patients. Yet there is no consensus on which methods have the most promise to speed trials and lower costs. During her keynote address, Dr. Woodcock will explore which of the latest progressive designs she believes are best suited to demonstrate the efficacy of personalized medicine based on past successes and proposed reforms.

  • Introduction: Steve BMS
  • Keynote: Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
  1. Not just the trial, but the Development Program
  2. knowledge that underpins the program vs Novel Trial Design
  3. +50%  of Trials FAIL at Phase 3 – simulate  best and worse scenarios
  4. Cut off points – affect the result of the Human Trial
  5. Outcome measures for disease have never or rarely, been tested
  6. Murphy’s law operate
  7. robust vs fragile design
  8. DEsign – conduct a seamless, adaptive development program
  9. Trade off – for benefits vs burden of Disease – Functionality vs Longevity – give up life for better functionality when aive
  10. More patient enroll or tril goes longer, treatment gets better and Endpoints needs to be revised
  11. heterogenious progression, fast progression
  12. DIsease heterogeniety — REDUCTION by beter Patient selection – more homogenious to reach similar progression
  13. Natural History: rare diseas vs heterogeneous
  14. Progression not known because longitudinal studies are limited or study is not representative
  15. projection of results of trial may be difict: Pharmacodynamic Markers (efficacy) dificult to reproduce
  16. Trial Design: Phase 1,2,3
  17. Alternatives: “Extended Phase 1 COhort” –>> Approval
  18. Endpoint (cancer): Response rate, Progression Free Survival (PFS) Time
  19. Mechanistics hypothesis, natural history data on non responding patients
  20. N-of -1 looking at disease trajectory
  21. Oncology: “basket” trials with biomarker defined targets across histologic diagnoses : NCI “MATCH” trial
  22. Emergencies: EBOLA trial
  23. DOse-finding can be randomized, adaptive,, include placedbo arm
  24. Serious, Rare or Uncommon DIsease wiht Existing Standard of Care (SOC) vs Placebo
  25. Common Diseaase with SOC
  26. Biomarkers: Predictive of Response: Magnitude of the response not Yes or No — Highest Biomarket Cutoff
  27. RWE – Rare WOrld Evidence
  28. Knowledge tht UNDERLIE – biological knowledge
  29. CUrative therappy with PM – promise is there

2:15 p.m. — Fireside Chat

  • Moderator: Alexander Vadas, Ph.D., Managing Director and Partner, L.E.K. Consulting
  1. Companion Diagnostics
  • Peer M. Schatz, M.B.A., CEO, QIAGEN (15 years around)
  1. PM and experience
  2. Value chain of PM does not work – Diagnostics is 2% of the HealthCare expenses. Reimbursement by COst of Production
  3. 30x smaller then Pharmaceuticals
  4. Standards to evaluate the value of diagnostics
  5. Biomarkers – 60,000 distinctive tests
  6. Benefits of Diagnostics not recognized
  7. HealthCare 2% spent on DIagnostics and Monitoring
  8. Value of information of Molecualr DIagnostics
  9. Lower quality evidence
  10. Reward value of diagnostics – Patients
  11. For LABs  diagnostics is a profit looser
  12. Molecular Diagnostics – new, PM is known in 1999 – AMP launched its Journal
  13. Value based Medicine, systems of rewards is to blame
  14. Reimbursement – Diagnostics and regulatory
  15. 30 Pharma Partnerships
  16. Industry organization
  17. Biopsy to Microbiome
  18. 70% of Cancer care is done at COmmunity Hospitals
  19. Human genomics data doubles annoually
  20. Data needs, 34% in accordance, 70% accordance with diagnosis – CONCORDANCE POOR CROSS GENOMICS OR AXON LABS
  21. PM recognize the value of information DIagnosis, improvemnet in Patient Diagnosis

Reply to interview by Alexander Vadas, Ph.D., Managing Director and Partner, L.E.K. Consulting

  • Education of Pathologists in Genomic Pathology
  • Approval of Companion Diagnostics in China requires infrastructure in regulatory interface with each Country
  • Seamless interaction with Pharma
  • If tests in Pathology are too expensive, LABS will not be able to be profitable
  • NGS – Variance – vs a frontline test Designed for Reimbursability and profitaility

2:45 p.m. — Networking Break

 

 

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LIVE 11/16 8AM – noon The 12th Annual Personalized Medicine Conference, HARVARD MEDICAL SCHOOL, Joseph B. Martin Conference Center, 77 Avenue Louis Pasteur, Boston

Leaders in Pharmaceutical Business intelligence (LPBI) Group

Covering in Real Time using Social Media this Event on

Personalized Medicine

Aviva Lev-Ari, PhD, RN, Founder LPBI Group & Editor-in-Chief

http://pharmaceuticalintelligence.com

Streaming LIVE @ HARVARD MEDICAL SCHOOL,

Joseph B. Martin Conference Center

@pharma_BI

@AVIVA1950

 

November 16

#PMConf

Joseph B. Martin Conference Center
77 Avenue Louis Pasteur
Boston, MA 02115

7:00 a.m. — Registration and Breakfast

8:00 a.m. — Opening Remarks – [Personalized Medicine = PM]

  • Edward Abrahams, Ph.D., President, Personalized Medicine Coalition
  • PM offers a more efficient HealthCare: Gov’t, Academia, research, pharma and Patients collaborations = working together
  • PMC goal – solutions in the field, stacks are high
  • Corporate sponsors: Genetech-Roache, astellas, Intermountain Precision Medicine

8:05 a.m. — The Personalized Medicine Report

  • William S. Dalton, Ph.D., M.D., CEO, M2Gen, Chairman, Personalized Medicine Coalition
  • Five medicines in 2016 are defined as PM
  • Efficiences in Care by Precision Medicine
  • Sequencing, molecular medicine, Genomics in all Medical Record

8:15 a.m. — Keynote Speaker

  • Greg Simon, Executive Director, Cancer Moonshot Task Force
  • 400 people in Washington at the Launch of Moonshot, 7,000 in audio/video attendance around the US
  • Video by all stackholders
  • Joe Bidan, today we are more ready than one year ago – ergency of now – a challenge we are not willing to postpone
  • Example CLL therapeutics today vs 20 years ago
  • Precision Medicine is the technology & Big Dat aand Science and Survivorship; PM is about the Patient
  • TASK FORCE on Cancer: added EPA, Council for the Arts, DoD
  • Double impact – How, Partnerships
  • VA – many Veterans with Cancers – Sequence effort, Watson-IBM, to review sequences from VA – new partnerships: IBM + VA – Walter Reed Initiative
  • VA tishue bank with 20 Companies – new Partnership
  • DoD and VA
  • Private initiatives on Moonshot topics: American Cancer SOciety – does their own, Foundations, Academia,
  • Health Systems, several not one — join together Dana Farbers, Sloan Katering
  • Accessible Medications is very important for Patients
  • The significance of 9 month to start changing the World

8:45 a.m. — Pioneering Precision: Charting a Course for Cutting-Edge Innovations

Many scientists believe innovations in personalized medicine are poised to yield major breakthroughs in coming years, but not all members of the health care system are clear on which research topics have the most potential. The participants in this panel will identify the most encouraging scientific directions for personalized medicine and point to the most promising topics for future research.

  • Moderator: Stephen Eck, M.D., Ph.D., Vice President, Oncology Medical Sciences, Astellas Pharma Global Development
  1. Oncology drugs were prescribed by histology no molecular diagnostics
  2. 2015 – 25% of FDA approved medicines are PM
  3. In 2016 – PM and molecular diagnostics – needs to be a SYSTEM not disjoint solutions
  4. Value for Price, small indications, cost containment
  5. Pricing treatment if several drugs are at use wihtout knowing the realtive contribution of each

 

  • David Altshuler, M.D., Ph.D., Executive Vice President, Global Research and Chief Scientific Officer, Vertex
  1. Prevent and modify the course, Human genetics and somatic genetics in cancer, discovery started with Mandelian, moved to somatic, now use of catelogue for common mutations,
  2. Test existing hypothesis on pathphysiology: LDL and heart disease vs HDL – genetics variance for Heart attack – predictor not modifier
  3. Predicting with Genome in PM, who respond to which medication
  4. New Hypothesis for new Paradigms
  5. Cyctic FIbrosis: Gene found, took a histology based disease CF – to foundational genomics – mutation in gene on cell surface – POTENTIATOR of opening the channel by a small molecule — these small molecule were developed in the last 15 years, follow Patients in Registries – reduction in rate of decline of lung function — Drug – is changing the course of the disease and intervention early
  6. small molecule in combination into cells that have only one copy, in a dish – two medications in Phase II
  7. Personalization is a statement of not ahving yet an effective therapy but it is identification of the Pathway
  8. Common genotype – small groupsidentification
  9. target diseases that will show doubling efficacy, curative therapies, new models: PENETRATION OF LYTHAL DISEASES
  10. Annuity model only in the future the outcome allows measurement – REGISTRIES are very important and need be used for outcomes in the future
  • Michael Panzara, M.D., Head of Neurology Franchise, WAVE Life Sciences
  1. Synthesis of nucleaic acid focusing on neurology – STEREOCHEMISTRY
  2. modify sulfor – phorphate new modification – mixure of isomeres stereo molecules – systhesize nuclaic acids
  3. design nucleic acids
  4. Pipeline: CNS, MUSCLES, EYE, Liver, Skin, GI
  5. HD – Huntington’s Disease [Mutant HTT] – cognitive decline
  6. WAVE approach: target only the mutant HTT while leaving wild-type intact – disease modifier
  7. Targeted therpy  mHTT RNA – transcript of mutant, SNP
  8. Complement activation assay vs HTT transcript – wtHTT: Patients with long CAG repeat associated with T isoform will be selected for trial.
  9. DMD – Exom Skipping,vs Dysfunctional Splicing: Dystrophin (increase production) vs Vinculin
  • Barbara Weber, M.D., Interim Chief Medical Officer, Neon Therapeutics
  1. Neoantigen biology: Native antigens (targets are expressed in both tumor and normal cells vs Neoantigens (Targets are specific to each Tumor)

Checkpoint inhibitor drug class: Neoantigen-based Therapies may expand ACTIVITY of Checkpoint Inhibitor – Nature 2013

  1. Ipilimumab in melanoma (NEJM 2014
  2. Pembrolizumab in NSCLC, Science 2015
  3. Pembrolizumab in colonorectal cancer, NEJM 2015

NEON: Personalized Neoantigens

  • personalized CAR-T
  • personalized Vaccines
  • Epitope Librart – Epitope selection
  • NT-001 PM Clinical Trial: combination of Neoantigen Vaccine wiht Checkpoint Inhibitor – PD-1
  • Monitor Immune response after insertion of neoantigents

9:45 a.m. — Networking Break

10:15 a.m. — Money Talks: The Future of Investment in Personalized Medicinemolecular diagnostics

Innovation requires investment. During this discussion, a panel of diverse investors will illuminate the most promising business opportunities for advancing personalized medicine, focusing on both macro and micro environments while also discussing the barriers to investment and potential solutions for removing them.

  • Moderator: Edward Winnick, Editor in Chief, GenomeWeb
  1. Beyond Ocology
  • Alexis Borisy, M.S., Partner, Third Rock Ventures
  1. What excite now – Breakthrough therapies in the RIGHT Patient populations
  2. FOundation Medicine – 1,000 Patients treated with FOundationOne across diagnostics and drug – genomics and disease and therapy in One
  3. New Diagnostics company creation – is very hard, not many, only if it is an extraordinary compelling idea, If FDA approves and the Payers do not endorse
  4. Therapeutics – have a solution, nucleaic based drug, gene editing, Payers are iterested, FDA is interested vs historical standdards, translational medicine – drug will drive diagnostics
  5. Reimbursement, Capital mobilization, new molecular diagnostics, challenges: Technical risk Clinical Risk
  6. Cut off for AstraZeneca vs BMS are different
  7. number tumor cells vs penetration of tumor cells vs infiltration of immune cells
  8. Merck  has a different strategy – precise indicator of I/O agents: Single vs. multiple combinations
  9. WHen population of patients is identified well: understand response rate go for 80% response rate inside this population identified
  10. beyond oncology MI and CVD, cardiomyopathy – genetic mutations, familial vs sporadic,
  11. Immunology: Inflammation, innume conditions, understand what is the microtrend in subpopulations
  12. Infectious diseases: organisms given narrow cast antibiotics and trade-offs PM antibiotics is a fundamental challengeresistence to antibiotics
  13. Diagnostics in Cancer – a small fraction vs the cost of Oncology drugs
  14. Longevity drive costs,
  15. Physician diagnostics is critical
  • Vamil Divan, M.D., M.B.A., Senior Research Analyst, Credit Suisse
  1. BMS is a leader in immunotherapy and Roache as well
  • Ryan Lindquist, M.B.A., Director, Investment Banking, Leerink Partners
  1. Diagnostics and Precision Medicine
  2. Diagnostics – reimbursement
  3. winners and loosers in Diagnostics
  4. Performance, cost reduction, Payers have rules of engagement
  5. Differentiation of generic drugs: COST OF TREATMENT FO RDRUG $30,000 COST RUNNING TEST $200, Pharma was willing to cut the cost creatively
  6. Drug pricing a point in investing in Biotech
  7. Neuroscience: many drugs are not effication – swab of mouth saliva allows to determine which pshychtropic drug will work for which patient on psych therapies
  8. Data companies with Regulatories
  9. Payers: Kaiser – has Outcome data and cost data
  10. Healthcare system: Payers and Delivery is separate
  11. Patient advocacy on reimbursement is critical and DOctor education on new Diagnostics at Academic Centers

Questions from the audience:

  1. Therapeutics: IP and reimbursement is straight forward
  2. Diagnostics: No IP therefore reimburcement more complex
  3. Data companies
  4. Foundation Medicine: Reimbursement is a challenge – what should the Payer do vs the DIagnostics companies, regulatory, American Pathology Guidelines
  5. Engagement Providers – what is their role?

 

11:15 a.m. — You + 999,999: How a One Million Person Cohort Can Pave the Way for Personalized Care

  • Introduction: Paolo Narvaez, Ph.D., Senior Principal Engineer, Director of Engineering, Intel Corporation
    1. How to use technology for PM – Intel Life Sciences

     

  • Keynote: Eric Dishman, Director, All of Us Research Program, National Institutes of Health
  1. PM Precision vs Personalized Health
  2. All of Us Research Program, National Institutes of Health funded – is PM 
  3. Framework Like the Framingham Study
  4. One million Patients,  – longitudinal: lifestyle, genes, environmental
  5. HUGE data not a study for one disease,  – accelerate Science
  6. Infectious,Neuro, mental, Heart& Lung, Musculo-Skeletal
  • DIrect Volunteer
  • Health Provider Org
  • Enroll & Consent
  • Surveys Journals
  • Baseline Measrement
  • Mission to accelerate Knowledge Turns & Breakthroughs
  • Participation – Transformational Approach
  • 1,000 vs One million
  • Diversity: People , geography health Status,
  • Data Types: Genomics, Claims – Data Access
  • High schools, community colleges
  • User-Centered approach
  • gathering Input
  • Personas: ready to go, determined, too much Govenment, No time, Suspitious
  • Platform Innovation Mindset & Process –>> Innovation Fannel
  • Baseline Physical Evaluation not a Physical Exam
  • PPI – Participant Provided Information
  • Mayo Clinic: Biospecimens: Blood and Urine

Research ROADMAP Workshops

  • Near Term
  • Mid Term
  • Long Term

Kind of Attendees: experts network (50 organization, co-funders, advoccy

Biobank – 35Million vials

IT infrafaces–>> security,  Enrollement 1-800-

New Eng PM Consortium, Bosotn, MA

 

 

 

12:00 p.m. — Luncheon

 

 

 

#PMConf

SOURCE

http://www.personalizedmedicinecoalition.org/Conference/November_16_Program

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LIVE 9/21 8AM to 10:55 AM Expoloring the Versatility of CRISPR/Cas9 at CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

http://www.discoveryontarget.com/

http://www.discoveryontarget.com/crispr-therapies/

Leaders in Pharmaceutical Business Intelligence (LPBI) Group is a

Media Partner of CHI for CHI’s 14th Annual Discovery on Targettaking place September 19 – 22, 2016 in Boston.

In Attendance, streaming LIVE using Social Media

Aviva Lev-Ari, PhD, RN

Editor-in-Chief

http://pharmaceuticalintelligence.com

#BostonDOT16

@BostonDOT

 

COMMENTS BY Stephen J Williams, PhD

EXPLORING THE VERSATILITY OF CRISPR/Cas9

 

8:00 Chairperson’s Opening Remarks

TJ Cradick , Ph.D., Head of Genome Editing, CRISPR Therapeutics

 

@CRISPRTX

 

8:10 Functional Genomics Using CRISPR-Cas9: Technology and Applications

Neville Sanjana, Ph.D., Core Faculty Member, New York Genome Center and Assistant Professor, Department of Biology & Center for Genomics and Systems Biology, New York University

 

CRISPR Cas9 is easier to target to multiple genomic loci; RNA specifies DNA targeting; with zinc finger nucleases or TALEEN in the protein specifies DNA targeting

 

  • This feature of crisper allows you to make a quick big and cheap array of a GENOME SCALE Crisper Knock out (GeCKO) screening library
  • How do you scale up the sgRNA for whole genome?; for all genes in RefSeq, identify consitutive exons using RNA-sequencing data from 16 primary human tissue (alot of genes end with ‘gg’) changing the bases on 3’ side negates crisper system but changing on 5’ then crisper works fine
  • Rank sequences to be specific for target
  • Cloned array into lentiviral and put in selectable markers
  • GeCKO displays high consistency betweens reagents for the same gene versus siRNA; GeCKO has high screening sensitivity
  • 98% of genome is noncoding so what about making a library for intronic regions (miRNA, promoter regions?)
  • So you design the sgRNA library by taking 100kb of gene-adjacent regions
  • They looked at CUL3; (data will soon be published in Science)
  • Do a transcription CHIP to verify the lack of binding of transcription factor of interest
  • Can also target histone marks on promoter and enhancer elements
  • NYU wants to explore this noncoding screens
  • sanjanalab.org

 

@nyuniversity

 

8:40 Therapeutic Gene Editing With CRISPR/Cas9

TJ Cradick , Ph.D., Head of Genome Editing, CRISPR Therapeutics

 

NEHJ is down and dirty repair of single nonhomologous end but when have two breaks the NEHJ repair can introduce the inversions or deletions

 

    • High-throughput screens are fine but can limit your view of genomic context; genome searches pick unique sites so use bioinformatic programs  to design specific guide Rna
    • Bioinformatic directed, genome wide, functional screens
    • Compared COSMID and CCTOP; 320 COSMID off-target sites, 333 CCtop off target
    • Young lab GUIDESeq program genome wide assay useful to design guides
    • If shorten guide may improve specificity; also sometime better sensitivity if lengthen guide

 

  • Manufacturing of autologous gene corrected product ex vivo gene correction (Vertex, Bayer, are partners in this)

 

 

They need to use a clones from multiple microarrays before using the GUidESeq but GUIDEseq is better for REMOVING the off targets than actually producing the sgRNA library you want (seems the methods for library development are not fully advanced to do this)

 

The score sometimes for the sgRNA design programs do not always give the best result because some sgRNAs are genome context dependent

9:10 Towards Combinatorial Drug Discovery: Mining Heterogeneous Phenotypes from Large Scale RNAi/Drug Perturbations

Arvind Rao, Ph.D., Assistant Professor, Department of Bioinformatics and Computational Biology, The University of Texas MD Anderson Cancer Center

 

Bioinformatics in CRISPR screens:  they looked at image analysis of light microscopy of breast cancer cells and looked for phenotypic changes

 

  • Then they modeled in a small pilot and then used the algorithm for 20,000 images (made morphometric measurements)
  • Can formulate training statistical algorithms to make a decision tree how you classify data points
  • Although their algorithms worked well there was also human input from scientists

Aggregate ranking of hits programs available on web like LINKS

 

@MDAndersonNews

 

10:25 CRISPR in Stem Cell Models of Eye Disease

Alexander Bassuk, M.D., Ph.D., Associate Professor of Pediatrics, Department of Molecular and Cellular Biology, University of Iowa

 

Blind athlete Michael Stone, biathlete, had eye disease since teenager helped fund and start the clinical trial for Starbardt disease; had one bad copy of ABCA4, heterozygous (inheritable in Ahkenazi Jewish) – a recessive inheritable mutation with juvenile macular degeneration

  • Also had another male in family with disease but he had another mutation in the RPGR gene
  • December 2015 paper Precision Medicine: Genetic Repair of retinitis pigmentosa in patient derived stem cells
  • They were able to correct the iPSCs in the RPGR gene derived from patient however low efficiency of repair, scarless repair, leaves changes in DNA, need clinical grade iPSCs, and need a humanized model of RPGR

@uiowa

10:55 CRISPR in Mouse Models of Eye Disease

Vinit Mahajan, M.D., Ph.D., Assistant Professor of Ophthalmology and Visual Sciences, University of Iowa College of Medicine

  • degeneration of the retina will see brown spots, the macula will often be preserved but retinal cells damaged but with RPGR have problems with peripheral vision, retinitis pigmentosa get tunnel vision with no peripheral vision (a mouse model of PDE6 Knockout recapitulates this phenotype)
  • the PDE6 is linked to the rhodopsin GTP pathway
  • rd1 -/- mouse has something that looks like retinal pigmentosa; has mutant PDE6; is actually a nonsense mutation in rd1 so they tried a crisper to fix in mice
  • with crisper fix of rd1 nonsense mutation the optic nerve looked comparible to normal and the retina structure restored
  • photoreceptors layers- some recovery but not complete
  • sequence results show the DNA is a mosaic so not correcting 100% but only 35% but stil leads to a phenotypic recovery; NHEJ was about 12% to 25% with large deletions
  • histology is restored in crspr repaired mice
  • CRSPR off target effects: WGS and analyze for variants SNV/indels, also looked at on target and off target regions; there were no off target SNVs indels while variants that did not pass quality control screening not a single SNV
  • Rhodopsin mutation accounts for a large % of patients (RhoD190N)
  • injection of gene therapy vectors: AAV vector carrying CRSPR and cas9 repair templates

CAPN mouse models

  • family in Iowa have dominant mutation in CAPN5; retinal degenerates
  • used CRSPR to generate mouse model with mutation in CAPN5 similar to family mutation
  • compared to other transgenic methods CRSPR is faster to produce a mouse model

To Follow LIVE CONFERENCE COVERAGE PLEASE FOLLOW ON TWITTER USING

Meeting #: #BostonDOT16

Meeting @: @BostonDOT

 

Overall good meeting #s:

#personalizedmedicine

#innovation

#cancer

#immunology

#immunooncology

#pharmanews

#CRSPR

#geneediting

#crisper

#biotech

 

AND FOLLOW these @

@pharma_BI

@AVIVA_1950

@BiotechNews

@CHI

@FierceBiotech

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LPBI Group’s Channels for e-Marketing of Biotech Conferences involve PreConference, During the Conference, Post Conference tasks, they are:

Author: Aviva Lev-Ari, PhD, RN

 

UPDATED on 4/5/2018

 

 

 

Channels for e-Marketing of Biotech Conferences

  • Our Journal +1,031,000 eReaders

http://pharmaceuticalintelligence.com

  • Aviva’s – +6000 BIOTECH followers on LinkedIn

http://www.linkedin.com/in/avivalevari

  • LPBI Group’s FaceBook Page

http://www.facebook.com/LeadersInPharmaceuticalBusinessIntelligence

  • LPBI Group’s Twitter Account

http://twitter.com/pharma_BI

  • Aviva Manages three Groups on LinkedIn

Cardiovascular Biotech & Pharma UK & US Networking Group

906 members

https://www.linkedin.com/groups/4357927

 

Leaders in Pharmaceutical Business Intelligence

336 members

https://www.linkedin.com/groups/4346921

 

Innovation in Israel

172 members

https://www.linkedin.com/groups/2987122

  • LPBI Group’s Company’s Page on LinedIn

https://www.linkedin.com/company/9325543?trk=tyah&trkInfo=clickedVertical%3Acompany%2CclickedEntityId%3A9325543%2Cidx%3A1-1-1%2CtarId%3A1439226813927%2Ctas%3ALeaders%20in%20Pharmaceutica

 

Tasks done PreConference, During the Conference, Post Conference include:

What Leaders in Pharmaceutical Business Intelligence (LPBI) Group, Boston can provide Conference Organizers ?

Pre-Conference

  1. Co-marketing of Conferences six month prior to Conference date
  2. Conference Presentation Coverage in Real Time for Scientific Media and Business Media
  3. Conference Presentation Content is published in the Open Access Online Scientific Journal
  4. Social media posting (Twitter and LinkedIn groups) once a month before the event
  5. Pre-conference blog posting of the event
  6. Conference listing/web banner on our website homepage
  7. E-blast to our membership/viewership (with Conference Organizer’s discount code) regarding their conference at a preplanned Time Table presented to us.

 During conference

  1. Each Presentation following publication in the Journal is posted on Social media
  2. Generation of electronic Conference Proceedings [LPBI’s e-Proceedings]
  3. “LPBI’s e-Proceedings on DVD” made available for sale by Conference Organizers.
  4. PR Campaign for a Conference event include the following:
  5.  Publish the Event in the Journal, options for re-blogging — Using a message provided by the Event Organizer
  6.  Connect the Event Announcement to Facebook, Twitter and LPBI’s 56 Groups on LinkedIn — with repeats
  7.  Targeted e-mails to potential Speakers and potential Attendees in Aviva’s 6,000 LinkedIn first level connections in Biotech
  8.  Per Conference Organizer’s specification of locations, industries and function in the organization message broadcasting to batches of 100 e-mails at a time.
  9.  Option to Publish a JOINT Announcement, including the message provided by the Conference Organizers and a message by Leaders of Pharmaceutical Business Intelligence (LPBI) Group, involve private advertisement in channels

Post Conference

  1. Analysis of journal engagement of posts for each talk covered
  2. Social media analysis of conference including Twitter Analysis of meeting including
  3. Twitter engagement statistics of conference
  4. TweetReach analysis of live tweets from the conference
  5. WordCloud of all tweets to identify most engaged and tweeted topics
  6. Post-conference blog posting/report/summary of the event
  7. Twitter and web engagement statistics of live posts and tweets from conference talks 
  8. e-Proceeding of the Conference shared with Conference Organizers for their dissemination to all attendees

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Individualizing Medicine Conference, October 5-6, 2016, Rochester, MN, Hosted by Mayo Clinic Center for Individualized Medicine, Supported by the Satter Foundation held at MAYO CIVIC CENTER 30 Civic Center Drive SE, Rochester, MN

Reporter: Aviva Lev-Ari, PhD, RN

 

Individualizing Medicine 2016: Advancing Care Through Genomics focuses on how to translate the promise of genomic medicine to your practice. Expert speakers, focused breakout sessions, case studies and a poster session provide opportunities to discover and discuss emerging topics in applied genomics.

REGISTRATION

http://individualizingmedicineconference.mayo.edu/

WEDNESDAY, OCTOBER 5, 2016

  • 7:00 AMRegistration Desk Opens, Continental Breakfast Available
  • 8:00 AM –8:30 AMIntroductions, Welcome, Tour of conference app

    Cathy Wurzer, Minnesota Public Radio (Moderator)
    Keith Stewart, M.B., Ch.B., Mayo Clinic

  • 8:30 AM –9:15 AMShaping the Future of Precision Oncology and the Role of the Patient

    Kathy Giusti, M.B.A., Multiple Myeloma Research Foundation

  • 9:15 AM –10:00 AMTowards Precision Medicine

    Euan A. Ashley, M.R.C.P., D.Phil., Stanford

  • 10:00 AM –10:30 AMBreak with Refreshments
  • 10:30 AM –11:15 AMImproving Outcomes Through Clinical Implementation of Pharmacogenetics

    Julie A. Johnson, Pharm.D., University of Florida College of Pharmacy

  • 11:15 AM –12:00 PMTitle To Be Announced

    Kathy Hudson, Ph.D., National Institutes of Health (NIH)

  • 12:00 PM –1:00 PMLunch & Presentation

    Teri Manolio, M.D., Ph.D., National Human Genome Research Institute, National Institutes of Health

  • 1:00 PM –2:30 PMConcurrent Session 1A Pharmacogenomics in Your Practice

  • 1:00 PM –2:30 PMConcurrent Session 1B The New Frontier – Next Generation Sequencing Based Microbial Diagnostics

  • 1:00 PM –2:30 PMConcurrent Session 1C Novel Genetic Applications with Potential to Transform Healthcare

  • 1:00 PM –2:30 PMConcurrent Session 1D To Be Announced
  • 2:30 PM –2:45 PMBreak with Refreshments
  • 2:45 PM –4:15 PMConcurrent Session 2A Utility of Genetic Testing: Promises and Pitfalls

  • 2:45 PM –4:15 PMConcurrent Session 2B Early Career Investigators in Precision MedicineSupported by the Brandt Family Scholars Fund

  • 2:45 PM –4:15 PMConcurrent Session 2C Functional Studies to Disambiguate Variants of Uncertain Significance in Clinical Testing

  • 2:45 PM –4:15 PMConcurrent Session 2D Pharmacogenomics Guidelines: International and US Perspectives

  • 4:15 PM –4:30 PMBreak
  • 4:30 PM –5:15 PMTo Use or Not to Use:  Clinical Application of Genetic Risk Scores in Age-Related Macular Degeneration

    Jonathan L. Haines, Ph.D., Case Western Reserve University

  • 5:15 PM –7:15 PMPoster/Welcome Reception, Light Hors d’ Oeuvres and Beverages Available

THURSDAY, OCTOBER 6, 2016

  • 7:00 AMRegistration Desk Opens, Continental Breakfast Available
  • 8:00 AM –8:15 AMWelcome
  • 8:15 AM –9:00 AMThe 100,000 Genomes Project

    Mark Caulfield, FMedSci, Genomics England

  • 9:00 AM –9:45 AMGenomes, Autism, and More Genomes

    Stephen W. Scherer, Ph.D., D.Sc., University of Toronto McLaughlin Centre & Centre for Applied Genomics at SickKids

  • 9:45 AM –10:15 AMBreak with Refreshments
  • 10:15 AM –11:45 AMConcurrent Session 3A Clinical Genomics Management: From the Patient Perspective

  • 10:15 AM –11:45 AMConcurrent Session 3B Applying Pharmacogenomics: Changing Practice, Changing Lives

  • 10:15 AM –11:45 AMConcurrent Session 3C Transforming Patient Care with Microbiome Research

  • 10:15 AM –11:45 AMConcurrent Session 3D Advances in Genomic Laboratory Medicine

  • 11:45 AM –12:45 PMLunch
  • 12:45 PM –2:15 PMConcurrent Session 4A Genomic Case Review with the Experts

  • 12:45 PM –2:15 PMConcurrent Session 4B Predictive and Consumer Genomics

  • 12:45 PM –2:15 PMConcurrent Session 4C Cancer and Precision Medicine

  • 12:45 PM –2:15 PMConcurrent Session 4D Epigenomics to Therapy: Emerging Agents

  • 2:15 PM –2:30 PMBreak with Refreshments
  • 2:30 PM –3:15 PMMarshall Summar, M.D.
  • 3:15 PM –4:00 PMSystems Medicine and P4 Medicine: Transforming Healthcare Through Wellness

    LeRoy Hood, M.D., Ph.D.

  • 4:00 PM –4:45 PMConsumer Genomics Panel Discussion

    LeRoy Hood, M.D., Ph.D.
    Justin Kao
    Jill Hagenkord, M.D.
    Additional Panel Members to be Announced

  • 4:45 PM –5:00 PMClosing/Summary
  • Stay for our Friday Workshops! Click here for more information.

FRIDAY, OCTOBER 7, 2016

  • 8:00 AM –5:00 PMGeneticon Upper Midwest Clinical Genetics
  • 7:00 AM –8:00 AMContinental Breakfast
  • 7:00 AM –8:00 AMEpigenomics and Microbiome Poster Session

    Click here for abstract submission information.

  • 8:00 AM –12:30 PMWorkshop Sessions with a Break
  • 8:00 AM –12:30 PMBio-Marker Discovery TIGER-Bio: The Integrated Genome-analysis Enabled Response Biomarker Program

  • 8:00 AM –12:30 PMEpigenomics Epigenomics: Bridging the Translational Gap
  • 8:00 AM –12:30 PMMicrobiome All You Need Is Bugs

  • 8:00 AM –12:30 PMPharmacogenomics (PGx) Applying PGx with the Clinical Pharmacogenetics Implementation Consortium (CPIC) and the Dutch Pharmacogenetic Work Group (DPWG)

SOURCE

http://individualizingmedicineconference.mayo.edu/schedule/

http://individualizingmedicineconference.mayo.edu/

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