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Archive for the ‘Drug Development Process’ Category

Ido Sagi – PhD Student @HUJI, 2017 Kaye Innovation Award winner for leading research that yielded the first successful isolation and maintenance of haploid embryonic stem cells in humans.

Posted in Behavioral Genetics, Bio Instrumentation in Experimental Life Sciences Research, Biological Networks, Gene Regulation and Evolution, Cell Biology, Cell Processing System in Cell Therapy Process Development, Chemical Genetics, Circulating Progenitor Cells, Diagnostics and Lab Tests, Disease Biology, Drug Discovery Chemistry, Epigenetics and Environmental Factors, Gene Regulation and Evolution, Genetics & Innovations in Treatment, Genetics & Pharmaceutical, Genome Biology, Genomic Testing: Methodology for Diagnosis, Medical and Population Genetics, Personalized and Precision Medicine & Genomic Research, Scientist: Career considerations, Stem Cells for Regenerative Medicine on June 29, 2017| Leave a Comment »

Ido Sagi – PhD Student @HUJI, 2017 Kaye Innovation Award winner for leading research that yielded the first successful isolation and maintenance of haploid embryonic stem cells in humans.

Reporter: Aviva Lev-Ari, PhD, RN

 

Ido Sagi – PhD Student, Silberman Institute of Life Sciences, HUJI, Israel

  • Ido Sagi’s research focuses on studying genetic and epigenetic phenomena in human pluripotent stem cells, and his work has been published in leading scientific journals, including Nature, Nature Genetics and Cell Stem Cell.
  • Ido Sagi received BSc summa cum laude in Life Sciences from the Hebrew University, and currently pursues a PhD at the laboratory of Prof. Nissim Benvenisty at the university’s Department of Genetics in the Alexander Silberman Institute of Life Sciences.

The Kaye Innovation Awards at the Hebrew University of Jerusalem have been awarded annually since 1994. Isaac Kaye of England, a prominent industrialist in the pharmaceutical industry, established the awards to encourage faculty, staff and students of the Hebrew University to develop innovative methods and inventions with good commercial potential, which will benefit the university and society.

Publications – Ido Sagi

Comparable frequencies of coding mutations and loss of imprinting in human pluripotent cells derived by nuclear transfer and defined factors.
Cell Stem Cell 2014 Nov 6;15(5):634-42. Epub 2014 Nov 6.
The New York Stem Cell Foundation Research Institute, New York, NY 10032, USA; Naomi Berrie Diabetes Center & Department of Pediatrics, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA. Electronic address:

November 2014

 

Download Full Paper
Human oocytes reprogram adult somatic nuclei of a type 1 diabetic to diploid pluripotent stem cells.
Nature 2014 Jun 28;510(7506):533-6. Epub 2014 Apr 28.
The New York Stem Cell Foundation Research Institute, New York, New York 10032, USA.

June 2014

 

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Stem cells: Aspiring to naivety.
Nature 2016 12 30;540(7632):211-212. Epub 2016 Nov 30.
The Azrieli Center for Stem Cells and Genetic Research, Department of Genetics, Silberman Institute of Life Sciences, The Hebrew University of Jerusalem, Jerusalem 91904, Israel.
November 2016

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SOURCE

Other related articles on Genetic and Epigenetic phenomena in human pluripotent stem cells published by LPBI Group can be found in the following e-Books on Amazon.com

e-Books in Medicine

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    Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics

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    Genomics Orientations for Personalized Medicine (Frontiers in Genomics Research Book 1)

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    Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation: The Art of Scientific & Medical Curation

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‘Landmark FDA approval bolsters personalized medicine’ by Edward Abrahams, PhD, President, PMC

Posted in Cancer and Current Therapeutics, Drug Discovery Chemistry, FDA Regulatory Affairs, Genome Biology, Personalized and Precision Medicine & Genomic Research, Variation in human protein-coding regions on June 21, 2017| Leave a Comment »

Personalized Medicine been Positively affected by FDA Drug Approval Record

Reporter: Aviva Lev-Ari, PhD, RN

FDA to Clear Path for Drugs Aimed at Cancer-Causing Genes

By Anna Edney and Michelle Cortez

June 20, 2017, 10:41 AM EDT June 20, 2017, 3:02 PM EDT

https://www.bloomberg.com/news/articles/2017-06-20/fda-moves-to-clear-path-for-drugs-aimed-at-cancer-causing-genes

 

 

‘Landmark FDA approval bolsters personalized medicine’

PMC – An Op-Ed in STAT News

by Edward Abrahams

June 21, 2017

Our understanding of cancer has been morphing from a tissue-specific disease — think lung cancer or breast cancer — to a disease characterized more by specific genes or biomarkers than by location. A recent FDA decision underscores that transition and further opens the door to personalized medicine.

Two years ago, the director of the FDA’s Office of Hematology and Oncology Products told the Associated Press that there was no precedent for the agency to approve a drug aimed at treating tumors that generate a specific biomarker no matter where the cancer is in the body. Such a drug had long been seen as the epitome of personalized medicine. But with the rapid pace of progress in the field, director Dr. Richard Pazdur said, such an approval could one day be possible.

That day has arrived.

In a milestone decision for personalized medicine, the FDA approved Merck’s pembrolizumab (Keytruda) late last month for the treatment of tumors that express one of two biomarkers regardless of where in the body the tumors are located. The decision marks the first time FDA has approved a cancer drug for an indication based on the expression of specific biomarkers rather than the tumor’s location in the body.

Keytruda is designed to help the immune system recognize and destroy cancer cells by targeting a specific cellular pathway. The FDA notes that the two biomarkers — microsatellite instability-high (MSI-H) and mismatch repair deficient (dMMR) — affect the proper repair of DNA inside cells.

The approval represents an important first for the field of personalized medicine, which anticipates an era in which physicians use molecular tests to classify different forms of cancer based on the biomarkers they express, then choose the right treatment for it. In contrast to standard cancer treatments, which are given to large populations of patients even though only a fraction of them will benefit, Keytruda was approved only for the 4 percent of cancer patients whose tumors exhibit MSI-H or dMMR mutations. That may help the health system save money by focusing resources only on patients who are likely to benefit from Keytruda.

Such “personalized” strategies now dominate the landscape for cancer drug development. Personalized medicines account for nearly 1 of every 4 FDA approvals from 2014 to 2016, and the Tufts Center for the Study of Drug Development estimates that more than 70 percent of cancer drugs now in development are personalized medicines.

While this is encouraging, the U.S. research, regulatory, and reimbursement systems aren’t aligned to stimulate the development of personalized medicines, and may even deter progress.

The Trump administration’s proposal to cut biomedical research spending at the National Institutes of Health by 18 percent in fiscal year 2018, for example, would undermine its ability to fund more studies like the National Cancer Institute’s Molecular Analysis for Therapy Choice (MATCH) trial, which is designed to test targeted therapies across tumor types.

While the regulatory landscape for these targeted medicines is clear, the path to market for the molecular tests that do the targeting is not. That uncertainty continues to stifle investment in the innovative tests that make personalized medicine possible. The result is a clinical environment in which the patients who could benefit from personalized medicines are often never identified because the necessary tests aren’t available to them.

Finally, increasing pressure on pharmaceutical and diagnostic companies to decrease prices without considering their value to individual patients and the health system could also deter investment in innovative solutions that address unmet medical needs, particularly for smaller patient populations.

Confronted with unprecedented opportunities in personalized medicine, policymakers would do well to ensure that our research, regulatory, and reimbursement systems facilitate the development of and access to these promising new therapies. Only then can we ensure that Keytruda’s groundbreaking approval represents the beginning of a new era that promises better health and a more cost-effective health system.

Edward Abrahams, Ph.D., is president of the Personalized Medicine Coalition.

 

 

 

SOURCE

From: <cwells@personalizedmedicinecoalition.org>

Date: Wednesday, June 21, 2017 at 1:38 PM

To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: PMC in STAT: “Landmark FDA Approval Bolsters Personalized Medicine”

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Pharmacotyping Pancreatic Cancer Patients in the Future: Two Approaches – ORGANOIDS by David Tuveson and Hans Clevers and/or MICRODOSING Devices by Robert Langer

Posted in 3D Printing for Medical Application, CANCER BIOLOGY & Innovations in Cancer Therapy, Cancer Genomics, Circulating Tumor Cells (CTC), Drug Delivery Platform Technology, Drug Development Process, Drug Development using MultiOrgan Chip, Drug Development/Formulation using 3D Printing, Genomic Expression, Genomic Testing: Methodology for Diagnosis, Genomics Pharmacy, Molecular Genetics & Pharmaceutical, Organoids, Pancreatic cancer, Patient-centered Medicine, Personalized and Precision Medicine & Genomic Research, Tissue Engineering and Regenerative Medicine on April 27, 2017| Leave a Comment »

Pharmacotyping Pancreatic Cancer Patients in the Future: Two Approaches – ORGANOIDS by David Tuveson and Hans Clevers and/or MICRODOSING Devices by Robert Langer

Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED on 4/5/2018

Featured video: Magical Bob

A fascination with magic leads Institute Professor Robert Langer to solve world problems using the marvels of chemical engineering.Watch Video

MIT News Office
March 27, 2018

http://news.mit.edu/2018/featured-video-magical-bob-langer-0327

 

This curation provides the resources for edification on Pharmacotyping Pancreatic Cancer Patients in the Future

 

  • Professor Hans Clevers at Clevers Group, Hubrecht University

https://www.hubrecht.eu/onderzoekers/clevers-group/

  • Prof. Robert Langer, MIT

http://web.mit.edu/langerlab/langer.html

Langer’s articles on Drug Delivery

https://scholar.google.com/scholar?q=Langer+on+Drug+Delivery&hl=en&as_sdt=0&as_vis=1&oi=scholart&sa=X&ved=0ahUKEwixsd2w88TTAhVG4iYKHRaIAvEQgQMIJDAA

  • Professor David Tuveson, Director of the Cancer Center at Cold Spring Harbor Laboratory
A MODEL FOR LIFE – Waging war against pancreatic cancer: an interview with David Tuveson
David Tuveson
Disease Models & Mechanisms 2017 10: 353-357; doi: 10.1242/dmm.029975

organoids, which I know you’re pretty involved in with Hans Clevers. What are your plans for organoids of pancreatic cancer?

Organoids are a really terrific model of a patient’s tumour that you generate from tissue that is either removed at the time of surgery or when they get a small needle biopsy. Culturing the tissue and observing an outgrowth of it is usually successful and when you have the cells, you can perform molecular diagnostics of any type. With a patient-derived organoid, you can sequence the exome and the RNA, and you can perform drug testing, which I call ‘pharmacotyping’, where you’re evaluating compounds that by themselves or in combination show potency against the cells. A major goal of our lab is to work towards being able to use organoids to choose therapies that will work for an individual patient – personalized medicine.

Organoids could be made moot by implantable microdevices for drug delivery into tumors, developed by Bob Langer. These devices are the size of a pencil lead and contain reservoirs that release microdoses of different drugs; the device can be injected into the tumor to deliver drugs, and can then be carefully dissected out and analyzed to gain insight into the sensitivity of cancer cells to different anticancer agents. Bob and I are kind of engaged in a friendly contest to see whether organoids or microdosing devices are going to come out on top. I suspect that both approaches will be important for pharmacotyping cancer patients in the future.

From the science side, we use organoids to discover things about pancreatic cancer. They’re great models, probably the best that I know of to rapidly discover new things about cancer because you can grow normal tissue as well as malignant tissue. So, from the same patient you can do a comparison easily to find out what’s different in the tumor. Organoids are crazy interesting, and when I see other people in the pancreatic cancer field I tell them, you should stop what you’re doing and work on these because it’s the faster way of studying this disease.

SOURCE
http://dmm.biologists.org/content/10/4/353

Other related articles on Pancreatic Cancer and Drug Delivery published in this Open Access Online Scientific Journal include the following:

 

Pancreatic Cancer: Articles of Note @PharmaceuticalIntelligence.com

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/05/26/pancreatic-cancer-articles-of-note-pharmaceuticalintelligence-com/

Keyword Search: “Pancreatic Cancer” – 275 Article Titles

https://pharmaceuticalintelligence.com/wp-admin/edit.php?s=Pancreatic+Cancer&post_status=all&post_type=post&action=-1&m=0&cat=0&paged=1&action2=-1

Keyword Search: Drug Delivery: 542 Articles Titles

https://pharmaceuticalintelligence.com/wp-admin/edit.php?s=Drug+Delivery&post_status=all&post_type=post&action=-1&m=0&cat=0&paged=1&action2=-1

Keyword Search: Personalized Medicine: 597 Article Titles

https://pharmaceuticalintelligence.com/wp-admin/edit.php?s=Personalized+Medicine&post_status=all&post_type=post&action=-1&m=0&cat=0&paged=1&action2=-1

  • Cancer Biology & Genomics for Disease Diagnosis, on Amazon since 8/11/2015

http://www.amazon.com/dp/B013RVYR2K

 

 

VOLUME TWO WILL BE AVAILABLE ON AMAZON.COM ON MAY 1, 2017

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Exosomes: Natural Carriers for siRNA Delivery using extracellular vesicles through endocytic pathway.

Posted in Drug Delivery Platform Technology, Drug Development Process, Drug Discovery Chemistry, Exosomes: Natural Carriers for siRNA Delivery on April 24, 2017| Leave a Comment »

Exosomes: Natural Carriers for siRNA Delivery using extracellular vesicles through endocytic pathway.

Reporter: Aviva Lev-Ari, PhD, RN

 

Exosomes: Natural Carriers for siRNA Delivery

Author(s): Lalit Kumar, Shivani Verma, Bhuvaneshwar Vaidya, Vivek Gupta.

Abstract:

Various cells of the human physiological system have the capability to release extracellular vesicles (EVs) involved in intercellular transport of proteins and nucleic acids. Exosomes are a subtype of extracellular vesicles having their origin through endocytic pathway. While being involved in intercellular transport of macromolecules, exosomes, due to their presence in several body fluids, can also be utilized as a system to commute RNA molecules and proteins in the body. Recent advances in gene therapy have provided a new outlook in disease therapeutics by modulation of gene expression using oligonucleotide based approach and exosomes have been reported a potential carrier for nucleic acid based therapeutic moieties. In recent years, small interfering RNA (siRNA) has emerged as promising therapeutic alternative for diseases with gene-based pathophysiology, however poor bioavailability limits its therapeutic potential. For effective delivery and enhancement of bioavailability of siRNA, several carriers including dendrimers, liposomes, siRNA conjugates, and siRNA aptamer chimeras, to name a few, have been explored. Exosomes can be considered a promising carrier for effective delivery of siRNA due to their existence in body’s endogenous system and high tolerance. The present review focuses on delivering knowledge about exosomes, siRNA, and capability of exosomes to act as natural carriers for siRNA delivery.

Keywords: Extracellular vesicles, endogenous, exosomes, oligonucleotide, small interfering RNA.

Order Reprints Order Eprints Rights & PermissionsPrintExport

Article Details

VOLUME: 21
ISSUE: 31
Year: 2015
Page: [4556 – 4565]
Pages: 10
DOI: 10.2174/138161282131151013190112
Price: $58

SOURCE

http://www.eurekaselect.com/135748/article

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VC Investment in BioTech MegaHubs and Top R&D Spenders among Big Pharma

Posted in BioTechnology - Venture Creation, BioTechnology - Venture Creation, Venture Capital, Drug Development Process, FDA Regulatory Affairs, Venture Capital on April 24, 2017| Leave a Comment »

VC Investment in BioTech MegaHubs and Top R&D Spenders among Big Pharma

Reporter: Aviva Lev-Ari, PhD, RN

UPDATED on 4/26/2017

The top 10 pharma R&D budgets in 2016

The Top 10 Pharma R&D Budgets in 2016

http://www.fiercebiotech.com/special-report/top-10-pharma-r-d-budgets-2016?utm_medium=nl&utm_source=internal&mrkid=993697&mkt_tok=eyJpIjoiT1RSbE9ESTRNR1pqWTJFNCIsInQiOiJFcUx4MFhxSFVGbVZhUkRGdUdRMTJMUGxFSEkrR0VTMEdXbjRvZkxmdXM4em4wRkg5QXZIOWJJWTgwNHR1a1dVbTRIUFwvNWRIXC9ZTkF5dHlpUUZ4bG1lS2c2NkszQk9oeGtRczhLcnYyalRSZEFjOEl6U3dUY2VaakxUbDdkNGNwIn0%3D

 

 

 

Table SOURCE: Thomson Reuters abd ENDPOINTS

According to both sources:

  • $3.5 billion for Silicon Valley plus the Bay Area and
  • $2.8 billion for New England.

Broken down by city, $6.1 billion went to

  • Boston ($2.7 billion),
  • San Jose ($2.5 billion) and
  • San Francisco/Berkeley ($1 billion).
  • San Diego ($725 million),
  • New York ($454 million) and
  • the Great Lakes area ($412 million)

SOURCE

Where the money is: Biotech’s megahubs command VC’s billions by john carroll, June 30, 2016 10:41 AM EDT, Updated: November 17, 2016 07:32 PM

https://endpts.com/where-the-money-is-biotechs-megahubs-command-vcs-billions/

The top 15 spenders in the global drug R&D business: 2017

by john carroll, 

April 24, 2017 05:22 AM EDT, Updated: 05:27 AM

The top five in the business saw their collective spending jump by more than $5 billion, from 2015 to 2016, based on the annual numbers filed largely — though not entirely — with the SEC and gathered by Endpoints News. Two of those companies,

  • Roche and the new number 2, a hard charging
  • Merck, accounted for the lion’s share of the increase. (To be sure, some onetime non-R&D spending, such as Merck’s patent settlement with Bristol-Myers on Keytruda, figured in. But so did bread and butter spending.)
  • Gilead also saw a significant increase in research costs, with
  • Eli Lilly — now off course following two bad setbacks for solanezumab and baricitinib — and the ever aggressive
  • Celgene joining the action as they pressed the accelerator on new drug programs.

Curiously, the added spending coincided with a bad drop in new drug approvals in 2016. But they don’t correlate, and we’ve already seen that turnaround under way as regulators get busy with a brand new year — and soon a brand new FDA commissioner.

SOURCE

https://endpts.com/special/top-research-budgets-in-pharma-and-biotech/

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Promising research for a male birth control pill

Posted in Affordable Care Act, Bioengineering & reverse engineering design, Biological Networks, Biological Networks, Gene Regulation and Evolution, Cell Biology, Cell Biology, Signaling & Cell Circuits, Cell Processing System in Cell Therapy Process Development, Chemical Genetics, Clinical & Translational, Clinical Genomics, CRISPR/Cas9 & Gene Editing, Developmental biology, Diagnostics and Lab Tests, Drug Development Process, Drug Discovery Chemistry, Gene Regulation, Gene Therapy & Gene Editing Development, Genetics & Innovations in Treatment, Genetics & Pharmaceutical, Genome Biology, Genomic Endocrinology, Genomic Testing: Methodology for Diagnosis, Genomics Pharmacy, Medical and Population Genetics, Pharmaceutical Drug Discovery, Population Health Management, Population Health Management, Genetics & Pharmaceutical, Reproductive Andrology, Embryology, Genomic Endocrinology, Preimplantation Genetic Diagnosis and Reproductive Genomics, Reproductive Biology & Bio Instrumentation, tagged , , , , , on March 23, 2017| Leave a Comment »

Reporter and Curator: Dr. Sudipta Saha, Ph.D.

2.1.5.5

2.1.5.5   Promising research for a male birth control pill, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair

Scientists think excessive population growth is a cause of scarcity and environmental degradation. A male pill could reduce the number of unintended pregnancies, which accounts for 40 percent of all pregnancies worldwide.

But, big drug companies long ago dropped out of the search for a male contraceptive pill which is able to chemically intercept millions of sperm before they reach a woman’s egg. Right now the chemical burden for contraception relies solely on the female. There’s not much activity in the male contraception field because an effective solution is available on the female side.

Presently, male contraception means a condom or a vasectomy. But researchers from Center for Drug Discovery at Baylor College of Medicine, USA are renewing the search for a better option—an easy-to-take pill that’s safe, fast-acting, and reversible.

The scientists began with lists of genes active in the testes for sperm production and motility and then created knockout mice that lack those genes. Using the gene-editing technology called CRISPR, in collaboration with Japanese scientists, they have so far made more than 75 of these “knockout” mice.

They allowed these mice to mate with normal (wild type) female mice, and if their female partners don’t get pregnant after three to six months, it means the gene might be a target for a contraceptive. Out of 2300 genes that are particularly active in the testes of mice, the researchers have identified 30 genes whose deletion makes the male infertile. Next the scientists are planning a novel screening approach to test whether any of about two billion chemicals can disable these genes in a test tube. Promising chemicals could then be fed to male mice to see if they cause infertility.

Female birth control pills use hormones to inhibit a woman’s ovaries from releasing eggs. But hormones have side effects like weight gain, mood changes, and headaches. A trial of one male contraceptive hormone was stopped early in 2011 after one participant committed suicide and others reported depression. Moreover, some drug candidates have made animals permanently sterile which is not the goal of the research. The challenge is to prevent sperm being made without permanently sterilizing the individual.

As a better way to test drugs, Scientists at University of Georgia, USA are investigating yet another high-tech approach. They are turning human skin cells into stem cells that look and act like the spermatogonial cells in the testes. Testing drugs on such cells might provide more accurate leads than tests on mice.

The male pill would also have to start working quickly, a lot sooner than the female pill, which takes about a week to function. Scientists from University of Dundee, U.K. admitted that there are lots of challenges. Because, a women’s ovary usually release one mature egg each month, while a man makes millions of sperm every day. So, the male pill has to be made 100 percent effective and act instantaneously.

References:

https://www.technologyreview.com/s/603676/the-search-for-a-perfect-male-birth-control-pill/

https://futurism.com/videos/the-perfect-male-birth-control-pill-is-coming-soon/?utm_source=Digest&utm_campaign=c42fc7b9b6-EMAIL_CAMPAIGN_2017_03_20&utm_medium=email&utm_term=0_03cd0a26cd-c42fc7b9b6-246845533

http://www.telegraph.co.uk/women/sex/the-male-pill-is-coming—and-its-going-to-change-everything/

http://www.mensfitness.com/women/sex-tips/male-birth-control-pill-making

http://health.howstuffworks.com/sexual-health/contraception/male-bc-pill.htm

http://europe.newsweek.com/male-contraception-side-effects-study-pill-injection-518237?rm=eu

http://edition.cnn.com/2016/01/07/health/male-birth-control-pill/index.html

http://www.nhs.uk/Conditions/contraception-guide/Pages/male-pill.aspx

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CHI’s Twelfth Annual Drug Discovery Chemistry Optimizing Small Molecules for Tomorrow’s Therapeutics April 23-27, 2017 | San Diego, CA

Posted in Drug Development Process, Drug Discovery Chemistry on February 15, 2017| Leave a Comment »

CHI’s Twelfth Annual Drug Discovery Chemistry Optimizing Small Molecules for Tomorrow’s Therapeutics April 23-27, 2017  |  San Diego, CA

Reporter: Aviva Lev-Ari, PhD, RN

Twelfth Annual

Drug Discovery Chemistry

Optimizing Small Molecules for Tomorrow’s Therapeutics

April 23-27, 2017  |  San Diego, CA

DrugDiscoveryChemistry.com

Register

Save up to $200 —

Register by March 10

Hear Over 40 Presentations from Top Pharma Companies

Cambridge Healthtech Institute’s Twelfth Annual Drug Discovery Chemistry is a dynamic conference for medicinal chemists working in pharma and biotech. As in the past, participation by industry is once again very strong this year, with presentations from AbbVie, AstraZeneca, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Eli Lilly, EMD Serono, Genentech, GlaxoSmithKline, Janssen, Merck, Novartis, Pfizer, and Takeda. Preview more than 40 of these presentations below:

AbbVie

» Important Aspects of Fragment Screening Collection Design

Ashley Adams, Ph.D., Senior Scientist, Discovery Chemistry and Technology

» Comparison of Methods for Determination of Drug-Target Engagement in Live Cells

Aleksandra Baranczak, Ph.D., Senior Scientist, Discovery Chemistry and Technology

» Bruton’s Tyrosine Kinase (BTK) – Considerations for the Design and Profiling of Irreversible Covalent Inhibitors

Michael Friedman, Ph.D., Principal Scientist

» Targeting the PRC2 Complex through a Novel Protein-Protein Interaction Inhibitor of EED

Chaohong Sun, Ph.D., Senior Principal Research Scientist; Head, Fragment Based Drug Discovery, and Global Protein Sciences-Small Molecule

» Fragment-Based, Structure-Enabled Approach to the Discovery of Novel Inhibitors of the BET Family of Proteins: ABBV-075 and Others

Le Wang, Ph.D., Principal Research Scientist, Oncology Discovery, Chemistry

» Kinetic and Thermodynamic Profiling in Drug Discovery: Are We There Yet?

Ying Wang, Ph.D., Principal Research Scientist, Department of Chemistry and Technology

AstraZeneca

» Clinical Candidate AZD5153 Is a Novel Bivalent Inhibitor of BET Bromodomains

Huawei (Ray) Chen, Ph.D., Principal Scientist II, Oncology iMed

» Targeting Inducible Nitric Oxide Synthase (iNOS)

Fredrik Edfeldt, Ph.D., Associate Principal Scientist, Biophysics, Discovery Sciences

Biogen

» Germinal-Center Kinase-Like Kinase Co-Crystal Structure Reveals a Swapped Activation Loop and C-Terminal Extension

Laura Silvian, Ph.D., Principal Scientist and Head, Physical Biochemistry

Boehringer Ingelheim

» Discovery of Novel Spiro[3H-Indole-3,2′-Pyrrolidin]-2(1H)-One Compounds as Chemically Stable and Orally Active Inhibitors of the MDM2–p53 Interaction

Andreas Gollner, Ph.D., Laboratory Head, Medicinal Chemistry

» Small Molecule Modulators of RORγ

Robert Hughes, Ph.D., Senior Associate Director, Small Molecule Discovery Research

» LTA4H Case Study: Parallel Core and Substituent FBDD to Clinical Compounds

Matthew R. Netherton, Ph.D., Senior Principal Scientist, Small Molecule Discovery Research, Medicinal Chemistry

Bristol-Myers Squibb

» Applications of the Thermal Shift Assay: More than Melts the Eye

Mary Harner, Ph.D., Research Investigator II, Leads Discovery & Optimization

» Compound Mechanism of Action: “The Known Unknown” and How That Affects Lead Optimization

Brian J. Murphy, Ph.D., Senior Principal Scientist, Fibrosis Drug Discovery, Disease Sciences and Biologics, R&D

» A Twisted Road to the Discovery of BMS-986142: Using Locked Atropisomers to Drive Potency in a Reversible Inhibitor of Bruton’s Tyrosine Kinase (BTK)

Joseph Tino, Ph.D., Senior Principal Scientist, Immunoscience Discovery Chemistry

» A First-in-Class Allosteric Inhibitor of TYK2 as a Potential Treatment for Inflammatory Autoimmune Diseases

Stephen Wrobleski, Principal Investigator, Immunoscience Chemistry

Celgene

» Ligand-directed Degradation of GSPT1 by a Novel Cereblon Modulator

Philip Chamberlain, Ph.D., Principal Scientist, Biochemistry and Structural Biology

» Targeting Inducible Nitric Oxide Synthase (iNOS)

Fredrik Edfeldt, Ph.D., Associate Principal Scientist, Biophysics, Discovery Sciences

» Ozanimod: An S1P1,5R Modulator that Targets Lymphocyte Trafficking in Autoimmune and Inflammatory Disease

Kristin Taylor Meadows, Ph.D., Scientist III, Biology

Eli Lilly & Co

» Inhibiting Interaction of IL17A and Its Receptor

Sepideh Afshar, Ph.D., Principal Research Scientist, Department of Protein Engineering

EMD Serono

» Structure-Based Optimization of a Potent, Selective and CNS penetrable p70S6K/AKT Inhibitor M2698 for the Treatment of Tumors with PAM Pathway Genomic Alterations

Igor Mochalkin, Ph.D., Associate Director, Medicinal Chemistry & Lead Optimization

Genentech

» The Development of Orally Bioavailable Antagonists of Inhibitor of Apoptosis Proteins (IAPs) for the Treatment of Cancer

Lewis Gazzard Ph.D., Senior Scientist, Discovery Chemistry

» Small Molecule Kinase Inhibitors for Brain Cancer: Limitations, Challenges and Opportunities

Timothy P. Heffron, Ph.D., Senior Scientist, Discovery Chemistry

GlaxoSmithKline

» Affinity Selection Mass Spectrometry for Target Validation and Hit Triage

Jeff Messer, Director, NCE Molecular Discovery

» Using Encoded Library Technologies to Discover Small Molecule Inhibitors of RSV Protein Complexes

Christopher Phelps, Ph.D., Manager, Drug Design & Selection Boston, RD Platform Technology & Science

Janssen

» Determination of a Focused Mini-Kinase Panel for Early Identification of Selective Kinase Inhibitors

Scott Bembenek, Ph.D., Principal Scientist, Computer-Aided Drug Discovery

» Discovery of a Series of Thiazole RORγt Inverse Agonists

Steven Goldberg, Ph.D., Associate Scientific Director, Immunology

» Combining Biophysical Techniques for Difficult Targets: Case Vignettes

Kevin Lumb, Ph.D., Scientific Director, Discovery Sciences

Merck & Co

» GPCR Structural Biology for Drug Discovery: Through the Protein Science Lens

Sujata Sharma, Ph.D., Director, Screening and Protein Science

Novartis

» Design of Technology-Compatible Cyclic Peptide Scaffolds with Oral Bioavailability

Lauren Monovich, Ph.D., Senior Investigator, Global Discovery Chemistry

» Natural Products: Promising Starting Points for Fragment-Based Screening

Tim Schuhmann, Ph.D., Investigator III, Natural Products Unit

» Leniolisib (CDZ173) – The Discovery of a New Generation of Selective PI3Kδ Inhibitors

Nicolas Soldermann, Ph.D., Senior Investigator and Group Leader, Global Discovery Chemistry

Pfizer

» Inhibition of Autoimmune Pathways with Dual Inhibition of JAK1 and TYK2

Brian Gerstenberger, Ph.D., Principal Scientist, Medicinal Chemistry

» Advancing a Clinical Candidate Targeting IRAK4 from a Fragment Lead

Seungil Han, Ph.D., Associate Research Fellow, Structural Biology & Biophysics, WorldWide Research & Development

» Allosteric Activators of AMP-Activated Protein Kinase for the Treatment of Diabetic Nephropathy

Ravi G. Kurumbail, Ph.D., Research Fellow and Structural Biology Laboratory Head

» Macrocyclic Secondary Structure: Permeability and Chemical Biology

Spiros Liras, Ph.D., Vice President, Head, Cardiovascular Metabolism, RDRU and Discovery Network

» Discovery and Synthesis of the Macrocyclic EML4-ALK Inhibitor, Lorlatinib (PF-06463922)

Paul Richardson, Ph.D., Director, Process and Analytical Technologies, Oncology Medicinal Chemistry

» CASE STUDY: Discovery of a Potent and Selective Sphingosine Kinase 1 Inhibitor through the Molecular Combination of Chemotype Distinct Screening Hits

Mark E. Schnute, Ph.D., Medicine Design

Takeda

» FBDD: Part of an Integrated Drug Discovery Platform

Derek Cole, Ph.D., Director, Medicinal Chemistry

» Discovery of NF-kappa-B-Inducing Kinase (NIK) Inhibitors

Walter Keung, Ph.D., Senior Scientist, Medicinal Chemistry

» Biophysical Characterization of GPCRs: SPR and Other Techniques

Phillip Schwartz, Ph.D., Senior Scientist, Biophysical Chemistry

» Fragments and SPR for GPCRs

Shuo Wang, Ph.D., Principal Scientist, Biophysical Chemistry

» Structure-Based Design, Synthesis, and Dermal Application of Novel Tyrosine Kinase 2 (TYK2) Inhibitors

Takatoshi Yogo, Ph.D., Principal Scientist, Research, Immunology Unit

Learn More | Present A Poster | Sponsor & Exhibit | Download Brochure

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Event-At-A-Glance

 

April 24-25

April 25-26

April 27

Inflammation Inhibitors

Kinase Inhibitor Chemistry

Biophysical Approaches

Protein-Protein Interactions, Part 1

Protein-Protein Interactions, Part 2

Small Molecules for Cancer Immunotherapy

GPCR-Targeted Drug Design

Fragment-Based Drug Discovery

Blood-Brain Penetrant Inhibitors

Macrocyclics & Constrained Peptides


SOURCE

From: Small Molecules <katieo@healthtech.com>

Date: Wednesday, February 15, 2017 at 11:11 AM

To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: Hear 40+ Presentations from Top Pharma Companies at Drug Discovery Chemistry

Read Full Post »

Real Time Coverage and eProceedings of Presentations on 9/19-9/21 @CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

Posted in BioTechnology - Venture Creation, BioTechnology - Venture Creation, Venture Capital, Conference Coverage with Social Media, CRISPR/Cas9 & Gene Editing, Drug Delivery Platform Technology, Drug Development Process, Drug Toxicity, FDA, Genetics & Innovations in Treatment, Genetics & Pharmaceutical, Genome Biology, Genomic Testing: Methodology for Diagnosis, Global Partnering & Biotech Investment on September 25, 2016| Leave a Comment »

Real Time Coverage and eProceedings of Presentations on 9/19-9/21 @CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

Curator: Aviva Lev-Ari, PhD, RN

2.1.5.11

2.1.5.11   Real Time Coverage and eProceedings of Presentations on 9/19-9/21 @CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair

LIVE 9/19 8AM – 10AM USING CRISPR/Cas9 FOR FUNCTIONAL SCREENING at CHI’s 2nd Annual Symposium CRISPR: Mechanisms and Applications @CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/19/live-919-8am-10am-using-crisprcas9-for-functional-screening-at-chis-2nd-annual-symposium-crispr-mechanisms-and-applications-chis-14th-discovery-on-target-919-9222/

LIVE 9/19 9:40 – noon CRISPR Engineering Lymphoma Lines & Will Interference from CRISPR Silence RNAi? CHI’s 2nd Annual Symposium CRISPR: Mechanisms and Applications @ CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/19/live-919-940-noon-crispr-engineering-lymphoma-lines-will-interference-from-crispr-silence-rnai-chis-2nd-annual-symposium-crispr-mechanisms-and-applications-chis-14th/

LIVE 9/19 1:40 – 3:20 EMERGING APPLICATIONS OF CRISPR/CAS9 at CHI’s 2nd Annual Symposium CRISPR: Mechanisms and Applications @ CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/19/live-919-140-320-emerging-applications-of-crisprcas9-at-chis-2nd-annual-symposium-crispr-mechanisms-and-applications-chis-14th-discovery-on-target-919-9222016/

LIVE 9/19 4PM – 5:30PM NK CELL-BASED CANCER IMMUNOTHERAPY @CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/19/live-919-4pm-530pm-nk-cell-based-cancer-immunotherapy-chis-14th-discovery-on-target-919-9222016-westin-boston-waterfront-boston/

LIVE 9/20 8AM to noon GENE THERAPIES BREAKTHROUGHS at CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/20/live-920-8am-to-noon-gene-therapies-breakthroughs-at-chis-14th-discovery-on-target-919-9222016-westin-boston-waterfront-boston/

LIVE 9/20 2PM to 5:30PM New Viruses for Therapeutic Gene Delivery at CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/20/live-920-2pm-to-530pm-new-viruses-for-therapeutic-gene-delivery-at-chis-14th-discovery-on-target-919-9222016-westin-boston-waterfront-boston/

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

https://pharmaceuticalintelligence.com/2016/09/21/live-921-8am-to-1055-am-expoloring-the-versatility-of-crisprcas9-at-chis-14th-discovery-on-target-919-9222016-westin-boston-waterfront-boston/

LIVE 9/21 8AM to 2:40PM Targeting Cardio-Metabolic Diseases: A focus on Liver Fibrosis and NASH Targets at CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/21/live-921-8am-to-240pm-targeting-cardio-metabolic-diseases-a-focus-on-liver-fibrosis-and-nash-targets-at-chis-14th-discovery-on-target-919-9222016-westin-boston-waterfront-b/

LIVE 9/21 12:50 pm Plenary Keynote Program at CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/21/live-921-1250-pm-plenary-keynote-program-at-chis-14th-discovery-on-target-919-9222016-westin-boston-waterfront-boston/

LIVE 9/21 3:20PM to 6:40PM KINASE INHIBITORS FOR CANCER IMMUNOTHERAPY COMBINATIONS & KINASE INHIBITORS FOR AUTOIMMUNE AND INFLAMMATORY DISEASES at CHI’s 14th Discovery On Target, 9/19 – 9/22/2016, Westin Boston Waterfront, Boston

https://pharmaceuticalintelligence.com/2016/09/21/live-921-320pm-to-640pm-kinase-inhibitors-for-cancer-immunotherapy-combinations-kinase-inhibitors-for-autoimmune-and-inflammatory-diseases-at-chis-14th-discovery-on-target-919/

Read Full Post »

Hashtags and Handles for CHI’s 14th Annual Discovery on Target, September 19 – 22, 2016 in Boston, Westin Boston Waterfront

Posted in Conference Coverage with Social Media, CRISPR/Cas9 & Gene Editing, Drug Delivery Platform Technology, Drug Development Process, Drug Discovery Chemistry, Genome Biology on September 18, 2016| Leave a Comment »

Hashtags and Handles for CHI’s 14th Annual Discovery on Target, September 19 – 22, 2016 in Boston, Westin Boston Waterfront

Curator: Stephen J Williams, PhD

 

CAMBRIDGE HEALTHTECH INSTITUTE’S 2016 Discovery on Target

attend

http://www.DiscoveryOnTarget.com

DOT-150x150

  • CHI’s Discovery on Target in Boston, September 19-22, 2016,

  • CRISPR: Mechanisms to Applications on 9/19/2016

 

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

 

TALK SPECIFIC # and @

 

Monday Sept. 19th Understanding CRISPR: Mechanisms and Applications

 

Day Time Talk Title # @
Monday Sept. 19, 2016 8:00 AM 8:00 Chairperson’s Opening Remarks

Scott Martin, Ph.D., Group Lead, Functional Genomics, Genentech Inc

 

 #BostonDOT16

#personalizedmedicine

#oncology

#Boston

#immunology

#biotech

#CRSPR

#geneediting

#genomics

 @BostonDOT

@CHI

@genentech

@Boston

@BiotechNews

@pharma_BI

@AVIVA_1950

@GeneEditing

@Genomeresearch

 

 

 
         
  8:10 AM 8:10 Comparing Arrayed siRNA and CRISPR Approaches Towards Functional Genomics Screening

Scott Martin, Ph.D., Group Lead, Functional Genomics, Genentech Inc.

 

 #Crisper

#CRSPR

#Cas9

#geneediting

#genomics

 

 

 @genentech

 

 
  8:40 AM 8:40 Getting from Alpha to Omega: Successfully Conceptualizing, Starting and Finishing CRISPR/Cas Screens

Ralph Garippa, Ph.D., Director, RNAi & Gene Editing Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

 

 

 

 #Crisper

#CRSPR

#Cas9

#geneediting

#genomics

 @CHI

@Boston

@BiotechNews

@pharma_BI

@AVIVA_1950

 
  9:10 AM 9:10 Genome Editing-Enabled HTS Assays for Genetically Inherited Disease Drug Discovery

James Inglese, Ph.D., Head Assay Development & Screening Technologies, National Center for Advancing Translational Sciences, National Institutes of Health

 

 

 #geneediting

#raredisease

#genetics

#AssayDevelopment

#NIH

#CMT

#drugdiscovery

 

 

 @BostonDOT

@NIH

@BiotechNews

@PharmaNews

@pharma_BI

@AVIVA_1950

@DrugDiscover365

 

 

 

 
  9:40 AM

 

 

 

 

 

 

 

 

 

10:40

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

11:40

 9:40 Use of CRISPR and Other Genomic Technologies to Advance Drug Discovery

Namjin Chung, Ph.D., Head, Functional Genomics Platform, Discovery Research, AbbVie, Inc.

10:40 Vignettes From the Bench: CRISPR Engineering Lymphoma Lines

Arthur L. Shaffer, III, Ph.D., Staff Scientist, Laboratory of Dr. Louis Staudt, Lymphoid Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health

 

11:10 PANEL DISCUSSION: Will Interference from CRISPR Silence RNAi?

Moderator: Scott Martin, Ph.D., Group Lead, Functional Genomics, Genentech Inc.

 

 

 

 #geneediting

#CRISPR

#Cas9

#NIH

#CMT

#drugdiscovery

#pharmanews

#CRSPR

#genomics

 @BostonDOT

@abbvie

@BiotechNews

@PharmaNews

@pharma_BI

@AVIVA_1950

@DrugDiscover365

 
  1:40 PM

 

 

 

 

 

 

 

 

 

 

 

 

1:50 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

2:20

 

 

 

 

 

 

 

 

 

 

 

2:50

 EMERGING APPLICATIONS OF CRISPR/CAS9

1:40 Chairperson’s Opening Remarks

James Inglese, Ph.D., Head Assay Development & Screening Technologies, National Center for Advancing Translational Sciences, National Institutes of Health

 

1:50 MicroRNA Target Site Editing of Chondrocyte Master-Regulators in Primary Human Cells Using CRISPR-Cas9

Christine Seidl, Ph.D., Post-Doctoral Research Associate, Cell Signaling, Kennedy Institute of Rheumatology, Oxford University

 

2:20 Massively Parallel Combinatorial Genetic Perturbation Screening with CRISPR-Cas9 in Human Cells

Cheryl H. Cui, Ph.D. Candidate, Harvard-MIT Division of Health Science and Technology, MIT

 

2:50 The Scientist’s Guide to CRISPR Law

Paul Enríquez, J.D., LL.M., Ph.D. Candidate, Structural and Molecular Biochemistry, North Carolina State University

 

 

 

 note use above hashtags including these specific ones

 

 

#CMT

 

 

 

 

 

 

 

#inflammation

 

 

 

 

 

 

 

 

 

 

 

 

 

 

#systembiology

#genetics

 

 

 

 note use above @ and these talk specific ones

 

 

@NIH

 

 

 

 

 

 

 

@UniofOxford

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@MIT

@Harvard

 

 

 

 

 

 

 

 

 

 

@NCarolinaSt

 

 
 

 

 

 12-3:00 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

3:30-6 PM

 Special Conference Short Courses

SC7: Using IP Landscape Studies to Improve Your Confidence While Navigating a Crowded IP and Technology Space – Detailed Agenda

 

Instructors:

David Berry, M.D., Ph.D., General Partner, Flagship Ventures

Ananda Chakrabarty, Ph.D., Department of Microbiology & Immunology, University of Illinois College of Medicine

Anu Daniel, Ph.D., Licensing Manager, Innovation, Partners Healthcare

Drew Lowery, Ph.D., Director of Life Sciences and Group Leader, Biotechnology Pharmaceuticals Group, Global Prior Art, Inc.

Amy Mendel, J.D., SVP, Intellectual Property, Evelo Biosciences

Daniel Neuman, Ph.D., Group Leader, Chemistry & Materials, Global Prior Art, Inc.

 

SC12: Introduction to Gene Editing – Detailed Agenda

Instructors:

Stephanie Mohr, Ph.D., Lecturer, Genetics & Director, Drosophila RNAi Screening Center at Harvard Medical School

Claire Yanhui Hu, Ph.D., Senior Bioinformatician, Drosophila RNAi Screening Center, Department of Genetics, Harvard Medical School

Paul Enríquez, J.D., LL.M., Ph.D. Candidate, Structural and Molecular Biochemistry, North Carolina State University

 

 #IP

#patent

#partnership

#innovation

#biotech

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

#genetics

#CRSPR

#Cas9

#molecularscreen

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 @FlagshipVenture

@PartnersNews

@GlobalArtAgency

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@harvardmed

@NCarolinaSt

 

 
     

 

    

 
Tuesday Sept. 21   Advances in Gene Editing and Gene Silencing   Conference Part 1

 

    

 
    KEYNOTE SESSION:

GENOME EDITING FOR IN VIVO APPLICATIONS

 

  
  8:05 AM 8:05 Chairperson’s Opening Remarks

Bryan R. Cullen, Ph.D., James B. Duke Professor of Molecular Genetics and Microbiology and Director, Center for Virology, Duke University

 

 #genetherapy

#virology

#adenovirus

 

 @Duke

 
  8:20 AM

 

 

 

 

 

 

 

 

 

 

 

10:35 AM

 

 

 

 

 

 

 

 

 

 

 

 

11:05 AM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

11:35 AM

 

 8:20 AAV for Gene Therapy and Genome Editing

James Wilson, M.D., Ph.D., Professor, Department of Pathology and Laboratory Medicine, Perelman School of Medicine; Director, Orphan Disease Center and Director, Gene Therapy Program, University of Pennsylvania

 

10:35 Targeted Endonucleases as Antiviral Agents: Promises and Pitfalls

Keith R. Jerome, M.D., Ph.D., Member, Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center; Professor and Head, Virology Division, Department of Laboratory Medicine, University of Washington

 

11:05 Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing

Daniel Anderson, Ph.D., Professor, Department of Chemical Engineering, Institute for Medical Engineering & Science, Harvard-MIT Division of Health Sciences & Technology and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

 

11:35 PANEL DISCUSSION: CRISPR/Cas: A Realistic and Practical Look at What the Future Could Hold

Moderator: Bryan R. Cullen, Ph.D., James B. Duke Professor of Molecular Genetics and Microbiology and Director, Center for Virology, Duke University

Participants: Session Speakers

 

 

 

 

 #genetherapy

#virology

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

#drugdelivery

#genedelivery

#cancer

 

 @PennMedicine

 

 

 

 

 

 

 

 

 

 

@UW

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@MIT

@kochinstitute

@Duke

 

 
  12:05 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

12:45 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 12:05 pm CRISPR/Cas9 for the Screening of the Human Kinome – A Pilot Study in an Aggressive Pediatric Cancer Cell Line

Simone T. Sredni, M.D., Ph.D., Research Assistant Professor, Neurological Surgery, Northwestern University Feinberg School of Medicine, Ann and Robert H. Lurie Children’s Hospital of Chicago

 

12:45 Luncheon Presentation: Building a Better Research Story: Screening with shRNA and CRISPR

Ryan Raver, Ph.D., Global Product Manager, Functional Genomics, MilliporeSigma

 

 

 

 

 

 

 #cancer

#kinome

#brain

#ChildhoodCancerAwareness

 

 

 

 

 

 

 

 

 

 

 

 

 

#genomics

 

 

 

 @NorthwesternMed

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@MilliporeSigma

 

 

 

 

 

 
     

COMPLEMENTING THE USE CRISPR & RNAi FOR DISEASE MODELING

 

    
  2:05 PM

 

 

 

 

 

 

 

 

 

2:15 PM

 

 

 

 

 

 

 

 

 

 

2:45 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

3:15 PM

 

 

 

 

 

 

 

 

4:25 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

4:55 PM

 2:05 Chairperson’s Remarks

Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

 

2:15 Comparing Arrayed siRNA and CRISPR Approaches Towards Functional Genomics Screening

Scott Martin, Ph.D., Group Lead, Functional Genomics, Genentech Inc.

 

2:45 Use of CRISPR/Cas9-Based Gene Editing to Model and Treat Retinal Degenerative Disease

Donald Zack, M.D., Ph.D., Guerrieri Professor of Genetic Engineering and Molecular Ophthalmology, Johns Hopkins University

 

3:15 HP Inkjet Technology for Enhancing Gene Editing Experiments

Erica Squires, Ph.D., Senior Applications Scientist, HP Inc.

 

4:25 Harnessing the Versatile CRISPR-Cas9 Systems for Cancer Modeling Platforms

Geoffrey Bartholomeusz, Ph.D., Associate Professor and Director, Target Identification and Validation Program, Department of Experimental Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center

 

4:55 Technology Panel: Trends in CRISPR & RNAi Technologies

Moderator: Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

Panelists:

Louise Baskin, Senior Product Manager, Dharmacon, GE Healthcare

Paul Diehl, Ph.D., Director, Business Development, Cellecta Inc.

 

 

 

 

  

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

#eyedisease

 

 

 

 

 

 

 

 

 

 

 

 

#bioprinting

#3D_printing

#tech

#innovation

#geneediting

 

 

 

 

 

#endcancer

#cancer

#drugdiscovery

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 @sloan_kettering

@MSKCC_OncoNotes

 

 

 

 

 

 

 

 

 

@genentech

 

 

 

 

 

 

 

 

 

 

@MacularHope

@HopkinsMedicine

 

 

 

 

 

 

 

 

 

 

 

@HP

@My3DPrinting

 

 

 

 

 

 

@MDAndersonNews

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@MMSK

@sloan_kettering

 

 

 

 

 

 

 

 

 

 

 

 

@CELLECTA

 

 

 

 

 

 
Wednesday Sept. 21 2016   EXPLORING THE VERSATILITY OF CRISPR/Cas9    
   

8:00 AM

 

 

 

 

 

8:10 AM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

8:40 AM

 

 

 

 

 

 

 

9:10 AM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

10:25 AM

 

 

 

 

 

 

 

 

 

 

10:55 AM

 8:00 Chairperson’s Opening Remarks

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

 

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

 

8:40 Therapeutic Gene Editing With CRISPR/Cas9

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

 

9:10 Towards Combinatorial Drug Discovery: Mining Heterogenous 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

 

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

 

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

 

 

 

 

 

 

   @CRISPRTX

 

 

 

 

 

 

@nyuniversity

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@CRISPRTX

 

 

 

 

 

 

@MDAndersonNews

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@uiowa

 
         
Wednesday 21, 2016 12:55 – 2:40 Plenary Keynote Sessions

 

    
  12:55 PM

 

 

 

 

 

 

 

1:15 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

2:00 PM

 12:55 Event Chairperson’s Opening Remarks

Cindy Crowninshield, RDN, LDN, Conference Director, Cambridge Healthtech Institute

 

1:15 Open Innovation Partnerships to Bridge the Gap from GWAS to Drug Targets Jeffrey Barrett, D.Phil., Founding Director, Open Targets; Group Leader, Wellcome Trust Sanger Institute

 

Aaron Day-Williams, Ph.D., Biogen Scientific Lead, Open Targets; Associate Director and Head, Statistical Genetics, Biogen

 

2:00 Cell-Penetrating Mini-Proteins Gregory L. Verdine, Ph.D., Erving Professor, Chemistry, Departments of Stem Cell and Regenerative Biology, Chemistry and Chemical Biology, and Molecular and Cellular Biology, Harvard University and Harvard Medical School

 

 

 

  

 

 

 

 

 

 

 

 

#openinnovation

#openscience

#bioinformatics

 

 

 

 

 

 

 

#openinnovation

 

 @CHI

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

@Biogen

 

 

 

 

 

 

 

 

@HarvardMed

 

 

 

 

 
         
Thursday September 22,2016 Part 2 USING CRISPR/RNAi FOR TARGET DISCOVERY & PATHWAY ANALYSIS    
   

8:30 AM

 8:30 Chairperson’s Remarks

John Doench, Ph.D., Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT

 

8:45 Strategies and Applications Using shRNA and CRISPR Technology for Identification of New Druggable Targets

Donald Apanovitch, Ph.D., Director, Functional Genomics (Oncology), Pfizer Research

9:15 High Throughput Phenotypic Screening in Drug Discovery Using the CRISPR-Cas9 System

Greg Hoffman, Ph.D., Investigator III, Developmental & Molecular Pathways Department, Novartis Institutes for Biomedical Research

 

9:45 CRISPR Libraries for Functional Genomics: Optimizing On-Target Activity and Avoiding Off-Target Effects

John Doench, Ph.D., Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT

 

11:10 A High Throughput Functional Genomics Screening Approach to Identify Modulators of Nonsense-Mediated mRNA Decay to Treat Mendelian Disorders

Madhu Lal-Nag, Ph.D., Group Leader, Trans-NIH RNAi Facility, National Center for Advancing Translational Sciences, National Institutes of Health

 

11:40 Fas-Mediated Apoptosis Overcomes Resistance to Kras-Silencing in Lung Cancer Cells

Haiwei Mou, Ph.D., Postdoctoral Fellow, Laboratory of Dr. Wen Xue, RNA Therapeutics Institute and Program in Molecular Medicine, University of Massachusetts Medical School

 

12:10 pm Arrayed CRISPR Screening with Synthetic crRNA Libraries for High-Throughput Loss-of-Function Studies

Louise Baskin, Senior Product Manager, Dharmacon, GE Healthcare

 

 

 

    
Thursday

Sept. 22 2016

 12:50 PM 12:50 Luncheon Presentation: Optimizing CRISPR for in vitro and in vivo Pooled Functional Genetic Screens

Paul Diehl, Ph.D., Director, Business Development, Cellecta, Inc.

 

 

    
    CRISPR-BASED FUNCTIONAL SCREENING FOR ONCOLOGY    
  2:15 PM

 

 

 

 

 

 

 

 

 

2:20 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

2:50 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

3:30 PM

 

 

 

 

 

 

 

 

 

 

 

4:00 PM

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 2:15 Chairperson’s Remarks

Roderick Beijersbergen, Ph.D., Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center

 

2:20 Large Scale CRISPR Screens for Discovery of Genotype Specific Combination Therapies

Roderick Beijersbergen, Ph.D., Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center

 

2:50 GPCR-Mediated cAMP as an Immune Checkpoint in Cancer Identified by RNAi Screening

Tillmann Michels, Head of Research Group, Immune Checkpoint Inhibitors, Department of Interventional Immunology, Regensburg Center for Interventional Immunology; Member, Department of Translational Immunology, German Cancer Research Center

 

3:30 CRISPR-Based Mutagenesis Approach for Cancer Drug Target Identification

Junwei Shi, Ph.D., Assistant Professor, Department of Cancer Biology, University of Pennsylvania School of Medicine

 

4:00 Applying Functional Genomics in Mouse Models of Human Cancer

Yejing Ge, Ph.D., Postdoctoral Fellow, Laboratory of Dr. Elaine Fuchs, Department of Mammalian Cell Biology and Development, Rockefeller University

 

4:30 A CRISPR/Cas9 System to Increase Homologous Recombination Repair

Ciro Bonetti, Ph.D., Postdoctoral Scientist, Laboratory of Dr. Andrea Ventura, Memorial Sloan-Kettering Cancer Center

 

 

 

 

    
         
         
         
         
         
         
         
         
         
         
         
         
         

 

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physiology-cover-seriese-vol-3individualsaddlebrown-page2

Milestones in Physiology

Discoveries in Medicine, Genomics and Therapeutics

Patient-centric Perspective 

http://www.amazon.com/dp/B019VH97LU 

2015

 

 

Author, Curator and Editor

Larry H Bernstein, MD, FCAP

Chief Scientific Officer

Leaders in Pharmaceutical Business Intelligence

Larry.bernstein@gmail.com

Preface

Introduction 

Chapter 1: Evolution of the Foundation for Diagnostics and Pharmaceuticals Industries

1.1  Outline of Medical Discoveries between 1880 and 1980

1.2 The History of Infectious Diseases and Epidemiology in the late 19th and 20th Century

1.3 The Classification of Microbiota

1.4 Selected Contributions to Chemistry from 1880 to 1980

1.5 The Evolution of Clinical Chemistry in the 20th Century

1.6 Milestones in the Evolution of Diagnostics in the US HealthCare System: 1920s to Pre-Genomics

 

Chapter 2. The search for the evolution of function of proteins, enzymes and metal catalysts in life processes

2.1 The life and work of Allan Wilson
2.2  The  evolution of myoglobin and hemoglobin
2.3  More complexity in proteins evolution
2.4  Life on earth is traced to oxygen binding
2.5  The colors of life function
2.6  The colors of respiration and electron transport
2.7  Highlights of a green evolution

 

Chapter 3. Evolution of New Relationships in Neuroendocrine States
3.1 Pituitary endocrine axis
3.2 Thyroid function
3.3 Sex hormones
3.4 Adrenal Cortex
3.5 Pancreatic Islets
3.6 Parathyroids
3.7 Gastointestinal hormones
3.8 Endocrine action on midbrain
3.9 Neural activity regulating endocrine response

3.10 Genomic Promise for Neurodegenerative Diseases, Dementias, Autism Spectrum, Schizophrenia, and Serious Depression

 

Chapter 4.  Problems of the Circulation, Altitude, and Immunity

4.1 Innervation of Heart and Heart Rate
4.2 Action of hormones on the circulation
4.3 Allogeneic Transfusion Reactions
4.4 Graft-versus Host reaction
4.5 Unique problems of perinatal period
4.6. High altitude sickness
4.7 Deep water adaptation
4.8 Heart-Lung-and Kidney
4.9 Acute Lung Injury

4.10 Reconstruction of Life Processes requires both Genomics and Metabolomics to explain Phenotypes and Phylogenetics

 

Chapter 5. Problems of Diets and Lifestyle Changes

5.1 Anorexia nervosa
5.2 Voluntary and Involuntary S-insufficiency
5.3 Diarrheas – bacterial and nonbacterial
5.4 Gluten-free diets
5.5 Diet and cholesterol
5.6 Diet and Type 2 diabetes mellitus
5.7 Diet and exercise
5.8 Anxiety and quality of Life
5.9 Nutritional Supplements

 

Chapter 6. Advances in Genomics, Therapeutics and Pharmacogenomics

6.1 Natural Products Chemistry

6.2 The Challenge of Antimicrobial Resistance

6.3 Viruses, Vaccines and immunotherapy

6.4 Genomics and Metabolomics Advances in Cancer

6.5 Proteomics – Protein Interaction

6.6 Pharmacogenomics

6.7 Biomarker Guided Therapy

6.8 The Emergence of a Pharmaceutical Industry in the 20th Century: Diagnostics Industry and Drug Development in the Genomics Era: Mid 80s to Present

6.09 The Union of Biomarkers and Drug Development

6.10 Proteomics and Biomarker Discovery

6.11 Epigenomics and Companion Diagnostics

 

Chapter  7

Integration of Physiology, Genomics and Pharmacotherapy

7.1 Richard Lifton, MD, PhD of Yale University and Howard Hughes Medical Institute: Recipient of 2014 Breakthrough Prizes Awarded in Life Sciences for the Discovery of Genes and Biochemical Mechanisms that cause Hypertension

7.2 Calcium Cycling (ATPase Pump) in Cardiac Gene Therapy: Inhalable Gene Therapy for Pulmonary Arterial Hypertension and Percutaneous Intra-coronary Artery Infusion for Heart Failure: Contributions by Roger J. Hajjar, MD

7.3 Diagnostics and Biomarkers: Novel Genomics Industry Trends vs Present Market Conditions and Historical Scientific Leaders Memoirs

7.4 Synthetic Biology: On Advanced Genome Interpretation for Gene Variants and Pathways: What is the Genetic Base of Atherosclerosis and Loss of Arterial Elasticity with Aging

7.5 Diagnosing Diseases & Gene Therapy: Precision Genome Editing and Cost-effective microRNA Profiling

7.6 Imaging Biomarker for Arterial Stiffness: Pathways in Pharmacotherapy for Hypertension and Hypercholesterolemia Management

7.7 Neuroprotective Therapies: Pharmacogenomics vs Psychotropic drugs and Cholinesterase Inhibitors

7.8 Metabolite Identification Combining Genetic and Metabolic Information: Genetic association links unknown metabolites to functionally related genes

7.9 Preserved vs Reduced Ejection Fraction: Available and Needed Therapies

7.10 Biosimilars: Intellectual Property Creation and Protection by Pioneer and by

7.11 Demonstrate Biosimilarity: New FDA Biosimilar Guidelines

 

Chapter 7.  Biopharma Today

8.1 A Great University engaged in Drug Discovery: University of Pittsburgh

8.2 Introduction – The Evolution of Cancer Therapy and Cancer Research: How We Got Here?

8.3 Predicting Tumor Response, Progression, and Time to Recurrence

8.4 Targeting Untargetable Proto-Oncogenes

8.5 Innovation: Drug Discovery, Medical Devices and Digital Health

8.6 Cardiotoxicity and Cardiomyopathy Related to Drugs Adverse Effects

8.7 Nanotechnology and Ocular Drug Delivery: Part I

8.8 Transdermal drug delivery (TDD) system and nanotechnology: Part II

8.9 The Delicate Connection: IDO (Indolamine 2, 3 dehydrogenase) and Cancer Immunology

8.10 Natural Drug Target Discovery and Translational Medicine in Human Microbiome

8.11 From Genomics of Microorganisms to Translational Medicine

8.12 Confined Indolamine 2, 3 dioxygenase (IDO) Controls the Homeostasis of Immune Responses for Good and Bad

 

Chapter 9. BioPharma – Future Trends

9.1 Artificial Intelligence Versus the Scientist: Who Will Win?

9.2 The Vibrant Philly Biotech Scene: Focus on KannaLife Sciences and the Discipline and Potential of Pharmacognosy

9.3 The Vibrant Philly Biotech Scene: Focus on Computer-Aided Drug Design and Gfree Bio, LLC

9.4 Heroes in Medical Research: The Postdoctoral Fellow

9.5 NIH Considers Guidelines for CAR-T therapy: Report from Recombinant DNA Advisory Committee

9.6 1st Pitch Life Science- Philadelphia- What VCs Really Think of your Pitch

9.7 Multiple Lung Cancer Genomic Projects Suggest New Targets, Research Directions for Non-Small Cell Lung Cancer

9.8 Heroes in Medical Research: Green Fluorescent Protein and the Rough Road in Science

9.9 Issues in Personalized Medicine in Cancer: Intratumor Heterogeneity and Branched Evolution Revealed by Multiregion Sequencing

9.10 The SCID Pig II: Researchers Develop Another SCID Pig, And Another Great Model For Cancer Research

Epilogue

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