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Archive for the ‘Personalized Medicine Coalition’ Category


 

Dear Colleague:


Recognizing that almost 40 percent of those who responded to the strategic planning survey underpinning the plan did not agree that “it is inevitable that all doctors will someday practice personalized medicine [because] the science and technology will demand it,” the Coalition has outlined robust efforts to encourage and nurture medical progress through education, advocacy, and evidence development.
On behalf of the Personalized Medicine Coalition (PMC), I am pleased to share PMC’s Strategic Plan for 2019.

Unless payers, providers, patients, and policymakers are aware of personalized medicine, it will be harder to increase research funding and propose smart public policies that encourage integrating diagnostics into therapeutic decisions.

And unless we develop the evidence base necessary to demonstrate the clinical and economic value of personalized medicine, those decision-makers are not going to embrace it, and, in fact, may propose policies, like “step therapy” — paying for what costs least first — that fly in the face of the principles of personalized medicine.

In short, in keeping with the scientific and technological progress of recent years, we need to redouble our efforts to deliver the promise of personalized medicine — better clinical outcomes and more efficient health systems. The stakes are very high.

We welcome your participation in PMC this year. Without it, progress will be much slower than any of us would like.

Please contact PMC Membership & Development Director Kayla Smith at ksmith@personalizedmedicinecoalition.org with questions about how to get involved with any of the projects listed in the plan.

Sincerely yours,

Edward Abrahams

President

SOURCE

rom: Personalized Medicine Coalition <messages@app.production.membersuite.com>

Reply-To: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>

Date: Wednesday, January 9, 2019 at 9:36 AM

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

Subject: PMC Strategic Plan for 2019

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Role of Informatics in Precision Medicine: Notes from Boston Healthcare Webinar: Can It Drive the Next Cost Efficiencies in Oncology Care? Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 1: Next Generation Sequencing (NGS)

Role of Informatics in Precision Medicine: Notes from Boston Healthcare Webinar: Can It Drive the Next Cost Efficiencies in Oncology Care?

Reporter: Stephen J. Williams, Ph.D.

 

Boston Healthcare sponsored a Webinar recently entitled ” Role of Informatics in Precision Medicine: Implications for Innovators”.  The webinar focused on the different informatic needs along the Oncology Care value chain from drug discovery through clinicians, C-suite executives and payers. The presentation, by Joseph Ferrara and Mark Girardi, discussed the specific informatics needs and deficiencies experienced by all players in oncology care and how innovators in this space could create value. The final part of the webinar discussed artificial intelligence and the role in cancer informatics.

 

Below is the mp4 video and audio for this webinar.  Notes on each of the slides with a few representative slides are also given below:

Please click below for the mp4 of the webinar:

 

 


  • worldwide oncology related care to increase by 40% in 2020
  • big movement to participatory care: moving decision making to the patient. Need for information
  • cost components focused on clinical action
  • use informatics before clinical stage might add value to cost chain

 

 

 

 

Key unmet needs from perspectives of different players in oncology care where informatics may help in decision making

 

 

 

  1.   Needs of Clinicians

– informatic needs for clinical enrollment

– informatic needs for obtaining drug access/newer therapies

2.  Needs of C-suite/health system executives

– informatic needs to help focus of quality of care

– informatic needs to determine health outcomes/metrics

3.  Needs of Payers

– informatic needs to determine quality metrics and managing costs

– informatics needs to form guidelines

– informatics needs to determine if biomarkers are used consistently and properly

– population level data analytics

 

 

 

 

 

 

 

 

 

 

 

 

What are the kind of value innovations that tech entrepreneurs need to create in this space? Two areas/problems need to be solved.

  • innovations in data depth and breadth
  • need to aggregate information to inform intervention

Different players in value chains have different data needs

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Data Depth: Cumulative Understanding of disease

Data Depth: Cumulative number of oncology transactions

  • technology innovators rely on LEGACY businesses (those that already have technology) and these LEGACY businesses either have data breath or data depth BUT NOT BOTH; (IS THIS WHERE THE GREATEST VALUE CAN BE INNOVATED?)
  • NEED to provide ACTIONABLE as well as PHENOTYPIC/GENOTYPIC DATA
  • data depth more important in clinical setting as it drives solutions and cost effective interventions.  For example Foundation Medicine, who supplies genotypic/phenotypic data for patient samples supplies high data depth
  • technologies are moving to data support
  • evidence will need to be tied to umbrella value propositions
  • Informatic solutions will have to prove outcome benefit

 

 

 

 

 

How will Machine Learning be involved in the healthcare value chain?

  • increased emphasis on real time datasets – CONSTANT UPDATES NEED TO OCCUR. THIS IS NOT HAPPENING BUT VALUED BY MANY PLAYERS IN THIS SPACE
  • Interoperability of DATABASES Important!  Many Players in this space don’t understand the complexities integrating these datasets

Other Articles on this topic of healthcare informatics, value based oncology, and healthcare IT on this OPEN ACCESS JOURNAL include:

Centers for Medicare & Medicaid Services announced that the federal healthcare program will cover the costs of cancer gene tests that have been approved by the Food and Drug Administration

Broad Institute launches Merkin Institute for Transformative Technologies in Healthcare

HealthCare focused AI Startups from the 100 Companies Leading the Way in A.I. Globally

Paradoxical Findings in HealthCare Delivery and Outcomes: Economics in MEDICINE – Original Research by Anupam “Bapu” Jena, the Ruth L. Newhouse Associate Professor of Health Care Policy at HMS

Google & Digital Healthcare Technology

Can Blockchain Technology and Artificial Intelligence Cure What Ails Biomedical Research and Healthcare

The Future of Precision Cancer Medicine, Inaugural Symposium, MIT Center for Precision Cancer Medicine, December 13, 2018, 8AM-6PM, 50 Memorial Drive, Cambridge, MA

Live Conference Coverage @Medcity Converge 2018 Philadelphia: Oncology Value Based Care and Patient Management

2016 BioIT World: Track 5 – April 5 – 7, 2016 Bioinformatics Computational Resources and Tools to Turn Big Data into Smart Data

The Need for an Informatics Solution in Translational Medicine

 

 

 

 

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LIVE eProceedings Day Two – The 14th Annual Personalized Medicine Conference: The Business of Personalization, November 15, 2018, HMS, Boston

Real Time Coverage: Aviva Lev-Ari, PhD, RN

 

PART II

 

The Business of Personalization

The successful implementation of [personalized medicine] will depend on the embrace of [its] principles in the business community.

 RAJU KUCHERLAPATI, PH.D.
Paul C. Cabot Professor of Genetics, Harvard Medical School

*** Speakers will be added to the schedule on a rolling basis as they are confirmed. ***

7:00 a.m.
Registration and Continental Breakfast

Joseph B. Martin Conference Center at Harvard Medical School
77 Avenue Louis Pasteur, Boston, MA 02115

8:00 a.m.
Opening Remarks

SPEAKER | Stephen L. Eck, M.D., Ph.D., Chief Medical Officer, Immatics U.S. Inc; Board Chair, Personalized Medicine Coalition

8:10 a.m.
Pioneering Precision: Inside the Pharmaceutical Industry’s Push Toward Personalized Medicine — A Fireside Chat

MODERATOR | Meg Tirrell, Reporter, CNBC

Daniel O’Day, CEO, Roche Pharmaceuticals

8:55 a.m.
Considering Costs: Evaluating Emerging Pharmaceutical and Insurance Industry Business Models in Personalized Medicine

The pharmaceutical industry is deeply invested in commercializing personalized therapies that must recoup fixed development costs from smaller patient populations covered by health insurance companies that are increasingly concerned about rising health care costs. In that context, this diverse panel will explore the viability of the business model for developing and paying for personalized medicines, tackling issues related to costs, prices, and access.

MODERATOR | Meg Tirrell, Reporter, CNBC

Peter Juhn, M.D., M.P.H., Global Head of Value-Based Partnerships, Amgen

Nick Leschly, CEO, Bluebird Bio

Michael Sherman, M.D., Chief Medical Officer, Senior Vice President, Harvard Pilgrim Health Care

Sean Tunis, M.D., Founder, CEO, Center for Medical Technology Policy

9:55 a.m.
Networking Break

Light refreshments provided.

Sponsored By

10:25 a.m.
Reinventing Research: Are Adaptive Platform Trials the Model of the Future? (A Harvard Business School Case Study)

Recognizing that traditional randomized controlled clinical trials can only study the safety and efficacy of a single therapy in one large population of patients, researchers in personalized medicine increasingly hope that “adaptive platform trials,” which employ advanced statistical techniques to simultaneously test the effectiveness of several personalized treatments in multiple sub-populations of patients, may be the key to new drug approvals in the future. Adaptive platform trials may make drug development more efficient by revealing which of several drug candidates are most promising for which patients, but maximizing the potential of these trials requires unprecedented collaboration among the institutions conducting and sponsoring research on various personalized treatments — and no obvious business models have emerged.

During this interactive case study discussion, professors from Harvard Business School will help us examine how researchers at the Dana-Farber Cancer Institute considered and addressed myriad challenges in their effort to design and operationalize an adaptive platform trial for glioblastoma patients, a deadly disease state for which there are few existing treatment options.

PRESENTED BY

Richard Hamermesh, D.B.A., Co-Faculty Chair, Harvard Business School Kraft Precision Medicine Accelerator; and

Ariel D. Stern, Ph.D., Assistant Professor, Technology and Operations Management Unit, Harvard Business School

11:40 a.m.
The 14th Annual Leadership in Personalized Medicine Award

INTRODUCTION | Steven D. Averbuch, M.D., Vice President, Head of Precision Medicine, Bristol-Myers Squibb

  • Ellen V. Sigal head of Friends of Cancer Research
  • Advanced science by Diagnostics Tests
  • Cancer Moonshot Program
  • Revolution therapies brought to market by Sigel’s sponsorship

AWARDEE | Ellen V. Sigal, Ph.D., Chairperson, Founder, Friends of Cancer Research

Friendly conversation:

  • Thanks to PMC
  • sister die on breast cancer at 40 with child of 4 1/2.
  • appointed to celebrate 20th year of American Cancer Association – Funding for Research, money spend in Washington is for Patients.
  • After ten years, interested in measurement of achieving evaluation, FDA structure was of interest.
  • Precision Medicine: biomarkers and targets for patients to define success for each patient, WHat is the right population for any drug, responders to drug therapy, if no response, change the drug.
  • Patient perspective: Challenges: 90% are treated in the Community and they need a second opinion, insurance, access, clinical trials done out of the community in Academic hospitals – patients are scared to death. Patients are asking for options: Right testing, access to testing involve insurance
  • combination therapy  – 6-8 months in advance,
12:10 p.m.
Bag Lunch
1:10 p.m.
Predicting and Preventing: Evaluating Progress Toward Personalized Medicine

The original architects of the personalized medicine paradigm envisioned an era in which clinicians could predict, prevent and treat disease based on an improved understanding of how human biology interacts with external environments. During this session, a panel of experts will examine our progress on each of these fronts during a wide-ranging conversation about personalized medicine’s past, present and future.

MODERATOR | Cynthia Casson Morton, Ph.D., William Lambert Richardson Professor of Obstetrics, Gynecology and Reproductive Biology, Harvard Medical School

  • 1 Million volunteer participants for genome sequencing and microbiome data
  • PM Past, Present and Future
  • Issues that are rapidly evolving: Physician, Patients

Birgit Funke, Ph.D., F.A.C.M.G., Vice President, Clinical Affairs, Veritas Genetics; Associate Professor of Pathology (Part-Time), Harvard Medical School

  • Risk prevention,
  • driving DOWN operating cost curation of the Genome

Luba Greenwood, J.D., Strategic Business Development and Corporate Ventures, Verily (an Alphabet company)

  • view on diagnostics from Roche, CHemist and lawyer, Venture capital, beyond Genomics, from diagnosis to prognosis,at Verily projects tapping into the entire life span aspect of health
  • treatment solution therapeutics except og Oncology threatment is a strugle in the genomics field and pharmaco-genomics
  • Power the patients vs Genomics in Diabetes
  • Diagnostics in use to keep patients OUT of hospitals – management of chronic diseases
  • Patient need to own the genome data not a Databank

Keith Stewart, M.B., CH.B., Carlson and Nelson Endowed Director, Center for Individualized Medicine, Mayo Clinic

  • Hematologist, genomics apply genomics for detection of predisposition, inherited , Health genome sequencing,
  • Barriers to deploy genomics: Knowledge, readiness of providers, cost of uninsured,
  • Diagnostics high value low cost
  • drug adherence, pharmacists to be involved in drug adherence before refill
2:10 p.m.
Assessing the Assays: Determining the Clinical and Economic Utility of Genomic Sequencing

Advocates for personalized medicine have contended that genomic sequencing can deliver clinical and economic value to patients and the health system by allowing providers to more efficiently diagnose disease and develop treatment plans. Following increased use of genomic sequencing in clinical settings, many stakeholders, including payers, have begun to examine that value proposition more closely. During this session, a pharmaceutical industry representative, a payer, and a health economist will discuss the status and future of the emerging evidence regarding the clinical and economic utility of genomic sequencing, including studies recently commissioned by the Personalized Medicine Coalition.

MODERATOR Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, Personalized Medicine Coalition

  • genetic profiling, adopt policy and procedures for mass deployment of NGS
  • show that it works – demonstrate value, payers and providers
  • a little more that evidence exist for payer to cover
  • rare diagnosed disease

Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana

  • labs, payers, providers, pharma — the GAP to be bridged
  • opportunities to prevent and treat disease
  • Payer, MDs, cost and impact, markers,
  • Humana has a research division Use Testing to find value, pharmacogenomics  – on Medicare, Medicaid patients
  • cost of doing the test vs not doing this test – assess value
  • pharmacisit, economist, statisticians – CMS – provide data on what is covered and what is not Humana: any missed opportunities, MD order tests of no impact per medical record
  • What test needed to be ordered? Patient stay healthy
  • NGS $650 – $2000 in 2018, in 2016 it was $25,000 cost of testing, cost of drugs
  • show us any value as good value – avoiding patient going to MDs Office, Hospital, ER – cost increase due to Pharmacogenomics testing $5K per test
  • Guidelines on ordering genomic testing, AI can assist providers, MDs need to catch up on a weekly basis
  • CMS Guideline: every test ordered must guide treatment otherwise not covered

Scott Ramsey, M.D., Ph.D., Full Member, Fred Hutchinson Cancer Research Center; Director, Hutchinson Institute for Cancer Outcomes Research

  • Value and utility are interconnected
  • cost effectiveness of NGS in melanoma: single gene testing – EGFR vs NGS – help clinicians to evaluate Lung Cancer
  • Flariton Database, 300 centers  – 140,000 – Patients got NGS – 7% ADDITIONAL patients founded mutations beyond EGFR
  • Survival in this cohort NGS vs EGFR – improved survival 6 month longer, mean survival 3 weeks long, not significant.
  • Increased survivals, why? cost of sequencing  – #14 most influential – cost does not drive value
  • #1 drug cost was the factor
  • #2 survival
  • marginal cost in platform comparison
  • Pricing of Testing NGS and Targeted therapy represent a threat to adoption of Genomics in Medicine
  • disparities and access – cost and patients: Partners and Mayo clinic patients are lucky

 

3:10 p.m.
PhRMA Foundation Challenge Awards: Developing Value Assessment Strategies That Align With Personalized Medicine

INTRODUCTION | Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, Personalized Medicine Coalition

PRESENTER | Shreeram Aradhye, M.D., Head of Global Medical Affairs, Chief Medical Officer, Pharmaceuticals, Novartis; Board Member, PhRMA Foundation

#1 Prize $50,000 – Dr. Garrison, UK

#2 Prize $25,000 – Dr. Robim Hayeems, Hospital for Sick Children Institute, Toronto, Canada

#3 Prize @ $10,000  – Dr. A Le, PharmD., PhD, Western University of Health Sciences

3:20 p.m.
Networking Break

Light refreshments provided.

3:50 p.m.
Impasse or Inflection Point? — An Investment Analysis

Sustaining the pace of innovation in personalized medicine will require continued investment in new initiatives, but the financial outlook for the field remains unclear. In that context, this panel of investors will examine whether personalized medicine is at an impasse, an inflection point or somewhere in between.

MODERATOR | William A. Sahlman, Ph.D., Baker Foundation Professor, Harvard Business School

  • market – can it sustain the opportunity – winners and losers
  • innovative financial models
  • Biotech IPO, VC, windows slam shut, drug failure – drivers and non
  • Increasing return to scale: AI, NGS, screening, – foreign money, China
  • Tsinghua University went back to China from Silicon Valley

Cary Pfeffer, M.D., Partner, Third Rock Ventures

  • was a decade at Biogen, MS indication drug, no biomarkers for patients – efficacy was in 50% non respondents 25%
  • Genomic sequencing to identify patient populations – no good effective medicine without target therapy
  • Mayocardia – drug in CVD for patients identified by Genomics
  • Genomics information needed to develop drugs

Michael Pellini, M.D., Managing Partner, Section 32; Board Member, Personalized Medicine Coalition

  • Impasse or Inflection Point? it s Inflection Point NOT an Impasse
  • Diagnostics component inside 4.8 Trillion in the therapeutics selection in the system as a whole
  • Foundation Medicine saw Roche as Big brother with International reach
  • Patients and Consumers will force in five years figuring out – every diagnosis of cancer will be sequenced and the infrastructure to interpret results and paid for

Salveen Richter, C.F.A., Vice President, Research Division, Goldman Sachs

  • innovative and disruptive, orphan drugs, Health IT, US Market 3 trillion – size of the opportunity 80% genetically driven
  • Cancer, CART therapy, easier to pay by performance, cost of the drug itself. profit in the 1st generation od Pharma manufacturers
  • One time pricing vs further indications, annuity type system, Hemophilia – $19Million market,
  • Europe successful in financing Health care — in the US — system must change – investment will flee, to fund pricing drug is key in changing the system CART Pricing is still difficult to pay for
  • Sequencing cost plunged, public investors placing funding in start ups even without return in the horizon, companies with multiple modalities spurring innovation – confusing in the investment side, technologies become obsolete very fast
  • Europe vs US, China is different no regulation like FDA,talent from US Pharma went back to China

 

4:50 p.m.
Closing Remarks

SPEAKER | Edward Abrahams, Ph.D., President, Personalized Medicine Coalition

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Contact: Christopher J. Wells

Personalized Medicine Coalition

cwells@personalizedmedicinecoalition.org

202-589-1770

FOR IMMEDIATE RELEASE

 

New York Times vs. Personalized Medicine?

PMC President: Times’ Critique of Streamlined Regulatory Approval for Personalized Treatments ‘Ignores Promising Implications’ of Field

WASHINGTON (June 13, 2018) — In response to an editorial published on June 9 by the New York Times titled “Easier Drug Approval, at What Price?,” Personalized Medicine Coalition (PMC) President Edward Abrahams today defends a series of decisions by the U.S. Food and Drug Administration (FDA) over the last several years that have streamlined the regulatory review process for personalized medicines.

“Unlike FDA, which has been an engine for innovation under the direction of Scott Gottlieb and his predecessors, ‘Easier Drug Approval, at What Price?’ ignores the promising implications of reforms in regulatory science that FDA has put in place to facilitate a new appreciation of how different individuals respond to selected treatments,” Abrahams said.

As PMC underlined earlier this year in a report titled Personalized Medicine at FDA: 2017 Progress Report, personalized medicines now account for one of every four drugs the agency approves. The Times’ editorial, which was also published online under the headline “Easier Drug Approval Isn’t Cutting Drug Prices,” contends that “it’s not clear that people, as opposed to drug companies, are feeling much benefit” from the streamlined regulatory review pathways that bring personalized treatments to market faster.

Abrahams disagrees, noting that in non-small cell lung cancer, for example, a disease that was nearly untreatable 20 years ago, there are now multiple drugs on the market that target a patient’s particular tumor. As indicated by the U.S. National Cancer Institute (NCI), these treatments “improve the survival of subsets of patients with metastatic disease.”

Furthermore, because targeted therapies zero in on specific cancerous mutations, doctors can use diagnostic tests to identify with much greater certainty the patients who will likely benefit from them, sparing expenses and side effects for those will not. That logic underpins FDA’s decision to streamline its regulatory processes, to ensure that patients who will benefit from promising targeted therapies — many of whom have few remaining options — can access those treatments without unnecessary delay.

“By putting in place smarter policies to encourage the efficient development of personalized drugs whose safety and efficacy profiles are often higher than one-size-fits-all, trial-and-error treatments, FDA serves the interests not only of patients but also the health system, which spends too much money on ineffective treatments,” Abrahams said.

To evaluate the American public’s interest in personalized medicine, PMC and GenomeWeb recently commissioned Public Perspectives on Personalized Medicine: A Survey of U.S. Public Opinion, which was published in May. Two-thirds of Americans indicated in the survey that they appreciate personalized medicine’s potential, and the majority expressed concerns about whether they will have access to personalized tests and treatments in the future.

###

About the Personalized Medicine Coalition:
The Personalized Medicine Coalition, representing innovators, scientists, patients, providers and payers, promotes the understanding and adoption of personalized medicine concepts, services and products to benefit patients and the health system. For more information, please visit www.personalizedmedicinecoalition.org.

SOURCE

From: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>

Date: Wednesday, June 13, 2018 at 3:15 PM

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

Subject: Re: New York Times vs. Personalized Medicine?

 

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PMC Comment Letter on Centers for Medicare & Medicaid Services: Innovation Center New Direction

Guest Author: Cynthia A. Bens, Vice President, Public Policy, PMC

cbens@personalizedmedicinecoalition.org

NOVEMBER 27, 2017

In response to a Wall Street Journal op-ed and request for information about innovative ways to pay for and deliver health care in the U.S., the Personalized Medicine Coalition has encouraged the Centers for Medicare & Medicaid Services (CMS) to spearhead models that empower physicians to move away from the current standard of care when patient outcomes can be improved by tailoring care to a patient’s genetics and other factors:

In keeping with PMC’s mission to underline the significance of personalized medicine to patients and the health system, the Coalition’s comment letter contends that personalized medicine products and services can increase the overall value of dollars spent by improving health outcomes.

CMS’ previous efforts to advance new payment models, the letter notes, were met with resistance largely because they focused on reducing overall health care costs without adequately considering whether those reductions would result in a disproportionate decrease in the outcomes that matter to patients.

PMC indicates in the letter that the guiding principles put forth in CMS’ request for information provide “reasonable assurance” that the agency plans to proceed at “a more measured pace” going forward.

“We believe that personalized medicine has the potential to help CMS deliver on its goal of [affordable, accessible health care] if [the agency] focuses on maximizing individual patient outcomes, if new models are fully evaluated before large-scale implementation, if payment is not rooted in current standard of care, and if physicians have the flexibility to tailor care based on a patient’s genetics and other factors,” the letter reads.

Please contact Cynthia A. Bens, Vice President, Public Policy, at cbens@personalizedmedicinecoalition.org with questions about PMC’s comment letter.

###

Personalized Medicine Coalition
1710 Rhode Island Ave. NW; Suite 700
Washington, D.C. 20036
Blog: Education & Advocacy
Twitter: @PerMedCoalition
www.PersonalizedMedicineCoalition.org

SOURCE

From: Personalized Medicine Coalition <messages@app.production.membersuite.com>
Reply-To: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>
Date: Monday, November 27, 2017 at 2:58 PM
To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>
Subject: PMC to CMS: To Increase Value, Empower Physicians to Tailor Care, Optimize Outcomes

 and

From: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>

Date: Tuesday, November 28, 2017 at 7:34 AM

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

Subject: Re: PMC to CMS: To Increase Value, Empower Physicians to Tailor Care, Optimize Outcomes

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UPDATED 12/05/2020

 

 

 

 

 

 

In the future, George Church believes, almost everything will be better because of genetics. If you have a medical problem, your doctor will be able to customize a treatment based on your specific DNA pattern. When you fill up your car, you won’t be draining the world’s dwindling supply of crude oil, because the fuel will come from microbes that have been genetically altered to produce biofuel. When you visit the zoo, you’ll be able to take your children to the woolly mammoth or passenger pigeon exhibits, because these animals will no longer be extinct. You’ll be able to do these things, that is, if the future turns out the way Church envisions it—and he’s doing everything he can to see that it does.

UPDATED 12/05/2020

George Church backs a startup solution to the massive gene therapy manufacturing bottleneck

Source: https://endpts.com/george-church-backs-a-startup-solution-to-the-massive-gene-therapy-manufacturing-bottleneck/
Jason Mast: Associate Editor
George Church and his graduate students have spent the last decade seeding startups on the razor’s edge between biology and science fiction: gene therapy to prevent aging, CRISPRed pigs that can be used to harvest organs for transplant, and home kits to test your poop for healthy or unhealthy bacteria. (OK, maybe they’re not all on that razor’s edge.)

But now a new spinout from the Department of Genetics’ second floor is tackling a far humbler problem — one that major company after major company has stumbled over as they tried to get cures for rare diseases and other gene therapies into the clinic and past regulators: How the hell do you build these?

CEO Lex Vovner of 64x Bio

 

 

 

“There’s a lot happening for new therapies but not enough attention around this problem,” Lex Rovner, who was a post-doc at Church’s lab from 2015 to 2018, told Endpoints News. “And if we don’t figure out how to fix this, many of these therapies won’t even reach patients.”

This week, with Church and a couple other prominent scientists as co-founders, Rovner launched 64x Bio to tackle one key part of the manufacturing bottleneck. They won’t be looking to retrofit plants or build gene therapy factories, as Big Pharma and big biotech are now spending billions to do. Instead, with $4.5 million in seed cash, they will try to engineer the individual cells that churn out a critical component of the therapies.

George Church
The goal is to build cells that are fine-tuned to do nothing but spit out the viral vectors that researchers and drug developers use to shuttle gene therapies into the body. Different vectors have different demands; 64x Bio will look to make efficient cellular factories for each.

“While a few general ways to increase vector production may exist, each unique vector serotype and payload poses a specific challenge,” Church said in an emailed statement. “Our platform enables us to fine tune custom solutions for these distinct combinations that are particularly hard to overcome.”

Before joining Church’s lab, Rovner did her graduate work at Yale, where she studied how to engineer bacteria to produce new kinds of protein for drugs or other purposes. And after leaving Church’s lab in 2018, she initially set out to build a manufacturing startup with a broad focus.

Yet as she spoke with hundreds of biotech executives on LinkedIn and in coffee shops around Cambridge, the same issue kept popping up: They liked their gene therapy technology in the lab but they didn’t know how to scale it up.

“Everyone kept saying the same thing,” Rovner said. “We basically realized there’s this huge problem.”

The issue would soon make headlines in industry publications: bluebird delaying the launch of Zynteglo, Novartis delaying the launch of Zolgensma in the EU, Axovant delaying the start of their Parkinson’s trial.

Part of the problem, Rovner said, is that gene therapies are delivered on viral vectors. You can build these vectors in mammalian cell lines by feeding them a small circular strand of DNA called a plasmid. The problem is that mammalian cells have, over billions of years, evolved tools and defenses precisely to avoid making viruses. (Lest the mammal they live in die of infection).

There are genetic mutations that can turn off some of the internal defenses and unleash a cell’s ability to produce virus, but they’re rare and hard to find. Other platforms, Rovner said, try to find these mutations by using CRISPR to knock out genes in different cells and then screening each of them individually, a process that can require hundreds of thousands of different 100-well plates, with each well containing a different group of mutant cells.

“It’s just not practical, and so these platforms never find the cells,” Rovner said.

64x Bio will try to find them by building a library of millions of mutant mammalian cells and then using a molecular “barcoding” technique to screen those cells in a single pool. The technique, Rovner said, lets them trace how much vector any given cell produces, allowing researchers to quickly identify super-producing cells and their mutations.

The technology was developed partially in-house but draws from IP at Harvard and the Wyss Institute. Harvard’s Pam Silver and Wyss’s Jeffrey Way are co-founders.

The company is now based in SoMa in San Francisco. With the seed cash from Fifty Years, Refactor and First Round Capital, Rovner is recruiting and looking to raise a Series A soon. They’re in talks with pharma and biotech partners, while they try to validate the first preclinical and clinical applications.

Gene therapy is one focus, but Rovner said the platform works for anything that involves viral vector, including vaccines and oncolytic viruses. You just have to find the right mutation.

“It’s the rare cell you’re looking for,” she said.

AUTHOR
Jason Mast
Associate Editor
jason@endpointsnews.com
@JasonMMast
Jason Mas

 

In 2005 he launched the Personal Genome Project, with the goal of sequencing and sharing the DNA of 100,000 volunteers. With an open-source database of that size, he believes, researchers everywhere will be able to meaningfully pursue the critical task of correlating genetic patterns with physical traits, illnesses, and exposure to environmental factors to find new cures for diseases and to gain basic insights into what makes each of us the way we are. Church, tagged as subject hu43860C, was first in line for testing. Since then, more than 13,000 people in the U.S., Canada, and the U.K. have volunteered to join him, helping to establish what he playfully calls the Facebook of DNA.

 

Church has made a career of defying the impossible. Propelled by the dizzying speed of technological advancement since then, the Personal Genome Project is just one of Church’s many attempts to overcome obstacles standing between him and the future.

 

“It’s not for everyone,” he says. “But I see a trend here. Openness has changed since many of us were young. People didn’t use to talk about sexuality or cancer in polite society. This is the Facebook generation.” If individuals were told which diseases or medical conditions they were genetically predisposed to, they could adjust their behavior accordingly, he reasoned. Although universal testing still isn’t practical today, the cost of sequencing an individual genome has dropped dramatically in recent years, from about $7 million in 2007 to as little as $1,000 today.

 

“It’s all too easy to dismiss the future,” he says. “People confuse what’s impossible today with what’s impossible tomorrow.”, especially through the emerging discipline of “synthetic” biology. The basic idea behind synthetic biology, he explained, was that natural organisms could be reprogrammed to do things they wouldn’t normally do, things that might be useful to people. In pursuit of this, researchers had learned not only how to read the genetic code of organisms but also how to write new code and insert it into organisms. Besides making plastic, microbes altered in this way had produced carpet fibers, treated wastewater, generated electricity, manufactured jet fuel, created hemoglobin, and fabricated new drugs. But this was only the tip of the iceberg, Church wrote. The same technique could also be used on people.

 

“Every cell in our body, whether it’s a bacterial cell or a human cell, has a genome,” he says. “You can extract that genome—it’s kind of like a linear tape—and you can read it by a variety of methods. Similarly, like a string of letters that you can read, you can also change it. You can write, you can edit it, and then you can put it back in the cell.”

 

This April, the Broad Institute, where Church holds a faculty appointment, was awarded a patent for a new method of genome editing called CRISPR (clustered regularly interspersed short palindromic repeats), which Church says is one of the most effective tools ever developed for synthetic biology. By studying the way that certain bacteria defend themselves against viruses, researchers figured out how to precisely cut DNA at any location on the genome and insert new material there to alter its function. Last month, researchers at MIT announced they had used CRISPR to cure mice of a rare liver disease that also afflicts humans. At the same time, researchers at Virginia Tech said they were experimenting on plants with CRISPR to control salt tolerance, improve crop yield, and create resistance to pathogens.

 

The possibilities for CRISPR technology seem almost limitless, Church says. If researchers have stored a genetic sequence in a computer, they can order a robot to produce a piece of DNA from the data. That piece can then be put into a cell to change the genome. Church believes that CRISPR is so promising that last year he co-founded a genome-editing company, Editas, to develop drugs for currently incurable diseases.

Source: news.nationalgeographic.com

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