The Vector

Volume 9, Issue 3: March 2020


Editorial Team

Melvin Rincon, M.D., Ph.D. – Editor, The Vector
Edith Pfister, Ph.D. – Associate Editor, The Vector
Karen Bulaklak, Ph.D. – Junior Editor, The Vector

Inside This Issue

Leadership Message
Breaking Through
Society News
Career Center
Public Policy
Industry News

Leadership Message

COVID-19 Update

At this time, there is no plan to cancel or postpone ASGCT’s 23rd Annual Meeting. Society staff and elected leadership are tracking all current travel restrictions and ongoing guidance developments regarding COVID-19 from the World Health Organization (WHO), the Centers for Disease Control and Prevention (CDC), and the Massachusetts Department of Public Health. The health and safety of our attendees is our top priority, and we will continue to follow state and federal guidances related to COVID-19 and update our members and attendees in accordance.

We recognize that this situation is evolving rapidly, as are the recommendations from public heath authorities. If any future recommendations trigger any changes to our plans, we will communicate those changes immediately. As always, we will prioritize the health and safety of all of our meeting attendees.

Vote in the Board of Directors Election Starting March 16

Along with the clocks and the seasons, ASGCT springs forward with our 2020 elections and the last chance to renew your membership for 2020. ASGCT members will elect five of their own to the Society’s Board of Directors from Monday, March 16, through Friday, April 10. All full and transitional members are eligible to vote, and I hope all of you will take a moment to ensure your membership is current before the ballot is sent next week. I’m proud to say we have many accomplished candidates vying for spots on the Board, and I look forward to seeing the incoming Board members guide our Society forward.

Here are the candidates running for spots on the Board:

Vice President (Term of Office: 2020-2021):

Hans-Peter Kiem, MD, PhD
Fred Hutchinson Cancer Research Center

David Schaffer, PhD
University of California-Berkeley       

Treasurer (2020-2023):

Paloma Giangrande, PhD
Moderna Therapeutics

Rachel Salzman, DVM
SwanBio Therapeutics

At-Large Director (2020-2023):

Marcela Maus, MD, PhD
Harvard University

Feng Zhang, PhD
Massachusetts Institute of Technology

At-Large Director (2020-2023):

Alessandro Aiuti, MD, PhD
Ospedale San Raffaele

Federico Mingozzi, PhD
Spark Therapeutics

At-Large Director, Scientific & Research Council Chair (2020-2023):

Alberto Auricchio, MD
Telethon Institute of Genetics and Medicine

Anastasia Khvorova, PhD
University of Massachusetts Medical School

Also, if you have not done so already, please renew your membership by tomorrow, March 13. Make sure you maintain uninterrupted access to your exclusive member benefits, like subscriptions to Molecular Therapy, eligibility for Annual Meeting registration discounts, and more.

Thank you all for your continued support of ASGCT, and I look forward to an eventful spring and a wonderful Annual Meeting!

Best,

Terence R. Flotte, M.D.
ASGCT Secretary

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Breaking Through

GPR108 Is a Highly Conserved AAV Entry Factor

Dudek AM, Zabaleta N, Zinn E, Pillay S, Zengel J, Porter C, Franceschini JS, Estelien R, Carette JE, Zhou GL, Vandenberghe LH

DOI: https://doi.org/10.1016/j.ymthe.2019.11.005

Summary by Edith Pfister, Ph.D.‚Äč

Despite the success of adeno-associated virus (AAV) in gene therapy, many questions about their basic biology remain. How these vectors enter the cell and deliver their cargo to the nucleus is of interest. This process may influence the cellular trophism and immune response to various AAV vectors and could enable engineering of new and useful vectors or novel ways to enhance transduction. Some, but not all AAVs rely on a conserved receptor, AAVR, for cellular entry but other factors have remained elusive. In a paper from the February issue of Molecular Therapy, Dudek et al used a clever approach to identify GPR108 as an upstream factor in the pathway with AAVR.

Using rh32.33, which does not require AAVR, the authors performed a genome-wide CRISPR screen to identify GPR108. Using GPR108 knockout cells, they show that most AAV capsids, including PHP-B and engineered capsids, require GPR108 for efficient transduction. The notable exception is the evolutionarily divergent AAV5. Dependence on GPR108 requires a region in the VP1 domain. GPR108 functions at a step downstream of binding to the cell surface and internalization but upstream of nuclear entry, suggesting that GPR108 may facilitate escape from the endosome or from the trans Golgi network.

There are still many gaps in our knowledge of AAV, but this paper fills one such gap. New techniques like the ones used here will continue to provide insight into the basic biology of AAVs and may allow a fine mapping of AAV tropism throughout the body. As we continue to refine our knowledge, AAVs can be engineered more precisely and effectively.

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Society News


Save the Date for the 2020 Policy Summit

The second annual ASGCT Policy Summit will be held Sept. 23-25, 2020 at the Renaissance Washington, DC Downtown Hotel. We'll host two and a half days of programming, once again focusing on the regulatory, payment policy, and ethical aspects of gene and cell therapy. Please watch the ASGCT website for more updates in the coming months.

Nominate a Colleague for the Jerry Mendell Award

Do you know an ASGCT member who has supported the translation of gene and cell therapy from a preclinical to clinical research settings? Nominate him or her for the inaugural Jerry Mendell Award for Translational Science. The award recognizes the extensive work required to bring gene and cell therapies to clinical trial.

Renew Your ASGCT Membership by March 13

Tomorrow, March 13, is the deadline to renew your ASGCT membership for 2020. Make sure you maintain ininterrupted access to your benefits, including publication discounts and subscriptions to Molecular Therapy, as well as eligibility for Annual Meeting registration discounts.

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Career Center


Are you looking for a job in the field of gene and cell therapy? Check out the ASGCT Job Bank and sign up to receive alerts for open jobs in your area.

If you're from a recruiting institution, advertise in the Featured Jobs section to target the 3,000+ audience of The Vector.

Featured Jobs

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Public Policy


Attend Policy Workshops Before the Annual Meeting

ASGCT is hosting two policy-focused Pre-Meeting Workshops on May 11. During Commercialization I: Opportunities and Challenges for Global Clinical Trials, the morning session, regulators will provide recommendations for executing clinical trials in a variety of countries. Wilson Bryan, MD, director of the Office of Tissues and Advanced Therapies and Jan Mueller-Berghaus, MD, from the EMA Committee for Advanced Therapies will discuss international regulatory alignment on clinical program design recommendations.

In the afternoon, Commercialization II: Value, Evidence, and Reimbursement will cover new perspectives on valuing gene and cell therapies in the U.S. and Europe, and offer a provider perspective on coverage and reimbursement. The speaker lineup includes:

  • Peter Marks, M.D., Ph.D., director of FDA CBER, who will discuss FDA evidence requirements for gene therapy approval, with implications for labeled indications and coverage
  • John O’Brien, former senior advisor to HHS Secretary Alex Azar, who will discuss coverage and reimbursement from the viewpoint of the Medicare program
  • Priscilla Parrilla, director of pharmacy operations with the Texas Vendor Drug Program, who will discuss state Medicaid plan amendments that allow milestone-based contracts

This year we’re also bringing back the Clinical Trials Training workshops as two half-day events. The morning workshop will focus on how to conduct early-phase clinical trials, and will be of most use to novice clinical investigators. Barry Byrne, M.D., Ph.D., director of the University of Florida, Powell Gene Therapy Center, and Chris Doering, Ph.D., associate professor of pediatrics at Emory University School of Medicine, will discuss the pathway to obtaining funding and the ingredients for a strong IND. Jeffrey Medin, Ph.D., MACC Fund professor at the Medical College of Wisconsin, will talk about his experience moving preclinical research into clinical trials for Fabry disease.

The afternoon workshop goes in-depth on launching trials in academic and commercial settings. Michelle Berg, president – GMP nucleic acids at Aldevron, will explain how to plan for manufacturing success in early gene therapy development. Additionally, Angela Schulz, M.D., head of the research group for childhood neurodegenerative disease at the University Medical Centre Hamburg-Eppendorf, will talk about successful clinical endpoints.

ASGCT Supports Patient Access to CAR T-Cell Therapy

ASGCT was among 15 supporting groups of a congressional letter requesting CMS to establish a new Medicare Severity-Diagnosis Related Group (MS-DRG) for CAR T-cell therapy, as the new technology add-on payment (NTAP) is anticipated to expire at the end of September. The letter, signed by 76 bipartisan members of Congress, states that a permanent payment mechanism will be necessary to avoid leaving providers with large deficits in reimbursement that would likely halt patient access. ASGCT supports adequate reimbursement to providers so that patient access to gene and cell therapies is as broad as possible.

Industry News


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2020

23rd Annual Meeting

May 12-15 | Boston