The Vector

Volume 8, Issue 11: November 2019


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

Meetings, Membership, and More

There’s an incredible energy surrounding ASGCT as we dive head-first into winter this year. Last week’s Policy Summit was a resounding success in both attendance and enthusiasm surrounding the topics; the Call for Abstracts is ready to go out to members with submissions opening on Nov. 18; and we set ASGCT membership records in total members, new members, and member retention. This is the best time in ASGCT’s history to be involved with the Society, and I’m excited to be the one who tells you why.

With a fantastic Policy Summit, including a revelatory talk from NIH Director Francis Collins, M.D., Ph.D., behind us, much of the Society is shifting into what’s shaping up to be another record-setting ASGCT Annual Meeting. Abstract submission for the 23rd Annual Meeting opens on Monday, Nov. 18, and as always Associate Members who are the first and presenting authors on an abstracts are offered free registration to the meeting.

Even taking into account our member discounts, it’s difficult to beat free. I know a great many of our Associate Members will submit their work and take advantage of what will be an incredible Annual Meeting—the Hynes Convention Center offers more than double the space available at the 2019 meeting, and is ASGCT’s first meeting in a convention center since 2013.

With all that extra space, ASGCT is expecting record representation from our membership at the Annual Meeting. For the 2019 membership year, we ended with 3,530 members and more than 1,400 new members, both shatter previous Society records. And now is the time to maintain this incredible growth, because the ASGCT renewal campaign is in full swing. You’ve likely received an initial notification from ASGCT staff if you’re membership is coming due, but they’ll be following up in the coming weeks to keep everyone in the loop. Renew your membership to maintain the great benefits that come with being a part of ASGCT!

Finally, thank you to the nearly 400 attendees who made the 2019 ASGCT Policy Summit a resounding success, and to the dedicated ASGCT staff who put it on. A policy discussion open to the public was a new initiative to ASGCT this year and the interest from our attendees, and attendance grew by more than four-times compared to the 2018 Value Summit, the invite-only precursor to the Policy Summit. The response to the event was overwhelmingly positive, and I’m looking forward to announcing dates and locations for the 2020 Policy Summit in the near future.

November is a time to be thankful, and I thank you all for everything you do to support and promote ASGCT.

Best,
Guangping Gao, Ph.D.
ASGCT President

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Breaking Through


A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion

DOI: https://doi.org/10.1016/j.ymthe.2019.09.003

Research and summary by Yu-Chen Chen, Ning-Xin Ma, Zi-Fei Pei, Zheng Wu, Fabricio H. Do-Monte , Susan Keefe, Emma Yellin, Miranda S. Chen, Jiu-Chao Yin, Grace Lee, AngĂ©lica Minier-Toribio, Yi Hu, Yu-Ting Bai, Kathryn Lee, Gregory J. Quirk, and Gong Chen

When our brain and spinal cord are attacked by an injury, neurons (the functional cells of our central nerve system) may be lost permanently. This is because neurons can’t divide so they lose the ability to regenerate themselves. Our lab has been trying to replace the lost neurons for years and they focus on billions of supportive glial cells inside the brain. Unlike neurons, glial cells divide and become activated after brain injury. In a work published in 2014 in Cell Stem Cell, we successfully showed it was possible to reprogram glial cells in the brain into functional neurons by delivering a single protein called NeuroD1. However, it remains unknown whether this strategy can generate a sufficient number of new cells needed to repair damaged brain tissue following a stroke.

In this work recently published in Molecular Therapy, we took advantage of adeno-associated viral vectors (AAVs) for the cell conversion purpose and the results were quite remarkable. In a mouse ischemic stroke model, we showed the NeuroD1 AAV treatment can regenerate about a third of the total lost neurons via preferentially targeting reactive, scar-forming glial cells. The conversion of those reactive glial cells into neurons also protected another third of the neurons lost from injury. We also showed that replaced neurons were fully functional and formed the needed neural connections in the brain. Importantly, NeuroD1 gene therapy led to significant functional improvements of the mice after a stroke. We performed several tests of fine movements with the forelimbs of the stroke mice, which showed about a 60 percent improvement up to two months after receiving the NeuroD1 therapy. Our collaborator Gregory Quirk and his group from the University of Puerto Rico showed similar improvements in the ability of rats to recover from stroke-related deficits in memory. Our work suggests that cell conversion can potentially help the functional recovery of stroke injury in the brain for the first time.

Irreversible loss of neurons is a major obstacle for the recovery of many neurodegenerative diseases and brain injuries. Our work provides a potential therapeutic method to restore the neurons lost from disease or injuries and to help recovery from functional deficits. While further study is needed, these findings in rodents offer encouraging evidence that treatments to repair the brain after a stroke or other injury may be on the horizon.

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Society News


Submit Your Abstracts Starting November 18

Abstract submission for ASGCT’s 23rd Annual Meeting opens Monday, Nov. 18. Submitting an abstract is a great opportunity to present and discuss your research with peers and leaders in the field of gene and cell therapy. For more information on submitting an abstract, including important dates and award opportunities, visit the abstracts section on the Annual Meeting website.

Renew Your ASGCT Membership

Don't forget to renew your ASGCT membership before the end of the year! Make sure you don't lose your benefits; online membership renewal is quick and secure.

Congratulations to the Career Development Award Winners

Last month, ASGCT selected six members to receive $50,000 Career Development Awards, a total distribution of $300,000. This group of awardees is ASGCT’s second class of Career Development Award recipients, a program in which ASGCT recognizes members who are transitioning toward independence in their careers.

Look Out for Remaining Patient Education Disease Resources

Have you seen ASGCT’s new disease-specific pages, videos, and infographics? As part of the second round of resources launching from our Patient Education program, we’ve been releasing new material weekly for diseases like Parkinson’s, hemophilia, muscular dystrophy, and more. Check out our Patient Education section to learn more.

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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


We’d like to thank everyone who attended the first annual ASGCT Policy Summit Nov. 4-6 in Washington, D.C.! The Summit brought nearly 400 attendees together for three unique days of discussions on global regulatory issues, payment policies, and ethical concerns surrounding gene therapy.

Day 1: Global Regulatory Issues in Gene Therapy Development

On the first day, speakers from Brazil, Japan, and Canada talked about the current gene therapy regulatory environments in their respective countries. Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research (FDA CBER), gave the keynote address, where he emphasized the need for regulators to work towards a globally harmonized framework to facilitate safe and ethical development of gene therapies. Liz Anne Gillham-Eisen, a director in the Health Products and Food Branch, Health Canada, discussed a new pathway that will help guide regulation for advanced therapeutic products in Canada.

Day 2: Perspectives on Payment Policies for Gene Therapies

Speakers on the second day discussed innovative payment strategies, including Cigna’s Embarc program and MIT’s Orphan Reinsurer and Benefit Manager. These models will hopefully help offset the cost of gene therapies and help more people gain access, speakers said. In the afternoon, Medicaid experts from Massachusetts and Virginia shared their perspectives on developing coverage for gene therapies.

Day 3: Ethical, Societal, and Policy Issues in Germline Gene Editing

On the third and final day, NIH Director Francis Collins, M.D., Ph.D., called for a worldwide moratorium on clinical uses of germline gene editing in humans. Following Collins’ keynote address, a panel of patients and patient advocates presented their views of gene editing, disability, and disease. Margaret Hamburg, M.D., foreign secretary of the National Academy of Medicine, gave the final keynote address about her work as co-chair of the WHO advisory committee in governance and oversight of human genome editing.

Industry News


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2020

23rd Annual Meeting

May 12-15 | Boston