Volume 9, Issue 11: November 2020


Editorial Team

Edith Pfister, Ph.D. – Editor, The Vector
Karen Bulaklak, Ph.D. – Associate Editor, The Vector
Jon Brudvig, Ph.D. – Junior Editor, The Vector

Inside This Issue

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

Leadership Message


AM21 Call for Abstracts Opens Next Week

Hello, ASGCT Members,

Since November is upon us, I’m excited to remind you that the Call for Abstracts for the 24th Annual Meeting will open on Monday, November 16. If you have not submitted an abstract before, I hope you’ll consider doing so for next year’s meeting. Submitting an abstract is a great opportunity to present and discuss your cutting-edge research with peers and leaders in the field. And, as always, ASGCT is offering free registration for Associate Members who are the first and presenting authors on an abstract. You may also be eligible for an award based on your abstract score. You can read about all of the award opportunities, abstract topic categories, and submission information on the Annual Meeting website. More information will be added as it becomes available. We are consistently impressed by the research you all submit to the meeting every year, and we’re very much looking forward to seeing your research for the 2021 meeting! The submission window will close on January 27.

As we approach the end of the year, I would also like to remind you to renew your membership for 2021. By renewing now, you’ll maintain uninterrupted access to your exclusive membership benefits, including discounts on registration for events like the Annual Meeting and Policy Summit, access to Molecular Therapy, ability to serve on ASGCT committees, and more. We are grateful to have you all as members and continue to be amazed at your contributions to the field.

I hope you and your loved ones are staying safe and well,

Sincerely,

Terry Flotte, M.D.
ASGCT Secretary

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Breaking Through


COVID-19-Induced ARDS is Associated with Decreased Frequency of Activated Memory/Effector T cells Expressing Tissue Migration Molecule CD11a++

Anft M, Paniskaki K, Blazquez-Navarro A, Doevelaar A, Seibert FS, Hölzer B, Skrzypczyk S, Kohut E, Kurek J, Zapka J, Wehler P, Kaliszczyk S, Bajda S, Thieme CJ, Roch T, Konik MJ, Berger MM, Brenner T, Kölsch U, Meister TL, Pfaender S, Steinmann E, Tempfer C, Watzl C, Dolff S, Dittmer U, Abou-El-Enein M, Westhoff TH, Witzke O, Stervbo U, Babel N

DOI: https://doi.org/10.1016/j.ymthe.2020.10.001

Summary by Karen Bulaklak, Ph.D.

As the country closes in on a year since the first reported COVID-19 case, the scientific community and general population have seen unprecedented treatment and regulatory advancements to fight the enigmatic SARS-CoV-2 virus. One of the most puzzling aspects of this pandemic is the range of disease severity in patients, underscoring the complex nature of SAR-CoV-2 pathogenesis. Furthermore, a significant amount of work has been aimed at understanding the contribution of the host response in both mitigating as well as exacerbating disease progression. In a recent report, Anft et al. looked at Spike (S) protein-reactive T cells in mild/moderate patients and critical patients with acute respiratory distress syndrome (ARDS) to better understand the involvement of the immune response in COVID-19.

In their initial observations, the research group looked at immune cell population frequencies, as lymphopenia has been linked to severe cases. While certain populations were initially lower in ARDS patients compared to mild/moderate control patients, these decreases appeared to resolve with improving condition. When the group looked at specific S-protein reactive T cell populations, they observed higher frequencies of CD4+CD154+ and CD8+CD137+ T cells in ARDS patients compared to mild/moderate patients, with a higher percentage of CD4+CD154+ T cells producing IL-2. Furthermore, all CD4+ from ARDS patients expressed at least two cytokines (compared to 70-80% of control patients), such as IL-2, TNF-α, IFN-γ, and Granzyme B, suggestive of protective immunity. Interestingly, healthy unexposed donors also showed some S protein reactivity, but had lower frequencies of the T cell subsets previously mentioned. A correlation was also found between the presence of CD4+CD154+ and antibody titers in the range of 50% neutralizing dose. Finally, the group looked at markers of activated effector T cells and found that ARDS patients expressed significantly lower levels of MHC-Class II HLA-DR, which is expressed on activated and proliferating T cells. Expression of CD11a, which is a T cell integrin critical for activation and migration, was also markedly lower in both CD4+ and CD8+ cells from ARDS patients, suggesting that CD11a could be used as a prognostic marker for severe COVID-19 cases.

While the authors were able to find differences in specific immune cell populations between mild/moderate and severe patients, it is difficult to make broad conclusions. There is much to learn about their exact roles in the manifestation of COVID-19 symptoms. Still, such efforts can aid in finding better benchmarks for determining efficacy as the possibility of a vaccine draws closer.

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Society News


Three ASGCT Members Elected to National Academy of Medicine

Last month, ASGCT Past-President Cynthia E. Dunbar, M.D., and Members David R. Liu, Ph.D., and Jerry R. Mendell, M.D. were elected to the National Academy of Medicine (NAM), which is considered one of the highest honors in the fields of health and medicine. Drs. Dunbar, Liu, and Mendell joined 87 regular members and 10 international members elected this year who have demonstrated outstanding professional achievement and commitment to service. You can read our Q&A with Drs. Dunbar and Mendell here.

Submit Your Abstracts Starting November 16

The Call for Abstracts for ASGCT's 24th Annual Meeting opens on Monday, November 16! Submit your cutting-edge research and you may be able to present your work to peers and leaders in the field of gene and cell therapy. Approximately 25 percent of the abstracts submitted will be eligible for oral presentation, and all accepted abstracts will be published in the May supplement to Molecular Therapy. Associate Members who are the first and presenting author will receive free registration. More information is available here.

Renew Your Membership for 2021

If you haven't already done so, make sure you renew your ASGCT membership for the 2021 member year to maintain your exclusive benefits. Our programming would not be possible without your membership, so we hope you'll continue to support our efforts to advance the field!

Gene Therapy Resources Available From NHLBI-Funded Programs 

Several NHLBI-supported programs, including the National Gene Vector Biorepository, the Gene Therapy Resource Program, and the Primate Center for Gene Therapy, provide low- to no-cost resources enabling translation of gene therapy research into clinical products. Read our blog post about these resources here.

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Career Center


Are you looking for a job in the field of gene and cell therapy? Check out the new ASGCT Career Center for great opportunities with industry, government, and academic organizations. 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 4,000+ audience of The Vector.

Featured Jobs

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Public Policy


ASGCT Advocates for Gene and Cell Therapy Research and Access

ASGCT submitted comments last month to a request for additional feedback from Representatives Fred Upton (R-MI) and Diana DeGette (D-CO) on several policy issues relevant to gene and cell therapies for the 21st Century Cures 2.0 legislation they are developing. The original Cures Act, passed with broad bipartisan support in 2016, provided $5.3 billion to the National Institutes of Health and the Food and Drug Administration to accelerate medical development and increase patient access to innovative new treatments.

ASGCT comments included recommending Cures 2.0 legislation extend funding for the Regenerative Medicine Innovation Project, which funds early translational regenerative medicine research, including study of gene therapy-related topics such as vector design and preclinical models. This innovation project, created by the original Cures Act, assists in prioritizing gene and cell therapy research funding.

The Society also supported funding of the Bespoke Gene Therapy Consortium’s Accelerating Medicines Partnership in Gene Therapy program, a public-private partnership that the Foundation for the NIH will lead. The program will aim to fund pilot research on gene therapy for four to six ultrarare diseases (with patient populations of less than ~100), using a standard menu of vectors, vector manufacturing processes, delivery methods, and clinical protocols, with the hope of advancing these therapies into clinical trials.

Other high-level highlights are ASGCT’s recommendations to improve patient access to gene and cell therapies by expediting public payer coverage decisions and enforcing Medicaid program coverage for FDA labeled indications; developing a consistent, transparent, and timely approach to coding decisions; reforming the Medicare inpatient payment system to improve reimbursement levels for providers; and enabling novel payment models such as value-based payment over time.

Industry News


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2025

Class Considerations on Immunogenicity for AAV GT Products

January 22-23, 2025 | Virtual

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