The Vector
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
From Molecular Therapy
Society News
Career Center
Public Policy
Industry News
Leadership Message
Abstract Submission Opens Next Week
Hello ASGCT Members,
We’ve reached that exciting point in the year once again—the start of Annual Meeting abstract submission! Beginning this Monday, November 15, you can submit your gene and cell therapy research for presentation at the 25th Annual Meeting next May. As the largest meeting of gene and cell therapy professionals, the ASGCT Annual Meeting is truly the best place to showcase your innovative research. Submitting an abstract gives you the opportunity to present and discuss your findings with colleagues and leaders in the field from across the world. You may also be eligible for one of these awards recognizing your work.
For 2022, we are asking anyone whose abstract is accepted for oral presentation to present live on site at the Walter E. Washington Convention Center in Washington, D.C. Accommodations for virtual presentations may be made by request based on employer travel and participation mandates or for personal reasons as we are able. More information about this will be posted on the Annual Meeting site as it’s confirmed. If your abstract is selected for poster presentation, you will have the option to present in person in the on-site poster hall, virtually, or both. For the first time ever, ASGCT will be accepting late-breaking abstracts in 2022, and more information will be posted on this page as it becomes available. As always, all Associate Members who are first and presenting abstract authors will receive free registration for the meeting.
Since we are also in the thick of renewal season at ASGCT, I’d like to encourage you to join me in renewing your membership. If you renew right now, you’ll maintain your access to all of your ASGCT benefits—event and Molecular Therapy publication discounts, on-demand professional development content, and much more—through 2022. Your membership is so important to the work we do at ASGCT, so please renew today!
I have a feeling that 2022 will be extra special because we’ll (hopefully) be together again to learn from each other in person. I look forward to seeing you all at the meeting in May!
Sincerely,
Terry Flotte, MD
ASGCT Secretary
Breaking Through
AAV5 delivery of CRISPR/Cas9 supports effective genome editing in mouse lung airway
Liang S, Walkey CJ, Martinez AE, Su Q, Dickinson ME, Wang D, Lagor WR, Heaney JD, Gao G, Xue W
DOI: https://doi.org/10.1016/j.ymthe.2021.10.023
Summary by Karen Bulaklak, PhD
A critical step in safe and effective gene therapy is successful delivery. While AAV vectors can transduce target tissues very efficiently, therapeutic gene expression can be impacted over time by loss of vector genomes, particularly in highly proliferative cell types. A solution to this problem is using gene editing technologies, such as CRISPR/Cas9, to induce permanent modification to elicit a therapeutic response. Even with high turnover, genetic changes can be passed on to daughter cells that share the beneficial effects of the therapy. To achieve this, delivery of gene editing molecules must occur at high enough levels to ensure that enough cells are transduced and can maintain the corrective edit over time.
To support explorative efforts into questions of gene editing efficacy and delivery, programs such as the NIH Somatic Cell Genome Editing Consortium (SCGE) have been created. In a recent report, researchers from the University of Massachusetts Medical School and Baylor College of Medicine described work supported by the NIH SCGE where they explored the ability of AAV serotype 5 (AAV5) to deliver CRISPR/Cas9 and effectively edit cells in proliferative airway epithelial cells in the lung. Previously, no attempts had been made to use AAV-mediated delivery of CRISPR/Cas9 into the lung airways. The UMass group utilized the Ai9 reporter mouse line, which harbors a lox-STOP-lox-tdTomato reporter cassette. CRISPR/Cas9 components were packaged into AAV5 in a dual vector conformation, where SpCas9 was in one vector and guide RNAs targeting the lox sites were in a separate vector. After intrathecal administration of these AAV5-CRISPR vectors into Ai9 mice, tdTomato expression was found in the airway at a rate of ~24% cells, indicating efficient gene editing. To examine reproducibility of these results, the same vectors were used to treat Ai9 mice at Baylor College. This group found that ~19% and ~22% of cells in the large and small airways were tdTomato+, respectively. Furthermore, tdTomato expression was rarely found in non-target tissues such as the liver and no expression was observed in reproductive organs. Additional investigation revealed reporter expression in both club and ciliated cells, as well as the alveolar region of the lung.
While further investigation of AAV5-CRISPR is needed to understand efficacy in a disease model, this proof-of-concept study holds promise for future lung-directed therapies. Moreover, this study is an example of fruitful scientific collaboration, which will be critical to the success of potentially lifesaving treatments.
From Molecular Therapy
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News from Molecular Therapy—Methods and Clinical Development:
Call for papers: Submit a paper to a special issue on evidence generation and reproducibility in cell and gene therapy research. Learn more about this special issue by reading this editorial and submit a paper by February 28, 2022.
Expanded scope: MTM has expanded its scope to include advances in gene and cell therapy and how they are translated and applied in the clinical setting. Novel methods of gene delivery into immune cells and hematopoietic and other stem cells using integrating and non-integrating vectors, non-viral vectors, and gene editing methods are featured. Additional focus areas include manufacturing of such products under Good Manufacturing Practice (GMP) conditions, including regulatory science and possible commercialization of these novel products. The editor-in-chief, Gerhard Bauer, PhD, encourages authors to submit their research to this high quality journal with an impact factor of 6.698.
Check out the latest issue of Molecular Therapy here, and don't miss the most recent content from other issues in the MT family:
Society News
Paloma Giangrande, PhD, Appointed MTNA Editor-in-Chief
Paloma Giangrande, PhD, will become the next editor-in-chief of Molecular Therapy Nucleic Acids (MTNA) on January 1, 2022. Giangrande, who has been serving as one of the journal’s associate editors-in-chief, will replace current editor John Rossi, PhD, to become the first female heading one of the Society’s journals. Read more on our blog.
Two ASGCT Members Elected to National Academy of Medicine
Congratulations to Past ASGCT President Helen Heslop, MD, DSc (Hon) and ASGCT Member Yuman Fong, MD, editor-in-chief of Molecular Therapy Oncolytics, on their election to the National Academy of Medicine! Heslop and Fong joined 88 regular members and 10 international members also elected this year. View the full list of newly elected members on NAM's website.
Read the Latest Gene, Cell, & RNA Therapy Landscape Report
Check out the new issue of our report, created in partnership with Informa Pharma Intelligence, that covers the therapeutics pipeline, clinical targets, developer progress, and more. In the third quarter, highlights include approvals of two new gene therapies in the European Union (EU) and China, growth in the RNA therapeutic space, and a spotlight on non-oncology indications. Read it here.
Stay Connected to Your Professional Hub by Renewing Your Membership
The best way to maintain uninterrupted access to your membership benefits is to renew your membership today! When you renew, you'll continue to enhance your knowledge through access to on-demand professional development cafés, stay on top of the latest research with your subscription to Molecular Therapy, and learn from peers during discounted events (including the Annual Meeting), through 2022. Stay connected to your professional hub and renew today!
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
Public Policy
ASGCT Backs Expediting of Funding Increases for NIH and FDA
ASGCT joined 207 other organizations in a letter led by Research!America urging action from Congressional leadership to complete the Fiscal Year 2022 (FY22) appropriations process before the current continuing resolution (CR) expires on December 3. While Congress is proposing robust funding for FDA and NIH in FY22, the government is operating under a CR, which is a temporary measure by which Congress provides flat funding for a limited amount of time. The letter urges Congress to work quickly to attain increased funding for agencies such as the FDA and NIH, which play roles in the development of treatments for the many people living with illnesses such as rare diseases and cancer. ASGCT supports continued innovation of gene and cell therapies by encouraging expeditious funding increases toward research and regulation of clinical development.
Comments to FDA Support CBER Staff Increase, Engagement with Sponsors
ASGCT submitted comments last month to support the Prescription Drug User Fee Act (PDUFA) commitment letter, released in August as part of the reauthorization process of the act for FYs 2023-2027 (PDUFA VII). The act authorizes FDA to collect user fees from companies that produce certain human drug and biological products, and the reauthorization process involves FDA engagement with stakeholders to establish FDA performance goals for the upcoming five-year period. ASGCT’s comments expressed eagerness to assist the FDA in working toward proposed goals, including:
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Bolstering support for the Center for Biologics Evaluation and Research (CBER) by hiring 228 new FTEs to meet the increasing challenges and demands in this growing field.
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Improving engagement and communication between FDA and sponsors through creation of a new meeting type (Type D).
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Providing common questions and answers regarding gene and cell therapy development.
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Investing in modernizing CBER’s digital infrastructure.
For more details on how the proposed PDUFA goals align with ASGCT’s regulatory policy priorities, subscribe to our policy newsletter, The Advocate.
Bespoke Gene Therapy Consortium Will Accelerate Gene Therapy Development for Rare Diseases
ASGCT has joined forces with FDA, 11 NIH institutes and centers, and more than a dozen other nonprofit groups and private companies to help accelerate the delivery of gene therapies for rare disease patients through the newly launched Bespoke Gene Therapy Consortium (BGTC). This public-private collaboration, managed by the Foundation for the National Institutes of Health (FNIH), aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with many rare diseases, in particular those diseases that are so rare that they challenge the typical commercial development model. Read more on our blog.
Industry News
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