The Vector

Volume 8, Issue 12: December 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

Happy Holidays from ASGCT

As we get closer to the end of what was an extremely successful year for ASGCT, I want to wish you all a happy and healthy holiday season. Looking back at events like the record-breaking 2019 Annual Meeting and the enthusiasm that we saw at the inaugural three-day Policy Summit, I hope you’ll join me in celebrating the Society’s accomplishments this year. More than that, thank you for being a part of it.

We could not have achieved what we were able to in 2019 without the enthusiastic participation and tireless effort of our members. That’s why I hope that you’ll renew your ASGCT membership by December 31 to ensure that you’ll continue having access to exclusive member benefits, like event discounts and journal subscriptions. Don’t let your membership expire! Your support is critical to us as we plan activities strategically for the coming year, and we want to provide the best programming we can to our members. If you have suggestions for us, we’d love to hear your feedback.

As we move into 2020, we are anticipating our largest-ever annual meeting as we return to a convention center in Boston. Please make sure you register before April 10 to receive an early registration discount, and secure your housing by booking a room in the ASGCT housing block. Make your reservations soon, as there’s no guarantee attendees will be able to get the same room rate once the hotel block is completely full. In addition to making your meeting plans, we still need your research! Abstract submission is open through January 29, and it’s a great opportunity to present and discuss your work with peers and leaders in the field. If you’re an Associate Member and a first or presenting author, you can also earn free registration. Learn more about abstract awards and other incentives here.

Last but not least, the 2019 Annual Meeting videos are now available on our YouTube channel. Watch videos from Education and Plenary Sessions, Scientific Symposiums, and more. If you’ve never been to an ASGCT annual meeting before, I hope the videos will inspire you to join us next year!

Best,
Guangping Gao, Ph.D.
ASGCT President

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Breaking Through

Systemic Exosomal Delivery of shRNA Minicircles Prevents Parkinsonian Pathology

Izco M, Blesa J, Schleef M, Schmeer M, Porcari R, Al-Shawi R, Ellmerich S, de Toro M, Gardiner C, Seow Y, Reinares-Sebastian A, Forcen R, Simons JP, Bellotti V, Cooper JM, Alvarez-Erviti L

DOI: https://doi.org/10.1016/j.ymthe.2019.08.010

Summary by Edith Pfister, Ph.D.

Parkinson’s disease (PD) is the second most common neurodegenerative disease, affecting about one out of 175 people in the U.S. Almost everyone knows someone who is affected by the disease so novel or innovative approaches to treatment generate significant interest. Although the most common forms of PD cannot be linked genetically to a single mutation, alpha-synuclein aggregation and pathology exist in both familial and sporadic forms of the disease and downregulation of alpha-synuclein shows promise in animal models of the disease. In the current study, Izco et. al. combined several interesting approaches to reduce alpha synuclein in the central nervous system.

Exosomes are small extracellular vesicles released by cells when the endosome, in the form of a multivesicular body, fuses with the external cell membrane. They are of interest as biomarkers of disease, as they carry information about their cellular origin. In addition, they show promise as delivery vessels for therapeutic cargo, as they appear to distribute widely throughout the body. In this study, the authors used exosomes tagged with the rabies virus glycoprotein (RVG) peptide to deliver DNA across the blood brain barrier. To solve the problem of stability and duration of effect, the authors employed shRNA minicircles, small double stranded DNA vectors containing only the transgene and its regulatory sequences. These shRNA minicircles can be loaded into exosomes more efficiently than their larger plasmid cousins, but like plasmids, they are capable of sustained expression. When injected intravenously, RVG-exosomes loaded with shRNA minicircles reduced alpha-synuclein protein and mRNA 30 days post-injection in a mouse model of PD and also reduced neuronal loss.

These are interesting new technologies, and worth pursuing. One caveat to this study is that the mice were treated very early, at the onset of pathology, which might be difficult in the context of human disease. This current work provides the tools to further examine this question.

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Society News


Annual Meeting and Membership Renewal Reminders

The year is winding down, so make sure you keep your ASGCT membership benefits in 2020. Renew your membership by Dec. 31. Additionally, abstract submission for the 23rd Annual Meeting is open through Jan. 29; submit your research today for the opportunity to present your work to leaders in the field. Finally, take advantage of early registration for the meeting by April 10. Visit the annual meeting website for more information about abstracts, registration, and award opportunities.

ASGCT Welcomes New Editors to Molecular Therapy Family

ASGCT announced the appointment of Roland W. Herzog, Ph.D. and Gerhard Bauer as editors in chief of the Society’s field-leading journals, Molecular Therapy and Molecular Therapy—Methods & Clinical Development, respectively. Both editors will begin their positions effective January 1, 2020. Read more on our blog.

CAR T-Cell Therapy Resources Complete 2019 Patient Education Releases

Earlier this month, ASGCT released the final disease-specific resources from its Patient Education program for 2019, this time focusing on using CAR T-cell therapy to treat blood cancers. The web page, infographic, and video about CAR T-cell therapy complete the second round of material from the Patient Education program, a new initiative in 2019.

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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


ASGCT Co-Hosts Congressional Briefing on Sickle Cell Disease

ASGCT co-hosted a second Congressional briefing on Capitol Hill to highlight how policymakers can support sickle cell disease (SCD) screening, treatment development, and access to new treatments. After co-hosting a high-interest briefing with Senate offices in June, ASGCT hosted this briefing in conjunction with House offices—Representatives Barbara Lee (D-CA), Michael C. Burgess, MD (R-TX), and Danny K. Davis (D-IL). Reps. Burgess and Davis gave remarks emphasizing the importance of supporting SCD research.

ASGCT Holds Second Annual FDA Liaison Meeting

ASGCT held the second annual liaison meeting with the FDA’s Center for Biologics Evaluation and Research (CBER) on November 18. This year’s meeting was co-chaired by Adora Ndu, PharmD, J.D., chair of the ASGCT Regulatory Affairs Committee; and Maritza McIntyre, Ph.D., a member of both the ASGCT Board of Directors and the Regulatory Affairs Committee. ASGCT members presented on three topics to staff of the Office of Tissues and Advanced Therapies (OTAT), providing ASGCT recommendations to the Agency on regulatory topics of mutual interest in the field. In addition, OTAT staff provided useful information on common investigational new drug deficiencies and recommendations for improvement. Visit the blog to view presentation slides and to read the summary of recommendations.

Society Signs On to Support National Biomedical Research Act

One priority of ASGCT's policy and advocacy program is supporting a robust level of NIH funding, especially for initiatives that include gene therapy research. To that end, ASGCT signed on in November as a supporter of the National Biomedical Research Act, authored by Senator Elizabeth Warren (D-MA) and co-sponsored by an anticipated 12 additional senators. The bill would create a fund to support innovative research and development through NIH and FDA initiatives. When it is introduced, it will be a re-introduction of  the ASGCT-supported S.2212 (2017). A companion bill has been developed in the House, sponsored by Rep. Yvette Clarke (D-NY) and Dave Loebsack (D-IA).

Industry News


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