The Vector

Volume 11, Issue 2: February 2022


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


Open Monday: Late-Breaking Abstract Submission

Hello ASGCT Members,

I hope you’re staying warm and safe this winter while also looking forward to exciting times ahead, including the ASGCT Annual Meeting! I’m proud to report that we received more than 1,200 abstract submissions for 2022. Thank you all who shared your research with us during this submission cycle; I can’t wait to learn more about your work during the meeting and I hope you’re looking forward to presenting your findings to your gene and cell therapy colleagues.

We may have closed the first submission window, but we aren’t done collecting abstracts just yet. Beginning Monday, Feb. 14, for the first time in ASGCT’s history, you can submit your late-breaking abstracts for possible presentation at the Annual Meeting. The second window provides the opportunity for you to report more recently generated research, but is not intended to be an extension of the general submission deadline. Late-breaking abstracts should present data that is high impact, groundbreaking, innovative, and newsworthy. Submit on this page through Friday, March 18. No changes or withdrawals will be allowed for late-breaking abstracts. Notifications will be sent on Monday, April 18 and abstracts will be published online Friday, May 13.

While we’re on the subject of the Annual Meeting, please make sure you register by Feb. 28 to receive the early-bird rate. If you still need to renew your ASGCT membership for 2022, you can do that when you register.

Finally, I hope you’ve enjoyed the first two sessions of ASGCT’s Insight Series! Watch them on demand and register for the February events that focus on AAV.

 

Sincerely,

Terry Flotte, MD
ASGCT Secretary

 

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Breaking Through


Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors

Becker J, Stanifer ML, Leist SR, Stolp B, Maiakovska O, West A, Wiedtke E, Börner K, Ghanem A, Ambiel I, Tse LV, Fackler OT, Baric RS, Boulant S, Grimm D

DOI: https://doi.org/10.1016/j.ymthe.2022.01.024

Summary by Karen Bulaklak, PhD

To combat the ongoing pandemic, numerous therapeutic modalities have been utilized and evaluated at unprecedented speed. In addition to vaccines, the efficacy of multiple small molecules, oligonucleotide therapies, and monoclonal antibodies have been investigated for their ability to reduce viral load and improve disease severity. Still, the emergence of new variants of concern (VOCs) is a looming possibility. As many of the treatments target the spike (S) glycoprotein of SARS-CoV-2, their effectiveness against new VOCs can be hampered. A recent example is the omicron variant, which contains 30 mutations in S, impacting vaccine efficacy and necessitating additional boosters. Thus, alternative approaches that shift away from targeting the spike protein, or a single aspect of the viral life cycle, could be beneficial.

To explore such alternative strategies, a group of researchers at the University of Heidelberg sought to disarm the virus at the genetic level, limiting its ability to rapidly mutate and avoid suppression. The researchers first identified short hairpin RNAs (shRNAs) against conserved genomic regions in vitro, focusing on the viral RdRp and N genes, which play crucial roles in replication and packaging. They also evaluated shRNAs against the human angiotensin-converting enzyme 2 (hACE2), which is the entry receptor for SARS-CoV-2. These shRNAs were packaged into AAV and used to treat Vero E6 cells that were subsequently infected with SARS-CoV-2. Through this experiment, they found that viral infection, replication, and production of infectious particles were greatly reduced by shRNA treatment. This led to the creation of a combinatorial approach, which they termed “SAVIOR.” SAVIOR contained the three most potent shRNAs in an AAV expression cassette. Interestingly, when they compared this approach with single shRNA treatment in infected Vero E6 cells, they found viral rebound in the single shRNA-treated samples. Sequencing revealed point mutations in the shRNA binding sites, suggesting that a multiplexed approach is effective against escape mutants. SAVIOR was also assessed in vivo in mouse models of SARS-CoV-2 infection. SAVIOR-treated mice displayed a reduction in viral copy number and infectious units in the lungs, as well as improvements in airway obstruction and lung hemorrhage. The viral genomes from these mice also revealed the absence of any escape mutants, supporting earlier in vitro findings.

While the research is in its early stages, the findings from this study demonstrate that a multifaceted approach against SARS-CoV-2 can feasibly improve disease outcomes and prevent escape variants. With the rapid pace of drug development, such combinatorial therapies may become more common, and even necessary, in the fight to finally overcome this pandemic.

From Molecular Therapy


Now open: Search for Molecular Therapy—Oncolytics editor-in-chief: ASGCT has opened its search for the next editorial leader of MTO as the inaugural editor-in-chief Dr. Yuman Fong’s term ends Dec. 31, 2022. MTO is an online, open-access journal focusing on the development and clinical testing of viral, cellular, and other biological therapies targeting cancer. Its 2020 impact factor is 7.200.

Applications must include a letter of intent detailing relevant experience and vision for the journal as well as a current C.V. Interested ASGCT members may submit inquiries to ASGCT CEO David Barrett.

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 April 30, 2022.

Check out the latest issue of Molecular Therapy here, and don't miss the most recent content from other issues in the MT family:

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Society News


#ASGCT22 Early-Bird Rate Available Through Feb. 28

Whether you choose to attend virtually or join us (safely) in Washington, D.C., make sure you register for the 25th Annual Meeting by Feb. 28 to secure the lowest rate! No matter which option you choose, you'll have live and on-demand access to plenary lectures, scientific symposia, educational sessions, and more! Register now and we'll see you in person or virtually in May!

Focus on AAV This Month During ASGCT's Insight Series

Join us for two more (free for members) events this month from ASGCT's Insight Series: AAV Vector Design on Feb. 16 and Immune Responses to Gene Therapy for Musculo-Skeletal and Cardiac Diseases on Feb. 23. Register for one or both events in this new monthly series and watch the January events on demand on our website!

Renew By March 15 to Maintain Your 2022 Benefits

If you want to keep receiving exclusive member benefits, make sure you renew your membership! When you renew, you'll maintain access to our live and on-demand events, receive a discount when you register for the Annual Meeting, keep your Molecular Therapy subscription, and more. Renew your membership today to continue enjoying these benefits through 2022.

 

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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


Comments on PREVENT Pandemics Act Call for Platform Technology Support

Earlier this month, ASGCT submitted comments supporting the inclusion of advanced platform technology language in the bipartisan PREVENT Pandemics Act, which was released as a discussion draft. As it relates to the gene therapy field, the draft PREVENT Pandemics Act would facilitate the development of gene and cell therapies across more disease areas by harnessing the power of platforms and promote greater patient access to approved therapies. ASGCT’s comments on the Act support the need for new Food and Drug Administration (FDA) designations to enable greater use of data across products that share common chemistries, including gene delivery vectors or common processes. The Society also reiterated support for ARPA-H as a way to invest in platform technologies and manufacturing innovation, both of which are critical to the future of gene and cell therapy. ASGCT will continue to engage on these issues as the Act moves through Congress.

Read Our Final 2021 Landscape Report with Pharma Intelligence

The final 2021 installment of ASGCT and Pharma Intelligence’s quarterly publication, the Gene, Cell, & RNA Therapy Landscape Report, is now available! The report covers the therapeutics pipeline, clinical targets, developer progress, and more. In the fourth quarter, highlights include the approval of a new cell and RNA therapy in the U.S., the expansion of two RNA therapies for COVID-19 to new countries, and the expanded approval of a gene therapy for a new indication. Read the Q4 report, and find our archive of past publications, here.  

Industry News


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2022

ASGCT Policy Summit

September 26-27, 2022

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