The Vector

Volume 7, Issue 10: October 2018

Editorial Team

Phillip Doerfler, PhD - Editor, The Vector
Melvin Rincon, MD, PhD - Associate Editor, The Vector
Edith Pfister, PhD - Junior Editor, The Vector

Inside This Issue

President's Message
Breaking Through
Society News
Public Policy
Industry News



President's Message

As the autumn leaves fall and a briskness enters the air, activities ramp up at ASGCT as we officially kick off Annual Meeting season. We’ve launched a new website, released our annual Exhibitor & Sponsor Prospectus, and both registration and housing at our host hotel open in the near future. Most importantly, however, the Society is set to open abstract submission for the 2019 Annual Meeting on Wednesday, October 17—less than one week from today!

All publishing and submission guidelines are in the Call for Abstracts.

This collection of field-leading research is the lifeblood of ASGCT’s meeting, and the Society is excited to feature the future of our field at the Washington Hilton next year. I encourage all of you to consider submitting your research to the ASGCT Annual Meeting.

In an effort to grow ASGCT’s influence even further beyond the Annual Meeting, the Society hosted our first-ever Value Summit in Washington, D.C. on September 24. Through a full day of sessions, stakeholders with various interests united by their commitment to advancing gene and cell therapy discussed strategies for maximizing patient access to durable, potentially curative treatments while maintaining the level of innovation that made them possible.

Many of the presenters from the ASGCT Value Summit have agreed to let ASGCT post their presentation materials online for those who were unable to attend the summit. I would like to thank those who shared their knowledge, both in-person and those who have made their expertise available online.


Finally, I invite all of our members to seek out the ASGCT booth at the European Society of Gene & Cell Therapy’s upcoming Annual Congress, October 16-19, in Lausanne, Switzerland. We look forward to this opportunity to connect with many of our members outside of our own meeting every year and look forward to seeing you in Switzerland!


Michele Calos, Ph.D.
ASGCT President

Breaking Through

Translating SOD1 Gene Silencing Toward the Clinic: A Highly Efficacious, Off-Target-Free, and Biomarker-Supported Strategy for fALS       

Iannitti et al., Mol Ther Nucleic Acids
Written by Phillip A. Doerfler and Edith Pfister

Published: May 19, 2018 | DOI:

In a recent publication in Molecular Therapy Nucleic Acids, Iannitti and colleagues pursued a novel treatment strategy for amyotrophic lateral sclerosis (ALS) that significantly advances upon previous iterations of ALS gene therapy. Their target was the copper/zinc superoxide dismutase (SOD1).

Mutations in SOD1 account for many familial ALS (fALS) diagnoses. The authors developed AAV9-based vectors to deliver an shRNA targeting SOD1 to the central nervous system (CNS). A single injection of the AAV9 targeting SOD1 into the cisterna magna reduced SOD1 throughout the brain and in the CSF and alleviated the symptoms of ALS in a transgenic mouse model expressing mutant SOD1.

When compared to mice injected with a control shRNA, mice injected with the SOD1 shRNA showed improvements in onset and disease severity, improved performance on the rotorod, decreased neurological score, and increased survival. AAV9-shRNASOD1 treated mice displayed improved survival of motor neurons and reduced gliosis in the lumbar cord ventral horn.

Using microarray analysis, the authors examined the specificity of targeting of the SOD1 shRNA, showing that SOD1 is the only gene affected by their vector. These results are significant as this vector can be directly translated to humans. The clinical candidate shows remarkable efficacy in a mouse model of fALS and proof-of-principle of widespread CNS gene transfer via intrathecal injection, which is translatable to humans.

The lack of significant off-target effects is reassuring and the ability to measure silencing of SOD1 in the CSF provides an accessible biomarker to monitor therapeutic targeting and efficacy in humans. The results coalesce into a promising future for ALS treatments.


Society News

Free Hematopoietic Stem Cell Webinar Recording Online

Molecular Therapy and CellPress hosted the Evolution of Hematopoietic Stem Cell Gene Therapy webinar on October 3, and the recording of the session is now available for free with registration through CellPress.

The expert panel of ASGCT Board of Directors member Alessandra Biffi (Harvard Medical School), Adrian Thrasher (UCL Great Ormond Street Institute of Child Health), and David A. Williams (Boston Children's Hospital) were led through the discussion by Rob Frederickson, executive editor of ASGCT's journal, Molecular Therapy.

ASGCT Holds First-Ever Value Summit

The American Society of Gene & Cell Therapy (ASGCT) held the first-ever ASGCT Value Summit: Advancing Patient Access to the Benefits of Gene Therapy on Monday, September 24. Throughout the full-day summit, a series of speakers focused on current efforts to enhance patient access to approved therapies, solutions to barriers to patient access, and the need for collaborative support on proposed solutions.

View The Presentations

A full account of the Value Summit is currently in production and will be published in an upcoming issue of Molecular Therapy.

Public Policy

PAHPA Bill Passes, National Health Security Meeting to Follow

The Pandemic and All-Hazards Preparedness and Advancing Innovation Act of 2018 (PAHPA) passed both chambers of Congress in September. The PAHPA Act contains a provision, which ASGCT supported, that calls for a meeting within a year from enactment to discuss the potential role that genomic engineering technologies, including gene editing, may have in advancing national health security.

ASGCT recognizes that gene editing technology has the potential to contribute to other societal benefits, in addition to therapeutic ones. The Society therefore supported the upcoming discussion of the potential for additional uses of gene editing, and promoted the language in the provision that calls for inclusion of representatives from academic, private, non-profit, and other entities with expertise in genome engineering technologies, in this meeting.

UPDATE (10/15): The preparedness programs have continued into this month, with increased funding, due to last month’s approval of fiscal year 2019 funding for the Health and Human Services Department. However, the Senate still needs to vote on the PAHPA bill (HR 6278), which would technically authorize the preparedness programs, including the provision calling for a meeting on the role of genomic engineering in advancing national health security. Senate action is expected after the midterm elections.

Presentation Materials From Two ASGCT Federal Meetings Available

Select presentation materials from ASGCT's FDA Liaison Meeting with the FDA Center for Biologics Evaluation and Research (CBER) and the Society's first-ever value summit, ASGCT Value Summit: Advancing Patient Access to the Benefits of Gene Therapy are now available online. Many Society members who presented at the events were gracious enough to make their knowledge available for all members.

More Policy Updates:

Industry News

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September 23-24, 2024 | Washington, D.C.

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