The Vector

Volume 8, Issue 8: August 2019


Editorial Team

Melvin Rincon, MD, PhD – Editor, The Vector
Edith Pfister, PhD – Associate Editor, The Vector

Inside This Issue

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

 

 

Leadership Message

Expanding Our Reach to Patients

Following the overwhelmingly positive feedback for our first round of programming, ASGCT is preparing to release seven new patient education resources next month. These resources were made possible through contributions from ASGCT staff, our supporters, and by multiple experts within our society’s membership. We are very proud of our efforts to produce content that is easily accessible and is informative for all education levels. In the last week of September, we will be releasing a pair of treatment-specific resources designed to inform patient networks about gene and cell therapies. The first rollout will debut animated videos, infographics, and interactive content for Parkinson’s and Huntington’s diseases. Content packages profiling treatments for muscular dystrophy, hemophilia, mucopolysaccharidosis (MPS), Pompe disease, and CAR T-cell therapies will soon follow.

I highly encourage all of our members to check out the current educational resources from ASGCT, including “Gene Therapy Basics” and “How Clinical Trials Work”—they are excellent resources for anyone looking for an introduction to what gene therapy is and how we are working together as basic scientists, clinicians, public advocates, and patient groups to discover and develop treatments for human disease.

Engaging patients and the general public have become an essential part of ASGCT’s mission. More and more gene and cell therapy strategies are moving to clinical testing stages. We have a duty as a community to educate people on the benefits and value these potentially-curative treatments have for patients with limited options.

In addition to our Patient Education program, we are proud to partner with the National Organization for Rare Disorders (NORD) for a series of free educational webinars starting this month. On August 29, Cenk Sumen, Ph.D. and I will lead a session entitled, “Gene Therapy: Then, Now, Later” that touches on the history of our field, currently approved therapies, and the immediate future for gene therapy. Upcoming webinars in October, November, and December will cover the science behind these strategies, the long-term effects of these treatments, and patient experiences.

ASGCT’s engagement in patient communities is expanding as more gene and cell therapies continue to reach the clinic. We are honored to help spread awareness of our collective advances in this new frontier and I hope the effects we can have on the lives of patients move many of you to become involved with future ASGCT efforts.

Best,

Phillip Tai, Ph.D.

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Breaking Through


Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

Jacob T. Cain, Shibi Likhite, Katherine A. White, Derek J. Timm, Samantha S. Davis, Tyler B. Johnson, Cassandra N. Dennys-Rivers, Federica Rinaldi, Dario Motti, Sarah Corcoran, Pablo Morales, Christopher Pierson, Stephanie M. Hughes, Stella Y. Lee, Brian K. Kaspar, Kathrin Meyer, Jill M. Weimer

DOI: https://doi.org/10.1016/j.ymthe.2019.06.015

Summary by Melvin Rincon, M.D., Ph.D.

CLN6-Batten disease is a rare, fatal, neurodegenerative disease that typically affects children. Beginning from 18 months to 6 years of age, children show gradual declines in motor capabilities, vision, and cognitive ability, eventually becoming wheelchair bound, reliant on a feeding tube, and succumbing to early death around 12 to 15 years of age. Here, Cain and colleagues evaluated the therapeutic potential of an AAV mediated gene therapy for CLN6-Batten disease when delivered through cerebrospinal fluid. For most of their experiments they used a mouse model of CLN6-Batten disease, carrying a spontaneous mutation in the Cln6 gene. The team, led by Drs. Jill Weimer (Sanford Research), Kathrin Meyers (Nationwide Children’s Hospital) and Brian Kaspar (formerly of Nationwide Children’s Hospital), found:

  • Administration of the viral mediated CLN6 gene therapy was safe and well tolerated in both mouse models and non-human primates when delivered through cerebrospinal fluid
  • Administration of the gene therapy led to efficient CLN6 protein expression throughout the brain of Cln6 mutant mice
  • Administration of the gene therapy prevented many of the pathological hallmarks of CLN6-Batten disease in Cln6 mutant mice, including a reduction in neuroinflammation and preservation of neuronal connections
  • Administration of the gene therapy led to remarkable improvements in motor function and memory/learning capabilities in Cln6 mutant mice

Lastly, administration of the gene therapy allowed Cln6 mutant mice to live a full lifespan

In summary, these preclinical data show that AAV mediated gene delivery of the CLN6 gene through cerebrospinal fluid is a highly promising therapeutic option for CLN6-Batten disease patients.

The mice in this study were followed for 24 months, making this one of the most comprehensive pre-clinical gene therapy studies published to date. Additionally, this work paves the way for future clinical trials for gene therapies of rare, neurological diseases. In fact, the impressive efficacy data laid the ground work for initiation of a phase I/II clinical trial for CLN6-Batten disease patients (NCT02725580) being conducted at the Abigail Wexner Research Institute at Nationwide Children’s Hospital. Amicus Therapeutics has recently reported disease stabilization and a favorable safety profile in the first patients in this clinical trial.

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Society News


What’s It Like to Attend the ASGCT Annual Meeting?

The ASGCT Annual Meeting attracts gene and cell therapy professionals from all ends of the field, but many members still haven’t made it to the Annual Meeting. The Society profiled four attendees, a clinician, a researcher, an exhibitor, and an ASGCT volunteer, with vastly differing roles to tell the story of what each gets from the Annual Meeting.

Special thanks to Rayne Rouce, M.D.; Clague Hodgson, Ph.D.; Casey Maguire, Ph.D.; and Guangping Gao, Ph.D.

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Career Center—NEW!


Recruit and connect with top gene and cell therapy job seekers on the ASGCT Job Bank.

Advertising opportunities are also available monthly via the Featured Jobs section and allow recruiting institutions to target the 3,000+ audience of The Vector.

Featured Jobs

Public Policy


ASGCT Sponsors NIH Genetics Summit

ASGCT recently committed to making global access to gene and cell therapies a policy priority. As a first step in these efforts, we are a sponsor of the Foundation for the National Institutes of Health’s International Summit in Human Genetics and Genomics. ASGCT Past-President Ken Cornetta M.D. (Indiana University) will be speaking on Sept. 6 as part of this month-long training program at the National Human Genome Research Institute.

Cornetta will provide an overview of clinical gene therapy for 30 early career PhDs, MDs, dentists, nurses, and trainees from developing countries. This five-year initiative aims to assist researchers from developing countries to expand knowledge and infrastructure related to the research and treatment of genetic diseases. Following the program, more than 140 articles have been published and more than 25 have received grants from NIH or other funding institutions.

Industry News


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2019
ASGCT Policy Summit
November 4 – 6 | Washington D.C.
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