Oral Presentation Details:
Oral Presentation: First-In-Human Gene Therapy for Tay-Sachs Disease: Report of Two Infants Treated on an Expanded Access Clinical Trial of rAAVrh8-HexA/HexB (AXO-AAV-GM2)
Abstract Number: 1299
Presenter: Terence Flotte, MD, Provost, Executive Deputy Chancellor and Dean, School of Medicine, University of Massachusetts Medical School and Principal Investigator
Session: Clinical Trials Spotlight Symposium
Date and Time: May 15, 2020, 8:30-8:45am
Invited Lecture: AXO-Lenti-PD: A Trans-Atlantic Collaboration on Gene Therapy for Parkinson's Disease
Presenter: Gregory R Stewart, PhD, SVP, Scientific Affairs
Session: Gene and Cell Therapeutic Strategies to Treat Disorders of the CNS - An International Perspective
Date and Time: May 15, 2020, 8:35-9:10am
Poster Presentation Details:
Title: Surgical Technique for Bilateral Intrathalamic Infusion of rAAVrh8-HEXA/ HEXB Gene Therapy in Infant with Tay-Sachs Disease
Abstract Number: 666
Presenter: Dr. Oguz Cataltepe, MD, Professor of Neurological Surgery and Pediatrics, University of Massachusetts Medical School; Director of Pediatric Neurosurgery, UMass Memorial Medical Center
Session: Synthetic/Molecular Conjugates and Physical Methods for Delivery
Date and Time: May 13, 2020, 5:30-6:30pm
About Axovant Gene Therapies
Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinson's disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit www.axovant.com.
In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases. A three-way Cooperative Research and Development Agreement (CRADA) among Axovant, the NHGRI, and the University of Massachusetts Medical School was established in 2019 to support the conduct of the clinical program.
About the University of Massachusetts Medical School
The mission of the University of Massachusetts Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery.
Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School and Auburn University has led to significant advances in the field. Miguel Sena-Esteves, PhD, associate professor of neurology at UMass Medical School; Heather Gray-Edwards, PhD, DVM, formerly of Auburn and currently assistant professor of radiology at UMass Medical School; and Douglas Martin, PhD, professor of anatomy, physiology and pharmacology in the College of Veterinary Medicine and the Scott-Ritchey Research Center at Auburn University, have worked collaboratively for more than a decade on animal models and therapeutic approaches for these and similar disorders. For more information, visit www.umassmed.edu.
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. All forward-looking statements are based on estimates and assumptions by Axovant's management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected. Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory submissions and approvals; the continued development of its gene therapy product candidates and platforms; Axovant's scientific approach and general development progress; and the availability or commercial potential of Axovant's product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovant's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on February 10, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.
Media and Investor Contact:
Axovant Gene Therapies
To read more Press Release articles, click here.