Sarepta Announces Agreement with Nationwide Children’s Hospital for Rights to its Gene Therapy Program to Treat Charcot-Marie-Tooth (CMT) Neuropathy, Globally the Most Common Inherited Neuromuscular Disorder
10/05/18 8:30 AM EDT
-- Furthers the Company’s strategy to expand neuromuscular gene therapy franchise --
-- CMT is a group of hereditary, degenerative nerve diseases that in addition to causing extraordinary pain, can greatly impact peripheral motor skills and profoundly limit the ability to walk or use one’s hands --
-- Clinical trial in patients scheduled to start in 2019 in the most prevalent subtype of CMT type 1A --
The clinical trial to test NT-3 gene therapy is planned to commence dosing in 2019 for CMT type 1A. The delivery of the NT-3 gene may have applicability to other sub-types of CMT in addition to other muscle-wasting diseases. Pre-clinical research has shown the ability of the NT-3 gene construct to regenerate nerves. Further research is under way to explore its potential.
Dr. Sahenk is an attending neurologist at Nationwide Children's, Director of Clinical and Experimental Neuromuscular Pathology at
“Our agreement today further builds on our strategy to establish a gene therapy engine delivering potentially transformative therapies to treat life-altering rare disease,” stated
“Gene therapy represents a potential new pathway for the treatment of CMT Neuropathy. We look forward to collaborating with Sarepta, whose dedication to those impacted by neuromuscular disorders and to rigorous scientific exploration, echoes our own at Nationwide Children’s,” said Zarife Sahenk, M.D., Ph.D., attending neurologist and Director of Clinical and Experimental Neuromuscular Pathology at
About Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth or CMT neuropathies are an inherited, heterogeneous group of peripheral nerve disorders affecting 1 in 2,500 persons. CMT is caused by mutations in genes that produce proteins involved in the structure and function of either the peripheral nerve axon or the myelin sheath. The degeneration of motor nerves results in muscle weakness and atrophy in the extremities (arms, legs, hands, or feet), and in some cases the degeneration of sensory nerves results in a reduced ability to feel heat, cold, and pain. Different mutations can cause mild to severe symptoms, with some patients losing their ability to walk or use their hands. Most patients are diagnosed at infancy, while other patients develop symptoms at adolescence.
About Nationwide Children’s Hospital
Named to the Top 10 Honor Roll on
This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the plan to commence the clinical trial to test NT-3 gene therapy in 2019 for CMT type 1A; the potential applicability of the delivery of the NT-3 gene to other sub-types of CMT in addition to other muscle-wasting diseases; the potential of the NT-3 gene construct to regenerate nerves; Sarepta’s strategy to establish a gene therapy engine delivering potentially transformative therapies to treat life-altering rare disease; and gene therapy representing a potential new pathway for the treatment of CMT Neuropathy.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the expected benefits and opportunities related to the agreement with Nationwide Children’s may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; in particular, the agreement may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement, the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates, possible limitations of Company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta’s product candidates; and even if commercialization of any product under the agreement is achieved, this may not result in any significant revenues to the parties; if the actual number of patients suffering from CMT and/or CMT type 1A is smaller than estimated, Sarepta’s revenue and ability to achieve profitability may be adversely affected; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2017 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the
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Source: Sarepta Therapeutics, Inc.
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