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Sarepta and Selecta Enter Agreement for Use of Selecta’s Platform in Neuromuscular Diseases

June 18, 2020

Sarepta and Selecta Enter Research License and Option Agreement for Selecta’s Immune Tolerance Platform in Neuromuscular Diseases

Rare Daily Staff

Sarepta Therapeutics said it has entered into an agreement with  Selecta Biosciences that provides Sarepta with an option to license the rights to develop and commercialize Selecta’s immune tolerance platform, ImmTOR, for use in Duchenne muscular dystrophy and certain limb-girdle muscular dystrophies.  

In advance of exercising its option, Sarepta will conduct research and evaluate the utility of ImmTOR to minimize or prevent the formation of neutralizing antibodies to adeno-associated virus (AAV) in connection with the administration of Sarepta’s gene therapy candidates in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMD).

DMD is a rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. LGMDs are a group of more than 30 distinct diseases that cause weakness and wasting of the muscles around the hips and shoulders, eventually progressing to the arms and legs. LGMD can be caused by a single gene defect that affects specific proteins within the muscle cell, including those responsible for keeping the muscle membrane intact.

Sarepta’s late-stage gene therapy candidates are delivered using AAV. Currently, all systemic AAV-delivered constructs are one-time therapies that cannot be re-dosed due to the robust post-administration development of neutralizing antibodies specific to the AAV vector.

Under the terms of the research license and option agreement, Sarepta will make an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone fees. If Sarepta exercises its options to enter any commercial license agreements, Selecta will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales. Additional financial details were not disclosed.

Selecta has generated preclinical evidence to support the potential for re-dosing patients receiving gene therapy. Selecta has reported that in preclinical studies, when used in combination with AAV gene therapy vectors, Selecta’s ImmTOR immune tolerance platform inhibits the development of neutralizing antibodies to the vector, permitting re-dosing of the gene therapy. 

“If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing,” said Doug Ingram, president and CEO, Sarepta Therapeutics. “We are encouraged by the data generated on the ImmTOR platform and excited to join with Selecta to explore the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed.”

Photo: Doug Ingram, president and CEO, Sarepta Therapeutics

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