Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment – FierceBiotech

Sarepta Therapeutics has penned its second DMD gene therapy pact this year as it announces a tie-up with Frances Genethon, a nonprofit R&D org.

The research collaborationwill see the Franco-American pair jointly develop treatments for Duchenne muscular dystrophy and comes after Sareptas first FDA approval for DMD with its controversial med Exondys 51 (eteplirsen).

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Sarepta is looking to tap into Genethons preclinical microdystrophin gene therapy approach, which can target the majority of patients with DMD. Its current med can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.

It is hoping that with new tie-ups, it could produce a gene therapy that could treat many more, if not all, patients with the disease, although this is still some years off. DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease primarily affects young boysand occurs in about one out of every 3,600 male infants worldwide.

This builds on the pacts announced at the start of the year at the JPM conference, which saw it sign a deal with the Nationwide Childrens Hospital, which also focuses on the microdystrophin gene therapy program, as well as another form of gene therapy.

An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Childrens. It also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program, originally developed by researcher Paul Martin. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin. The goal will be to produce a muscle cell that can function normally even when dystrophin is absent, Sarepta said at the time.

Under the terms of its latest collaboration, Genethon will be responsible for the early development work. Sarepta has the option to co-develop Genethons microdystrophin program, which includes exclusive U.S. commercial rights. Financial terms, as is becoming more common with these pacts, have not been disclosed.

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Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industrys most comprehensive franchise in DMD, said Ed Kaye, Sareptas outgoing chief. This partnership brings together our collective experience in Duchenne drug development and Genethons particular expertise in gene therapy for rare diseases. We look forward to working with Genethon given their knowledge, large infrastructure and state-of the-art manufacturing capabilities to advance next generation therapies for DMD.

Frederic Revah, CEO of Genethon, added: Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. In order to accelerate the development of a treatment, we are very pleased to partner with Sarepta Therapeutics, which has demonstrated commitment and success for innovative therapies for Duchenne muscular dystrophy. This partnership brings together the highly complementary and synergistic expertises of Sarepta and Genethon, to the benefit of the patients.

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Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment - FierceBiotech