Sarepta Therapeutics, GenEdit collaborate on gene editing therapies

By The Science Advisory Board staff writers

February 2, 2022 -- Sarepta Therapeutics and GenEdit have released results from a research collaboration and option agreement under which the companies are employing GenEdit's NanoGalaxy platform and Sarepta's technology to develop gene editing therapeutics for the treatment of neuromuscular diseases.

As part of the agreement, Sarepta obtained exclusive option rights to license polymer nanoparticles developed by GenEdit for up to four neuromuscular indications selected by Sarepta.

Initial in vivo results from the research collaboration have demonstrated the potential of GenEdit's polymer nanoparticles to deliver therapeutic cargo to specific muscle tissue after systemic administration. The research collaboration and option agreement commenced in December 2020.

Gene editing has the potential to revolutionize the treatment of diseases caused by genetic mutations by permanently modifying the genes that lead to disease. Sarepta is pursuing a variety of approaches to genetic medicine, including gene editing, as a potentially curative treatment for rare neuromuscular diseases. GenEdit has demonstrated in preclinical studies that its NanoGalaxy platform can selectively deliver to different tissues a variety of functional genetic medicine cargos, including CRISPR-Cas9 ribonucleoprotein, for targeted in vivo gene editing.

In addition to research payments, under the terms of the agreement, GenEdit may receive up to $57 million in near-term payments. It is also eligible to receive future development, regulatory and commercial milestones, and tiered royalties on future product sales. Additional financial details were not disclosed.

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