Sarepta, GenEdit join forces on gene editing therapeutics

Sarepta Therapeutics and San Francisco-based startup GenEdit will collaborate on the development of gene editing therapeutics for neuromuscular disease.

Per the agreement, Massachusetts-based Sarepta — a precision genetics medicine company — will have exclusive access to GenEdit’s gene delivery technology. GenEdit will receive $57 million in near-term payments from Sarepta with future payments contingent on meeting unspecified development, regulatory and commercial milestones. 

GenEdit focuses on tissue-specific gene therapy. The company received $26 million in funding from Eli Lilly and other investors last September to advance its research, including development of NanoGalaxy, GenEdit’s proprietary polymer nanoparticles.

The two companies plan to use the NanoGalaxy platform to find cures for neuromuscular diseases, and to advance the development of gene editing treatments. In 2020, the companies began in vivo testing to see if the polymer nanoparticles could be delivered directly to specific muscle tissue. The results have been encouraging, with findings showing such treatment is possible.

Sarepta recently ended a once-promising partnership with Lysogene Therapeutics months before final data were expected to arrive. According to Lysogene, the partnership ended following "unsuccessful discussions" over transference of production responsibility back to Lysogene.