GenEdit Polymer Nanoparticles Rushed by Sarepta

In the near future, Sarepta Therapeutics will pay GenEdit up to $57 million for access to the Eli Lilly-backed biotech’s non-viral gene editing delivery platform. According to the firms, the gene therapy company will combine its gene editing technology with GenEdit NanoGalaxy platform to target up to four neuromuscular indications chosen by Sarepta.

Sarepta recently announced the cancellation of a gene treatment for a neurodegenerative illness that it had developed in collaboration with Lysogene. Sarepta’s stock fell roughly 15% after the biopharma indicated last month that it planned to approach the FDA for approval of its fourth Duchenne muscular dystrophy medication as early as next year. Sarepta is now collaborating with GenEdit in order to expand its gene editing workflow.

CEO Kunwoo Lee, Ph.D., cofounded the biotech after working on CRISPR-Cas9-based research in DMD with CRISPR pioneer Jennifer Doudna at the University of California, Berkeley. Nonviral polymer nanoparticles developed by GenEdit are designed to address safety concerns while delivering gene editing treatments to particular tissues, with functional activity sustained after many doses.

The Sarepta collaboration in neuromuscular disorders is a “great area to show how the platform works,” according to Lee, who has an internal specialty in neurology and immunology. The CEO of refused to say when the programmes will be tested in humans. The two businesses first collaborated in December 2020, and preliminary in vivo research has indicated that GenEdit polymer nanoparticles may transport genetic medicine cargos to particular muscle tissue after repeated injection.