Sarepta wagers $72.5 on neuromuscular disease collaboration

22 June 2020

US self-styled leader in precision genetic medicine for rare diseases Sarepta Therapeutics (Nasdaq: SRPT) aims to expand its gene therapy capability via a global research and option agreement with Codiak BioSciences to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing and RNA technologies for neuromuscular diseases.

The engineered exosome approach offers the potential to effectively deliver genetic therapeutics without triggering the adaptive immune response.

The two-year agreement includes up to five neuromuscular targets. Codiak, which in 2015 was founded in part on technology developed in the laboratories of Raghu Kalluri, Professor and chairman of the Department of Cancer Biology, MD Anderson, is eligible to receive up to $72.5 million in upfront and near-term license payments plus research funding. Sarepta is granted an option to any of the candidates developed pursuant to the research alliance.

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