On June 22, 2020, Sarepta Therapeutics, Inc. (NASDAQ:SRPT) (https://www.sarepta.com/), a leader in precision genetic medicine for rare diseases, and Codiak BioSciences, Inc., a company at the forefront of advancing engineered exosomes as a new class of biologic medicines, announced a global research and option agreement 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 is eligible to receive up to $72.5 million in up-front and near-term license payments plus research funding. Sarepta is granted an option to any of the candidates developed pursuant to the research alliance. Exosomes are natural nanoparticles that serve, in part, as the body’s intercellular communication system, facilitating the transfer of a wide variety molecular payloads between cells. Exosomes provide a unique advantage as a targeted delivery system for genetic medicines because they are believed to be inherently non-immunogenic. Through its proprietary, engEx Platform, Codiak can engineer exosomes with specific cargos and guide tropism to cell types of interest. The collaboration will leverage Codiak’s exosome engineering capabilities with Sarepta’s expertise in precision genetic medicine to develop next-generation therapeutics for patients with neuromuscular diseases that have few or no treatment options. “As Sarepta expands its leadership position in precision genetic medicine, this alliance with Codiak furthers our goal to deliver the most advanced therapies to patients.
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