On June 15, 2021 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and Capsida Biotherapeutics Inc., a biotechnology company dedicated to developing breakthrough gene therapies using fully integrated adeno-associated virus (AAV) engineering, cargo development and manufacturing, reported a strategic partnership to research, develop, manufacture and commercialize in vivo gene editing therapies delivered with engineered AAV vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia (Press release, CRISPR Therapeutics, JUN 15, 2021, View Source [SID1234584021]).

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Under the agreement, CRISPR Therapeutics will lead research and development of the Friedreich’s ataxia program and perform gene-editing activities for both programs, and Capsida will lead research and development of the ALS program and conduct capsid engineering for both programs. Capsida’s high-throughput AAV engineering platform generates capsids optimized to target specific tissue types and limits transduction of tissues and cell types that are not relevant to the target disease, potentially allowing for improved efficacy and safety. CRISPR Therapeutics and Capsida will each have the option to co-develop and co-commercialize the program that the other company leads. Following such option, the companies would equally share all research, development and commercialization costs and profits worldwide related to the collaboration product. As part of the collaboration, Capsida will also be responsible for process development and clinical manufacture of both programs and have the option to manufacture commercial products generated under the agreement.

"We are excited to enter this collaboration with Capsida. The combination of Capsida’s AAV engineering platform and CRISPR Therapeutics’ gene-editing platform has the potential to enable transformative gene-edited therapies for patients with neurological diseases," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "This new partnership is one more step in our overall strategy of bringing together innovative and complementary technologies to unlock the full potential of our core platform."

"Bringing together Capsida’s fully integrated, tissue targeting gene therapy platform with CRISPR Therapeutics’ leading gene-editing capabilities gives us the potential to develop first-in-class gene therapies for patients with severe neurological disorders and expand the reach of Capsida’s broadly enabling capabilities," said Robert Cuddihy, M.D., Chief Executive Officer of Capsida Biotherapeutics.