Rare Daily Staff

Gene editing company CRISPR Therapeutics and AAV gene therapy developer Capsida Biotherapeutics have entered a strategic partnership to research, develop, manufacture, and commercialize in vivo gene editing therapies delivered with engineered AAV vectors for the treatment of the neurodegenerative conditions familial amyotrophic lateral sclerosis and Friedreich’s ataxia.

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.

Each company will 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.

“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, CEO of Capsida Biotherapeutics.

Photo: Robert Cuddihy, CEO of Capsida Biotherapeutics

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