Capsida and CRISPR partner on ALS and Friedreich's ataxia research

Swiss-American biotech CRISPR Therapeutics (Nasdaq: CRSP) and USA-based Capsida Biotherapeutics have entered into 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.

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.