Partnership Targets Gene-editing Therapies for Familial ALS

Partnership Targets Gene-editing Therapies for Familial ALS

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Partnership Targets Gene-editing Therapies for Familial ALS

CRISPR Therapeutics and Capsida Biotherapeutics have partnered to develop gene-editing therapies for familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Capsida specializes in designing virus-based means of delivering therapies to specific cells, while CRISPR’s expertise lies in gene-editing technology, particularly the CRISPR gene-editing system. “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…

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