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Main focus: Gene editing for the treatment of central nervous system (CNS) disorders
Company stage: Pre-clinical
Diseases: ALS and Friedreich’s ataxia
Genome editing tool: Delivery using engineered adeno-associated virus (AAV)
Funding stage: Private
Location: Newbury Park, CA, USA
Partners: CRISPR Therapeutics
Capsida Biotherapeutics is developing adeno-associated viral (AAV) vectors for novel gene therapies. The company's involvement in the gene editing space comes from the collaboration with CRISPR Therapeutics. The two companies are developing novel in vivo gene-editing therapies for familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.