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Capsida Biotherapeutics

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.


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Company: Capsida Biotherapeutics
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