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Capsida Deal Focuses on Gene Editing

Newbury Park-based Capsida Biotherapeutics announced a collaboration with CRISPR Therapeutics to develop, manufacture and commercialize gene editing therapies for amyotrophic lateral sclerosis, or ALS, and Friedreich’s ataxia.

The two biotech companies will combine their respective specializations to produce the therapies for the severe and rare neurological diseases.

CRISPR will oversee research and development of the Friedreich’s ataxia program and head gene-editing for both programs. The company’s proprietary CRISPR/Cas9 platform is a gene editing technology that allows for accurate and directed changes to genomic DNA.

Capsida will lead research and development of the ALS program and run capsid engineering for both programs. Capsida’s adeno-associated virus, or AAV, engineering platform allows for the targeting of specific tissue, delivering improved safety and effectiveness.

“We are excited to enter this collaboration with Capsida,” Dr. Samarth Kulkarni, CRISPR’s chief executive, said in a statement. “The combination of Capsida’s AAV engineering platform and CRISPR Therapeutics’ gene-editing platform has the potential to enable transformative gene-edited therapies for patients with neurological diseases.”  Capsida Chief Executive Dr. Robert Cuddihy wrote in an email to the Business Journal that there is a strong cultural fit with CRISPR, “given both companies have the same nimble and flexible operating  model and operate  with the same sense  of urgency.”  The companies will have the option to co-develop and co-commercialize the program that the other company leads. If that option is exercised, Capsida and CRISPR would equally share research, development and commercialization costs and profits worldwide related to the products.

Also, Capsida will be responsible for both programs’ process development and clinical manufacturing. It will have the option to manufacture the final commercial products.   Addressing potential obstacles, Cuddihy said “efficient delivery remains one of the biggest challenges in enabling in vivo gene editing, and this collaboration helps overcome that challenge.”  The Capsida and CRISPR collaboration comes a month and a half after Capsida’s $140 million funding debut, which included $50 million in Series A money from Versant Ventures and Westlake Village BioPartners in Westlake Village plus a $90 million collaboration with pharma giant AbbVie in Chicago.The Series A financing will be instrumental in Capsida’s plan to finance its manufacturing facility in Thousand Oaks.

There is currently no timeline or roadmap for project milestones, but Cuddihy wrote that Capsida and CRISPR are working as diligently and expeditiously as possible.

Antonio Pequeño IV
Antonio Pequeño IV
Antonio “Tony” Pequeño IV is a reporter covering health care, finance and law for the San Fernando Valley Business Journal. He specializes in reporting on some of the biggest names in the Valley’s biotechnology sector. In addition to his work with the Business Journal, Tony has reported with BuzzFeed News on the unsupervised use of Clearview AI, a controversial facial recognition technology. Tony, who also conducts freelance reporting, graduated from the USC’s Master of Science in Journalism program in 2021. He is in his fifth year as a journalist as of 2021.
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