CRISPR, Capsida team up on gene-edited therapies for ALS, Friedreich's ataxia

CRISPR Therapeutics has inked a partnership with newly launched Capsida Biotherapeutics to develop and commercialise in vivo gene-editing therapies delivered with engineered adeno-associated virus (AAV) vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich's ataxia, the companies announced Tuesday. Financial terms of the deal were not disclosed.

Capsida CEO Robert Cuddihy suggested that pairing his company's fully integrated, tissue-targeting gene therapy platform with CRISPR's gene-editing capabilities "gives us the potential to develop first-in-class gene therapies for patients with severe neurological disorders."

Complementary technologies

Under the partnership, Capsida will lead R&D activities for the ALS programme and conduct capsid engineering for both programmes using its high-throughput AAV engineering platform. According to the companies, the platform generates capsids "optimised to target specific tissue types and limits transduction of tissues and cell types that are not relevant to the target disease," thereby potentially improving efficacy and safety. For its part, CRISPR will head up the Friedreich's ataxia programme, and handle gene-editing activities for both.

Meanwhile, CRISPR and Capsida will both have the option to co-develop and co-market the programme led by the other. If they choose to do so, the companies will equally split all R&D and commercialisation costs and profits worldwide related to the collaboration product. Capsida has also agreed to be responsible for process development and clinical manufacture of both programmes, and has the option to manufacture commercial products generated under the deal.

CRISPR CEO Samarth Kulkarni said the new tie-up "is one more step in our overall strategy of bringing together innovative and complementary technologies to unlock the full potential of our core platform."