CRISPR and Capsida Partner in Gene Edited Therapies for ALS, Friedreich’s Ataxia – BioSpace

Capsida CEO,Dr. Robert (Bob)Cuddihy/ Photo Courtesy of Capsida Biotherapeutics

Capsida Biotherapeutics, which debuted in April already holding a research alliance with AbbVie, has wasted no time in putting its fully integrated adeno-associated virus (AAV) platform to work in the highest need therapeutic spaces.

This morning, Capsida announced a strategic collaboration with gene therapy leader, CRISPR Therapeutics to develop, manufacture and commercialize in vivo gene-edited therapies for amyotrophic lateral sclerosis (ALS) and Friedreichs ataxia (FA).

Per the agreement, CRISPR will lead the research and development of the Friedreichs ataxia program, while Capsida will shepherd the R&D for the ALS effort. Capsida, of course, will conduct the capsid engineering for both programs and will also assume responsibility for the process development and clinical manufacturing of both programs.

These are two neurodegenerative targets with significant unmet needs, and Capsida Chief Executive Officer, Dr. Robert M. Cuddihy, is excited about the progress this collaboration could bring.

I think it's going to take a different and a novel approach, and I think we're combining two innovative approaches that hopefully can really be disruptive to the field and start to bring some benefit to patients, he said.

Maybe the lesser-known of the two indications, Friedreichs ataxia is a genetic, progressive neurodegenerative movement disorder caused by an expansion of a GAA trinucleotide repeat in the FXN gene. This bears similarity to a familial form of ALS that the partners will be looking at.

What we can do in both of those diseases with genome editing is essentially excise the pathogenic repeat out of the genome to return the genome to more or less its non-aberrant state, said CRISPR Chief Operating Officer, Dr. Lawrence Klein.

CRISPR has done extensive work on the gene-editing side of bothdiseases. The next challenge, Klein shared, is to deliver the machinery necessary for editing, and Capsida stood out as having these particular capabilities.

We looked at a number of different companies that are working on engineering AAV to be more neurotropic, and the data that Capsida has been able to generate through their engineering platform really stood out. The neurotropic capacity of their engineered AAV vectors is truly remarkable, he said.

Capsidas high-throughput engineering and cargo development platform is designed to improve the safety, specificity, and reach of gene therapies for currently inaccessible targets.

By selecting the delivery vehicle, a novel engineered capsid, that has significantly enhanced tissue specificity versus AAV9, while de-targeting the tissues not relevant to the targeted disease, we expect potentially enhanced efficacy and safety profiles, Cuddihy explained.

In two diseases with no approved disease-modifying treatment options, time is of the essence, and Capsida, which has its own GMP manufacturing facility, could help to expedite time-to-market for a successful therapy down the line.

We pride ourselves on really being a fully integrated gene therapy company. Not only are we selecting novel capsids, we're assessing them upfront for manufacturability, ability to scale. We have our GMP facility opening up this year, so we're raring to go, Cuddihy said.

In this evenly-yoked partnership, both CRISPR and Capsida will have the option to co-develop and co-commercialize each program. Following such an option, the two would equally share all research, development, and commercialization costs and profits of an approved product.

I think there is a high degree of unmet need where small molecules and antibodies haven't had as much success, and where gene therapy and genome engineering can really bring about new approaches to treat these diseases, Klein said. Delivery technologies are getting better and better where I think we can really start to access the targets in a way that doesnt bring toxicity challenges.

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CRISPR and Capsida Partner in Gene Edited Therapies for ALS, Friedreich's Ataxia - BioSpace