Setting its Sights on Neurological Disorders, CRISPR Therapeutics Turns to an AAV Startup That Just Emerged Out of Stealth

On June 15th, Swiss-American biotech company CRISPR Therapeutics signed a striking deal with Capsida Biotherapeutics, a Thousand Oaks, CA-based startup that recently emerged out of stealth.

The collaboration aims to build better targeted gene therapies to treat debilitating and life-threatening, rare familial neurological disorders, amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia (FA).

Both ALS and Friedreich’s ataxia are rare progressive neurodegenerative diseases that affect nerve cells in the brain and the spinal cord, resulting in the impairment of voluntary muscle coordination. As with many other nervous system disorders, there is currently no cure or effective treatment for either disease. Importantly, traversing the blood-brain barrier poses a great challenge to drug delivery in the clinic.

AAV Gene Therapies

AAV is a powerful and harmless drug delivery tool used by several top gene therapies, including Novartis’ Zolgensma, that was FDA approved for Spinal Muscular Atrophy (SMA). Interestingly, Capsida’s breakthrough technology is also based around potential high-throughput AAV strains designed to specifically target nerve cells in the central nervous system affected by neurodegeneration.

With rapid advancements in CRISPR-Cas9 gene-editing technology, most companies are venturing out to explore viral vectors with limited off-target side-effects, particularly harmless AAV strains to shuttle the gene editing system into the afflicted cells for efficacy, safety, and improved delivery across the blood-brain barrier.

The strategic collaboration between CRISPR and Capsida seeks to develop and manufacture better targeted in vivo gene editing therapies delivered via engineered next-generation AAV vectors.

“Bringing together Capsida’s fully integrated, tissue targeting gene therapy platform with CRISPR Therapeutics’ leading gene-editing capabilities gives us the potential to develop first-in-class gene therapies for patients with severe neurological disorders and expand the reach of Capsida’s broadly enabling capabilities,” said Robert Cuddihy, CEO of Capsida.

Agreement Terms

As part of their agreement, CRISPR Therapeutics and Capsida will take up the research and development for Friedreich’s ataxia and ALS, respectively. Additionally, CRISPR will develop gene editing candidates, and Capsida will perform high-throughput capsid design for both programs.

Further, both companies can also opt to co-develop and co-commercialize the program that the other company leads. Although the exact financial terms of the pact were not disclosed, the duo agreed to equally share all research, development, and commercialization costs besides worldwide profits.

A Strategic Partnership

CRISPR therapeutics’ cutting-edge transformative gene editing technology is among the most discussed in the biotech world today. The company has collaborated with leading players in the industry like Bayer, Vertex Pharmaceuticals, and ViaCyte to establish a therapeutic portfolio of altering the human cells outside the body across a broad spectrum of rare diseases such as sickle cell anemia, beta thalassemia, including others.

“We are excited to enter this collaboration with Capsida. 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,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics

Capsida’s engineered, best-in-class, nervous system-directed AAV, would significantly expand CRISPR therapeutics’ gene-editing portfolio, giving it an edge in the market.

Editor: Rajaneesh K. Gopinath, Ph.D.

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Author

Isha Kapoor