CRISPR Therapeutics, Capsida Collaborate to Develop Gene Therapies

CRISPR Therapeutics and Capsida Biotherapeutics recently entered into a collaboration to develop, manufacture, and commercialize gene therapies. 

The companies will leverage adeno-associated virus (AAV) vectors to develop gene therapies for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia, a genetic, progressive, neurodegenerative movement disorder.

“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,” Samarth Kulkarni, PhD, chief executive officer of CRISPR Therapeutics, said in the announcement. 

“This new partnership is one more step in our overall strategy of bringing together innovative and complementary technologies to unlock the full potential of our core platform,” Kulkarni continued.

Under the agreement, CRISPR Therapeutics will lead research and development of the Friedreich’s ataxia program and perform gene-editing activities for both programs. The company will provide its CRISPR/Cas9 platform, a gene-editing technology that allows for precise, directed changes to a patient’s DNA.

Meanwhile, Capsida will lead research and development of the ALS program and conduct capsid engineering for both programs. The company will provide its AAV engineering platform. The platform generates capsids optimized to target specific tissue types and limits transduction cell types not relevant to disease.

Capsida will also be responsible for the process development and clinical manufacture of both programs and will have the option to manufacture commercial products created under the collaboration. 

The companies will each have the option to co-develop and co-commercialize the program that the other company leads. The companies will also equally share all research, development, and commercialization costs and profits globally. 

“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, MD, chief executive officer of Capsida Biotherapeutics.

Experts believe that the next generation of cell therapies holds significant promise for patients because it creates a point-of-care that may cure many illnesses. 

CRISPR technology has become an increasingly popular tool used to edit human genes, also known as in vivo gene editing. 

Over the past few years, the tool has been used for many diseases, including inherited eye diseases, neurodegenerative conditions, including Alzheimer’s disease, and non-inherited diseases, including cancer and HIV.