Partnership Targets Gene-editing Therapies for Familial ALS

CRISPR Therapeutics and Capsida Biotherapeutics have partnered to develop gene-editing therapies for familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.

Capsida specializes in designing virus-based means of delivering therapies to specific cells, while CRISPR’s expertise lies in gene-editing technology, particularly the CRISPR gene-editing system.

“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,” Robert Cuddihy, MD, Capsida’s CEO, said in a press release.

CRISPR, an abbreviation for “clustered regularly interspaced short palindromic repeats,” comes from a defense mechanism that bacteria and some other single-celled organisms use to fight off viral infections.

In its natural setting, CRISPR is used to alter the DNA sequences a virus uses to carry out an infection. Scientists recently discovered how to repurpose CRISPR into a much broader and more tunable tool, enabling them to add, remove, or otherwise change specific stretches of DNA, such as mutations involved in specific diseases.

The CRISPR machinery can be delivered to cells via a type of harmless virus called an adeno-associated virus (AAV). Capsida’s proprietary AAV engineering platform is designed to generate viruses optimized to target specific tissue types. Limiting the virus’ ability to enter other cells is expected to improve the therapy’s safety and effectiveness.

Under the agreement, Capsida will lead research and development into familial ALS therapies; CRISPR will focus on Friedreich’s ataxia. Capsida will engineer the AAVs to be used in each program.

Each company has the option to co-develop and co-commercialize the program that the other company leads. Should one of the collaborators pursue this option, each would share in all research, development, and commercialization costs, as well as the profits.

Capsida, which is responsible for process development and manufacturing of both programs, also has the option to manufacture any commercialize products resulting from this partnership.

“We are excited to enter this collaboration with Capsida,” said Samarth Kulkarni, PhD, CEO of CRISPR Therapeutics. “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.”