CRISPR Partners with Capsida to Develop Gene-editing Therapies

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

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CRISPR Therapeutics has joined forces with Capsida Biotherapeutics to discover and develop new gene-editing therapies for Friedreich’s ataxia (FA) and familial amyotrophic lateral sclerosis (ALS).

The partnership seeks to combine CRISPR Therapeutics’ gene-editing expertise using its proprietary CRISPR/Cas9 platform with Capsida’s tissue-targeted, adeno-associated virus (AAV)-based gene therapies.

“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, CRISPR Therapeutics’ CEO, said in a press release.

“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 added.

The CRISPR-Cas9 system, originally discovered in bacteria as a defense mechanism, allows researchers to edit parts of the genome by adding, removing, or changing specific sections of the DNA sequence.

Modified and harmless versions of AAVs are often used in gene therapy to deliver the gene of interest to cells or, as in this case, the CRISPR/Cas9 machinery that will modify a patient’s DNA sequence.

Capsida’s high-throughput AAV engineering platform generates viral carriers optimized to target tissue types relevant for a given disease, potentially allowing for improved safety and effectiveness.

This type of gene-editing approach has the potential to correct the FA-causing excessive DNA expansion in the FXN gene, as well as the most common gene mutations associated with familial ALS, a serious neuromuscular disease.

Under the agreement, CRISPR Therapeutics will lead the FA program and conduct gene-editing activities for both programs, while Capsida will lead the ALS program and be responsible for AAV engineering, process development, and clinical manufacture for both programs.

Each company may opt to co-develop and co-commercialize the program that the other company leads, in which case they would equally share all research, development, and commercialization costs, as well as profits.

Capsida will also have the option to manufacture any commercial products resulting from this partnership.

Robert Cuddihy, MD, Capsida’s CEO, said that “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.”