Gene Therapies for ALS, Friedreich’s Ataxia the Focus of New CRISPR, Capsida Partnership


The news comes after CRISPR’s favorable data readouts in its sickle cell and beta thalassemia studies.

CRISPR Therapeutics and Capsida Biotherapeutics have announced a strategic partnership for the research and development of in vivo gene editing therapies delivered via AAV vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Freidreich’s ataxia.1

“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, PhD, chief executive officer, CRISPR Therapeutics, in a statement. “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.”

CRISPR Therapeutics will lead research and development of the Friedreich’s ataxia program and develop gene-editing strategies for both programs, while Capsida will lead research and development of the ALS program and develop capsids for both programs using their high-throughput AAV engineering platform designed to generate optimized and specific capsids while limiting transduction of tissues and cell types irrelevant to the target disease. The targeted capsids may allow for improved efficacy and safety over other capsid designs. Capsida will also be responsible for clinical manufacture and development of both programs and will have the option to manufacture commercial products generated under the agreement.

READ MORE: FDA Clears IND Application for SOD1-ALS Gene Therapy

While the financial terms of the deal have not been disclosed, each company will have the option to co-develop and co-commercialize both programs, in which case the companies would equally share all research, development, and commercialization costs and profits related to the product.

“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,” added Robert Cuddihy, MD, chief executive officer, Capsida Biotherapeutics.

CRISPR Therapeutics

CRISPR Therapeutics has previously established strategic collaborations with companies such as Bayer, Vertex Pharmaceuticals, and ViaCyte. The company, with the use of its proprietary CRISPR/Cas9 platform, currently has a therapeutic portfolio that includes hemoglobinopathies, oncology, regenerative medicine, and rare diseases. 

CRISPR recently read out data from 2 studies from its partnership with Vertex for the treatment of sickle cell disease and beta thalassemia. Patients dosed with the experimental drug CTX001 showed a “consistent and sustained” response for at least 3 months after dosing. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all 7 with severe sickle cell disease were pain free, the company announced.

One patient not included in the data cutoff experienced a cerebellar hemorrhage less than 3 months after being treated. The serious adverse event was determined to be related to the busulfan conditioning gene therapy patients undergo before receiving treatment and has since resolved, according to Vertex.

Capsida Therapeutics launched in April 2021 and is aiming initially at broad rare and neurological diseases. It has a current partnership with AbbVie with the goal of identifying 3 central nervous system targets.

1. CRISPR Therapeutics and Capsida Biotherapeutics announce strategic collaboration to develop gene-edited therapies for Amyotrophic Lateral Sclerosis and Friedreich’s ataxia. News release. CRISPR Therapeutics. June 15, 2021.
2. Vertex and CRISPR Therapeutics present new data in 22 patients with greater than 3 months follow-up post-treatment with investigational CRISPR/Cas9 gene-editing therapy, CTX001™ at European Hematology Association Annual Meeting. News release. CRISPR Therapeutics. June 11, 2021.
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