CRISPR Therapeutics and Capsida Biotherapeutics Announce Strategic Collaboration to Develop Gene-Edited Therapies for Amyotrophic Lateral Sclerosis and Friedreich’s Ataxia

- Collaboration leverages expertise and capabilities of both companies towards developing life-changing treatments for severe neurological diseases -

ZUG, Switzerland and CAMBRIDGE, MA, USA and THOUSAND OAKS, CA, USA I June 15, 2021 ICRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and Capsida Biotherapeutics Inc., a biotechnology company dedicated to developing breakthrough gene therapies using fully integrated adeno-associated virus (AAV) engineering, cargo development and manufacturing, today announced a strategic partnership to research, develop, manufacture and commercialize in vivo gene editing therapies delivered with engineered AAV vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.

Under the agreement, CRISPR Therapeutics will lead research and development of the Friedreich’s ataxia program and perform gene-editing activities for both programs, and Capsida will lead research and development of the ALS program and conduct capsid engineering for both programs. Capsida’s high-throughput AAV engineering platform generates capsids optimized to target specific tissue types and limits transduction of tissues and cell types that are not relevant to the target disease, potentially allowing for improved efficacy and safety. CRISPR Therapeutics and Capsida will each have the option to co-develop and co-commercialize the program that the other company leads. Following such option, the companies would equally share all research, development and commercialization costs and profits worldwide related to the collaboration product. As part of the collaboration, Capsida will also be responsible for process development and clinical manufacture of both programs and have the option to manufacture commercial products generated under the agreement.

“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. “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.”

“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, M.D., Chief Executive Officer of Capsida Biotherapeutics.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit

CRISPR THERAPEUTICS® word mark and design logo are registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is developing tissue-targeted gene therapies using its biologically driven, high-throughput adeno-associated virus (AAV) engineering and proprietary cargo development platform. As a fully integrated gene therapy company, Capsida is combining its differentiated AAV engineering and screening capabilities with cargo development and state-of-the-art manufacturing to establish a proprietary pipeline of groundbreaking gene therapies across a range of therapeutic areas for indications that are unreachable with current technologies. The company’s leadership is backed by decades of successful biologics manufacturing experience and deep AAV biology expertise. Visit us at to learn more.

SOURCE: Capsida Biotherapeutics

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