Regeneron and Mammoth Biosciences Collaborate to Pursue Next-Generation CRISPR-Based Gene Editing for Multiple Diseases

Mammoth’s proprietary ultracompact CRISPR-based gene editing platform and Regeneron’s proprietary delivery technologies set to advance in vivo programs in multiple tissue and cell types

Mammoth to receive $100 million total upfront payment and equity investment from Regeneron at signing

TARRYTOWN, N.Y., April 25, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and Mammoth Biosciences, Inc., today announced a collaboration to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types. Regeneron is developing adeno-associated viral vectors (AAVs) using antibody-based targeting to enhance delivery of genetic medicine payloads to specific tissues and cell types. Mammoth is developing novel ultracompact nucleases and associated gene editing systems, with a variety of editing functionalities at a significantly smaller size than other CRISPR-based systems, including first generation Cas9 nucleases. By leveraging Regeneron’s expertise in AAV and antibody engineering and Mammoth’s expertise in ultracompact gene editing systems, the teams will endeavor to create disease-modifying medicines that can be delivered to tissues beyond the liver, to which most gene editing treatments are currently limited.

“We believe in the incredible power of gene editing, which we are utilizing in our diverse preclinical and clinical genetic medicines pipeline. After years spent developing our next-generation delivery approaches, we are eager to combine them with Mammoth’s gene editing systems to better match payload, delivery system and disease type,” said Christos Kyratsous, Ph.D., Senior Vice President and co-Head of Regeneron Genetic Medicines at Regeneron. “Together, we have the potential to overcome significant delivery hurdles and effectively reach tissues around the body, impact multiple diseases and dramatically increase the number of patients who could benefit from gene editing treatments.”

“Mammoth brings over a decade of scientific expertise in CRISPR, beginning with our co-founders’ work in the Doudna Lab at University of California Berkeley. We believe we can further our mission to transform the lives of patients by accelerating the discovery and development of genetic medicines in collaboration with Regeneron. Mammoth’s ultracompact CRISPR systems address the size constraints of viral delivery and complement Regeneron’s targeted AAV technologies. We look forward to working with Regeneron to enable all-in-one AAV delivery and unlock the true potential of in vivo gene editing,” said Trevor Martin, Ph.D., co-founder and Chief Executive Officer of Mammoth Biosciences.