On April 25, 2024 Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and Mammoth Biosciences, Inc., reported a collaboration to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types (Press release, Regeneron, APR 25, 2024, View Source [SID1234642352]). 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.

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"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.

"This exciting collaboration pairs two teams with a shared commitment to translating high science into ground-breaking in vivo genetic medicines that can potentially address the needs of more patients and more prevalent conditions," said Jennifer Doudna, Ph.D., co-founder and Chair of the Scientific Advisory Board at Mammoth Biosciences, CRISPR genome editing co-inventor and winner of the 2020 Nobel Prize in Chemistry.

Under the terms of the agreement, Mammoth will receive $100 million inclusive of $95 million in equity investment at signing, and an upfront payment, and is eligible to receive up to $370 million per target in development, regulatory and commercial milestone payments, and royalty rates ranging from single digits to mid-teens on future net sales of all collaboration products. In addition, Mammoth has the right to opt-in to co-funding and sharing profits on a majority of collaboration programs in lieu of receiving milestones and royalties. In exchange, for a period of five and a half years, Regeneron is obtaining broad access to Mammoth’s editing technologies, other than certain excluded targets, with the option to extend such access for an additional two years upon the payment of a research extension fee. The parties will jointly select and research collaboration targets, and then Regeneron will lead development and commercialization.