Regeneron and Mammoth Biosciences to collaborate on CRISPR gene therapies

Regeneron Pharmaceuticals and Mammoth Biosciences have announced a collaboration for the research, development and commercialisation of in vivo CRISPR-based gene-editing therapies for multiple tissue and cell types.
The collaboration will utilise Regeneron’s expertise in adeno-associated viral vectors (AAVs) and antibody engineering, as well as Mammoth’s experience in ultacompact gene-editing systems. It is intended that the two companies will ‘create disease-modifying medicines that can be delivered to tissues beyond the liver, to which most gene editing treatments are currently limited’, according to Regeneron’s press release.
Under the terms of this agreement, Mammoth will receive $100m, which includes $95m in equity investment at signing and an upfront payment, as well as be eligible for up to $370m per target in development, regulatory and commercial milestone payments, and royalty rates on future net sales of the collaboration products.
Christos Kyratsous PhD, senior vice president and co-head of Regeneron Genetic Medicines at Regeneron, commented: “We believe in the incredible power of gene editing, which we are utilising 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. 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.”
Trevor Martin PhD, co-founder and chief executive officer of Mammoth Biosciences, added: “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, US. 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.”
“This exciting collaboration pairs two teams with a shared commitment to translating high science into groundbreaking in vivo genetic medicines that can potentially address the needs of more patients and more prevalent conditions,” stated Jennifer Doudna PhD, 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.