The collaboration will combine Regeneron's expertise in AAVs and antibodies with Mammoth's knowledge in compact gene editing to develop therapies for multiple tissues and cell types
Regeneron partner on gene editing therapies with Mammoth Biosciences. (Credit: National Cancer Institute on Unsplash)
Regeneron Pharmaceuticals and Mammoth Biosciences have partnered to research, develop and commercialise in vivo CRISPR-based gene editing therapies for multiple tissues and cell types.
US-based Regeneron is developing adeno-associated viral vectors (AAVs) that utilise antibody-based targeting methods to deliver genetic medicines to specific tissues and cells.
Mammoth Biosciences is a biotechnology company working on ultracompact nucleases and related gene editing systems. These systems offer a range of editing capabilities compared to traditional CRISPR-based systems.
The collaboration will combine Regeneron’s expertise in AAVs and antibodies with Mammoth’s knowledge in compact gene editing. Both entities will develop treatments for diseases that affect tissues beyond the liver.
Regeneron senior vice president and Regeneron Genetic Medicines co-head Christos Kyratsous said: “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.”
Under the agreement, Mammoth will initially receive $100m, which includes $95m in equity investment upon signing, along with an upfront payment.
Mammoth stands to gain up to $370m per target via various milestone payments during development, regulatory approvals, and commercialisation.
The company will also receive royalties ranging from single digits to mid-teens on future net sales of collaborative products.
Additionally, the biotechnology company retains the option to participate in co-funding and profit-sharing arrangements for most collaboration projects instead of receiving milestone payments and royalties.
In return, Regeneron Pharmaceuticals gains broad access to Mammoth’s editing technologies, excluding certain targets, for five and a half years. This access may be extended for an additional two years upon payment of a research extension fee.
Both parties will collaborate in selecting and researching targets, with Regeneron leading the development and commercialisation activities.
Mammoth Biosciences co-founder and CEO Trevor Martin said: “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.”