NEW YORK – Regeneron Pharmaceuticals said Thursday that it will collaborate with Mammoth Biosciences on the research, development, and commercialization of therapies for multiple tissues and cell types using in vivo CRISPR-based gene editing.
As part of the deal, Mammoth will receive a $95 million equity investment and a $5 million upfront payment from Regeneron at signing.
Mammoth, cofounded by Jennifer Doudna, who won the Nobel Prize for her gene editing work in 2020, is developing small CRISPR-based systems, while Regeneron is developing adeno-associated viral vectors using antibody-based targeting to deliver genetic therapy to specific cells and tissues.
Under the terms of the deal, the firms will jointly select and research targets, and Regeneron will lead development and commercialization.
Mammoth will also be eligible to receive up to $370 million per target in development as well as regulatory and commercial milestone payments and royalty rates "ranging from single digits to mid-teens on future net sales of all collaboration products," Regeneron said in a statement. Mammoth may also decide to co-fund and share profits on most of the collaboration programs in lieu of milestone and royalty payments, the Tarrytown, New York-based pharma company said.
In exchange, for a period of five and a half years, Regeneron will obtain broad access to Mammoth's gene editing technologies, excluding certain targets, with the option to extend access for an additional two years upon the payment of an extension fee.
"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, senior VP and co-head of Regeneron Genetic Medicines.
Trevor Martin, cofounder and CEO of Mammoth Biosciences, said that the firm's "ultracompact CRISPR systems address the size constraints of viral delivery and complement Regeneron's targeted AAV technologies," adding that the collaboration can "enable all-in-one AAV delivery and unlock the true potential of in vivo gene editing."