Regeneron to collaborate on gene editing therapies with Doudna-founded Mammoth

By Thomson Reuters Apr 25, 2024 | 6:19 AM

(Reuters) – Regeneron Pharmaceuticals and Mammoth Biosciences will collaborate to research, develop and commercialize gene editing therapies for multiple diseases, the companies said on Thursday.

Mammoth’s CRISPR-based gene editing platform and Regeneron’s delivery technologies will be used to create disease-modifying medicines that can be delivered to tissues beyond the liver.

CRISPR, discovered by Jennifer Doudna and CRISPR Therapeutics co-founder Emmanuelle Charpentier, uses molecular “scissors” to trim faulty parts of genes that can then be disabled or replaced with new strands of normal DNA.

In 2023, the U.S. FDA approved five gene therapies, including a sickle cell disease treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that uses the latter’s gene editing technology.

Regeneron joins drugmakers Bayer and Vertex Pharmaceuticals in striking partnership deals with Mammoth for development of gene therapies.

Mammoth, which was founded by Doudna, will receive $100 million in total upfront payment and equity investment from Regeneron and could receive up to $370 million per therapy in development, regulatory and commercial milestone payments.

The parties will jointly select and research for the therapies, and then Regeneron will lead development and commercialization.

(Reporting by Christy Santhosh in Bengaluru; Editing by Krishna Chandra Eluri)