Regeneron Pharmaceuticals and Mammoth Biosciences to Collaborate on Gene Editing Therapies

New York-based Regeneron Pharmaceuticals is partnering with Brisbane, California-based Mammoth Biosciences (founded by Jennifer Doudna) to research, develop, and commercialize gene editing therapies for various diseases. The companies disclosed the collaboration plan on Thursday, April 25, 2024.

Regeneron’s delivery technologies and Mammoth’s CRISPR-based gene editing platform will be used to create disease-altering medications that will be delivered straight to the tissues, beyond the liver. This will help to reduce the toxicity burden on the liver.

“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.”

CRISPR is a novel gene editing technique that Jennifer Doudna and Emmanuelle Charpentier, CRISPR Therapeutics co-founder discovered. The technique makes use of molecular “scissors” to snip faulty parts of genes that can be deactivated afterward or replaced with new strands of normal DNA. 

Last year was a golden year for gene therapy

The United States FDA approved five gene therapies last year. This includes a treatment for sickle cell disease developed by CRISPR Therapeutics and Vertex Pharmaceuticals that make use of gene editing technologies.

Regeneron is the latest drugmaker to strike a deal with Mammoth for the development of gene therapies. Mammoth has previously entered similar collaborations with Vertex Pharmaceuticals and Bayer.

“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.

The financial details of the collaborations show that Mammoth will receive an upfront payment and equity investment of $100 million from Regeneron including a $95 million equity investment at signing. A further $370 million per therapy could be paid to Mammoth if they hit development, regulatory, and commercial milestones. Although both parties will jointly choose a therapy to research, Regeneron will lead the development and commercialization. 

For five and a half years, Regeneron will have access to Mammoth’s editing technologies with the option to pay and extend it for another two years