Regeneron Pharmaceuticals has entered a significant collaboration with Mammoth Biosciences to develop in vivo CRISPR-based gene editing therapies, in a deal that includes an upfront payment of $100 mn and potential milestone payments totaling $370 mn per target. The partnership will leverage Regeneron's viral vector delivery technologies and Mammoth's compact nucleases for precise genetic modifications with the aim to enhance therapeutic applications across various diseases.

The following article originally appeared in BioPharma Dive.

Gene therapy developers have long been hamstrung by drug delivery, limited in which tissues they can reach and slowed by concerns of unintended safety problems.

Regeneron is one of many companies working on solutions around that bottleneck. It aims to develop better viral vectors — the engineered shells that shuttle genetic material into the body — by leaning on its expertise in antibody drugs.

“We’re finally at the tipping point where we’re ready to move into trying to develop our technologies into true medicines to help patients,” said Leah Sabin, executive director of Regeneron’s genetic medicines division.

The Mammoth deal is one of several forays by Regeneron into genetic medicine. Other deals include a longstanding research pact with Intellia Therapeutics that has led to a program for the rare disease transthyretin amyloidosis, and the purchase of a gene therapy biotech named Decibel Therapeutics.

“In the beginning, we were really careful to try to partner with external companies and forging collaborations to get our feet wet,” Sabin said. “With each passing year, we're more committed to becoming a serious player in the genetic medicine space.”

Regeneron has also expanded into cell therapy with a buyout of 2seventy Bio’s pipeline and a collaboration with Sonoma Biotherapeutics, following on earlier work in autoimmune drug development.

Thursday’s deal gives Mammoth $95 million in an equity investment and $5 million in upfront cash. The biotech could receive up to $370 million more per target if certain development, regulatory and commercial milestones are met.

“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,” Trevor Martin, Mammoth’s CEO, said in a statement.

Martin co-founded Mammoth in 2018 with a pair of University of California, Berkeley researchers, Janice Chen and Lucas Harrington, who had worked with CRISPR pioneer Jennifer Doudna on a paper outlining gene editing enzymes.

The Brisbane, California-based company began testing CRISPR technology for COVID-19 diagnostic tests. In 2023, it switched gears to develop CRISPR-based therapies, focusing on smaller DNA-cutting enzymes that it claims could be more easily delivered into tissues across the body.

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