Regeneron Pharmaceuticals and California-based biotech Mammoth Biosciences have partnered to develop CRISPR-based gene editing therapies aimed creating in vivo programs targeting multiple tissue and cell types beyond the liver, a current limitation in gene editing treatments.
Mammoth will receive an initial $100 million from Regeneron, comprising an upfront payment and equity investment. Future payments may reach up to $370 million per target, depending on development milestones, regulatory approvals, and commercial sales, with additional royalty payments on net sales.
The two will leverage Mammoth’s ultracompact CRISPR systems, which are considerably smaller than first-generation Cas9 nucleases, allowing for more versatile gene editing capabilities with Regeneron’s expertise in adeno-associated viral vectors (AAVs) and antibody engineering to enhance the delivery of these genetic therapies to targeted cells.
Mammoth's technology suite includes a range of innovative CRISPR tools, such as ultracompact Cas enzymes, characterized by a smaller size, enhanced temperature stability, quicker reaction kinetics, and flexible sequences for targeting. These properties support the development of genetic medicine capable of performing diverse edits, including double-stranded breaks, epigenetic modifications to silence genes, single-letter base editing and versatile RNA-encoded edits.
