Vertex and Mammoth Collaborate to Develop In Vivo Gene-Editing Therapies for Serious Diseases

Vertex Pharmaceuticals has added to its gene-editing capabilities through a new partnership with Mammoth Biosciences to develop in vivo gene-editing therapies for two genetic diseases using Mammoth’s next-generation CRISPR systems.

Photo: David Altshuler, chief scientific officer of Vertex Pharmaceuticals

“We believe our novel ultra-small CRISPR systems have the potential to be game changers when it comes to systemic and targeted delivery of in vivo gene-editing therapies,” said Peter Nell, chief business officer and head of Therapeutic Strategy at Mammoth Biosciences. “The combination of Mammoth’s unique technology with Vertex’s unmatched experience in serious disease research and development will only accelerate programs with the goal of reaching patients with high unmet medical need.”

Mammoth’s CRISPR platform consists of a proprietary toolbox of novel, ultracompact Cas enzymes, including Cas14 and Casɸ. The small size of these Mammoth systems, together with further optimized parameters, have the potential to facilitate advanced delivery, which may increase the scope of in vivo gene-editing for genetic diseases.

Under the terms of the agreement, Mammoth Biosciences will receive upfront payments of $41 million, including an investment in the form of a convertible note, and is eligible to receive up to $650 million in potential future payments based upon the successful achievement of prespecified research, development, and commercial milestones across two potential programs. In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration.

“Vertex and Mammoth share the same commitment to developing therapies that have the potential to be transformative for people with serious diseases,” said David Altshuler, chief scientific officer of Vertex Pharmaceuticals. “We look forward to expanding our cell and genetic therapies capabilities with the addition of Mammoth’s ultra-small CRISPR systems for in vivo genome editing, which will provide us with another set of tools to tackle many of the diseases we’re interested in.”

Author: Rare Daily Staff