Vertex and Mammoth Enter into Gene-Editing Partnership Worth Up to $691 Million

Vertex Pharmaceuticals and Mammoth Biosciences announced a partnership on Oct. 26, 2021, to develop in-vivo gene-editing therapies. In a deal that could be worth as much as $691 million, Vertex will use Mammoth’s ultra-small clustered regularly interspaced short palindromic repeats (CRISPR) systems to discover and develop novel in-vivo gene-editing therapies for two genetic diseases.

Mammoth’s CRISPR platform consists of various novel, ultracompact Cas enzymes, including Cas12 and Casɸ. Because of their small size, they have the potential to increase the scope of in-vivo gene-editing by facilitating advanced delivery techniques.

Vertex has done significant work with cystic fibrosis in the past, which has led to multiple approved medications. Their current pipeline features small molecule medicines for alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. Additionally, the pipeline also has cell and genetic therapies for sickle cell disease, beta thalassemia, Duchenne muscular dystrophy, and type 1 diabetes mellitus.

“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, in a Vertex press release. “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.”

“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 office of Vertex Pharmaceuticals, in the press release. “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.”

Source: Vertex Pharmaceuticals