Vertex Pharmaceuticals has collaborated with Mammoth Biosciences to develop in vivo gene-editing therapies for two genetic diseases.
As part of the partnership, the companies will use Mammoth’s next-generation CRISPR systems to discover and develop new in vivo gene-editing therapies.
The CRISPR platform comprises a toolbox of novel, ultracompact Cas enzymes, including Casɸ and Cas14.
The company believes that the small size CRISPR systems combined with optimised parameters, will have the potential to accelerate the delivery of therapies.
Mammoth Biosciences Therapeutic Strategy head and chief business officer Peter Nell said: “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.
“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.”
Under the deal, Vertex will make a $41m upfront payment to Mammoth, which will also be eligible to receive up to $650m if the two potential programmes achieve research, development and commercial milestones.
Furthermore, Mammoth will also receive tiered royalties on future net sales on any products.
Vertex chief scientific officer David Altshuler said: “Vertex and Mammoth share the same commitment to developing therapies that have the potential to be transformative for people with serious diseases.
“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.”