The partners have inked a new non-exclusive licensing deal for the use of CRISPR’s gene editing platform, CRISPR/Cas9, to accelerate the development of Vertex’s hypoimmune cell therapies for type 1 diabetes (T1D).
CRISPR will also be eligible for up to $230m in R&D milestones and receive royalties on any future products resulting from this deal.
The partners have been in a strategic research collaboration since 2015, one focused on the use of CRISPR’s CRISPR/Cas9 gene editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease.
Exa-cel, developed for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), represents the first potential treatment to emerge from that joint research program. The therapy is awaiting regulatory backing in the EU and the US.
Vertex has multiple programs in its T1D portfolio including VX-880 and VX-264, which are in the clinic, as well as its hypoimmune program, which is in preclinical development.
Bastiano Sanna, executive vice president and chief of cell and genetic therapies at Vertex, said CRISPR’s gene editing technolgoy will allow it to further accelerate its goal of generating fully differentiated, insulin-producing hypoimmune islet cells for T1D.
The deal does not give CRISPR any interest in Vertex’s existing clinical-stage diabetes treatments.