August 24, 2021 -- Vertex Pharmaceuticals and Arbor Biotechnologies are teaming up to develop ex vivo engineered cell therapies based on Arbor's CRISPR gene editing technologies.
The agreement, which builds on a previous partnership from 2018, grants Vertex rights to use Arbor's technology to research and develop cell therapies, with the goal of generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia, and for the treatment of other diseases, according to the companies.
For Vertex, the deal further expands its cell and genetic therapy toolkit, as well as its efforts to develop cell therapies for the treatment of multiple serious diseases, said Bastiano Sanna, PhD, executive vice president and chief of cell and genetic therapies, in a statement.
In addition to an upfront cash payment, Arbor will be eligible to receive up to $1.2 billion in potential payments for achieving specified research, development, regulatory, and commercial milestones across up to seven potential programs. Vertex will also invest in Arbor in the form of a convertible note and will pay tiered royalties on future net sales of products resulting from the collaboration.