Novel ex vivo engineered cell therapies to be developed by Vertex Pharma and Arbor Biotechnologies

Cambridge, Massachusetts : Vertex Pharmaceuticals Incorporated, a global biotechnology company, and Arbor Biotechnologies (Arbor), an early-stage life sciences company, announced a new collaboration to enhance efforts in developing ex vivo engineered cell therapies, using Arbor’s proprietary CRISPR gene-editing technology for select diseases.

Under this new partnership, Vertex will receive rights to use Arbor’s technology to research and develop ex vivo engineered cell therapies towards Vertex’s 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.

“This new collaboration further expands our toolkit in cell and genetic therapies and, specifically, our work to discover and develop cell therapies for the treatment of multiple serious diseases,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex.

“We are excited to bring Arbor’s technology together with Vertex’s ongoing programs and capabilities in diabetes, hemoglobinopathies and other diseases to create improved cell replacement therapies for broad populations of patients," Sanna added.

“Arbor and Vertex share a common goal to improve the lives of people with life-threatening diseases through the discovery and development of innovative therapies, and Vertex has proven to be an ideal partner in that effort,” said Devyn Smith, Ph.D., CEO of Arbor.

“This agreement demonstrates the versatility and strength of Arbor’s platform and reinforces our strategic vision of expanding our engineered cell therapy capabilities through partnerships with leading organizations in our industry, while we focus our internal portfolio efforts on genetic medicines," Smith said.