Vertex will use Arbor’s CRISPR gene-editing technology in developing novel cell therapies for the treatment of serious diseases

lab-2813892_640(1)

Vertex partners with Arbor to develop cell therapies. (Credit: Michal Jarmoluk from Pixabay.)

Vertex Pharmaceuticals has teamed up with Arbor Biotechnologies to use the latter’s technology in developing ex vivo engineered cell therapies for certain diseases.

The current cell therapies collaboration builds on a previous partnership between Arbor and Vertex, initiated in 2018.

Under the new agreement, Vertex obtains the rights to leverage Arbor’s CRISPR gene-editing technology to research and develop ex vivo engineered cell therapies.

Arbor is entitled to receive an upfront cash payment and to receive up to $1.2bn in potential research, development, regulatory and commercial milestones payments

The company will also receive tiered royalties on future net sales on any products produced from the collaboration.

Furthermore, Vertex has agreed to invest in Arbor, in the form of a convertible note.

Vertex executive vice president and cell and genetic therapies chief Bastiano Sanna said: “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.

“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.”

Vertex aims will use cell therapies to generate fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes.

Also, it will use the technology for next-generation approaches in sickle cell disease and beta thalassemia, along with the treatment of other diseases.

Arbor Biotechnologies is an early-stage life sciences company engaged in discovering and developing next-generation genetic medicines.

The firm leverages its unique discovery engine to discover CRISPR-based genetic modifiers with differentiated genetic editing capabilities.

Arbor CEO Devyn Smith said: “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.

“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 organisations in our industry, while we focus our internal portfolio efforts on genetic medicines.”