Rare Daily Staff

Vertex Pharmaceuticals and Arbor Biotechnologies entered a new collaboration to enhance efforts in developing ex vivo engineered cell therapies using Arbor’s proprietary CRISPR gene-editing technology for select diseases.

The new agreement between Arbor and Vertex builds upon the companies’ first partnership established in 2018. 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, executive vice president and chief of Cell and Genetic Therapies at Vertex.

Arbor uses its proprietary discovery engine to discover unique and wholly owned CRISPR-based genetic modifiers with differentiated genetic editing capabilities that can be custom tailored to address the underlying pathology of genetic diseases.

Under the terms of the agreement, Arbor will receive an upfront cash payment and is eligible to receive up to $1.2 billion in potential payments based upon the successful achievement of specified research, development, regulatory and commercial milestones across up to seven potential programs. In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration. Vertex will also make an investment in Arbor in the form of a convertible note.

“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,” said Devyn Smith, CEO of Arbor.

Photo: Bastiano Sanna, executive vice president and chief of Cell and Genetic Therapies at Vertex

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