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Arbor Biotechnologies, Vertex Pharmaceuticals Partner for Gene-Editing Protein Discovery

NEW YORK (GenomeWeb) – Arbor Biotechnologies and Vertex Pharmaceuticals announced today that they have partnered to discover novel proteins, including DNA endonucleases, for the development of new gene editing-based therapies for cystic fibrosis and four other diseases to be selected later.

Arbor will contribute its protein biodiscovery platform — which includes integrating machine learning, genome sequencing, gene synthesis, and high-throughput screening — while Vertex will contribute its own expertise in the development of gene editing-based approaches for the treatment of serious diseases, the firms said.

Researchers from both firms will specifically focus on the discovery of novel programmable DNA endonucleases or nickases with high fidelity and catalytic activity, as well as novel transduction approaches. Arbor will primarily conduct the discovery activities. Vertex will provide Arbor with an undisclosed up-front cash commitment, fund research activities, and will pay research, development, regulatory, and commercial milestones, as well as royalties on future net sales. Vertex will also make an investment in Arbor in the form of a convertible note and will obtain an observer seat on the company's board.

"Vertex has a proven track record in discovering and developing innovative medicines and is an ideal partner for Arbor," Arbor Cofounder David Walt said in a statement. "We believe that by using our powerful biodiscovery platform we will be able to identify complementary and next generation tools to enhance Vertex's pipeline of transformative medicines and new gene editing therapies."