Vertex and Arbor Partner to Develop Novel Engineered Cell Therapies

Vertex Pharmaceuticals and Arbor Biotechnologies have 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. The new agreement 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.

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.

Founded in 1989 in Cambridge, Vertex is a global biotechnology company that has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines and a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus. Vertex’s global headquarters is now located in Boston’s Innovation District and its international headquarters is in London.

Arbor Biotechnologies is an early-stage life sciences company based in Cambridge that uses its proprietary discovery engine to discover unique and wholly owned CRISPR-based genetic modifiers with differentiated genetic editing capabilities. This portfolio of gene editors can be custom tailored to address the underlying pathology of genetic diseases. (Source: Vertex Website, 24 August, 2021)