Vertex and Arbor Collaborate to Develop Novel ex vivo Engineered Cell Therapies

Vertex Pharmaceuticals Incorporated and Arbor Biotechnologies unveiled they have entered into a new cooperation to accelerate the development of novel ex vivo engineered cell therapies. Through the partnership, Vertex will use Arbor’s patented CRISPR gene-editing technology to study and develop ex vivo engineered cell treatments for serious diseases.

Vertex will use Arbor’s patented CRISPR gene-editing technology

Arbor and Vertex’s new arrangement expands on their previous collaboration, which began in 2018. Moreover, as part of this extended agreement Vertex’s objective is to create fully differentiated, insulin-producing hypo immune islet cells for type 1 diabetes therapy, as well as next-generation treatments in sickle cell disease, beta-thalassemia, and other life-threatening medical disorders.

Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex stated:

“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.”

Devyn Smith, Ph.D., CEO of Arbor 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 organizations in our industry, while we focus our internal portfolio efforts on genetic medicines.”

Terms of the Collaboration

Arbor will get an upfront cash payment and will be eligible for up to $1.2 billion in future payments based on the successful fulfillment of defined research, development, regulatory, and commercial targets across up to seven potential programs, according to the terms of the deal. Furthermore, Vertex will pay tiered royalties on any future net trade of products resulting from this cooperation and will also make a convertible note investment in Arbor.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has 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. Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters are now located in Boston’s Innovation District and its international headquarters are in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America.

About Arbor

Arbor Biotechnologies is an early-stage life sciences company discovering and developing the next generation of genetic medicines. Co-founded by Feng Zhang and David Walt, Arbor 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. Arbor’s pipeline of genetic medicines is focused on bringing curative therapies to all patients with a genetic disease. Following its strategic partnership with Vertex Pharmaceuticals to accelerate the path to the clinic for Arbor’s technologies, Arbor also recently announced an agreement with Lonza. These partnerships further validate the breadth of applications of Arbor’s gene-editing platform.


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