Agreement provides access to Arbor’s precision editing capabilities

CAMBRIDGE, MA, USA I January 11, 2023 I Arbor Biotechnologies (Arbor) today announced the expansion of an existing strategic collaboration covering in vivo genetic medicines by which Vertex Pharmaceuticals Incorporated (Vertex) will receive rights to Arbor’s novel precision editing technology for up to three diseases.

Under the terms of the agreement, Arbor is eligible to receive payments based upon the successful achievement of specified research, development, regulatory and commercial milestones. In addition, Vertex will pay tiered royalties on future net sales of any products that may result from this collaboration. 

This marks a significant expansion of the relationship between the two companies, who first partnered on gene-editing therapies in 2018 and entered into a new relationship in 2021 around next-generation cell therapy approaches in diabetes and hemoglobinopathies, among other diseases.

“We are thrilled to build upon our collaboration with Vertex, who continues to be a valued partner,” said Devyn Smith, Ph.D., CEO of Arbor. “Cell and gene therapy are immensely promising fields in which there has been incredible progress, particularly in the past several years, but it has become increasingly evident that there is a continuous need for additional editing technology beyond first-generation approaches to meet the complexity of genetic diseases. Our precision editors have been optimally designed to provide more precise editing while being smaller in size to allow for further streamlined delivery and manufacturing, making them an attractive tool for use in treating a broad range of diseases.”

“This expanded collaboration brings us additional access to tools and expertise to help us advance targets of interest across our cell and genetic therapy portfolio,” said David Altshuler, M.D., Ph.D., Chief Scientific Officer of Vertex Pharmaceuticals. “We look forward to continuing to work with Arbor as a valued partner as we use scientific innovation to create transformative medicines for people with serious diseases.”

About Arbor Biotechnologies

Arbor Biotechnologies is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry to date. Founded by Feng Zhang, David Walt, David Scott, and Winston Yan, our proprietary discovery engine is focused on discovering and evolving technologies that enable gene knockout, RT editing, precise excisions and large insertions into endogenous loci. Leveraging our wholly owned nucleases as the chassis for genetic modification, we have the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question, with a focus on areas of high unmet need. As Arbor continues to advance its pipeline toward the clinic with an initial focus in liver and CNS disease, the Company has also secured several partnerships around gene editing and ex vivo cell therapy programs to broaden the reach of its novel nuclease technology. For more information, please visit:

SOURCE: Arbor Biotechnologies