Therapy Areas: Hereditary Disorders
Vertex, Arbor Biotechnologies Establish Collaboration to Discover Novel Proteins to Advance Discovery of Gene-Editing Therapies
7 January 2019 - - US-based biotechnology companies Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) and Arbor Biotechnologies have entered into a strategic research collaboration focused on the discovery of novel proteins including DNA endonucleases to advance the development of new gene-editing therapies for cystic fibrosis and four other diseases to be selected later, the companies said.

Arbor has developed a protein biodiscovery platform that includes a comprehensive set of technologies and techniques, integrating machine learning, genome sequencing, gene synthesis, and high-throughput screening that will augment Vertex's efforts to develop gene-editing approaches for the treatment of serious diseases.

Under the terms of the collaboration, scientists from Vertex and Arbor will work closely together to discover novel proteins and tools that Vertex may use in the development of new medicines.

The focus of the collaboration is to discover novel programmable DNA endonucleases or nickases with high fidelity and catalytic activity as well as novel transduction approaches. Discovery activities will be conducted primarily by Arbor and funded by Vertex.

Vertex will provide Arbor with an 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 obtain an observer seat on the Arbor board of directors.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.

In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programmes focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District.

Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America. Vertex is consistently recognised as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for nine years in a row.

Arbor Biotechnologies is an early-stage company pushing the boundaries of biodiscovery. Based in Cambridge, MA, Arbor is unlocking nature's genetic diversity to create transformative products in human health and sustainability.

The company's proprietary platform integrates a computational search engine with high-throughput experimental capabilities to enable both rapid protein characterization and product generation.


Related Headlines