Arbor Biotechnologies

Arbor Biotechnologies specializes in gene editing to develop novel therapeutics for a variety of indications, starting with liver-related diseases. It operates an AI-powered biodiscovery platform to discover novel Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) enzymes, which are sequences of repeating genetic code. To do this, its platform deploys computational search techniques, genome sequencing, gene synthesis, and high-throughput screening. This can eventually be turned into drugs or even applied to agriculture and other industrial uses.

As of March 2024, the company had five drug discovery programs, one was in IND-enabling stage, while two were in the lead optimization stage, and two were in discovery stages. In 2018, the company announced the discovery of Cas13d, a new type of gene editing enzyme that helps to alter RNA to treat genetic diseases. 

Key customers and partnerships

Most recently, in January 2024 , the company announced a strategic collaboration with 4D Molecular Therapeutics that would focus on advancing new AAV-based gene-editing therapies for central nervous system (CNS) diseases with high unmet medical needs in both rare and common disease populations.

The company entered into a strategic partnership worth USD 1.2 billion with Vertex Pharmaceuticals in August 2021 to develop ex vivo therapies (gene editing done in a specialized laboratory and returned to the body). The duo originally partnered in 2019 to develop proteins for gene-edited therapies. The companies expanded this partnership in January 2023 , to give Vertex Pharmaceuticals the rights to Arbor’s novel precision editing technology for up to three diseases.

Arbor also entered into a non-exclusive licensing agreement with EdiGene Inc . in February 2022 to leverage Arbor’s platform for ex vivo oncology therapies and a strategic research and non-exclusive licensing agreement with TCR2 Therapeutics in January 2022 to develop cell therapies for the latter. 

Funding and financials

In its latest Series B funding round in November 2021, which was led by Ally Bridge Group, TCG Crossover, and Temasek, Arbor Biotechnologies raised USD 215 million. The proceeds of the round were earmarked to fuel the company’s clinical development pipeline.