Cambridge gene-editing startup Arbor Biotechnologies raises $215 million

With the oversubscribed second financing round, Arbor Biotechnologies has now raised more than $300 million to discover and develop a new generation of CRISPR editing tools.

Over the past decade, CRISPR has emerged as one of the most promising approaches to treating deadly genetic diseases, with scientists discovering enzymes that can act as molecular scissors to delete or fix disease-causing genes.

“The CRISPR technology is absolutely amazing,” said Arbor chief executive Devyn Smith, a former executive at the Flagship Pioneering biotech Sigilon Therapeutics of Cambridge and at the New York-based pharmaceutical giant Pfizer. “It’s going to make differences in patients’ lives for a long, long time.”

The original CRISPR Cas9 enzyme was developed partly by Nobel Prize laureate Jennifer Doudna of the University of California at Berkeley. Smith said Arbor has engineered a “significant number” of other CRISPR enzymes that can more precisely target genetic defects.

Among the neurological diseases the company hopes to treat, he said, is Huntington’s disease, a fatal disorder that causes the progressive breakdown of nerve cells in the brain. Perhaps the most famous person diagnosed with Huntington’s was Woody Guthrie, the towering folk singer who influenced Bob Dylan and died in 1967 at the age of 55. The faulty gene that causes Huntington’s was identified in 1993.

Arbor also hopes to use gene-editing tools to treat primary hyperoxaluria, a group of rare genetic metabolic disorders marked by the accumulation of a substance known as oxalate in the kidneys and other organs.

Smith said the firm, which has about 60 employees and was founded in 2016, hopes to begin a clinical trial of its experimental treatments in early 2024.

Since 2018, Arbor has signed two deals with Boston-based Vertex Pharmaceuticals to use CRISPR technology for a variety of genetic diseases, including cystic fibrosis, the disorder for which Vertex has developed four blockbuster treatments. The companies are also collaborating on treatments for diabetes, sickle cell disease, and beta thalassemia.

In addition to Zhang, the scientific cofounders of Arbor include David Walt, a professor of bioinspired engineering and pathology at Harvard Medical School and Brigham and Women’s Hospital.

The latest fundraising round was led by Temasek, Ally Bridge Group, and TCG Crossover. The round also featured new and old investors, including Vertex and ARCH Venture Partners.


Jonathan Saltzman can be reached at jonathan.saltzman@globe.com.

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