CRISPR Biotech Editor Arbor Bio Adds $ 215 Million for Treatment of Liver and CNS Diseases


Arbor Biotechnologies, a genetic drug company that claims to have the most comprehensive gene editing toolkit in the industry, is also building a substantial war chest. The company has raised $ 215 million in funding as it strives to move its core programs forward into clinical trials.

The Series B funding announced on Tuesday was led by Temasek, Ally Bridge Group and TCG Crossover.

Arbor, based in Cambridge, Mass., Uses machine learning techniques and artificial intelligence to mine its database of billions of proteins. This allows the company to discover enzymes that have potential use in editing CRISPR genes. Cas9, a type of enzyme called nuclease, might be the best known of the cutting enzymes used in CRISPR editing. Arbor claims to have discovered more than 60 families of nucleases as well as more than 70 CRISPR transposases, another type of enzyme. Arbor’s CRISPR toolkit includes DNA nucleases, RNA nucleases and transposases. The company uses these nucleases in its genetic drugs, which are designed to modify the genetic errors that cause disease.

So far, Arbor is preclinical. The company’s main programs relate to diseases of the liver and central nervous system. In a prepared statement, Arbor CEO Devyn Smith said Arbor plans to use the new capital to advance these programs into clinical trials. The money will also be used to advance pipeline programs and to support further investments in its discovery technology.

“While our primary focus has been to develop our bespoke CRISPR nucleases, we are also looking to advance our other precision editing innovations, such as CRISPR transposases,” said Smith.

Funding is the latest development in what has been a busy year for Arbor. The company has recruited a wave of new hires, including Smith, who joined the team in April. The company has also made progress in business development. In August, Vertex Pharmaceuticals entered into a new partnership with Arbor, expanding an alliance initially started in 2018.

As part of the new deal, Boston-based Vertex will use Arbor’s CRISPR gene editing technology for research and development of new ex-vivo cell therapies. At the top of Vertex’s wishlist is the engineering of islet cells of Langerhans, a type of pancreatic cell that produces insulin, as a potential new therapy for type 1 diabetes. Vertex is interested. also developing therapies for blood disorders, sickle cell anemia and beta thalassemia. The upfront payment to Arbor was not disclosed, but the companies said potential milestone payments could reach up to $ 1.2 billion.

Applications of CRISPR in therapeutic applications have evolved into smaller cutting enzymes that leave more room for additional cargo. Arbor has published research on Cas13d from the company’s technology platform. The enzyme is part of the Cas13 family. Its smaller size makes it applicable for RNA manipulation and detection.

However, Arbor faces competition in the downsizing race, particularly as biotech pursues gene-editing therapies that do their job in vivo, inside the patient. Mammoth Biosciences boasts a toolkit that includes the enzymes Cas14 and CasPhi, both of which are smaller than Cas9. South San Francisco-based Mammoth announced a $ 150 million Series D funding round in September. Late last month, Mammoth and Vertex announced an alliance focused on the development of in vivo gene editing therapies for two undisclosed genetic diseases. The deal gives Mammoth $ 41 million upfront, with an additional $ 650 million tied to milestones.

Arbor was founded in 2016, based on research from MIT. Biotechnology raised a $ 15.6 million Series A funding round in 2017. The latest funding added new investors Arrowmark Partners, Deep Track Capital, Great Point Ventures, Illumina Ventures, Janus Henderson Investors, Logos Capital, Ono Venture Investment, Piper Heartland Healthcare Capital, Ridgeback Capital Investments, Section 32, Surveyor Capital, T. Rowe Price Associates, Tao Capital Partners, funds managed by Tekla Capital Management, Woodline Partners and a global investment fund that has not been disclosed. In addition, Vertex has also invested, as well as the previous investor ARCH Venture Partners.

Photo by Flickr user Jason Baker via Creative Commons license


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