USA – Arbor Biotechnologies, a genome-editing company discovering and developing the next generation of genetic medicines, has announced the completion of a U$215 million Series B financing to advance the company’s programs to treat inherited diseases of the liver and CNS.

To date, the company has raised more than US$300 million, including this round. Arbor has raised new funds in order to maintain its lead in the search for new genetic medicines.

The Series B funding comes as programs in liver and central nervous system disorders near human testing.

Arbor, based in Cambridge, Massachusetts, mines its database of billions of proteins using machine learning and artificial intelligence.

This allows the company to discover enzymes that could be used in CRISPR gene editing. Cas9, a nuclease enzyme, is possibly the most well-known of the cutting enzymes used in CRISPR editing.

Arbor claims to have discovered over 60 nuclease families and over 70 CRISPR transposases, another type of enzyme.

Arbor’s CRISPR toolbox includes DNA and RNA nucleases, as well as transposases. These nucleases are used by the company in its genetic medicines, which are designed to correct genetic errors that cause disease.

Arbor is currently in the preclinical stage. The primary programs of the company are for liver and central nervous system diseases.

Arbor CEO Devyn Smith stated that the new capital will be used to advance those programs toward clinical testing.

The funds will also be used to advance pipeline programs and support additional investment in the company’s discovery gene editing technology.

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

The company has also made strides in business development. Vertex Pharmaceuticals announced a new partnership with Arbor in August, expanding on an alliance that began in 2018.

Vertex, based in Boston, will use Arbor’s CRISPR gene-editing technology for the research and development of new ex-vivo cell therapies under the terms of the new agreement.

Engineering islet cells, a type of pancreatic cell that produces insulin, as a potential new therapy for type 1 diabetes is at the top of Vertex’s wish list as well as developing treatments for sickle cell anemia and beta thalassemia.

CRISPR applications in therapeutics have shifted toward smaller cutting enzymes that leave more room for additional cargo. Arbor has published research on Cas13d that is based on the company’s technology platform.

The enzyme belongs to the Cas13 family. Because of its small size, it is suitable for RNA manipulation and detection.

Therapeutic gene-editing competitive landscape

Arbor, on the other hand, faces competition in the race to go smaller, especially as biotechs pursue gene-editing therapies that work in vivo—inside the patient.

Mammoth Biosciences promotes a toolbox that includes Cas14 and CasPhi enzymes, both of which are smaller than Cas9.

Mammoth, based in South San Francisco, announced a US$150 million Series D round of funding in September.

Mammoth and Vertex announced a collaboration last month to develop in vivo gene-editing therapies for two unnamed genetic diseases.

As per the agreement Mammoth receives US$41 million up front, with an additional US$650 million tied to milestones.

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