A developer of gene-edited precision therapies co-founded by CRISPR pioneer Feng Zhang, PhD, has more than doubled its total capitalization after completing a $215 million Series B financing today.

Arbor Biotechnologies said the financing brings its total capital raised to more than $300 million, and will enable it in part to advance lead programs in liver and CNS disease into clinical phases, as well as the rest of its pipeline, which the company has not publicly detailed.

Arbor also plans to use part of the financing proceeds to further develop its gene-editing technology platform, which applies a “toolbox” of CRISPR-based genomic editors as well as diverse technologies that include artificial intelligence, genome sequencing, gene synthesis and high-throughput screening, which the aim of identifying “impactful” peptides, proteins, and enzymes.

“By leveraging our proprietary discovery engine—which applies machine learning and AI to mine our protein database containing billions of proteins—we have successfully built the most extensive toolbox of wholly owned CRISPR genomic editors in the industry,” Arbor CEO Devyn Smith, PhD, said in a statement.

“This breadth of tools and IP enables us, and our partners, to approach numerous diseases with gene editing solutions that can be tailored to edit or rewrite the genetic errors that result in disease pathology,” according to Smith. “This provides us with the ability to target the root cause of genetic diseases.”

In 2018, Arbor researchers published a paper in the journal Molecular Cell detailing a new CRISPR enzyme, CRISPR-Cas13d, which was significantly smaller than other members of the CRISPR-Cas13 family, and which as a result was envisioned as having applications as diverse as RNA manipulation and highly sensitive diagnostics: “The small size, minimal targeting constraints, and modular regulation of Cas13d effectors further expands the CRISPR toolkit for RNA manipulation and detection,” the researchers concluded.

Beyond CRISPR Nucleases

In today’s announcement, Smith also disclosed that Arbor was ready to look beyond its primary focus until now of developing CRISPR nucleases.

“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,” he stated.

Zhang, a Core Institute Member of the Broad Institute of MIT and Harvard, joined Quanterix co-founder David Walt, PhD, David Scott (now the company’s Head of Innovation), and Winston Yan, MD, PhD, a onetime postdoc in Zhang’s lab, in co-founding Arbor in 2016.

A year later, the company emerged from stealth mode by announcing completion of a $15.6 million Series A financing that included among investors Keith Crandell, a co-founder and Managing Director of ARCH Venture Partners; Annie Hazlehurst, founder of Faridan Ventures; Alexandria Real Estate Equities; and several private investors.

The Series B financing was co-led by Temasek, Ally Bridge Group, and TCG Crossover. ARCH Venture Partners was among other investors—as was Vertex Pharmaceuticals, with which Arbor in August launched an up-to-$1.2 billion collaboration to develop ex vivo engineered cell therapies based on Arbor’s CRISPR gene-editing technology for select diseases. At the time, the companies said their goal was to help Vertex generate fully differentiated, insulin-producing hypoimmune islet cells for treating type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia, and for treating other diseases.

Also participating in the Series B round were additional new investors that included 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 (a Citadel company), T. Rowe Price Associates, Tao Capital Partners, funds managed by Tekla Capital Management LLC, Woodline Partners LP, and an undisclosed global investment fund.

As part of the Series B financing, Chen Yu, MD, MBA, Managing Partner at TCG Crossover, will join Arbor’s board.

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