Rare Daily Staff

Arbor Biotechnologies closed an oversubscribed and upsized $215 million series B financing, which it will use to advance its lead programs in liver and CNS disease into the clinic and progress a pipeline of precision editing therapeutics.

Arbor will also use the funds to continue to invest in its discovery engine to develop the next generation of gene editing technology.

“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,” said Devyn Smith, CEO of Arbor Biotechnologies. “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. This provides us with the ability to target the root cause of genetic diseases.”

In August, Arbor entered a collaboration worth up to $1.2 billion with Vertex Pharmaceuticals to enhance efforts in developing ex vivo engineered cell therapies using Arbor’s proprietary CRISPR gene-editing technology for select diseases. under which Vertex received rights to use Arbor’s technology to research and develop ex vivo engineered cell therapies towards generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia, and for the treatment of other diseases.

Co-founded by David Walt and Feng Zhang, Arbor has built an extensive toolbox of propriety genomic editors to tackle the underlying genetic pathology of the disease. Using the discovery platform, Arbor can discover, screen, and engineer novel editing enzymes and effectors that can then be tailored to the underlying cause of disease to result in potentially curative medicines for patients.

“With this investment, we are well-positioned to continue advancing toward the clinic with our initial focus in liver and first-in-class treatments for CNS diseases, as well as continue to build out our proprietary discovery engine. 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.”

Arbor’s lead preclinical candidate targets primary hyperoxaluria, a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys. There are three known subtypes of PH (PH1, PH2 and PH3), each resulting from a mutation in one of three different genes. These genetic mutations cause enzyme deficiencies that result in the overproduction of oxalate, an end-product of metabolism. Abnormal production and accumulation of oxalate leads to recurrent kidney stones, nephrocalcinosis and chronic kidney disease that may progress to end-stage renal disease requiring intensive dialysis. Compromised renal function eventually results in the accumulation of oxalate in a wide range of organs including the skin, bones, eyes, and heart. In the most severe cases, symptoms start in the first year of life.

Temasek, Ally Bridge Group, and TCG Crossover led the financing round. Additional new investors 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, Woodline Partners, and an undisclosed global investment fund. The round also included investment from strategic partner Vertex Pharmaceuticals and continued support from existing investors, such as ARCH Venture Partners.

As part of the Series B Financing, Chen Yu, managing partner at TCG Crossover, will join the Company’s Board of Directors.

Photo: Devyn Smith, CEO of Arbor Biotechnologies

X