NEW YORK – Arbor Biotechnologies on Tuesday said it closed a $73.9 million Series C funding round to advance its pipeline of gene-editing therapies targeting diseases of the liver and central nervous system (CNS), including its lead candidate in primary hyperoxaluria type 1 (PH1).
The funding round was led by ARCH Venture Partners and TCGX and included existing investors Vertex Pharmaceuticals, Ally Bridge Group, Arrowmark Partners, Deep Track Capital, Piper Heartland Healthcare Capital, Surveyor Capital, Temasek, T. Rowe Price Associates, and funds managed by Abrdn. New investors QIA, Partners Investment, Revelation Partners, and Kerna Ventures also participated in the funding round.
The Cambridge, Massachusetts-based firm will use the proceeds to advance its CNS-targeted gene-editing therapeutics and reverse transcriptase-based editing programs, including its lead program for PH1, ABO-101, which is currently undergoing testing in the Phase I/II RedePHine trial. PH1 is a genetic disorder in which oxalate buildup damages the kidneys and other organs. ABO-101 comprises a lipid nanoparticle that encapsulates mRNA expressing a Type V CRISPR Cas12i2 nuclease and an optimized guide RNA and is designed to knock down HAO1 gene expression in the liver and reduce oxalate production.
Arbor also has other pipeline candidates that it is advancing toward investigational new drug (IND) and clinical trial application (CTA) filings, including a program targeting amyotrophic lateral sclerosis (ALS) and one targeting a rare liver disease. The Series C financing extends Arbor's cash runway into 2027.