Arbor Biotechnologies Secures $73.9M in Series C for Gene Editing Therapies

Arbor Biotechnologies, a pioneering biotechnology firm, has successfully secured $73.9 million in a Series C funding round. This substantial financial boost, led by ARCH Venture Partners and TCGX, will propel the company's efforts in advancing its innovative gene editing therapeutics, specifically targeting liver and central nervous system (CNS) diseases. The round also saw significant contributions from new investors such as QIA, Partners Investment, Revelation Partners, and Kerna Ventures, alongside continued support from existing investors, including funds managed by abrdn Inc., Ally Bridge Group, Arrowmark Partners, Deep Track Capital, Piper Heartland Healthcare Capital, Surveyor Capital (a Citadel company), Temasek, T. Rowe Price Associates, and Vertex Pharmaceuticals Incorporated.

The newly acquired funds will primarily support Arbor's lead therapeutic candidate, ABO-101, which addresses primary hyperoxaluria type 1 (PH1), a rare genetic disorder. The financing will also enable the company to progress its first-in-class programs, including a unique reverse transcriptase (RT) editing initiative for a rare liver disease and a project targeting amyotrophic lateral sclerosis (ALS).

Devyn Smith, CEO of Arbor Biotechnologies, expressed gratitude for the robust support from the investor consortium, emphasizing the company's dedication to developing groundbreaking gene editing therapies aimed at treating severe genetic disorders. Smith noted that with this backing, Arbor is well-equipped to make meaningful advancements in delivering novel gene editing solutions, especially for CNS conditions where there is a significant unmet need.

Arbor's advanced pipeline is underpinned by a suite of proprietary genomic editors, designed to perform a range of editing functions with high specificity and broad therapeutic potential. The lead program, ABO-101, is undergoing evaluation in RedePHine, a Phase 1/2 clinical trial. This trial aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarker activity of the treatment in patients with PH1.

Keith Crandell, co-founder and partner at ARCH Venture Partners, commended Arbor's impressive track record of focusing on pipeline development, execution, and capital efficiency. Crandell remarked on the strong preclinical data supporting Arbor's pipeline, suggesting that the firm is well-positioned to realize the promise of CRISPR-based genetic medicines.

ABO-101 is a groundbreaking investigational gene editing treatment designed for a one-time liver-directed application. It aims to permanently deactivate the HAO1 gene in the liver, thereby reducing oxalate production associated with PH1. This rare genetic disorder is characterized by enzyme deficiencies in the liver, leading to excessive oxalate accumulation and subsequent kidney issues. The treatment employs a lipid nanoparticle (LNP) system licensed from Acuitas Therapeutics, which delivers messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA targeting the human HAO1 gene. Currently, ABO-101 is being tested in the multi-center RedePHine trial, having already received orphan drug designation (ODD) and rare pediatric disease designation (RPDD) from the US FDA for PH1 treatment.

Based in Cambridge, Mass., Arbor Biotechnologies is at the forefront of developing next-generation gene editing therapies for a variety of genetic conditions. The company's proprietary gene editing technologies extend beyond the limitations of early editing techniques, unlocking new gene targets and fostering a robust pipeline of first-in-class assets. With a strong focus on diseases with no existing cures, particularly those affecting the liver and CNS, Arbor continues to drive forward its mission of delivering transformative genetic medicines.