Arbor Biotechnologies Announces $73.9 Million Series C Financing to Advance Novel Gene Editing Therapeutics

Series C led by ARCH Venture Partners and TCGX with significant participation from existing and new investors Financing extends Arbor’s cash runway into 2027 and supports the clinical development of lead program ABO-101 and continued advancement of a broader portfolio of CNS-targeted gene editing therapeutics and reverse transcriptase (RT)-based editing programs

CAMBRIDGE, MA – March 18, 2025 – Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced the closing of a $73.9 million Series C financing to support the advancement of its pipeline of novel gene editing therapeutics targeting diseases in the liver and central nervous system (CNS). ARCH Venture Partners and TCGX led the financing, with participation from new investors QIA, Partners Investment, Revelation Partners, and Kerna Ventures and 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 proceeds will support clinical development of the company’s lead therapeutic candidate, ABO-101, in primary hyperoxaluria type 1 (PH1) and progression to IND/CTA filing of its first-in-class programs, including an RT editing program for a rare liver disease and a program targeting amyotrophic lateral sclerosis (ALS).

“This financing is a testament to the hard work of our team as well as our consistent focus and capital-efficient execution in developing a differentiated portfolio of gene editing therapeutics with the aim of realizing a new generation of potentially curative genetic medicines for patients,” said Devyn Smith, CEO of Arbor Biotechnologies. “We are grateful for the support of this top-tier investor syndicate and their confidence in the Arbor team. With their backing, we are well positioned to make significant strides toward delivering novel gene editing therapeutics, including those targeting CNS diseases with high unmet need.”

The company’s pipeline is built upon a suite of proprietary, wholly owned genomic editors that enable a variety of functions, unlocking sophisticated and precise ways of editing the genome that offer unique properties, high specificity and broad therapeutic applications. Arbor is advancing its lead asset ABO-101—a liver-targeted gene editing therapeutic for the treatment of PH1—in RedePHine, a Phase 1/2, multi-center, open-label, dose-escalation clinical trial designed to evaluate its safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarker activity in patients with PH1 (NCT06839235).

“Arbor is developing a differentiated portfolio with first-in-class potential to deliver on the promise of CRISPR-based genetic medicines,” said Keith Crandell, co-founder and partner at ARCH Venture Partners. “Arbor has established a track record of pipeline focus, coupled with execution and capital efficiency, to yield strong preclinical data supporting its pipeline. We are impressed with the team’s progress to date and are proud to support the advancement of these programs.”

 

About ABO-101
ABO-101 is a novel, investigational gene editing medicine designed to be a one-time liver-directed gene editing treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce primary hyperoxaluria type 1 (PH1)-associated oxalate production. PH1 is a rare genetic disorder in which enzyme deficiencies in the liver lead to the overproduction and buildup of oxalate, resulting in kidney stones eventually leading to end stage kidney disease and systemic oxalosis. ABO-101 is designed to knock down HAO1 gene expression in the liver, thereby providing durable reduction in oxalate production. ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene. ABO-101 is currently under evaluation in RedePHine, a Phase 1/2, multi-center, open-label, dose-escalation clinical trial designed to study its safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarker activity in patients with PH1 (NCT06839235). ABO-101 has been granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) by the US FDA for the treatment of PH1.

About Arbor Biotechnologies, Inc.
Arbor Biotechnologies™, a next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company’s unique suite of optimized gene editors, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. With Arbor’s lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, in clinical development, the company continues to focus their research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: arbor.bio.

 

Media Contact:
Peg Rusconi
Deerfield Group
prusconi@nulldeerfieldgroup.com