Arbor Biotechnologies has closed $73.9m in a Series C financing round, earmarked for advancing its gene editing therapeutic pipeline aimed at liver and central nervous system diseases.

TCGX and ARCH Venture Partners spearheaded the funding round, joined by new investors Partners Investment, QIA, Kerna Ventures and Revelation Partners.

Discover B2B Marketing That Performs

Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.

Find out more

Current investors including Arrowmark Partners, Vertex Pharmaceuticals, Ally Bridge Group and Deep Track Capital also contributed.

The capital injection will facilitate the clinical progression of Arbor’s ABO-101, the lead therapeutic candidate targeting the rare genetic disorder, primary hyperoxaluria type 1 (PH1).

It will also support the company’s move towards filings of the investigational new drug/clinical trial application for its programmes, which include a reverse transcriptase-editing initiative targeting rare liver conditions and a programme focused on amyotrophic lateral sclerosis.

Arbor Biotechnologies CEO Devyn Smith stated: “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 realising a new generation of potentially curative genetic medicines for patients.”

GlobalData Strategic Intelligence

US Tariffs are shifting - will you react or anticipate?

Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.

By GlobalData

Arbor’s pipeline is built on genomic editors that allow for various functions, paving the way for high-precision genome editing.

Arbor is currently progressing the therapy through a multi-centre, open-label, Phase I/II RedePHine trial.

This dose-escalation trial aims to assess the therapy’s pharmacokinetics, tolerability, safety, biomarker activity and pharmacodynamics in PH1 patients.

ABO-101 is a gene editing medicine to be used as a liver-directed treatment for a single time only. It seeks to permanently disable the functioning of the liver’s hydroxyacid oxidase 1 (HAO1) gene, thereby decreasing oxalate production associated with PH1.

The US Food and Drug Administration has awarded ABO-101 both orphan drug and rare paediatric disease designations for PH1.

Pharmaceutical Technology Excellence Awards - Nominations Closed

Nominations are now closed for the Pharmaceutical Technology Excellence Awards. A big thanks to all the organisations that entered – your response has been outstanding, showcasing exceptional innovation, leadership, and impact.

Excellence in Action
NorthWest EHealth has won three 2025 Pharmaceutical Technology Excellence Awards for Innovation, Safety and Diversity. Explore how its ConneXon platform is transforming SAE reporting and real-time safety oversight, while FARSITE delivers socio-economically inclusive recruitment to boost trial efficiency, data integrity and regulatory-grade representativeness.

Discover the Impact