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.
Go deeper with GlobalData
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.”
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.
