US-based next-generation gene editing company Arbor Biotechnologies has secured $73.9m in a Series C financing round, to advance its pipeline of gene editing therapeutics.
The financing was led by ARCH Venture Partners and TCGX, with participation from new investors such as Kerna Ventures, Partners Investment, QIA, and Revelation Partners.
Existing investors, including abrdn, Ally Bridge Group, Arrowmark Partners, Deep Track Capital, Piper Heartland Healthcare Capital, Surveyor Capital, Temasek, T. Rowe Price Associates, and Vertex Pharmaceuticals, also participated in the financing round.
The funding will primarily support the clinical development of Arbor’s lead candidate, ABO-101, and advance its programmes for liver and central nervous system diseases.
ARCH Venture Partners co-founder and partner Keith Crandell said: “Arbor is developing a differentiated portfolio with first-in-class potential to deliver on the promise of CRISPR-based genetic medicines.
“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.”
Arbor’s pipeline is built on proprietary genomic editors that enable precise genome editing.
Its lead asset, ABO-101, is a gene editing therapeutic targeting primary hyperoxaluria type 1 (PH1) and is currently being developed in the RedePHine Phase 1/2 clinical trial.
The 1/2 trial evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarker activity of ABO-101 in PH1 patients.
The investigational gene editing therapy is designed for a one-time liver-directed treatment to permanently inhibit the HAO1 gene, reducing oxalate production associated with PH1.
PH1 is a rare genetic disorder characterised by enzyme deficiencies in the liver, leading to kidney stones and systemic oxalosis.
ABO-101 works through a lipid nanoparticle encapsulating messenger RNA with a novel CRISPR Cas12i2 nuclease and optimised guide RNA targeting the HAO1 gene.
The drug has been granted orphan drug designation and rare paediatric disease designations from the US Food and Drug Administration (FDA), for treating PH1.
Arbor Biotechnologies CEO Devyn Smith said: “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.
“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.”
