Arbor Biotechnologies said it closed a $73.9 million series C financing to support the advancement of its pipeline of gene editing therapeutics targeting diseases in the liver and central nervous system.
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, 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.
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.
The proceeds will support clinical development of the company’s lead therapeutic candidate, ABO-101, in primary hyperoxaluria type 1 and progression to filing applications to begin clinical trials of its first-in-class programs, including a reverse transcriptase (RT) editing program for a rare liver disease and a program targeting amyotrophic lateral sclerosis.
Primary hyperoxaluria type 1 (PH1) is an ultra-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 a novel, experimental 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. ABO-101 is designed to knock down HAO1 gene expression in the liver, thereby providing a durable reduction in oxalate production.
ABO-101 consists of a lipid nanoparticle encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA that 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
The U.S. Food and Drug Administration granted ABO-101 orphan drug designation and rare pediatric disease designation for the treatment of PH1.
“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 well positioned to make significant strides toward delivering novel gene editing therapeutics, including those targeting CNS diseases with high unmet need.”
Photo: Devyn Smith, CEO of Arbor Biotechnologies
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