Arbor Biotechnologies secured $73.9 million in Series C funding, led by ARCH Venture Partners and TCGX, to advance its gene editing therapies for liver and central nervous system (CNS) diseases. The funding will propel the clinical development of ABO-101 for primary hyperoxaluria type 1 (PH1) and progress other programs towards Investigational New Drug (IND) applications, including a novel reverse transcriptase (RT) editing program for a rare liver disease and a program targeting amyotrophic lateral sclerosis (ALS). This investment significantly extends Arbor’s operational runway into 2027.
This influx of capital is crucial for the advancement of gene editing therapies, particularly for challenging targets like the CNS. Arbor’s progress in developing a diverse pipeline, including a first-in-class RT editing program and a therapy for ALS, positions them at the forefront of gene editing innovation. Successfully translating these preclinical programs into clinical trials represents a significant step towards addressing unmet medical needs in these complex disease areas.
Arbor’s lead program, ABO-101, is currently in a Phase 1/2 clinical trial for PH1. The company is also developing a preclinical RT editing program for an undisclosed rare liver disease and an ALS program, both of which are approaching IND-enabling studies. The Series C funding provides the resources to advance these programs and further expand Arbor’s proprietary gene editing platform.
This substantial investment underscores growing confidence in Arbor’s platform and its potential to deliver transformative gene editing therapies. The extended cash runway allows Arbor to focus on executing its clinical development strategy and potentially bring groundbreaking treatments to patients suffering from debilitating genetic diseases. The advancement of programs targeting the CNS, in particular, holds immense promise for addressing previously untreatable neurological conditions.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.