Arbor Biotechnologies received Orphan Drug and Rare Pediatric Disease designations from the U.S. FDA for ABO-101, a gene editing therapy for primary hyperoxaluria type 1 (PH1). This follows the FDA’s acceptance of the Investigational New Drug application, paving the way for a Phase 1/2 trial in the first half of 2025. Preclinical data and the trial design will be presented at the International Pediatric Nephrology Association Congress in February 2025.
These designations are crucial because they highlight the significant unmet need for PH1 treatments, especially for children who often experience early onset of severe symptoms. The potential for a one-time, curative treatment addresses the limitations of current PH1 management, which primarily involves managing symptoms and complications rather than addressing the underlying genetic cause. This represents a potential paradigm shift in PH1 treatment. The designations also provide Arbor with financial incentives, including tax credits and fee waivers, which can help accelerate the development and eventual commercialization of ABO-101.
ABO-101, delivered via a lipid nanoparticle, targets the •HAO1• gene in the liver. This gene is responsible for the overproduction of oxalate, the root cause of PH1 complications. By precisely editing the •HAO1• gene, ABO-101 aims to permanently reduce oxalate production and prevent the debilitating effects of the disease. The upcoming Phase 1/2 trial will evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics, and preliminary efficacy of ABO-101 in both adult and pediatric PH1 patients.
The FDA designations and upcoming clinical trial represent a critical step towards a potential cure for PH1. Positive trial results could validate Arbor’s gene editing platform and establish ABO-101 as a first-in-class therapy. This could lead to a significant improvement in the quality of life for PH1 patients and potentially transform the treatment landscape for other rare genetic diseases.
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.
