Arbor Biotechnologies has reached a significant clinical milestone, dosing the first patient at Mayo Clinic in the redePHine Phase 1/2 study for ABO-101. This groundbreaking therapy is an investigational CRISPR gene-editing treatment for Primary Hyperoxaluria Type 1 (PH1), an ultra-rare and debilitating genetic disease. PH1 is caused by a mutation in the AGXT gene, leading to a deficiency in a liver enzyme. This deficiency results in the overproduction of oxalate, which then builds up as crystals in the kidneys and other organs, often progressing to end-stage kidney disease and systemic oxalosis. Currently, patients rely on lifelong medications or face the arduous prospect of combined liver and kidney transplants for a long-term solution.

The excitement around ABO-101 stems from its potential as a one-time, intravenously administered gene-editing therapy. It's designed to precisely target the HAO1 gene in the liver, aiming to achieve a permanent reduction in oxalate production. Early indications from the redePHine study are positive; no serious adverse events were reported within the 28-day DLT (dose-limiting toxicity) period following the first patient's dosing, prompting the safety board to recommend continuing with the study. This early safety signal is crucial and provides strong encouragement for the ongoing clinical development.

This clinical trial marks a pivotal moment for Arbor Biotechnologies, showcasing their commitment to translating innovative genetic medicines into tangible hope for patients with rare genetic diseases. As Dan Ory, MD, Chief Medical Officer of Arbor Biotechnologies, emphasized, preclinical data suggests ABO-101 could offer a one-time therapeutic solution, potentially freeing patients from the burden of lifelong treatments. The Oxalosis and Hyperoxaluria Foundation (OHF) has also voiced strong support, highlighting the importance of seeing potential therapies advance from concept to clinical trials, bringing new possibilities to the PH1 community.

The redePHine study is an open-label, global dose escalation trial that will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of a single dose of ABO-101. While the initial dosing took place at Mayo Clinic under the direction of Dr. John Lieske, Arbor Biotechnologies plans to open additional sites in the UK and Europe in the third quarter of this year, expanding the global reach of this important research. ABO-101 has already received Orphan Drug (ODD) and Rare Pediatric Disease Designation (RPDD) from the FDA for PH1, underscoring the high unmet medical need and the therapy's potential significance. This comprehensive approach to clinical development, combined with the promising mechanism of action, positions ABO-101 as a potential game-changer for individuals living with PH1.