FDA Accepts Arbor Biotechnologies' IND for ABO-101 in Primary Hyperoxaluria Type 1 Treatment

Arbor Biotechnologies, a biotechnology firm renowned for its groundbreaking work in genetic medicine, recently announced a significant development in its research aimed at treating primary hyperoxaluria type 1 (PH1). The U.S. Food and Drug Administration (FDA) has given the green light to the Investigational New Drug (IND) application for ABO-101, a pioneering gene editing therapy designed to combat PH1. This approval sets the stage for the redePHine Phase 1/2 clinical study, which will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and initial effectiveness of ABO-101 in both adult and pediatric patients diagnosed with PH1.

Dan Ory, M.D., who serves as the Chief Medical Officer at Arbor, expressed enthusiasm about this advancement. He highlighted the potential of ABO-101 to emerge as a first-of-its-kind treatment for PH1, a rare condition with significant unmet therapeutic needs. ABO-101 is supported by a robust collection of preclinical data that demonstrates effective and long-lasting in vivo editing of the HAO1 gene. This editing leads to therapeutically meaningful reductions in urinary oxalate levels in PH1 models.

The innovative ABO-101 therapy targets the liver to address the root causes of PH1, a genetic disorder characterized by enzyme deficiencies. These deficiencies result in excessive production and accumulation of oxalate, leading to kidney stones and eventually progressing to end-stage kidney disease and systemic oxalosis. By specifically reducing HAO1 gene expression in the liver, ABO-101 aims to achieve a sustained reduction in oxalate production.

Kim Hollander, Executive Director of The Oxalosis and Hyperoxaluria Foundation, remarked on the FDA's approval of the ABO-101 IND as a momentous milestone for the hyperoxaluria community. She emphasized the foundation's commitment to collaborating with industry leaders like Arbor to enhance care, research, and patient-centric drug development for hyperoxaluria. Hollander noted that Arbor’s CRISPR-based methodology offers a transformative possibility in genomic medicine with the potential to significantly improve the lives of PH1 patients.

The CEO of Arbor, Devyn Smith, sees the initiation of this clinical study as a crucial step forward for PH1 patients and for the company itself. As Arbor advances its pipeline of first-in-class therapeutic programs, Smith underscores the critical need for innovative strategies to tackle genetic conditions, ranging from ultra-rare to the most common genetic diseases. He praised the dedication of Arbor's team in reaching this critical juncture.

ABO-101 is engineered as a single administration gene editing therapy that permanently inactivates the HAO1 gene in the liver to curtail oxalate production associated with PH1. This investigational therapy employs a lipid nanoparticle (LNP) system, licensed from Acuitas Therapeutics, which encapsulates messenger RNA. This RNA encodes a novel Type V CRISPR Cas12i2 nuclease along with an optimized guide RNA that accurately targets the human HAO1 gene.

Based in Cambridge, Massachusetts, Arbor Biotechnologies is a pioneering entity in next-generation gene editing, dedicated to developing a diverse portfolio of gene editing treatments for a wide array of genetic conditions. The company's suite of advanced gene editors surpasses the limitations of earlier technologies, opening doors to new genetic targets. This advancement has propelled the development of an extensive pipeline of first-in-class assets aimed at diseases with significant unmet needs. Arbor's leading program, ABO-101, is now entering clinical trials, as the company continues to focus its research and development on genomic diseases affecting the liver and central nervous system, for which there are currently no effective cures.