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FDA Clears IND for Arbor Biotechnologies’ ABO-101 Gene Editing Therapeutic for Primary Hyperoxaluria Type 1

NextEdge Admin

21 Dec 2024

2 min 22 sec

Arbor Biotechnologies™ announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for ABO-101, an innovative gene-editing therapeutic targeting primary hyperoxaluria type 1 (PH1). The redePHine Phase 1/2 study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ABO-101 in adult and pediatric PH1 patients.

PH1 is a rare genetic disorder caused by enzyme deficiencies in the liver, leading to excessive oxalate production, kidney stones, end-stage kidney disease, and systemic oxalosis. ABO-101 utilizes a CRISPR-based approach to knock down the HAO1 gene in the liver, providing durable reduction in oxalate levels. Supported by preclinical data showing specific and lasting in vivo HAO1 editing, ABO-101 demonstrates therapeutic reductions in urinary oxalate in PH1 models.

“We’re excited to advance ABO-101 to the clinic as a potential first-in-class treatment for PH1, addressing a high unmet need,” said Dan Ory, M.D., Chief Medical Officer of Arbor. Kim Hollander, Executive Director of The Oxalosis and Hyperoxaluria Foundation, highlighted the significant milestone this represents for the PH1 community, calling it a groundbreaking opportunity in genomic medicine.

ABO-101 is a one-time gene-editing therapy composed of lipid nanoparticles licensed from Acuitas Therapeutics. These encapsulate messenger RNA encoding a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA targeting the human HAO1 gene. Arbor CEO Devyn Smith commended the team’s efforts, emphasizing the broader potential of their genetic medicines for rare and common genetic disorders.