Arbor Biotechnologies Announces FDA Acceptance of IND Application for ABO-101 for the Treatment of Primary Hyperoxaluria Type 1
RedePHine phase 1/2 clinical trial initiation expected in first half of 2025
CAMBRIDGE, MA – December 19, 2024 – Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ABO-101, a novel gene editing therapeutic designed to address primary hyperoxaluria type 1 (PH1). The redePHine Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ABO-101 in both adult and pediatric patients with PH1.
“We are thrilled to advance ABO-101 to the clinic as we believe it has the potential to be a first-in-class treatment for PH1, a rare disease with a high unmet need, and reinforces the promise of our precisely tailored gene editing approach,” said Dan Ory, M.D., Chief Medical Officer of Arbor. “ABO-101 is supported by a strong suite of preclinical data demonstrating specific and durable in vivo editing of HAO1 and corresponding, therapeutically relevant reductions in urinary oxalate in PH1 disease models.”
ABO-101 is a liver-targeted gene editing therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1). PH1 is a rare genetic disorder in which enzyme deficiencies in the liver lead to the overproduction and buildup of oxalate, resulting in kidney stones eventually leading to end stage kidney disease and systemic oxalosis. ABO-101 is designed to knock down HAO1 gene expression in the liver, thereby providing durable reduction in oxalate production.
“The FDA approval of the ABO-101 IND marks a significant milestone for the hyperoxaluria community. At OHF, we are committed to partnering with industry leaders to advance care, research, and patient-focused drug development for hyperoxaluria. Arbor’s innovative CRISPR-based approach represents a groundbreaking opportunity in genomic medicine, with the potential to transform the lives of patients with PH1,” said Kim Hollander, Executive Director of The Oxalosis and Hyperoxaluria Foundation.
“The initiation of this study marks an important milestone both for patients with PH1 and in the growth of our company as we begin advancing our pipeline of first-in-class programs into the clinic,” said Devyn Smith, CEO of Arbor. “There is a tremendous need for innovative approaches to treat genetic conditions – from ultra-rare to the most common genetic diseases – and we’re excited for the potential of our next-generation genetic medicines that can address a vast spectrum of devastating diseases. I applaud the hard work of our team at Arbor for getting us to this stage.”
About ABO-101
ABO-101 is a novel, investigational gene editing medicine designed to be a one-time treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene.
About Arbor Biotechnologies, Inc.
Arbor Biotechnologies™, a next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company’s unique suite of optimized gene editors goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. With Arbor’s lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, progressing into clinical trials, the company continues to focus their research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: arbor.bio.
Media Contact:
Peg Rusconi
Deerfield Group
prusconi@nulldeerfieldgroup.com
