Arbor Biotechnologies Announces First Patient Dosed at Mayo Clinic in the redePHine Phase 1/2 Study of ABO-101, an Investigational Gene Editing Treatment for Primary Hyperoxaluria Type 1
- ABO-101 was well tolerated with no serious adverse events or dose-limiting toxicities reported in the 28-days following dosing
- Phase 1/2 redePHine study (NCT06839235) dose escalation ongoing with enrollment at Mayo Clinic in the US and plans to open additional sites in the UK and EU
CAMBRIDGE, MA – July 30, 2025 – Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced the first patient was dosed at Mayo Clinic in the redePHine study evaluating ABO-101, an investigational CRISPR gene editing therapy, in people living with primary hyperoxaluria type 1 (PH1). There were no serious adverse events reported in the 28-day DLT period following dosing, and the safety board recommended continuation of dosing.
“Preclinical data support the potential for ABO-101 to be a one-time therapy for patients living with PH1, who currently rely on lifelong medication, such as siRNA therapies, and for whom historically the only long-lasting treatment option was a combined liver and kidney transplant,” said Dan Ory, MD, Chief Medical Officer of Arbor Biotechnologies. “As Arbor’s first clinical trial, this study marks an important milestone in our commitment to advancing innovative genetic medicines into the clinic for people living with PH1 and other rare genetic diseases.”
“At OHF, we’re proud to stand alongside companies and researchers who prioritize the patient experience in their work. As a patient advocacy organization, we know how critical it is to see potential therapies move from concept to clinical trials. Dosing the first patient in the redePHine study marks an exciting milestone—and a meaningful step forward in bringing hope and new possibilities to those living with PH1,” said Kim Hollander, Executive Director, The Oxalosis and Hyperoxaluria Foundation (OHF).
PH1 is a rare genetic disease caused by a mutation in the AGXT gene that leads to liver enzyme deficiency, resulting in oxalate overproduction and crystal buildup in kidneys and other organs, and can progress to end-stage kidney disease. ABO-101 is a one-time IV-administered CRISPR gene editing therapy targeting the glycolate oxidase gene (HAO1). The redePHine study is an open-label global dose escalation study to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of a single dose of ABO-101 in people living with PH1. redePHine is anticipated to enroll globally with plans to open additional sites in the UK and Europe in Q3 of this year.
The trial, conducted at Mayo clinic under the direction of John Lieske, M.D., a board-certified nephrologist, was approved through a new accelerated activation lane for high impact clinical trials. Dr. Lieske practices in the Division of Nephrology and Hypertension at Mayo Clinic, where he treats patients with nephrolithiasis, chronic kidney disease, and hyperoxaluria. He leads a research team that seeks to improve treatment and prevention of chronic kidney disease and kidney stones.
About Primary Hyperoxaluria Type I (PH1)
Primary hyperoxaluria type 1 is an ultrarare lifelong genetic disease, where a mutation in the AGXT gene leads to an enzyme deficiency in the liver resulting in an overproduction of oxalate by the liver and eventual buildup of oxalate crystals in the kidney and other organ systems. As the disease progresses, it can cause recurring kidney stones, kidney damage and eventually lead to end-stage kidney disease (ESKD) and systemic oxalosis. While two siRNA therapies are available for lifelong treatment, historically the only treatment with a long-term effect for PH1 has been a combined liver and kidney transplant.
About ABO-101
ABO-101 is a novel, investigational gene editing medicine designed to be a one-time liver-directed gene editing treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce oxalate production. ABO-101 is currently being evaluated for PH1 in the redePHine Phase 1/2 clinical study (NCT06839235). 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. ABO-101 has received orphan drug (ODD) and rare pediatric disease designation (RPDD) from the FDA for the treatment of PH1. ABO-101 has not been approved for any use by the FDA or any other regulatory agency.
About the redePHine Study
The Phase 1/2 redePHine study is an open label global dose escalation study to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of a single dose of ABO-101 in participants with primary hyperoxaluria type 1. The trial will consist of 2 Study Periods. During the first Study Period, there will be 2 parts. In Part A, adult participants will be treated with a single ascending dose to identify a recommended dose. In Part B, pediatric participants will be treated with the recommended dose. Following the first Study Period, participants will start Study Period 2, a long-term monitoring program to comply with local and national requirements. For more information on redePHine (NCT06839235), please visit clinicaltrials.gov.
About Arbor Biotechnologies, Inc.
Arbor Biotechnologies™, a clinical stage, 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, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, 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 clinical program, ABO-101 for the treatment of primary hyperoxaluria type 1, the company continues to focus its 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.
Arbor Biotechnologies Media Contact:
Peg Rusconi
Deerfield Group
prusconi@nulldeerfieldgroup.com