Abstract: INFO08-FR
redePHine: A Global, Open-Label Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1
Session Information
- Informational Posters - 2
October 23, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- No subcategory defined
Authors
- Ory, Dan, Arbor Biotechnologies Inc, Cambridge, Massachusetts, United States
- Rozova, Anna, Chiesi Canada Corp., Woodbridge, Ontario, Canada
- Lieske, John C., Mayo Clinic Minnesota, Rochester, Minnesota, United States
Description
Background
Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder characterized by excessive hepatic oxalate production leading to abnormal increase in urinary oxalate (UOx) excretion, nephrocalcinosis, nephrolithiasis, progressive kidney failure and/or systemic oxalosis. Hydroxyacid oxidase 1 (HAO1), a key gene in hepatic oxalate production that encodes glycolate oxidase (GO), is a validated target for the treatment of PH1. ABO-101 is a novel, investigational CRISPR-Cas12 gene editing therapy that targets permanent loss of function of the HAO1 gene to functionally cure PH1.
Purpose
The redePHine study is recruiting patients with PH1 to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ABO-101.
Methods
redePHine (EUCT: 2024-518144-20-00; ClinicalTrials.gov: NCT06839235) is an ongoing phase 1/2, open-label, multicenter, global study. Current study locations include France, Germany, Netherlands, Tunisia, UK and US. The study has 3 parts: part A will enroll 3 sequential adult cohorts aged ≥18 to ≤64 years to receive ascending single intravenous doses of ABO-101 to identify a recommended dose (RD); parts B and C will enroll pediatric patients aged ≥6 to <18 years and siRNA-treated adult and pediatric patients aged ≥6 to <18 years, respectively, to receive ABO-101 at the RD (Figure). Participants will be followed for 24 months for safety and efficacy and then will enter long-term safety follow-up. Part A and B participants must not have received any siRNA therapy for PH1 24 months prior to enrollment, with hyperoxaluria ≥1.5x upper limit of normal. Eligibility criteria include a genetic PH1 diagnosis, estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73m2, and weight ≤90 kg. Key endpoints include the incidence and severity of treatment-emergent adverse events up to Month 6, change in 24-hour UOx from baseline to Month 6 and change in eGFR from baseline to Months 12 and 24.
Conclusion
redePHine is the first human trial of ABO-101 and currently recruiting to provide insights into the safety, tolerability, and preliminary efficacy of this novel gene editing therapy.
Acknowledgment
We would like to thank all investigators and patients involved in the redePHine study for their contribution to the trial. Medical writing support was provided by Cactus Life Sciences and funded by Chiesi USA Inc. and Arbor Biotechnologies.
Figure: redePHine Study Design
Funding
- The research presented in this abstract was funded by Chiesi USA Inc. and Arbor Biotechnologies.