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Gene Therapy Trial Report

Summary

Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)


NCTID NCT06839235 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Primary Hyperoxaluria Type 1 (PH1)
Disease Ontology Term DOID:0111670
Compound Name ABO-101
Sponsor Arbor Biotechnologies
Funder Type Industry
Recruitment Status
Enrollment Count 23 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant HAO1
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Intravenous
Drug Product Type MRNA, LNP
Delivery System Lipid encapsulation
Vector Type LNP
Editor Type Cas12i2
Dose 1 Undisclosed dose escalation

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-02-17
Completion Date 2043-02
Last Update 2026-02-12

Participation Criteria


Eligible Age 6 Years - 64 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria for Parts A and B * Documentation of PH1 as determined by genetic analysis confirming pathogenic mutations in the alanine-glyoxylate aminotransferase (AGXT) gene (valid historical laboratory data will be reviewed and approved by the Sponsor) * Age at time of signing the informed consent/assent form: * Part A: ≥18 years to ≤64 years * Part B: ≥6 years to \<18 years * 24-hour UOx ≥0.7 mmol/24 hours/1.73 m² * eGFR ≥30 mL/min/1.73m² * Weight ≤90 kg Key Exclusion Criteria for Parts A and B * Confirmed diagnosis of primary hyperoxaluria type 2 or type 3 * History of a liver, kidney or combined liver/kidney transplant * Currently on dialysis * Participant has previously used (within past 24 months) or is currently receiving an approved or investigational urinary oxalate lowering RNA interference (RNAi) or siRNA therapy * Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 7
Locations United States,United Kingdom,France,Tunisia,Germany

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates First patient dosed in July 2025

Resources/Links