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Clinical Trial Report

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
Recruiting
Enrollment Count 23
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
Target Tissue/Cell
Delivery System Lipid encapsulation
Vector Type LNP
Editor Type Cas12i2
Dose 1 Undisclosed dose escalation
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-02-17
Completion Date 2043-02
Last Update 2025-04-02

Participation Criteria


Eligible Age 6 Years - 64 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates IND accepted 12/19/24

Resources/Links