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Clinical Trial Report

Gene Therapy Trial Report

Summary

Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)


NCTID NCT06511349 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Type 1 Primary Hyperoxaluria
Disease Ontology Term DOID:0111670
Compound Name YOLT-203
Sponsor RenJi Hospital
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 7
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 YolCas12
Dose 1 0.3 mg/kg
Dose 2 0.6 mg/kg
Dose 3 1.0 mg/kg
Dose 4
Dose 5

Study Record Dates


Current Stage Early phase1
Submit Date 2024-07-04
Completion Date 2026-01-31
Last Update 2024-12-19

Participation Criteria


Eligible Age >=2 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates First patient dosed (8/5/24)

Resources/Links