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Gene Therapy Trial Report

Summary

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)


NCTID NCT06461702 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Familial Hypercholesterolemia
Disease Ontology Term DOID:13810
Compound Name YOLT-101
Sponsor The First Affiliated Hospital of Bengbu Medical University
Funder Type Other
Recruitment Status
Enrollment Count 13 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant PCSK9/LDLR
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Intravenous
Drug Product Type RNP
Target Tissue/Cell Hepatocyte
Delivery System LNP
Vector Type none
Editor Type hpABE5
Dose 1 0.2 mg/kg (n=1)
Dose 2 0.4 mg/kg (n=2)
Dose 3 0.6 mg/kg (n=3)
Dose 4
Dose 5

Study Record Dates


Current Stage Early phase1
Submit Date 2024-04-16
Completion Date 2025-12
Last Update 2024-06-17

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates FDA cleared IND application in June 2025

Resources/Links