Contact SCGE




Gene Therapy Trial Report

Summary

YOLT-204 in Patients With Hemoglobinopathies


NCTID NCT07190001 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)
Disease Ontology Term DOID:0081445; DOID:0080771
Compound Name YOLT-204
Sponsor Guangzhou Women and Children's Medical Center
Funder Type Other
Recruitment Status
Not yet recruiting
Enrollment Count 18 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant HBG1
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type MRNA, LNP
Target Tissue/Cell
Delivery System Lipid encapsulation
Vector Type
Editor Type ABE8e
Dose 1 Undisclosed
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Early phase1
Submit Date 2025-09-16
Completion Date 2027-04-30
Last Update 2025-09-24

Participation Criteria


Eligible Age 3 Years - 17 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links