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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
Delivery System 🔄 Lipid encapsulation
Editor Type 🔄 ABE8e
Dose 1 🔄 Undisclosed

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