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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Phase I/IIaļ¼ŒOpen-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)


NCTID NCT06539208 (View at clinicaltrials.gov)
Description
Indication Transthyretin Amyloidosis, with Polyneuropathy/Cardiomyopathy
Compound Name YOLT-201
Sponsor YolTech Therapeutics Co., Ltd
Funder Type Industry
Status
Recruiting
Enrollment Count 31

Therapy Information


Target Gene/Variant TTR
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 Liver
Delivery System Lipid encapsulation
Vector Type LNP
Editor Type Cas9 mRNA
Dose 1 Undisclosed dose ascension
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-08-01
Completion Date 2026-06-30
Last Update 2024-08-06

Participation Criteria


Eligible Age 18 Years - 80 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 3
Locations China

Regulatory Information


Has US IND False
Recent Updates First patient dosed (6/28/24)

Resources/Links