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Clinical Trial Report
Gene Therapy Trial Report
Summary
Therapy Information
Study Record Dates
Participation Criteria
Locations
Regulatory Information
Resources/Links
Summary
A Phase I/IIa,Open-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
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of YOLT-201 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM).
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Development Status
Active
Indication
Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
Disease Ontology Term
DOID:0050638
Compound Name
YOLT-201
Sponsor
YolTech Therapeutics Co., Ltd
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
31
Results Posted
Not Available
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
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Dose escalation completed December 2024
Resources/Links
News and Press Releases
YolTech Completes Dose Escalation in YOLT-201 Phase I Trial
Preclinical Publications
Library-Assisted Evolution in Eukaryotic Cells Yield Adenine Base Editors with Enhanced Editing Specificity