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Clinical Trial Report
Gene Therapy Trial Report
Summary
ASC618 Gene Therapy in Hemophilia A Patients
NCTID
NCT04676048
(View at clinicaltrials.gov)
Description
Currently, hemophilia A patients are managed with prophylactic or on-demand replacement therapy with recombinant FVIII or alternative therapeutics. The major challenges of current treatment regimens, such as the short half-life of hemophilia therapeutics with the need for frequent IV injections, encourage the current efforts for gene transfer therapy. This study will evaluate the safety and preliminary efficacy of ASC618, an AAV vector encoding B-domain deleted codon-optimized human factor VIII under a synthetic liver-directed promoter
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Development Status
Active
Indication
Hemophilia A
Disease Ontology Term
DOID:12134
Compound Name
ASC618
Compound Description
HCB-ET3-LCO
Sponsor
ASC Therapeutics
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
12
Results Posted
Not Available
Therapy Information
Target Gene/Variant
F8
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2/8
Editor Type
none
Dose 1
Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2020-12-15
Completion Date
2026-12
Last Update
2023-02-01
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Fast Track, Orphan Drug Designation
Recent Updates
First patient dosed January 2024
Resources/Links
News and Press Releases
ASC Therapeutics Doses First Patient with ASC618 Second-Generation Gene Therapy for Hemophilia A
Preclinical Publications
Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery
Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A
(Poster) Preclinical Development of ASC-618, an Advanced Human Factor VIII Gene Therapy Vector for the Treatment of Hemophilia A - ASGCT 2020