Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Initiatives
Therapeutic Leads
Multiple Disease Platforms
Platform Clinical Programs
Regulatory Documents
Gene Therapy Trial Browser
All Trials
Active Trials
Recruiting
Completed Trials
Phase I
Phase II
Phase III
Gene Therapy Trial Report
Summary
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
NCTID
NCT05682144
(View at clinicaltrials.gov)
Description
A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
(Show More)
Development Status
Active
Indication
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Disease Ontology Term
DOID:12802
Compound Name
ISP-001
Sponsor
Immusoft of CA, Inc.
Funder Type
Industry
Recruitment Status
Recruiting
(Click here for study contact information)
Enrollment Count
2 (ESTIMATED)
Results Posted
Not Available
Therapy Information
Target Gene/Variant
IDUA
Therapy Type
Gene transfer
Therapy Route
Ex-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD19 positive B cells
Delivery System
Electroporation
Vector Type
Sleeping Beauty
Editor Type
Dose 1
2.5E7 cells/kg
Dose 2
10E8 cells/kg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2022-12-12
Completion Date
2039-06-01
Last Update
2025-04-30
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
2
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates
First patient dosed December 2023, drug is redosable
Resources/Links
Clinical Publications
(Presentation) Safety and Initial Activity of Autologous Human B Cells Genetically Engineered to Express Human Iduronidase Using the Sleeping Beauty Transposon System: Results from a First-in-Human Clinical Trial in Subjects with MPS I - WORLDSymposium 2025
News and Press Releases
In a Major Breakthrough for Cell and Gene Therapy, Immusoft Has Re-dosed a Patient with ISP-001 in Its Lead Program for MPS I