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Gene Therapy Trial Report

Summary

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

NCTID NCT05682144 (View at clinicaltrials.gov)
Description
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
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
Dose 1 🔄 2.5E7 cells/kg
Dose 2 🔄 10E8 cells/kg

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 🔄
News and Press Releases 🔄