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

PHASE1RecruitingINTERVENTIONAL
Enrollment

2

Participants

Timeline

Start Date

April 12, 2023

Primary Completion Date

June 1, 2025

Study Completion Date

June 1, 2039

Conditions
Mucopolysaccharidosis IH/SMucopolysaccharidosis IS
Interventions
BIOLOGICAL

Autologous Plasmablasts (B cells)

Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.

Trial Locations (2)

55455

RECRUITING

University of Minnesota, Minneapolis

94609

RECRUITING

UCSF Benioff Children's Hospital Oakland, Oakland

All Listed Sponsors
lead

Immusoft of CA, Inc.

INDUSTRY