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GIVI-MPC
- Development stage
- Preclinical
- Lead developer
- IPS HEART
- Modality
- iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Intramuscular
01
Overview
GIVI-MPC is an induced pluripotent stem cell (iPSC)-derived cell therapy developed by IPS HEART for the treatment of Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD). The therapy utilizes the small molecule histone deacetylase (HDAC) inhibitor Givinostat to reprogram human iPSCs into myogenic/mesenchymal progenitor cells (MPCs). These MPCs are designed to engraft into skeletal muscle tissue and restore the expression of 100% full-length human dystrophin, addressing the underlying genetic deficiency in dystrophic patients. GIVI-MPC has received FDA Orphan Drug and Rare Pediatric Disease designations for DMD, as well as Orphan Drug designations for BMD in both the US and EU. It is currently in the preclinical stage, with a Phase I/II clinical trial design approved by the FDA following a pre-IND meeting.
- Other names
- Givinostat-reprogrammed myogenic progenitor cells
02
Targets
ITGA4 (Integrin alpha-4)
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