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Satgeno
- Development stage
- Preclinical
- Lead developer
- MyoPax
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intramuscular
01
Overview
Satgeno is an autologous, gene-edited cell therapy platform developed by MyoPax for the treatment of limb-girdle muscular dystrophies (LGMD). The therapeutic approach involves isolating Pericyte-derived Human Satellite-like cells (PHSats)—a specific population of muscle stem cells—from a patient's muscle biopsy. These cells are then genetically corrected ex vivo using CRISPR/Cas9 technology delivered via mRNA to address mutations in genes such as DYSF (dysferlin), SGCA (alpha-sarcoglycan), or LMNA (lamin A/C). The corrected PHSats are subsequently re-implanted into the patient to restore muscle regenerative capacity and functional protein expression. Satgeno is currently in preclinical development, with first-in-human clinical trials in the advanced planning stages.
- Other names
- Satgeno
02
Targets
DYSF (Dysferlin)Calpain-3 (CAPN3) c.550delA mutant locus
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