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VTA-100
- Development stage
- Preclinical
- Lead developer
- Vita Therapeutics
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Parenteral
01
Overview
**VTA-100** is an investigational autologous cell therapy developed by Vita Therapeutics for limb-girdle muscular dystrophy type 2A (LGMD2A/R1), the most common subtype of LGMD characterized by progressive proximal muscle weakness starting in childhood or adolescence, often leading to wheelchair dependence by age 35. It utilizes induced pluripotent stem cell (iPSC)-derived satellite cells, genetically corrected for the underlying mutation (typically in the CAPN3 gene), to enable muscle regeneration and repair by replacing defective muscle stem cells. Preclinical data presented in 2023 demonstrated efficacy in disease models, with IND-enabling studies ongoing as of 2025 and a first-in-human trial planned to recruit 15 patients soon.[1][2][3][7][9]
- Other names
- VTA 100VTA100VTA-100
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