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gc101
- Development stage
- Unknown
- Lead developer
- GeneCradle
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Recombinant Proteins and Enzymes
- Administration
- Intrathecal
01
Overview
GC101 is an investigational gene therapy developed for the treatment of spinal muscular atrophy (SMA), including types 1, 2, and 3. It is a recombinant adeno-associated virus type 9 (rAAV9) vector carrying a functional SMN1 gene expression cassette. The therapy is administered as a single intrathecal injection to deliver the SMN1 gene directly to motor neuron cells in the central nervous system. By restoring SMN1 expression in these cells, GC101 aims to improve motor function and survival outcomes in patients with SMA. Clinical trials have demonstrated good safety and preliminary efficacy across multiple SMA subtypes[5][6][7][8][9][10].
- Other names
- GC101 adeno-associated virus injectionGC-101 adeno-associated virus injectionGC 101 adeno-associated virus injection
02
Targets
SMN (Survival motor neuron protein)
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