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SMA gene therapy GC101 found safe, effective in animals
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SMA gene therapy GC101 found safe, effective in animals
Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy (SMA) type 3 and to be safe in non-human primates, supporting its move into clinical testing. Genecradle Therapeutics is sponsoring ongoing Phase 1/2 clinical trials in SMA type 1 (NCT05824169), SMA type 2 (NCT05901987), and SMA type 3 (NCT06421831). Each clinical trial is testing how safe a single dose of GC101 is and how well it works when…
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