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Genecombio Ltd.
Clinical trials sponsored by Genecombio Ltd., explained in plain language.
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Can a One-Time gene therapy fix the root cause of rett syndrome?
Cure By invitation onlyRett syndrome is a rare genetic disorder that mostly affects girls and stems from mutations in the MECP2 gene. This trial tests GCB-002, an experimental gene therapy given as a single injection into the spinal fluid, in 6 to 9 girls aged 2 to 10 who have a confirmed MECP2 mutatio…
Sponsor: Genecombio Ltd. • Aim: Cure
Last updated Sep 17, 2026 00:00 UTC
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One-Shot gene therapy aims to restore movement in children with SMA
Cure By invitation onlyResearchers test GCB-001, a gene therapy given as a single injection into the spinal fluid, in children aged 2 to 12 with type 2 spinal muscular atrophy who can sit alone but cannot walk. The trial checks safety and whether the treatment improves motor skills over 12 months. It i…
Sponsor: Genecombio Ltd. • Aim: Cure
Last updated Sep 13, 2026 00:00 UTC