Genecombio Ltd.

Rett 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…

Researchers 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…