Alström Syndrome (AS) Alström Syndrome (AS) is an ultra-rare genetic disorder characterized by a spectrum of progressive symptoms, including vision and hearing loss, obesity, type 2 diabetes, cardio...
Bardet-Biedl Syndrome (BBS) Bardet-Biedl Syndrome (BBS) is a rare, multisystem genetic disorder characterized by symptoms such as retinal degeneration, obesity, polydactyly, kidney abnormalities, and...
Gene Therapy Trial VGB-R04 for Hemophilia B Patients in China Trial ID: NCT05152732Study Title: Safety and Tolerability of VGB-R04 in Patients With Haemophilia BSponsor: Institute of Hematology & ...
Gene Therapy Clinical Trial for Hemophilia B: ZS801 in China Trial ID: NCT05641610Study Title: A Non-Randomized, Open-Label, Dose-Escalation, Phase I/II Study to Evaluate the Safety, Tolerability, Kin...
Clinical Trial Update: SKG0201 Gene Therapy for Spinal Muscular Atrophy Type 1 (SMA 1) Trial ID: NCT06191354Study Title: A Multicenter, Open, Dose-Escalation Clinical Study Evaluating the Safety, Init...
Gene Therapy for Spinal Muscular Atrophy Type 1 – Phase I/II Clinical Trial ClinicalTrials.gov Identifier: NCT05824169Sponsor: GeneCradle Inc.Study Title: Safety and Efficacy of GC101 Gene Therapy i...
GC101 Gene Therapy for Spinal Muscular Atrophy Type 2 – Phase I/II Clinical Trial ClinicalTrials.gov Identifier: NCT05901987Sponsor: GeneCradle Inc.Study Title: Safety and Efficacy of GC101 Gene The...
Phase III Trial: GC101 Gene Therapy for Type 2 Spinal Muscular Atrophy (SMA) ClinicalTrials.gov Identifier: NCT06971094Sponsor: GeneCradle Inc.Study Title: Safety and Efficacy Evaluation of GC101 Gen...
Phase III Trial: GC101 Gene Therapy for Type 2 Spinal Muscular Atrophy (SMA) ClinicalTrials.gov Identifier: NCT06971094Sponsor: GeneCradle Inc.Study Title: Safety and Efficacy Evaluation of GC101 Gen...
Gene Therapy for Wilson's Disease in China Introduction Wilson’s disease is a rare genetic disorder that causes excessive copper accumulation in the liver, brain, and other vital organs. With...