Last update 21 Nov 2024
GC-101
Last update 21 Nov 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms |
Target |
Mechanism SMN1 gene stimulants(survival of motor neuron 1, telomeric gene stimulants) |
Therapeutic Areas |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePhase 1/2 |
First Approval Date- |
Regulation- |
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Related
5
Clinical Trials associated with GC-101A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
评价GC101腺相关病毒注射液鞘内（IT）注射治疗2型脊髓性肌萎缩症（SMA）患者的安全性、耐受性以及疗效的开放、多中心、单臂Ⅰ/Ⅱ期临床试验
[Translation] An open, multicenter, single-arm Phase I/II clinical trial to evaluate the safety, tolerability, and efficacy of intrathecal (IT) injection of GC101 adeno-associated virus injection in the treatment of patients with type 2 spinal muscular atrophy (SMA)
100 Clinical Results associated with GC-101
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100 Translational Medicine associated with GC-101
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100 Patents (Medical) associated with GC-101
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100 Deals associated with GC-101
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R&D Status
10 top R&D records. to view more data
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Juvenile Spinal Muscular Atrophy | Phase 2 | CN | 10 May 2024 | |
| Muscular Atrophy, Spinal, Type II | Phase 2 | CN | 31 Jul 2023 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | CN | 14 Mar 2023 |
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