Last update 11 Dec 2024
GC-101
Last update 11 Dec 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms GC-101, RDGT-002 |
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- |
RegulationBreakthrough Therapy (CN) |
Login to view timeline
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
Login to view more data
100 Translational Medicine associated with GC-101
Login to view more data
100 Patents (Medical) associated with GC-101
Login to view more data
100 Deals associated with GC-101
Login to view more data
R&D Status
10 top R&D records. to view more data
Login
| 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 |
Login to view more data
Clinical Result
Clinical Result
Indication
Phase
Evaluation
View All Results
Login to view more data
Translational Medicine
Boost your research with our translational medicine data.
login
or
Deal
Boost your decision using our deal data.
login
or
Core Patent
Boost your research with our Core Patent data.
login
or
Clinical Trial
Identify the latest clinical trials across global registries.
login
or
Approval
Accelerate your research with the latest regulatory approval information.
login
or
Regulation
Understand key drug designations in just a few clicks with Synapse.
login
or
Chat with Hiro
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.
Bio
Bio Sequences Search & Analysis
Sign up for free
Chemical
Chemical Structures Search & Analysis
Sign up for free