Delving into the Latest Updates on GC-101 with Synapse

Overview

Basic Info

Drug Type

AAV based gene therapy

Synonyms

GC101腺相关病毒, GC-101, RDGT-002

Target

SMN1

Action

stimulants

Mechanism

SMN1 gene stimulants(survival of motor neuron 1, telomeric gene stimulants)

Therapeutic Areas

Nervous System DiseasesOther Diseases

Active Indication

Muscular Atrophy, Spinal, Type IIJuvenile Spinal Muscular AtrophyHMN (Hereditary Motor Neuropathy) Proximal Type I

Inactive Indication-

Originator Organization

Beijing GeneCradle Technology Co., Ltd.

Active Organization

Beijing GeneCradle Technology Co., Ltd.

Inactive Organization-

License Organization-

Drug Highest PhasePhase 3

First Approval Date-

RegulationOrphan Drug (United States), Breakthrough Therapy (China)

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This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA. The secondary objectives are to assess the efficacy, safety, and pharmacokinetic (PK) profile of GC101 in treating Type 2 SMA.

Start Date27 May 2025

Sponsor / Collaborator

Beijing GeneCradle Technology Co., Ltd.

NCT06421831

/ RecruitingPhase 1/2

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 3 Patients

The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.

Start Date10 May 2024

Sponsor / Collaborator

Beijing GeneCradle Technology Co., Ltd.

NCT05901987

/ Active, not recruitingPhase 1/2

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

The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.

Start Date01 Aug 2023

Sponsor / Collaborator

Beijing GeneCradle Technology Co., Ltd.

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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1

Literatures (Medical) associated with GC-101

World Journal of Pediatrics

Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study

Article

Author: Gu, Rui-Jie ; Ma, Xiu-Wei ; Dong, Xiao-Yan ; Zhuang, Lu ; Li, Chong-Yang ; Jiang, Xin-Yang ; Yu, Shuang-Qing ; Wang, Xiao-Dong ; Li, Qiu-Ping ; Zhou, Bing ; Zhong, Bai-Mao ; Yang, Xiao ; Ma, Wen-Hao ; He, Fang ; Li, Ting ; Zhang, Sheng ; Feng, Zhi-Chun ; Xu, Juan ; Wang, Yong-Xia ; Zhang, Yan-Ping ; Wu, Xiao-Bing ; Zhang, Shan ; Wu, Zhi-Jie ; Li, Zhong-Qiu ; Mao, Ying-Ying ; Zhu, Li-Na

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100 Deals associated with GC-101

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Muscular Atrophy, Spinal, Type II	Phase 3	China	Beijing GeneCradle Technology Co., Ltd.	27 May 2025
Juvenile Spinal Muscular Atrophy	Phase 2	China	Beijing GeneCradle Technology Co., Ltd.	10 May 2024
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	China	Beijing GeneCradle Technology Co., Ltd.	14 Mar 2023