Delving into the Latest Updates on VGN-R08 with Synapse

Overview

Basic Info

Drug Type

AAV based gene therapy

Synonyms

VGN-R08b, VGNR08

Target

GBA1

Action

stimulants

Mechanism

GBA stimulants(Beta-glucocerebrosidase stimulants), Gene transference(Gene transference)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]

Active Indication

Parkinson DiseasePrimary Parkinson's diseaseGaucher Disease, Type 2

Inactive Indication-

Originator Organization

Shanghai Vitalgen Biopharma Co., Ltd.

Active Organization

Shanghai Vitalgen Biopharma Co., Ltd.

Tianze Yuntai (Shenzhen) Biomedical Co., Ltd.

Inactive Organization-

License Organization-

Drug Highest PhasePhase 1/2

First Approval Date-

RegulationFast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States)

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主要目的: 评估VGN-R08b治疗GBA1突变型帕金森病患者的耐受性和安全性次要目的： 1. 评估VGN-R08b给药后（侧脑室注射）的药代动力学、药效学、免疫原性特征； 2. 评估VGN-R08b治疗GBA1突变型帕金森病患者的初步有效性，探索最佳有效剂量，为后续研究提供科学依据

[Translation]

Primary Objective: To evaluate the tolerability and safety of VGN-R08b in patients with GBA1-mutant Parkinson's disease. Secondary Objectives: 1. To evaluate the pharmacokinetic, pharmacodynamic, and immunogenic characteristics of VGN-R08b after administration (intraventricular injection); 2. To assess the preliminary efficacy of VGN-R08b in treating patients with GBA1-mutant Parkinson's disease, explore the optimal effective dose, and provide a scientific basis for subsequent studies.

Start Date-

Sponsor / Collaborator

Tianze Yuntai (Shenzhen) Biomedical Co., Ltd.

[+1]

100 Clinical Results associated with VGN-R08

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100 Translational Medicine associated with VGN-R08

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100 Patents (Medical) associated with VGN-R08

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100 Deals associated with VGN-R08

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Parkinson Disease	Phase 2	-	Shanghai Vitalgen Biopharma Co., Ltd.	01 Mar 2026
Primary Parkinson's disease	Phase 2	China	Shanghai Vitalgen Biopharma Co., Ltd.	12 Feb 2026
Gaucher Disease, Type 2	Phase 1	China	Shanghai Vitalgen Biopharma Co., Ltd.	01 Mar 2023