Delving into the Latest Updates on VGN-R09b with Synapse

Overview

Basic Info

Drug Type

AAV based gene therapy

Synonyms

VGN R09b, VGNR09b

Target

DDC

Action

activators

Mechanism

DDC activators(DOPA decarboxylase activators), Gene transference(Gene transference)

Therapeutic Areas

Congenital DisordersEndocrinology and Metabolic DiseaseNervous System Diseases

Active Indication

Aromatic Amino Acid Decarboxylase DeficiencyPrimary Parkinson's diseaseParkinson Disease

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 3

First Approval Date-

Regulation-

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第一部分主要目的：评估VGN-R09b治疗AADC缺乏症儿童的安全性、初步有效性。次要目的：评估VGN-R09b治疗AADC缺乏症儿童的有效性、药代动力学、药效学、免疫原性等特征。第二部分主要目的：评估VGN-R09b治疗AADC缺乏症儿童的有效性。次要目的：评估VGN-R09b治疗AADC缺乏症儿童的安全性。

[Translation]

Part I Main purpose: To evaluate the safety and preliminary efficacy of VGN-R09b in the treatment of children with AADC deficiency. Secondary purpose: To evaluate the efficacy, pharmacokinetics, pharmacodynamics, immunogenicity and other characteristics of VGN-R09b in the treatment of children with AADC deficiency. Part II Main purpose: To evaluate the efficacy of VGN-R09b in the treatment of children with AADC deficiency. Secondary purpose: To evaluate the safety of VGN-R09b in the treatment of children with AADC deficiency.

Start Date20 Jun 2024

Sponsor / Collaborator

Shanghai Taichang Biotechnology Co., Ltd.

[+1]

NCT06432140

/ Not yet recruitingPhase 1

An Open, Dose-escalating and Dose Confirmation Trial to Evaluate the Safety and Efficacy of VGN-R09b by Intra Putamen Injection in Patients With Severe Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

This trial includes dose-escalating part (phase 1) and dose confirming part, to prove the safety and efficacy of VGN-R09b to treat patients with severe AADC deficiency

Start Date01 May 2024

Sponsor / Collaborator

Shanghai Vitalgen Biopharma Co., Ltd.

100 Clinical Results associated with VGN-R09b

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100 Translational Medicine associated with VGN-R09b

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100 Patents (Medical) associated with VGN-R09b

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100 Deals associated with VGN-R09b

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Aromatic Amino Acid Decarboxylase Deficiency	Phase 3	China	Shanghai Vitalgen Biopharma Co., Ltd.	-
Primary Parkinson's disease	Phase 2	China	Shanghai Vitalgen Biopharma Co., Ltd.	12 Aug 2024
Parkinson Disease	Phase 2	-	Shanghai Vitalgen Biopharma Co., Ltd.	01 Jul 2024