Gaucher’s Disease - Drug Pipeline Landscape, 2023

Published Feb 07, 2023

Length 71 Pages

SKU # GIS17762182

Description

Gaucher's disease, or Gaucher disease (GD), is an inherited genetic disorder of the lysosomal storage that is caused by a genetic defect of the lysosomal enzyme "glucocerebrosidase." This enzyme accumulates in cells and certain organs, including the spleen and liver. The condition is mainly of 3 types: Gaucher disease type 1, Gaucher disease type 2, and Gaucher disease type 3.

Gaucher Disease's main cause is mutations in the GBA1 gene, resulting in a lysosomal deficiency.

Gaucher Disease symptoms include enlargement of the liver and spleen, a low number of red blood cells, easy bruising, and bone disease (bone pain and fractures).

Gaucher Disease is diagnosed by clinical symptoms and laboratory tests like an enzyme test called a beta-glucosidase leukocyte (BGL) test.

Gaucher Disease is treated with enzyme replacement therapy, a bone marrow transplant and procedures like spleen removal.

Report Highlights

Global Insight Service’s, Gaucher’s Disease - Drug Pipeline Landscape, 2023 report provides an overview of the Gaucher’s Disease pipeline drugs. This report covers detailed insights on Gaucher’s Disease drugs under development, assessment by target, mechanism of action, route of administration and molecule type.Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Gaucher’s Disease pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.

Methodology

The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.

Scope

The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clincial trials, and key upcoming milestones are included.

Reasons to Buy

Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.

Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.

Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.

Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.

Please Note: This report will be delivered by publisher within 3-4 business days of order confirmation.

List of Tables

List of Figures

1. Introduction

1.1 Gaucher Disease – Pipeline Drugs, 2023 - Coverage

2. Disease Overview – Gaucher Disease

2.1 Causes

2.2 Signs and Symptoms

2.3 Diagnosis

2.4 Disease Management

3. Gaucher Disease – Pipeline Drugs Development - Overview

3.1 Comparative Analysis by Stage of Development

3.2 Number of Products under Development by Companies, 2023

3.3 Products under Development by Companies, 2023

4. Assessment of Therapeutics

4.1 Assessment by Target

4.2 Assessment by Mechanism of Action

4.3 Assessment by Molecule Type

4.3 Assessment by Route of Administration

5. Drug Profiles

5.1 Clinical Stage Drugs – Phase III

5.1.1 Eliglustat

5.1.2 Velaglucerase Alfa

5.1.3 Venglustat

5.2 Clinical Stage Drugs – Phase II

5.2.1 AVR-RD-02

5.2.2 CAN103

5.2.3 FLT201

5.2.4 LY3884961

5.3 Early-Stage Drugs – Preclinical

5.3.1 AL00804

5.3.2 GBA1 Gene Therapy

5.3.3 Gene Therapy

5.3.4 Glucocerebrosidase

5.3.5 GT-02287

5.3.6 GT-02329

5.3.7 JCXH-301

5.3.8 LB-200

5.3.9 M 011

5.3.10 M 012

5.3.11 YH32138

5.4 Early-Stage Drugs – Discovery

5.4.1 AVV-Gcase D15

5.4.2 Enzyme Replacement Therapy

5.4.3 EV-products for Regenerative Medicine

5.4.4 Gene Therapy for Gaucher Disease

5.4.5 KSHN007

5.4.6 XB3-007

5.4.7 YH36425

6. Key Regulatory Designations

7. Key Deals

8. Key Upcoming Milestones

9. Key Companies Involved

9.1 AceLink Therapeutics Inc

9.2 AVROBIO

9.3 BioArctic AB

9.4 Bioasis Technologies Inc

9.5 CANbridge Bio-pharma Co Ltd

9.6 Freeline Therapeutics

9.7 Gaintherapeutics

9.8 Generation Bio Co

9.9 ILIAS Biologics Inc

9.10 Immorna Hangzhou Biotechnology Co Ltd

9.11 Jasper Therapeutics Inc

9.12 Kashiv BioSciences LLC

9.13 Lixte Biotechnology Holdings Inc

9.14 Lysogene SAS

9.15 M6P Therapeutics

9.16 Prevail Therapeutics

9.17 Sanofi

9.18 Takeda

9.19 Vanqua Bio Inc

9.20 Voyager Therapeutics Inc

9.21 Yuhan Corp

10. Dormant Drugs

10.1 Inactive Drugs

10.2 Discontinued Drugs

11. Appendix

List of Tables

Table 1.1 Number of Products Under Development for Gaucher Disease

Table 1.2 Number of Products Under Development by Companies

Table 1.3 Products Under Development by Companies

Table 1.4 Products by Targets

Table 1.5 Products by Mechanism of Action

Table 1.6 Products by Molecule Type

Table 1.7 Products by Route of Administration

Table 2.1 Clinical Trial Details - Eliglustat /Sanofi

Table 2.2 Clinical Trial Details - Velaglucerase Alfa/Takeda

Table 2.3 Clinical Trial Details - Venglustat/Sanofi

Table 2.4 Clinical Trial Details - AVR-RD-02/AVROBIO

Table 2.5 Clinical Trial Details - CAN103/CANbridge Bio-pharma Co Ltd

Table 2.6 Clinical Trial Details - FLT201/Freeline Therapeutics

Table 2.7 Clinical Trial Details - LY3884961/Prevail Therapeutics

Table 3.1 Regulatory Designations

Table 4.1 Inactive Drugs

Table 4.2 Discontinued Drugs

List of Figures

Figure 1.1 Number of Products under Development for Gaucher Disease, 2023

Figure 1.2 Products by Top 5 Targets and Stage of Development for Gaucher Disease, 2023

Figure 1.3 Products by Top 5 Mechanism of Action for Gaucher Disease, 2023

Figure 1.4 Products by Top 5 Molecule Type for Gaucher Disease, 2023

Figure 1.5 Products by Top 5 Route of Administration for Gaucher Disease, 2023

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