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Gaucher’s Disease pipeline analysis shows tremendous growth opportunities for the upcoming years in the domain

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Gaucher’s Disease pipeline analysis shows tremendous growth opportunities for the upcoming years in the domain

September 06
14:30 2021
Gaucher’s Disease pipeline analysis shows tremendous growth opportunities for the upcoming years in the domain
Gaucher’s Disease Pipeline
DelveInsight’s Gaucher’s Disease pipeline report provides comprehensive insights into the current clinical development scenario and growth prospects in the Gaucher’s disease market. A detailed picture of Gaucher’s disease pipeline landscape is provided, including a disease overview and treatment guidelines for Gaucher’s disease.

DelveInsight’s Gaucher’s Disease pipeline report provides comprehensive insights into the current clinical development scenario and growth prospects in the Gaucher’s disease market. A detailed picture of Gaucher’s disease pipeline landscape is provided, including a disease overview and treatment guidelines for Gaucher’s disease.

Some of the Key Highlights from the Gaucher’s Disease Pipeline Report

  • The Gaucher’s Disease pipeline comprises therapies that are in different stages of the clinical phase include Venglustat, Arimoclomol, PR-001, AVR-RD-02, M 011, GBA programme, and others that are expected to be available for Gaucher’s Disease treatment soon.

  • Several pharmaceutical companies are evaluating their drug candidates for Gaucher’s Disease treatment include Sanofi, Orphazyme, Prevail Therapeutics, AvroBio, M6P Therapeutics, Gain therapeutics, among others.

  • The therapies that are in late phase (Phase II/III) of development for Gaucher’s Disease include Venglustat (Sanofi), and others whereas Arimoclomol (Orphazyme) is in Phase II of Gaucher’s Disease clinical trials. 

  • Some therapies such as M 011, GBA programme, along with others are still in the early phase of development i.e. preclinical stage for Gaucher’s Disease treatment.

For more information, request sample @ Gaucher’s Disease Pipeline Analysis

Gaucher’s Disease: Overview

Gaucher Disease is a rare, inherited metabolic disorder characterized by a lack of the enzyme glucocerebrosidase, which causes an accumulation of harmful amounts of certain fats (lipids), specifically the glycolipid glucocerebroside, throughout the body, particularly in the bone marrow, spleen, and liver. Gaucher disease symptoms and physical findings vary greatly from patient to patient. There are three types of Gaucher’s Disease- Gaucher Disease Type 1, Gaucher Disease Type 2, and Gaucher Disease Type 3.

Gaucher’s Disease Symptoms

Gaucher’s Disease symptoms differ from person to person. Many people’s symptoms start in childhood. Some people experience only minor symptoms. Gaucher’s Disease symptoms can include: 

  • Enlarged Spleen

  • Enlarged Liver

  • Eye Movement Disorders

  • Yellow Spots in the Eyes

  • Seizures

  • Bone Pain and Fracture

Gaucher’s Disease Treatment

Gaucher Disease Type 1 is treatable with regular therapy. Gaucher Disease treatments either increase enzyme levels or decrease the fatty substance that accumulates in the body as a result of Gaucher Disease. The neurological damage caused by Gaucher Disease Types 2 and 3 has no treatment. Gaucher’s Disease treatments available include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).

Gaucher’s Disease Pipeline Analysis: Drug Profiles

Venglustat: Sanofi

Product Description

Venglustat developed by Sanofi is a small-molecule glucosylceramide synthase (GCS) inhibitor that is designed to reduce the production of glucosylceramide (GL-1) and thus the formation of glucosylceramide-based glycosphingolipids.

Phase II/III

NCT02843035: Sanofi launched a three-part study in January 2017 to assess the efficacy and safety of Venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease type 3 (GD3) receiving open-label long-term treatment.

Gaucher’s Disease Pipeline Therapies and Key Companies 

  • Venglustat: Sanofi 

  • Arimoclomol: Orphazyme

  • PR-001: Prevail Therapeutics

  • AVR-RD-02: AvroBio

  • M 011: M6P Therapeutics

  • GBA programme: Gain therapeutics

Get more details on the emerging therapies @ Gaucher’s Disease Treatment Gene Therapy

Gaucher’s Disease Therapeutics Assessment

  • By Product Type

    • Monotherapy

    • Combination Therapy

  • By Stage

    • Discovery

    • Pre-Clinical

    • Phase I

    • Phase II

    • Phase III

    • Pre-registration

  • By Route of Administration

    • Inhalation

    • Intravenous

    • Oral

    • Subcutaneous

  • By Molecule Type

    • Small Molecule

    • Stem Cell Therapy

    • Gene Therapy

  • Targets

    • Immune System

    • Multiple Kinase

  • By Mechanism of Action

    • Protease Inhibitors

    • Immunomodulatory

Scope of the Report

Coverage: Global

Gaucher’s Disease Key Players: Sanofi, Orphazyme, Prevail Therapeutics, AvroBio, M6P Therapeutics, Gain therapeutics, among others

Gaucher’s Disease Pipeline Therapies: Venglustat, Arimoclomol, PR-001, AVR-RD-02, M 011, GBA programme, and others

Table of Contents

1.

Introduction

2.

Executive Summary

3.

Gaucher’s Disease: Overview

4.

Gaucher’s Disease Pipeline Therapeutics

5.

Gaucher’s Disease Late Stage Products (Phase III)

6.

Gaucher’s Disease Mid Stage Products (Phase  II/III)

7.

Gaucher’s Disease Mid Stage Products (Phase II)

8.

Gaucher’s Disease Early Stage Products (Phase  I/II)

9.

Gaucher’s Disease Early stage products (Phase I/II)

10.

Gaucher’s Disease Preclinical and Discover Stage Products

11.

Gaucher’s Disease Preclinical and Discover Stage Products

12.

Gaucher’s Disease Therapeutic Assessment

13.

Gaucher’s Disease Inactive Products

14.

Gaucher’s Disease Company-University Collaborations (Licensing/Partnering) Analysis

15.

Gaucher’s Disease Key Companies

16.

Gaucher’s Disease Key Products

17.

Gaucher’s Disease- Unmet Needs

18.

Gaucher’s Disease- Market Drivers and Barriers

19.

Gaucher’s Disease- Future Perspectives and Conclusion

20.

Gaucher’s Disease Analyst Views

21.

Appendix

22.

About DelveInsight

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Key questions answered in the Gaucher’s Disease Pipeline Report

  • What are the treatment options for Gaucher’s Disease?

  • How many pharmaceutical companies are working on Gaucher’s Disease treatments?

  • Which of these companies’ pharmaceuticals is the most commonly used?

  • What is the total number of Gaucher’s Disease medications produced by each company?

  • How many drugs in the early, mid, or late stages of development for Gaucher’s Disease are there?

  • How many of the current therapies in development can be used alone or in combination with other treatments?

  • What are the most significant industry-industry and industry-academy collaborations, mergers and acquisitions, and licensing practices that have an impact on Gaucher’s Disease?

Related Reports

Gaucher Disease Type 1 Pipeline Insights

Get a comprehensive analysis of Gaucher Disease Type 1 pipeline therapies and key companies including Sanofi, Orphazyme, Prevail Therapeutics, among others.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance. 

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