Gaucher Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2021 Update

Pages: 143 Published: November 30, 2021 Report Code: GMDGDHC13172IDB

Gaucher disease is a rare genetic disorder that occurs due to a lack of an enzyme called glucocerebrosidase. Symptoms include cognitive impairment, enlarged spleen, severe swelling, lung disease, fatigue, and seizures. Treatment includes enzyme replacement therapy.

The Gaucher disease drugs in development market research report provides comprehensive information on the therapeutics under development for Gaucher disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Gaucher disease and features dormant and discontinued projects.

What are the key targets in the Gaucher disease pipeline products market?

The key targets in the Gaucher disease pipeline products market are glucosylceramidase, ceramide glucosyltransferase, glucosylceramide, acid ceramidase, beta galactosidase, glycosidase, and heat shock protein 70.

Gaucher Disease Pipeline Products Analysis Market by Targets

Gaucher Disease Pipeline Products Analysis Market by Targets

For more target insights, download a free report sample

What are the key mechanisms of action in the Gaucher disease pipeline products market?

The key mechanisms of action in the Gaucher disease pipeline products market are glucosylceramidase activator, glucosylceramidase replacement, ceramide glucosyltransferase inhibitor, glucosylceramidase inhibitor, acid ceramidase inhibitor, beta galactosidase activator, and heat shock protein 70 activator.

Gaucher Disease Pipeline Products Market Analysis by Mechanism of Actions

Gaucher Disease Pipeline Products Market Analysis by Mechanism of Actions

For more mechanism of actions insights, download a free report sample

What are the key routes of administration in the Gaucher disease pipeline products market?

The key routes of administration in the Gaucher disease pipeline products market are oral, intravenous, and intracisternal.

Gaucher Disease Pipeline Products Market Analysis by Routes of Administration

Gaucher Disease Pipeline Products Market Analysis by Routes of Administration

For more routes of administration insights, download a free report sample

What are the different molecule types in the Gaucher disease pipeline products market?

The different molecule types in the Gaucher disease pipeline products market are small molecule, gene therapy, recombinant enzyme, gene-modified cell therapy, protein, biologic, fusion protein, and oligonucleotide.

Gaucher Disease Pipeline Products Market Analysis by Molecule Type

Gaucher Disease Pipeline Products Market Analysis by Molecule TypeTo know more about molecule types, download a free report sample

Which are the major companies in the Gaucher disease pipeline products market?

Some of the major companies in the Gaucher disease pipeline products market are M6P Therapeutics, AVROBIO Inc, Eli Lilly and Co, Freeline Therapeutics Holdings Plc, ILIAS Biologics Inc, ISU ABXIS Co Ltd, Takeda Pharmaceutical Co Ltd, Yuhan Corp, Adienne Pharma & Biotech SA, and Apollo Therapeutics LLC

Gaucher disease Pipeline Products Market Analysis by Companies

Gaucher disease Pipeline Products Market Analysis by Companies

To know more about major companies, download a free report sample

Market Report Scope

Key targets Glucosylceramidase, Ceramide Glucosyltransferase, Glucosylceramide, Acid Ceramidase, Beta Galactosidase, Glycosidase, and Heat Shock Protein 70
Key mechanism of action Glucosylceramidase Activator, Glucosylceramidase Replacement, Ceramide Glucosyltransferase Inhibitor, Glucosylceramidase Inhibitor, Acid Ceramidase Inhibitor, Beta Galactosidase Activator, and Heat Shock Protein 70 Activator
Key routes of administration Oral, Intravenous, and Intracisternal
Molecule type Small Molecule, Gene Therapy, Recombinant Enzyme, Gene-Modified Cell Therapy, Protein, Biologic, Fusion Protein, and Oligonucleotide
Major companies M6P Therapeutics, AVROBIO Inc, Eli Lilly and Co, Freeline Therapeutics Holdings Plc, ILIAS Biologics Inc, ISU ABXIS Co Ltd, Takeda Pharmaceutical Co Ltd, Yuhan Corp, Adienne Pharma & Biotech SA, and Apollo Therapeutics LLC

Scope

  • The report provides a snapshot of the global therapeutic landscape of Gaucher disease
  • The report reviews pipeline therapeutics for Gaucher disease by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in Gaucher disease therapeutics and enlists all their major and minor projects
  • The report assesses Gaucher disease therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA), and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews the latest news related to pipeline therapeutics for Gaucher disease

Reasons to Buy

  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with a potentially strong product portfolio and create effective counter-strategies to gain a competitive advantage
  • Identify and understand important and diverse types of therapeutics under development for Gaucher disease
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and their most promising pipeline therapeutics
  • Devise corrective measures for pipeline projects by understanding Gaucher disease pipeline depth and focus on Indication therapeutics
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from the pipeline

Key Players

  • Adienne Pharma & Biotech SA

    Apollo Therapeutics LLC

    AVROBIO Inc

    Belrose Pharma Inc

    Bial – Portela & Ca SA

    Bioasis Technologies Inc

    Biosidus SA

    Blue Turtle Bio Technologies Inc

    CANbridge Life Sciences Ltd

    Centogene NV

    Coave Therapeutics

    Denali Therapeutics Inc

    Eli Lilly and Co

    Erad Therapeutics Inc

    Evox Therapeutics Ltd

    Freeline Therapeutics Holdings Plc

    Gain Therapeutics Inc

    Generation Bio Co

    Genzyme Corp

    Graphite Bio Inc

    ILIAS Biologics Inc

    ISU ABXIS Co Ltd

    Johnson & Johnson

    Kashiv BioSciences LLC

    M6P Therapeutics

    Orphazyme A/S

    Recursion Pharmaceuticals Inc

    Sanofi

    SmartPharm Therapeutics Inc

    Takeda Pharmaceutical Co Ltd

    Vanqua Bio Inc

    Vera Therapeutics Inc

    Voyager Therapeutics Inc

    Yuhan Corp

Table of Contents

Table of Contents

Introduction

Global Markets Direct Report Coverage

Gaucher Disease – Overview

Gaucher Disease – Therapeutics Development

Gaucher Disease – Therapeutics Assessment

Gaucher Disease – Companies Involved in Therapeutics Development

Gaucher Disease – Drug Profiles

Gaucher Disease – Dormant Projects

Gaucher Disease – Discontinued Products

Gaucher Disease – Product Development Milestones

Appendix

List of Tables

List of Tables

Number of Products under Development for Gaucher Disease, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Companies, 2021 (Contd..1)

Number of Products under Development by Universities/Institutes, 2021

Products under Development by Companies, 2021

Products under Development by Companies, 2021 (Contd..1)

Products under Development by Companies, 2021 (Contd..2)

Products under Development by Universities/Institutes, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Gaucher Disease – Pipeline by Adienne Pharma & Biotech SA, 2021

Gaucher Disease – Pipeline by Apollo Therapeutics LLC, 2021

Gaucher Disease – Pipeline by AVROBIO Inc, 2021

Gaucher Disease – Pipeline by Belrose Pharma Inc, 2021

Gaucher Disease – Pipeline by Bial – Portela & Ca SA, 2021

Gaucher Disease – Pipeline by Bioasis Technologies Inc, 2021

Gaucher Disease – Pipeline by Biosidus SA, 2021

Gaucher Disease – Pipeline by Blue Turtle Bio Technologies Inc, 2021

Gaucher Disease – Pipeline by CANbridge Life Sciences Ltd, 2021

Gaucher Disease – Pipeline by Centogene NV, 2021

Gaucher Disease – Pipeline by Coave Therapeutics, 2021

Gaucher Disease – Pipeline by Denali Therapeutics Inc, 2021

Gaucher Disease – Pipeline by Eli Lilly and Co, 2021

Gaucher Disease – Pipeline by Erad Therapeutics Inc, 2021

Gaucher Disease – Pipeline by Evox Therapeutics Ltd, 2021

Gaucher Disease – Pipeline by Freeline Therapeutics Holdings Plc, 2021

Gaucher Disease – Pipeline by Gain Therapeutics Inc, 2021

Gaucher Disease – Pipeline by Generation Bio Co, 2021

Gaucher Disease – Pipeline by Genzyme Corp, 2021

Gaucher Disease – Pipeline by Graphite Bio Inc, 2021

Gaucher Disease – Pipeline by ILIAS Biologics Inc, 2021

Gaucher Disease – Pipeline by ISU ABXIS Co Ltd, 2021

Gaucher Disease – Pipeline by Johnson & Johnson, 2021

Gaucher Disease – Pipeline by Kashiv BioSciences LLC, 2021

Gaucher Disease – Pipeline by M6P Therapeutics, 2021

Gaucher Disease – Pipeline by Orphazyme A/S, 2021

Gaucher Disease – Pipeline by Recursion Pharmaceuticals Inc, 2021

Gaucher Disease – Pipeline by Sanofi, 2021

Gaucher Disease – Pipeline by SmartPharm Therapeutics Inc, 2021

Gaucher Disease – Pipeline by Takeda Pharmaceutical Co Ltd, 2021

Gaucher Disease – Pipeline by Vanqua Bio Inc, 2021

Gaucher Disease – Pipeline by Vera Therapeutics Inc, 2021

Gaucher Disease – Pipeline by Voyager Therapeutics Inc, 2021

Gaucher Disease – Pipeline by Yuhan Corp, 2021

Gaucher Disease – Dormant Projects, 2021

Gaucher Disease – Dormant Projects, 2021 (Contd..1)

Gaucher Disease – Discontinued Products, 2021

List of Figures

List of Figures

Number of Products under Development for Gaucher Disease, 2021

Number of Products under Development by Companies, 2021

Number of Products by Targets, 2021

Number of Products by Stage and Targets, 2021

Number of Products by Mechanism of Actions, 2021

Number of Products by Stage and Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

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