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Gaucher Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Publisher Global Markets Direct
Published Nov 30, 2022
Length 105 Pages
SKU # GMD17776509

Description

Gaucher Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease - Drugs In Development, 2022, provides an overview of the Gaucher Disease (Genetic Disorders) pipeline landscape.

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

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Gaucher Disease - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Gaucher Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Gaucher Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Gaucher Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 8, 1, 23 and 9 respectively. Similarly, the Universities portfolio in Discovery stages comprises 5 molecules, respectively.

Gaucher Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note:Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope
  • The pipeline guide provides a snapshot of the global therapeutic landscape of Gaucher Disease (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Gaucher Disease (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Gaucher Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Gaucher Disease (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Gaucher Disease (Genetic Disorders)
Reasons to Buy
  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Gaucher Disease (Genetic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Gaucher Disease (Genetic Disorders) pipeline depth and focus of 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

105 Pages
Introduction
Global Markets Direct Report Coverage
Gaucher Disease – Overview
Gaucher Disease – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Gaucher Disease – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Gaucher Disease – Companies Involved in Therapeutics Development
AceLink Therapeutics Inc
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
Graphite Bio Inc
ILIAS Biologics Inc
Immorna Hangzhou Biotechnology Co Ltd
ISU ABXIS Co Ltd
Jasper Therapeutics Inc
Johnson & Johnson
Kashiv BioSciences LLC
Lysogene SAS
M6P Therapeutics
Orphazyme A/S
Recursion Pharmaceuticals Inc
Sanofi
SmartPharm Therapeutics Inc
Takeda Pharmaceutical Co Ltd
Vanqua Bio Inc
Voyager Therapeutics Inc
Yuhan Corp
Gaucher Disease – Drug Profiles
AAV-Gcase D15 – Drug Profile
ADN-LYS – Drug Profile
AL-00804 – Drug Profile
ambroxol – Drug Profile
arimoclomol citrate – Drug Profile
AVRRD-02 – Drug Profile
Biologic to Activate Glucocerebrosidase for Genetic Disorders – Drug Profile
CAN-103 – Drug Profile
CTx-GBA1 – Drug Profile
DOS-000011 – Drug Profile
eliglustat tartrate – Drug Profile
ETV:Gcase – Drug Profile
FLT-201 – Drug Profile
Gaucher Disease – Drug Profile
GBA1 Mutations – Drug Profile
Gene Therapy for Gaucher Disease – Drug Profile
Gene Therapy for Gaucher Disease Type I – Drug Profile
Gene Therapy to Activate Glucosylceramidase for Gaucher Disease and Parkinson's Disease – Drug Profile
GPH-301 – Drug Profile
GT-02287 – Drug Profile
GT-02329 – Drug Profile
imiglucerase biosimilar – Drug Profile
JCXH-301 – Drug Profile
JSP-191 – Drug Profile
M-011 – Drug Profile
M-012 – Drug Profile
M-013 – Drug Profile
miglustat – Drug Profile
NCGC-607 – Drug Profile
PR-001 – Drug Profile
Proteins for Gaucher Disease Type I and III – Drug Profile
Proteins for Gaucher's Disease – Drug Profile
Proteins for Gaucher's Disease Type II – Drug Profile
Recombinant Glucosylceramidase Replacement for Type I and Type III Gaucher's Disease – Drug Profile
Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis – Drug Profile
Small Molecule to Inhibit Acid Ceramidase for Gaucher's and Krabbe's Diseases – Drug Profile
Small Molecules for Gaucher Disease – Drug Profile
Small Molecules for Gaucher Disease, Lewy Body Dementia and Parkinson's Disease – Drug Profile
Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease – Drug Profile
velaglucerase alfa – Drug Profile
venglustat malate – Drug Profile
xB-3007 – Drug Profile
YH-36425 – Drug Profile
YHC-1116 – Drug Profile
YHNCE-10 – Drug Profile
Gaucher Disease – Dormant Projects
Gaucher Disease – Discontinued Products
Gaucher Disease – Product Development Milestones
Featured News & Press Releases
Dec 07, 2022: AVROBIO to share comprehensive Gaucher disease program update
Oct 27, 2022: AVROBIO receives rare pediatric disease designation from the U.S. FDA for first-in-class gene therapy for Gaucher Disease
Oct 14, 2022: M6P Therapeutics presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress
May 11, 2022: Gain Therapeutics presents positive preclinical data on its Gaucher Disease program at IWGGD Symposium
Feb 10, 2022: M6P Therapeutics presents promising preclinical data in Gaucher disease at the 18th Annual WORLDSymposium 2022
Feb 08, 2022: Gain Therapeutics presents preclinical data on its structurally targeted allosteric regulators in GBA1 Gaucher Disease at the WORLDSymposium 2022
Feb 08, 2022: Freeline presents on its Gaucher disease AAV-based gene therapies at the 18th Annual WORLDSymposium
Jan 27, 2022: Freeline to present on its gaucher disease AAV-based gene therapies at the 18th Annual WORLDSymposium
Jan 06, 2022: Freeline announces FDA clearance of investigational New Drug Application for FLT201 for Gaucher Disease Type 1
Nov 08, 2021: AVROBIO to present clinical data on AVRRD-02 at the 14th ICIEM Conference
Oct 19, 2021: AVROBIO reports new interim safety data across investigational gene therapies for Gaucher disease type 1
Sep 09, 2021: Freeline announces Orphan Drug Designations for FLT201 for the treatment of Gaucher Disease
Feb 08, 2021: Freeline presents data on its Gaucher disease and Fabry disease AAV-based gene therapies at the 17th Annual WORLDSymposium
Feb 01, 2021: AVROBIO announces clinical data presentation on AVRRD-02 at 17th Annual WORLDSymposium 2021
Nov 18, 2020: Prevail Therapeutics granted composition of matter patent for experimental gene therapy program PR001
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Gaucher Disease, 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Number of Products under Development by Companies, 2022 (Contd..1)
Table 4: Number of Products under Development by Universities/Institutes, 2022
Table 5: Products under Development by Companies, 2022
Table 6: Products under Development by Companies, 2022 (Contd..1)
Table 7: Products under Development by Companies, 2022 (Contd..2)
Table 8: Products under Development by Universities/Institutes, 2022
Table 9: Number of Products by Stage and Target, 2022
Table 10: Number of Products by Stage and Mechanism of Action, 2022
Table 11: Number of Products by Stage and Route of Administration, 2022
Table 12: Number of Products by Stage and Molecule Type, 2022
Table 13: Gaucher Disease – Pipeline by AceLink Therapeutics Inc, 2022
Table 14: Gaucher Disease – Pipeline by Adienne Pharma & Biotech SA, 2022
Table 15: Gaucher Disease – Pipeline by Apollo Therapeutics LLC, 2022
Table 16: Gaucher Disease – Pipeline by AVROBIO Inc, 2022
Table 17: Gaucher Disease – Pipeline by Belrose Pharma Inc, 2022
Table 18: Gaucher Disease – Pipeline by Bial - Portela & Ca SA, 2022
Table 19: Gaucher Disease – Pipeline by Bioasis Technologies Inc, 2022
Table 20: Gaucher Disease – Pipeline by Biosidus SA, 2022
Table 21: Gaucher Disease – Pipeline by Blue Turtle Bio Technologies Inc, 2022
Table 22: Gaucher Disease – Pipeline by CANbridge Life Sciences Ltd, 2022
Table 23: Gaucher Disease – Pipeline by Centogene NV, 2022
Table 24: Gaucher Disease – Pipeline by Coave Therapeutics, 2022
Table 25: Gaucher Disease – Pipeline by Denali Therapeutics Inc, 2022
Table 26: Gaucher Disease – Pipeline by Eli Lilly and Co, 2022
Table 27: Gaucher Disease – Pipeline by Erad Therapeutics Inc, 2022
Table 28: Gaucher Disease – Pipeline by Evox Therapeutics Ltd, 2022
Table 29: Gaucher Disease – Pipeline by Freeline Therapeutics Holdings Plc, 2022
Table 30: Gaucher Disease – Pipeline by Gain Therapeutics Inc, 2022
Table 31: Gaucher Disease – Pipeline by Generation Bio Co, 2022
Table 32: Gaucher Disease – Pipeline by Graphite Bio Inc, 2022
Table 33: Gaucher Disease – Pipeline by ILIAS Biologics Inc, 2022
Table 34: Gaucher Disease – Pipeline by Immorna Hangzhou Biotechnology Co Ltd, 2022
Table 35: Gaucher Disease – Pipeline by ISU ABXIS Co Ltd, 2022
Table 36: Gaucher Disease – Pipeline by Jasper Therapeutics Inc, 2022
Table 37: Gaucher Disease – Pipeline by Johnson & Johnson, 2022
Table 38: Gaucher Disease – Pipeline by Kashiv BioSciences LLC, 2022
Table 39: Gaucher Disease – Pipeline by Lysogene SAS, 2022
Table 40: Gaucher Disease – Pipeline by M6P Therapeutics, 2022
Table 41: Gaucher Disease – Pipeline by Orphazyme A/S, 2022
Table 42: Gaucher Disease – Pipeline by Recursion Pharmaceuticals Inc, 2022
Table 43: Gaucher Disease – Pipeline by Sanofi, 2022
Table 44: Gaucher Disease – Pipeline by SmartPharm Therapeutics Inc, 2022
Table 45: Gaucher Disease – Pipeline by Takeda Pharmaceutical Co Ltd, 2022
Table 46: Gaucher Disease – Pipeline by Vanqua Bio Inc, 2022
Table 47: Gaucher Disease – Pipeline by Voyager Therapeutics Inc, 2022
Table 48: Gaucher Disease – Pipeline by Yuhan Corp, 2022
Table 49: Gaucher Disease – Dormant Projects, 2022
Table 50: Gaucher Disease – Dormant Projects, 2022 (Contd..1)
Table 51: Gaucher Disease – Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development for Gaucher Disease, 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products by Targets, 2022
Figure 4: Number of Products by Stage and Targets, 2022
Figure 5: Number of Products by Top 10 Mechanism of Actions, 2022
Figure 6: Number of Products by Stage and Mechanism of Actions, 2022
Figure 7: Number of Products by Routes of Administration, 2022
Figure 8: Number of Products by Stage and Routes of Administration, 2022
Figure 9: Number of Products by Molecule Types, 2022
Figure 10: Number of Products by Stage and Molecule Types, 2022

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