Home > > > > Report Detail

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

Publisher Name :
Date: 30-Nov-2022
No. of pages: 108
Inquire Before Buying

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.

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

Table of Contents
List of Tables
List of Figures
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
Gaucher Disease - Drug Profiles
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
Number of Products under Development for Gaucher Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Companies, 2022 (Contd..1)
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Companies, 2022 (Contd..2)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Gaucher Disease - Dormant Projects, 2022
Gaucher Disease - Dormant Projects, 2022 (Contd..1)
Gaucher Disease - Discontinued Products, 2022

List of Figures
Number of Products under Development for Gaucher Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products by Targets, 2022
Number of Products by Stage and Targets, 2022
Number of Products by Top 10 Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022
  • Global Histone Deacetylase (HDAC) Inhibitors Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 87
    The global Histone Deacetylase (HDAC) Inhibitors market was valued at US$ 615.2 million in 2023 and is anticipated to reach US$ 1223.2 million by 2030, witnessing a CAGR of 8.8% during The forecast period 2024-2030. North American market for Histone Deacetylase (HDAC) Inhibitors is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Histone Deacetylase (HDAC) Inhibitor......
  • Global Esomeprazole Magnesium Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 74
    The global Esomeprazole Magnesium market was valued at US$ 5287 million in 2023 and is anticipated to reach US$ 9451.5 million by 2030, witnessing a CAGR of 8.3% during The forecast period 2024-2030. North American market for Esomeprazole Magnesium is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Esomeprazole Magnesium is estimated to increase from $ million in 2......
  • Global Combination Therapy Drug Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 86
    The global Combination Therapy Drug market was valued at US$ 105 million in 2023 and is anticipated to reach US$ 166.7 million by 2030, witnessing a CAGR of 4.1% during The forecast period 2024-2030. The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during The next six years. The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, The chemical drug market is......
  • Global Axitinib Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 68
    The global Axitinib market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Axitinib is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Axitinib is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The......
  • Global Lyme Disease Vaccine Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 70
    The lyme disease vaccine can reduce lyme disease incidence, reduce symptoms and reduce mortality. Lyme disease is a tick-borne infection of spirochete, a natural disease caused by borrelia burgdorferi. The global Lyme Disease Vaccine market was valued at US$ 257 million in 2023 and is anticipated to reach US$ 763 million by 2030, witnessing a CAGR of 8.8% during The forecast period 2024-2030. The global pharmaceutical market is 1475 billion USD in 2022, growing at......
  • Global Transdermal Absorption Patch Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 85
    The global Transdermal Absorption Patch market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Transdermal Absorption Patch is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Transdermal Absorption Patch is estimated to increase from $ million i......
  • Global Ketoprofen Patches Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 78
    Ketoprofen patch, indications for The treatment of symptoms caused by The following diseases: rheumatoid arthritis and rheumatoid arthritis The global Ketoprofen Patches market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Ketoprofen Patches is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast per......
  • Global Methyl Salicylate Patches Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 82
    The global Methyl Salicylate Patches market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Methyl Salicylate Patches is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Methyl Salicylate Patches is estimated to increase from $ million in 2023 to......
  • Global Diclofenac Patches Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 94
    The global Diclofenac Patches market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Diclofenac Patches is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Diclofenac Patches is estimated to increase from $ million in 2023 to reach $ million by 2......
    • SERVICES
    Value for Money
    We believe in "optimum utilization of available budget and resources". While servicing our clients' (your) market research requirements, we keep the same approach in focus to help you get the best value for your $$s.
    Ever Growing Inventory
    Ranging from the smallest feasible / required data (datasheets, data facts, SWOT analysis, company profiles, etc) to full research reports that help you make decisions, our inventory is updated almost on a daily basis with the latest industry reports from domain experts that track more than 5000 niche sectors.
    One Stop Solution
    Need a custom research report on medical devices market? Require all available business intelligence on 3D printing industry? Exploring F&B sector of a particular country/region? RnRMarketResearch.com is your one-stop-solution to all market intelligence needs. We not only offer custom research and consulting services, we also "bundle" reports to meet your needs and help you fetch the data analysis you require for your business.
    Dedicated Client Engagement
    Not limited to only "finding" relevant reports for you, our client engagement team dedicates its efforts to understand your "business need" and accordingly maps available research data to help you move forward. Call "your" client engagement executive any time of your day and get your questions answered in order to make the correct business decision.
    Saving Time and Efforts
    Simply share your research requirement details with us and let us do all the hard work to find required intelligence for you. When you add up our "one stop solution" and "dedicated client engagement" services mentioned above, you obviously know the time and effort saving you do by working with us.
    Payment Flexibility
    Working with Fortune 500 organizations, we understand the importance of being flexible for payments. Share your payment terms with us and we will surely match up to them to ensure you get access to required business intelligence data without having to wait for the payment to be done.
    Post-Purchase Research Support
    Have questions after reading a report / datasheet bought through us? Not sure about the methodology used for data available in the research? Talk to us / Share your questions with us and if required, we will connect you with the analyst(s)/author(s) of the report(s) and ensure you get satisfactory answers for the same. Need more data / analysis / report(s) on the topic of your research/project? The RnRMarketResearch.com team is here for you 24X7 to support you with your post-purchase requirements. Subscription Offers & Packages (Get in touch with us for more details - [email protected] / +1 888 391 5441 )
    • Ad Hoc
    • Pay - as - you - go / Bucket Subscriptions
    • Fixed Cost for #of reports
    • Customize / Personalize as per your needs