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Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2019

  • ID: 4901360
  • Drug Pipelines
  • December 2019
  • Region: Global
  • 101 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • E-scape Bio Inc
  • IntraBio Ltd
  • Lauren Sciences LLC
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • MORE
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2019

Summary

This latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2019, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) pipeline landscape.

Niemann-Pick C disease is one of a group of lysosomal storage diseases that affect metabolism and that are caused by genetic mutations. It involves the accumulation of sphingolipids in cells throughout the body, particularly reticuloendothelial cells (the mononuclear phagocyte system). Symptoms include enlarged liver, brain damage, difficulty walking and swallowing, increased sensitivity to touch, difficulty speaking, loss of muscle tone, learning difficulties.

Report Highlights

This Pharmaceutical and Healthcare latest pipeline guide Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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, Discovery and Unknown stages are 3, 1, 2, 9, 2 and 1 respectively. Similarly, the Universities portfolio in Phase 0, Preclinical and Discovery stages comprises 1, 2 and 2 molecules, respectively.

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (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 the publisher'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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (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.
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • E-scape Bio Inc
  • IntraBio Ltd
  • Lauren Sciences LLC
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • MORE
Introduction
Report Coverage
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development
Amicus Therapeutics Inc
Cyclo Therapeutics Inc
E-scape Bio Inc
Evox Therapeutics Ltd
IntraBio Ltd
Jewell Laboratories LLC
Lauren Sciences LLC
Mallinckrodt Plc
Merck & Co Inc
Neurotrope Bioscience Inc
Okklo Life Sciences BV
Oraxion Therapeutics Inc
Orphazyme A/S
Perlara PBC
Ranedis Pharmaceuticals LLC
SOM Biotech SL
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drug Profiles
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Discontinued Products
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones
Appendix

List of Tables
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2019
Number of Products under Development by Companies, H2 2019
Number of Products under Development by Universities/Institutes, H2 2019
Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Products under Development by Universities/Institutes, H2 2019
Number of Products by Stage and Target, H2 2019
Number of Products by Stage and Mechanism of Action, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Amicus Therapeutics Inc, H2 2019
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H2 2019

List of Figures
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2019
Number of Products under Development by Companies, H2 2019
Number of Products under Development by Universities/Institutes, H2 2019
Number of Products by Targets, H2 2019
Number of Products by Stage and Targets, H2 2019
Number of Products by Mechanism of Actions, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Molecule Types, H2 2019
Number of Products by Stage and Molecule Types, H2 2019
Note: Product cover images may vary from those shown
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  • Amicus Therapeutics Inc
  • Cyclo Therapeutics Inc
  • E-scape Bio Inc
  • Evox Therapeutics Ltd
  • IntraBio Ltd
  • Jewell Laboratories LLC
  • Lauren Sciences LLC
  • Mallinckrodt Plc
  • Merck & Co Inc
  • Neurotrope Bioscience Inc
  • Okklo Life Sciences BV
  • Oraxion Therapeutics Inc
  • Orphazyme A/S
  • Perlara PBC
  • Ranedis Pharmaceuticals LLC
  • SOM Biotech SL
Note: Product cover images may vary from those shown
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