Gaucher Disease - Pipeline Review, H1 2017

  • ID: 4311840
  • Report
  • 74 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Actelion Ltd
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Orphazyme ApS
  • Pharming Group NV
  • Shire Plc
  • MORE
Gaucher Disease - Pipeline Review, H1 2017

Summary:

The latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease - Pipeline Review, H1 2017, 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:

The latest Pharmaceutical and Healthcare latest pipeline guide Gaucher Disease - Pipeline Review, H1 2017, 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 II, Phase I, Preclinical and Discovery stages are 4, 2, 11 and 2 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 1 and 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 our 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.
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Note: Product cover images may vary from those shown
2 of 5

FEATURED COMPANIES

  • Actelion Ltd
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Orphazyme ApS
  • Pharming Group NV
  • Shire Plc
  • MORE
  1. Introduction
  2. Gaucher Disease - Overview
  3. Gaucher Disease - Therapeutics Development
  4. Pipeline Overview
  5. Pipeline by Companies
  6. Pipeline by Universities/Institutes
  7. Products under Development by Companies
  8. Products under Development by Universities/Institutes
  9. Gaucher Disease - Therapeutics Assessment
  10. Assessment by Target
  11. Assessment by Mechanism of Action
  12. Assessment by Route of Administration
  13. Assessment by Molecule Type
  14. Gaucher Disease - Companies Involved in Therapeutics Development
  15. Actelion Ltd
  16. Genzyme Corp
  17. greenovation Biotech GmbH
  18. JCR Pharmaceuticals Co Ltd
  19. Lixte Biotechnology Holdings Inc
  20. Neuraltus Pharmaceuticals Inc
  21. Orphazyme ApS
  22. Pharming Group NV
  23. Protalix BioTherapeutics Inc
  24. Shire Plc
  25. Gaucher Disease - Drug Profiles
  26. ADN-LYS - Drug Profile
  27. Product Description
  28. Mechanism Of Action
  29. R&D Progress
  30. ambroxol - Drug Profile
  31. Product Description
  32. Mechanism Of Action
  33. R&D Progress
  34. arimoclomol - Drug Profile
  35. Product Description
  36. Mechanism Of Action
  37. R&D Progress
  38. AVRRD-02 - Drug Profile
  39. Product Description
  40. Mechanism Of Action
  41. R&D Progress
  42. Gene Therapy for Gaucher Disease - Drug Profile
  43. Product Description
  44. Mechanism Of Action
  45. R&D Progress
  46. Gene Therapy for Gaucher Disease - Drug Profile
  47. Product Description
  48. Mechanism Of Action
  49. R&D Progress
  50. ibiglustat - Drug Profile
  51. Product Description
  52. Mechanism Of Action
  53. R&D Progress
  54. imiglucerase biosimilar - Drug Profile
  55. Product Description
  56. Mechanism Of Action
  57. R&D Progress
  58. LB-201 - Drug Profile
  59. Product Description
  60. Mechanism Of Action
  61. R&D Progress
  62. LB-205 - Drug Profile
  63. Product Description
  64. Mechanism Of Action
  65. R&D Progress
  66. LTI-291 - Drug Profile
  67. Product Description
  68. Mechanism Of Action
  69. R&D Progress
  70. miglustat - Drug Profile
  71. Product Description
  72. Mechanism Of Action
  73. R&D Progress
  74. MOSS-GBA - Drug Profile
  75. Product Description
  76. Mechanism Of Action
  77. R&D Progress
  78. NCGC-607 - Drug Profile
  79. Product Description
  80. Mechanism Of Action
  81. R&D Progress
  82. NP-003 - Drug Profile
  83. Product Description
  84. Mechanism Of Action
  85. R&D Progress
  86. Pcgin - Drug Profile
  87. Product Description
  88. Mechanism Of Action
  89. R&D Progress
  90. PRX-112 - Drug Profile
  91. Product Description
  92. Mechanism Of Action
  93. R&D Progress
  94. Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility - Drug Profile
  95. Product Description
  96. Mechanism Of Action
  97. R&D Progress
  98. Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease - Drug Profile
  99. Product Description
  100. Mechanism Of Action
  101. R&D Progress
  102. Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile
  103. Product Description
  104. Mechanism Of Action
  105. R&D Progress
  106. RND-001 - Drug Profile
  107. Product Description
  108. Mechanism Of Action
  109. R&D Progress
  110. Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis - Drug Profile
  111. Product Description
  112. Mechanism Of Action
  113. R&D Progress
  114. Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile
  115. Product Description
  116. Mechanism Of Action
  117. R&D Progress
  118. Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease - Drug Profile
  119. Product Description
  120. Mechanism Of Action
  121. R&D Progress
  122. taliglucerase alfa - Drug Profile
  123. Product Description
  124. Mechanism Of Action
  125. R&D Progress
  126. velaglucerase alfa - Drug Profile
  127. Product Description
  128. Mechanism Of Action
  129. R&D Progress
  130. Gaucher Disease - Dormant Projects
  131. Gaucher Disease - Discontinued Products
  132. Gaucher Disease - Product Development Milestones
  133. Featured News & Press Releases
  134. Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
  135. Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease
  136. Sep 02, 2014: Shire’s VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease
  137. Jul 04, 2014: Shire’s VPRIV approved in Japan for the improvement of symptoms of Gaucher disease
  138. Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease
  139. Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection)
  140. Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance
  141. Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium
  142. Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients
  143. Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112
  144. Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease
  145. Jun 28, 2012: Shire’s Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease
  146. Jun 28, 2012: Shire’s Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease
  147. Feb 22, 2012: Shire Announces European Approval Of Manufacturing Facility For VPRIV
  148. Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease
  149. Appendix
  150. Methodology
  151. Coverage
  152. Secondary Research
  153. Primary Research
  154. Expert Panel Validation
  155. Contact Us
  156. Disclaimer
List of Tables:
  1. Number of Products under Development for Gaucher Disease, H1
  2. Number of Products under Development by Companies, H1
  3. Number of Products under Development by Universities/Institutes, H1
  4. Products under Development by Companies, H1
  5. Products under Development by Universities/Institutes, H1
  6. Number of Products by Stage and Target, H1
  7. Number of Products by Stage and Mechanism of Action, H1
  8. Number of Products by Stage and Route of Administration, H1
  9. Number of Products by Stage and Molecule Type, H1
  10. Gaucher Disease - Pipeline by Actelion Ltd, H1
  11. Gaucher Disease - Pipeline by Genzyme Corp, H1
  12. Gaucher Disease - Pipeline by greenovation Biotech GmbH, H1
  13. Gaucher Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1
  14. Gaucher Disease - Pipeline by Lixte Biotechnology Holdings Inc, H1
  15. Gaucher Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H1
  16. Gaucher Disease - Pipeline by Orphazyme ApS, H1
  17. Gaucher Disease - Pipeline by Pharming Group NV, H1
  18. Gaucher Disease - Pipeline by Protalix BioTherapeutics Inc, H1
  19. Gaucher Disease - Pipeline by Shire Plc, H1
  20. Gaucher Disease - Dormant Projects, H1
  21. Gaucher Disease - Discontinued Products, H1
List of Figures:
  1. Number of Products under Development for Gaucher Disease, H1
  2. Number of Products under Development by Companies, H1
  3. Number of Products under Development by Universities/Institutes, H1
  4. Number of Products by Targets, H1
  5. Number of Products by Stage and Targets, H1
  6. Number of Products by Top 10 Mechanism of Actions, H1
  7. Number of Products by Stage and Top 10 Mechanism of Actions, H1
  8. Number of Products by Routes of Administration, H1
  9. Number of Products by Stage and Routes of Administration, H1
  10. Number of Products by Molecule Types, H1
  11. Number of Products by Stage and Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Actelion Ltd
  • Genzyme Corp
  • greenovation Biotech GmbH
  • JCR Pharmaceuticals Co Ltd
  • Lixte Biotechnology Holdings Inc
  • Neuraltus Pharmaceuticals Inc
  • Orphazyme ApS
  • Pharming Group NV
  • Protalix BioTherapeutics Inc
  • Shire Plc
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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