Mucopolysaccharidosis III - Pipeline Review, H1 2017

  • ID: 4311850
  • Report
  • 61 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Abeona Therapeutics Inc
  • ArmaGen Inc
  • Lysogene SAS
  • MORE
Mucopolysaccharidosis III - Pipeline Review, H1 2017

Summary:

The latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis III - Pipeline Review, H1 2017, provides an overview of the Mucopolysaccharidosis III (Metabolic Disorders) pipeline landscape.

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to breakdown molecules called glycosaminoglycans (GAG). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT).

Report Highlights:

The latest Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis III - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (Metabolic 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 Mucopolysaccharidosis III (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 6, 1, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Mucopolysaccharidosis III (Metabolic 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 Mucopolysaccharidosis III (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis III (Metabolic 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 Mucopolysaccharidosis III (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Mucopolysaccharidosis III (Metabolic 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 Mucopolysaccharidosis III (Metabolic 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 Mucopolysaccharidosis III (Metabolic 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 Mucopolysaccharidosis III (Metabolic 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

  • Abeona Therapeutics Inc
  • ArmaGen Inc
  • Lysogene SAS
  • MORE
  1. Introduction
  2. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview
  3. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - 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. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment
  10. Assessment by Target
  11. Assessment by Mechanism of Action
  12. Assessment by Route of Administration
  13. Assessment by Molecule Type
  14. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development
  15. Abeona Therapeutics Inc
  16. Alexion Pharmaceuticals Inc
  17. ArmaGen Inc
  18. Axcentua Pharmaceuticals AB
  19. BioMarin Pharmaceutical Inc
  20. Laboratorios Del Dr Esteve SA
  21. Lysogene SAS
  22. Swedish Orphan Biovitrum AB
  23. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Drug Profiles
  24. ABO-101 - Drug Profile
  25. Product Description
  26. Mechanism Of Action
  27. R&D Progress
  28. ABO-102 - Drug Profile
  29. Product Description
  30. Mechanism Of Action
  31. R&D Progress
  32. AGT-184 - Drug Profile
  33. Product Description
  34. Mechanism Of Action
  35. R&D Progress
  36. AXP-10711 - Drug Profile
  37. Product Description
  38. Mechanism Of Action
  39. R&D Progress
  40. BMN-250 - Drug Profile
  41. Product Description
  42. Mechanism Of Action
  43. R&D Progress
  44. EGT-101 - Drug Profile
  45. Product Description
  46. Mechanism Of Action
  47. R&D Progress
  48. EGT-201 - Drug Profile
  49. Product Description
  50. Mechanism Of Action
  51. R&D Progress
  52. lesinidase alfa - Drug Profile
  53. Product Description
  54. Mechanism Of Action
  55. R&D Progress
  56. LYSSAF-301 - Drug Profile
  57. Product Description
  58. Mechanism Of Action
  59. R&D Progress
  60. LYSSAF-302 - Drug Profile
  61. Product Description
  62. Mechanism Of Action
  63. R&D Progress
  64. Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile
  65. Product Description
  66. Mechanism Of Action
  67. R&D Progress
  68. Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile
  69. Product Description
  70. Mechanism Of Action
  71. R&D Progress
  72. Small Molecule for Sanfilippo Syndrome - Drug Profile
  73. Product Description
  74. Mechanism Of Action
  75. R&D Progress
  76. SOBI-003 - Drug Profile
  77. Product Description
  78. Mechanism Of Action
  79. R&D Progress
  80. Stem Cell Therapy for Mucopolysaccharidosis Type IIIA - Drug Profile
  81. Product Description
  82. Mechanism Of Action
  83. R&D Progress
  84. Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile
  85. Product Description
  86. Mechanism Of Action
  87. R&D Progress
  88. Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile
  89. Product Description
  90. Mechanism Of Action
  91. R&D Progress
  92. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects
  93. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products
  94. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones
  95. Featured News & Press Releases
  96. Jan 19, 2017: Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B
  97. Oct 19, 2016: European Commission Grants SOBI003 Orphan Designation for the Treatment of MPS IIIA
  98. May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB)
  99. Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome
  100. Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome
  101. May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA
  102. Apr 02, 2014: Orphan designation granted for MPSIII
  103. Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease
  104. Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein
  105. Appendix
  106. Methodology
  107. Coverage
  108. Secondary Research
  109. Primary Research
  110. Expert Panel Validation
  111. Contact Us
  112. Disclaimer
List of Tables:
  1. Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), 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. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H1
  11. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Alexion Pharmaceuticals Inc, H1
  12. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H1
  13. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H1
  14. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc, H1
  15. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H1
  16. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H1
  17. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H1
  18. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H1
  19. Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H1
List of Figures:
  1. Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1
  2. Number of Products under Development by Companies, H1
  3. Number of Products by Targets, H1
  4. Number of Products by Stage and Targets, H1
  5. Number of Products by Mechanism of Actions, H1
  6. Number of Products by Stage and Mechanism of Actions, H1
  7. Number of Products by Routes of Administration, H1
  8. Number of Products by Stage and Routes of Administration, H1
  9. Number of Products by Molecule Types, H1
  10. Number of Products by Stage and Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Abeona Therapeutics Inc
  • Alexion Pharmaceuticals Inc
  • ArmaGen Inc
  • Axcentua Pharmaceuticals AB
  • BioMarin Pharmaceutical Inc
  • Laboratorios Del Dr Esteve SA
  • Lysogene SAS
  • Swedish Orphan Biovitrum AB
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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