Mucopolysaccharidosis I - Pipeline Review, H1 2017

  • ID: 4311833
  • Report
  • 73 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • AngioChem Inc
  • ArmaGen Inc
  • Bioasis Technologies Inc
  • CRISPR Therapeutics
  • Eloxx Pharmaceuticals Ltd
  • Immusoft Corp
  • MORE
Mucopolysaccharidosis I - Pipeline Review, H1 2017

Summary:

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

MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.

Report Highlights:

The latest Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (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 I (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler 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, Phase I, Preclinical and Discovery stages are 5, 1, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.

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

  • AngioChem Inc
  • ArmaGen Inc
  • Bioasis Technologies Inc
  • CRISPR Therapeutics
  • Eloxx Pharmaceuticals Ltd
  • Immusoft Corp
  • MORE
  1. Introduction
  2. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
  3. Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
  15. AngioChem Inc
  16. ArmaGen Inc
  17. Bioasis Technologies Inc
  18. CRISPR Therapeutics
  19. Eloxx Pharmaceuticals Ltd
  20. Immusoft Corp
  21. Inventiva
  22. OPKO Health Inc
  23. PTC Therapeutics Inc
  24. RegenxBio Inc
  25. Sangamo Therapeutics Inc
  26. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
  27. AGT-181 - Drug Profile
  28. Product Description
  29. Mechanism Of Action
  30. R&D Progress
  31. ataluren - Drug Profile
  32. Product Description
  33. Mechanism Of Action
  34. R&D Progress
  35. Cell Therapy for Mucopolysaccharidosis I - Drug Profile
  36. Product Description
  37. Mechanism Of Action
  38. R&D Progress
  39. Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile
  40. Product Description
  41. Mechanism Of Action
  42. R&D Progress
  43. ELX-02 - Drug Profile
  44. Product Description
  45. Mechanism Of Action
  46. R&D Progress
  47. JOT-102 - Drug Profile
  48. Product Description
  49. Mechanism Of Action
  50. R&D Progress
  51. MGTA-456 - Drug Profile
  52. Product Description
  53. Mechanism Of Action
  54. R&D Progress
  55. odiparcil - Drug Profile
  56. Product Description
  57. Mechanism Of Action
  58. R&D Progress
  59. Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
  60. Product Description
  61. Mechanism Of Action
  62. R&D Progress
  63. pentosan polysulfate sodium - Drug Profile
  64. Product Description
  65. Mechanism Of Action
  66. R&D Progress
  67. Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile
  68. Product Description
  69. Mechanism Of Action
  70. R&D Progress
  71. Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
  72. Product Description
  73. Mechanism Of Action
  74. R&D Progress
  75. Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
  76. Product Description
  77. Mechanism Of Action
  78. R&D Progress
  79. RGX-111 - Drug Profile
  80. Product Description
  81. Mechanism Of Action
  82. R&D Progress
  83. SB-318 - Drug Profile
  84. Product Description
  85. Mechanism Of Action
  86. R&D Progress
  87. Stem Cell Therapy for Hurler Sydrome - Drug Profile
  88. Product Description
  89. Mechanism Of Action
  90. R&D Progress
  91. Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
  92. Product Description
  93. Mechanism Of Action
  94. R&D Progress
  95. X-372 - Drug Profile
  96. Product Description
  97. Mechanism Of Action
  98. R&D Progress
  99. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
  100. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
  101. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
  102. Featured News & Press Releases
  103. Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
  104. Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium
  105. Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1)
  106. Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
  107. Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
  108. Mar 31, 2016: ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for the Treatment of Hurler Syndrome
  109. Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I)
  110. Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome
  111. Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
  112. Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome
  113. Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome
  114. Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I
  115. Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome
  116. Appendix
  117. Methodology
  118. Coverage
  119. Secondary Research
  120. Primary Research
  121. Expert Panel Validation
  122. Contact Us
  123. Disclaimer
List of Tables:
  1. Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H1
  11. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H1
  12. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc, H1
  13. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H1
  14. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Ltd, H1
  15. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H1
  16. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H1
  17. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H1
  18. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H1
  19. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H1
  20. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1
  21. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1
  22. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1
List of Figures:
  1. Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), 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 Mechanism of Actions, H1
  7. Number of Products by Stage and 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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  • AngioChem Inc
  • ArmaGen Inc
  • Bioasis Technologies Inc
  • CRISPR Therapeutics
  • Eloxx Pharmaceuticals Ltd
  • Immusoft Corp
  • Inventiva
  • OPKO Health Inc
  • PTC Therapeutics Inc
  • RegenxBio Inc
  • Sangamo Therapeutics Inc
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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