Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2017

  • ID: 4311862
  • Report
  • 40 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Lucane Pharma SA
  • PhaseRx Inc
  • Promethera Biosciences SA
  • RaNA Therapeutics Inc
  • Selecta Biosciences Inc
  • Unicyte AG
  • MORE
Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2017

Summary:

The latest Pharmaceutical and Healthcare disease pipeline guide Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2017, provides an overview of the Ornithine-Transcarbamylase Deficiency (Genetic Disorders) pipeline landscape.

Ornithine transcarbamylase (OTC) deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme ornithine transcarbamylase (OTC). OTC plays an important role in the break down and removal of nitrogen the body (urea cycle). The lack of the OTC enzyme results in excessive accumulation of nitrogen, in the form of ammonia (hyperammonemia), in the blood. Symptoms include vomiting, refusal to eat, progressive lethargy, and coma. Treatment includes nitrogen scavenging agents.

Report Highlights:

The latest Pharmaceutical and Healthcare latest pipeline guide Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Ornithine-Transcarbamylase Deficiency (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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Ornithine-Transcarbamylase Deficiency and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II and Preclinical stages are 1, 1, 1 and 5 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Ornithine-Transcarbamylase Deficiency (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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Ornithine-Transcarbamylase Deficiency (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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Ornithine-Transcarbamylase Deficiency (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 Ornithine-Transcarbamylase Deficiency (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 Ornithine-Transcarbamylase Deficiency (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 Ornithine-Transcarbamylase Deficiency (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

  • Lucane Pharma SA
  • PhaseRx Inc
  • Promethera Biosciences SA
  • RaNA Therapeutics Inc
  • Selecta Biosciences Inc
  • Unicyte AG
  • MORE
  1. Introduction
  2. Ornithine-Transcarbamylase Deficiency - Overview
  3. Ornithine-Transcarbamylase Deficiency - 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. Ornithine-Transcarbamylase Deficiency - Therapeutics Assessment
  10. Assessment by Target
  11. Assessment by Mechanism of Action
  12. Assessment by Route of Administration
  13. Assessment by Molecule Type
  14. Ornithine-Transcarbamylase Deficiency - Companies Involved in Therapeutics Development
  15. Dimension Therapeutics Inc
  16. Lucane Pharma SA
  17. PhaseRx Inc
  18. Promethera Biosciences SA
  19. RaNA Therapeutics Inc
  20. Selecta Biosciences Inc
  21. Unicyte AG
  22. Ornithine-Transcarbamylase Deficiency - Drug Profiles
  23. DTX-301 - Drug Profile
  24. Product Description
  25. Mechanism Of Action
  26. R&D Progress
  27. Gene Therapy to Activate Ornithine Transcarbamylase for Ornithine Transcarbamylase Deficiency - Drug Profile
  28. Product Description
  29. Mechanism Of Action
  30. R&D Progress
  31. Gene Therapy to Activate Ornithine Transcarbamylase for Ornithine Transcarbamylase Deficiency - Drug Profile
  32. Product Description
  33. Mechanism Of Action
  34. R&D Progress
  35. Heparesc - Drug Profile
  36. Product Description
  37. Mechanism Of Action
  38. R&D Progress
  39. HepaStem - Drug Profile
  40. Product Description
  41. Mechanism Of Action
  42. R&D Progress
  43. PRX-OTC - Drug Profile
  44. Product Description
  45. Mechanism Of Action
  46. R&D Progress
  47. SHP-641 - Drug Profile
  48. Product Description
  49. Mechanism Of Action
  50. R&D Progress
  51. sodium benzoate - Drug Profile
  52. Product Description
  53. Mechanism Of Action
  54. R&D Progress
  55. Stem Cell Therapy for Type 1 Diabetes, Urea Cycle Disorders and Acute Liver Failure - Drug Profile
  56. Product Description
  57. Mechanism Of Action
  58. R&D Progress
  59. Ornithine-Transcarbamylase Deficiency - Dormant Projects
  60. Ornithine-Transcarbamylase Deficiency - Product Development Milestones
  61. Featured News & Press Releases
  62. May 03, 2017: PhaseRx to Present Data at the American Society of Gene & Cell Therapy 20th Annual Meeting
  63. Apr 25, 2017: PhaseRx Receives Positive Opinion for Orphan Drug Designation for PRX-OTC from European Medicines Agency
  64. Feb 08, 2017: PhaseRx to Present Data for PRX-OTC at the 13th Annual WORLDSymposium
  65. Nov 28, 2016: PhaseRx Receives Orphan Drug Designation from FDA for PRX-OTC for the Treatment of Ornithine Transcarbamylase Deficiency
  66. Appendix
  67. Methodology
  68. Coverage
  69. Secondary Research
  70. Primary Research
  71. Expert Panel Validation
  72. Contact Us
  73. Disclaimer
List of Tables:
  1. Number of Products under Development for Ornithine-Transcarbamylase Deficiency, 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. Ornithine-Transcarbamylase Deficiency - Pipeline by Dimension Therapeutics Inc, H1
  11. Ornithine-Transcarbamylase Deficiency - Pipeline by Lucane Pharma SA, H1
  12. Ornithine-Transcarbamylase Deficiency - Pipeline by PhaseRx Inc, H1
  13. Ornithine-Transcarbamylase Deficiency - Pipeline by Promethera Biosciences SA, H1
  14. Ornithine-Transcarbamylase Deficiency - Pipeline by RaNA Therapeutics Inc, H1
  15. Ornithine-Transcarbamylase Deficiency - Pipeline by Selecta Biosciences Inc, H1
  16. Ornithine-Transcarbamylase Deficiency - Pipeline by Unicyte AG, H1
  17. Ornithine-Transcarbamylase Deficiency - Dormant Projects, H1
List of Figures:
  1. Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H1
  2. Number of Products under Development by Companies, H1
  3. Number of Products by Stage and Targets, H1
  4. Number of Products by Mechanism of Actions, H1
  5. Number of Products by Stage and Mechanism of Actions, H1
  6. Number of Products by Stage and Routes of Administration, H1
  7. Number of Products by Molecule Types, H1
  8. Number of Products by Stage and Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Dimension Therapeutics Inc
  • Lucane Pharma SA
  • PhaseRx Inc
  • Promethera Biosciences SA
  • RaNA Therapeutics Inc
  • Selecta Biosciences Inc
  • Unicyte AG
Note: Product cover images may vary from those shown
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Note: Product cover images may vary from those shown
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