Sickle Cell Disease Drug Development Pipeline Review, 2017

  • ID: 4434631
  • Report
  • 77 pages
  • GBI Research
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FEATURED COMPANIES

  • Acceleron Pharma
  • Epidestiny
  • Errant Gene Therapeutics
  • Gamida Cell
  • Global Blood Therapeutics
  • Novartis
  • MORE

Sickle cell anemia is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Signs and symptoms include anemia, delayed growth, vision problems, pain and frequent infections. There are a total of 62 products in development for this indication, by 45 companies and nine academic institutions.

Key companies operating in this pipeline space include Acceleron Pharma, Epidestiny, Errant Gene Therapeutics, Gamida Cell, Global Blood Therapeutics, Hillhurst Biopharmaceuticals, Novartis and Regenacy Pharmaceuticals. Hemoglobin and a range of histone deacetylases are the most common molecular targets being studied in sickle cell anemia.

Vaso-occlusive crisis associated with sickle cell disease is a painful episode that occurs in people who have sickle cell anemia. Symptoms include fever, pain, fatigue, abdominal pain and swelling, headaches, shortness of breath, dehydration and infection. There are a total of 10 products in development for this indication, by nine companies.

This report provides an overview of the Sickle Cell Disease pipeline landscape. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for sickle cell anemia and vaso-occlusive crisis associated with sickle cell disease, and features dormant and discontinued projects.

Scope

  • Which companies are the most active within each pipeline?
  • Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
  • To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
  • What are the most important R&D milestones and data publications to have happened in this disease area?

Reasons to buy

  • Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication
  • Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication, and a comprehensive picture of recent updates and milestones for each
  • Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these
  • Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Acceleron Pharma
  • Epidestiny
  • Errant Gene Therapeutics
  • Gamida Cell
  • Global Blood Therapeutics
  • Novartis
  • MORE

1 Table of Contents
1 Table of Contents
1.1 List of Tables
1.2 List of Figures

2 Introduction
2.1 Sickle Cell Disease Report Coverage
2.2 Sickle Cell Disease – Overview
2.3 Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Overview

3 Therapeutics Development
3.1 Sickle Cell Disease
3.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease

4 Therapeutics Assessment
4.1 Sickle Cell Disease
4.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease

5 Companies Involved in Therapeutics Development
5.1 Sickle Cell Disease
5.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease

6 Dormant Projects
6.1 Sickle Cell Disease
6.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease
7 Discontinued Products
7.1 Vaso-Occlusive Crisis Associated With Sickle Cell Disease

8 Product Development Milestones
8.1 Sickle Cell Disease
8.2 Vaso-Occlusive Crisis Associated With Sickle Cell Disease

9 Appendix
9.1 Methodology
9.2 Coverage
9.3 Secondary Research
9.4 Primary Research
9.5 Expert Panel Validation
9.6 Contact Us
9.7 Disclaimer

List of Tables
Table 1: Number of Products under Development for Sickle Cell Disease
Table 2: Number of Products under Development by Companies, Sickle Cell Disease
Table 3: Number of Products under Development by Universities/Institutes, Sickle Cell Disease
Table 4: Products under Development by Companies, Sickle Cell Disease
Table 5: Products under Development by Universities/Institutes, Sickle Cell Disease
Table 6: Number of Products under Development for Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Table 7: Number of Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Table 8: Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Table 9: Number of Products by Stage and Target, Sickle Cell Disease
Table 10: Number of Products by Stage and Mechanism of Action, Sickle Cell Disease
Table 11: Number of Products by Stage and Route of Administration, Sickle Cell Disease
Table 12: Number of Products by Stage and Molecule Type, Sickle Cell Disease
Table 13: Number of Products by Stage and Target, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Table 14: Number of Products by Stage and Mechanism of Action, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Table 15: Number of Products by Stage and Route of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Table 16: Number of Products by Stage and Molecule Type, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Table 17: Sickle Cell Disease – Pipeline by Acceleron Pharma Inc
Table 18: Sickle Cell Disease – Pipeline by Addmedica SAS
Table 19: Sickle Cell Disease – Pipeline by Angiocrine Bioscience Inc
Table 20: Sickle Cell Disease – Pipeline by Annexin Pharmaceuticals AB
Table 21: Sickle Cell Disease – Pipeline by ArQule Inc
Table 22: Sickle Cell Disease – Pipeline by Bio Products Laboratory Ltd
Table 23: Sickle Cell Disease – Pipeline by Bioverativ Inc
Table 24: Sickle Cell Disease – Pipeline by bluebird bio Inc
Table 25: Sickle Cell Disease – Pipeline by Bristol-Myers Squibb Co
Table 26: Sickle Cell Disease – Pipeline by Calimmune Inc
Table 27: Sickle Cell Disease – Pipeline by CRISPR Therapeutics
Table 28: Sickle Cell Disease – Pipeline by CSL Ltd
Table 29: Sickle Cell Disease – Pipeline by Daiichi Sankyo Co Ltd
Table 30: Sickle Cell Disease – Pipeline by Editas Medicine Inc
Table 31: Sickle Cell Disease – Pipeline by Errant Gene Therapeutics LLC
Table 32: Sickle Cell Disease – Pipeline by Gamida Cell Ltd
Table 33: Sickle Cell Disease – Pipeline by Genethon SA
Table 34: Sickle Cell Disease – Pipeline by Gilead Sciences Inc
Table 35: Sickle Cell Disease – Pipeline by Global Blood Therapeutics Inc
Table 36: Sickle Cell Disease – Pipeline by Incyte Corp
Table 37: Sickle Cell Disease – Pipeline by Ironwood Pharmaceuticals Inc
Table 38: Sickle Cell Disease – Pipeline by La Jolla Pharmaceutical Company
Table 39: Sickle Cell Disease – Pipeline by MaxCyte Inc
Table 40: Sickle Cell Disease – Pipeline by Merck & Co Inc
Table 41: Sickle Cell Disease – Pipeline by Morphogenesis Inc
Table 42: Sickle Cell Disease – Pipeline by NKT Therapeutics Inc
Table 43: Sickle Cell Disease – Pipeline by Novartis AG
Table 44: Sickle Cell Disease – Pipeline by Orphagen Pharmaceuticals Inc
Table 45: Sickle Cell Disease – Pipeline by Prolong Pharmaceuticals LLC
Table 46: Sickle Cell Disease – Pipeline by Protagonist Therapeutics Inc
Table 47: Sickle Cell Disease – Pipeline by ReveraGen BioPharma Inc
Table 48: Sickle Cell Disease – Pipeline by Sancilio & Company Inc
Table 49: Sickle Cell Disease – Pipeline by Sangamo Therapeutics Inc
Table 50: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by AstraZeneca Plc
Table 51: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Bristol-Myers Squibb Co
Table 52: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Gilead Sciences Inc
Table 53: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Modus Therapeutics Holding AB
Table 54: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Novartis AG
Table 55: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by NuvOx Pharma LLC
Table 56: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Pfizer Inc
Table 57: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Prolong Pharmaceuticals LLC
Table 58: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Pipeline by Seattle Genetics Inc
Table 59: Sickle Cell Disease – Dormant Projects
Table 60: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Dormant Projects
Table 61: Vaso-Occlusive Crisis Associated With Sickle Cell Disease – Discontinued Products

List of Figures
Figure 1: Number of Products under Development for Sickle Cell Disease
Figure 2: Number of Products under Development by Companies, Sickle Cell Disease
Figure 3: Number of Products under Development by Universities/Institutes, Sickle Cell Disease
Figure 4: Number of Products under Development for Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 5: Number of Products under Development by Companies, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 6: Number of Products by Top 10 Targets, Sickle Cell Disease
Figure 7: Number of Products by Stage and Top 10 Targets, Sickle Cell Disease
Figure 8: Number of Products by Top 10 Mechanism of Actions, Sickle Cell Disease
Figure 9: Number of Products by Stage and Top 10 Mechanism of Actions, Sickle Cell Disease
Figure 10: Number of Products by Routes of Administration, Sickle Cell Disease
Figure 11: Number of Products by Stage and Routes of Administration, Sickle Cell Disease
Figure 12: Number of Products by Top 10 Molecule Types, Sickle Cell Disease
Figure 13: Number of Products by Stage and Top 10 Molecule Types, Sickle Cell Disease
Figure 14: Number of Products by Targets, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 15: Number of Products by Stage and Targets, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 16: Number of Products by Mechanism of Actions, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 17: Number of Products by Stage and Mechanism of Actions, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 18: Number of Products by Routes of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 19: Number of Products by Stage and Routes of Administration, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 20: Number of Products by Molecule Types, Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Figure 21: Number of Products by Stage and Molecule Types, Vaso-Occlusive Crisis Associated With Sickle Cell Disease

Note: Product cover images may vary from those shown
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  • Acceleron Pharma
  • Epidestiny
  • Errant Gene Therapeutics
  • Gamida Cell
  • Global Blood Therapeutics
  • Hillhurst Biopharmaceuticals
  • Novartis
  • Regenacy Pharmaceuticals
Note: Product cover images may vary from those shown
5 of 4
Note: Product cover images may vary from those shown
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