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Sickle Cell Disease - Pipeline Review, H1 2017

Published By :

Global Markets Direct

Published Date : May 2017

Category :

Diseases & Conditions

No. of Pages : 191 Pages

Sickle Cell Disease - Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Sickle Cell Disease - Pipeline Review, H1 2017, provides an overview of the Sickle Cell Disease (Hematological Disorders) pipeline landscape.

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. Treatment includes antibiotics, pain relievers, blood transfusion and stem cell transplant.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Sickle Cell Disease - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Sickle Cell Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 5, 5, 9, 27, 6 and 1 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 5 and 2 molecules, respectively.

Sickle Cell Disease (Hematological 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 Global Markets Directs 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 Sickle Cell Disease (Hematological Disorders).
- The pipeline guide reviews pipeline therapeutics for Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological 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 its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Sickle Cell Disease (Hematological 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.
Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Sickle Cell Disease - Overview 8
Sickle Cell Disease - Therapeutics Development 9
Pipeline Overview 9
Pipeline by Companies 10
Pipeline by Universities/Institutes 14
Products under Development by Companies 15
Products under Development by Universities/Institutes 18
Sickle Cell Disease - Therapeutics Assessment 19
Assessment by Target 19
Assessment by Mechanism of Action 22
Assessment by Route of Administration 25
Assessment by Molecule Type 27
Sickle Cell Disease - Companies Involved in Therapeutics Development 29
Acceleron Pharma Inc 29
Addex Therapeutics Ltd 29
Addmedica SAS 30
Advinus Therapeutics Ltd 30
Alnylam Pharmaceuticals Inc 31
Angiocrine Bioscience Inc 31
ArQule Inc 32
Bio Products Laboratory Ltd 32
Bioverativ Inc 33
bluebird bio Inc 33
Bristol-Myers Squibb Company 34
Calimmune Inc 34
CRISPR Therapeutics 35
CSL Ltd 35
Editas Medicine Inc 36
Errant Gene Therapeutics LLC 36
Gamida Cell Ltd 37
Genethon SA 37
Gilead Sciences Inc 38
Global Blood Therapeutics Inc 38
Incyte Corp 39
Johnson & Johnson 39
La Jolla Pharmaceutical Company 40
MaxCyte Inc 40
Merck & Co Inc 41
Modus Therapeutics Holding AB 41
Morphogenesis Inc 42
NKT Therapeutics Inc 42
Novartis AG 43
Orphagen Pharmaceuticals Inc 43
Pfizer Inc 44
Prolong Pharmaceuticals LLC 44
Protagonist Therapeutics Inc 45
ReveraGen BioPharma Inc 45
Sancilio & Company Inc 46
Sangamo Therapeutics Inc 46
Sickle Cell Disease - Drug Profiles 47
(decitabine + tetrahydrouridine) - Drug Profile 47
AB-110 - Drug Profile 48
ACY-957 - Drug Profile 49
ALN-TMP - Drug Profile 52
ambrisentan - Drug Profile 54
apixaban - Drug Profile 57
ARQ-092 - Drug Profile 65
BB-305 - Drug Profile 68
benserazide - Drug Profile 76
CAL-H - Drug Profile 78
CNTO-530 - Drug Profile 79
CordIn - Drug Profile 80
crizanlizumab - Drug Profile 81
didox - Drug Profile 83
Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile 86
DRX-194 - Drug Profile 87
GBT-440 - Drug Profile 88
Gene Therapy for Sickle Cell Anemia - Drug Profile 94
Gene Therapy for Sickle Cell Disease - Drug Profile 95
Gene therapy for Sickle Cell Disease - Drug Profile 96
Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 97
Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile 98
Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia - Drug Profile 99
glutamine - Drug Profile 100
haptoglobin (human) - Drug Profile 104
haptoglobin + hemopexin - Drug Profile 105
HBI-002 - Drug Profile 106
HBI-137 - Drug Profile 107
hydroxyurea - Drug Profile 108
hydroxyurea - Drug Profile 109
IMR-687 - Drug Profile 110
INCB-59872 - Drug Profile 111
LJPC-401 - Drug Profile 112
luspatercept - Drug Profile 114
M-012 - Drug Profile 122
NiCord - Drug Profile 124
NKTT-120 - Drug Profile 128
panobinostat - Drug Profile 130
pentosan polysulfate sodium - Drug Profile 138
PF-04447943 - Drug Profile 139
PNQ-103 - Drug Profile 140
Protein for Sickle Cell Disease - Drug Profile 141
PTG-300 - Drug Profile 142
RCY-1497 - Drug Profile 143
RN-1 - Drug Profile 144
RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 145
Sanguinate - Drug Profile 146
SC-411 - Drug Profile 149
SCD-101 - Drug Profile 150
SCPF - Drug Profile 151
sevuparin sodium - Drug Profile 152
Small Molecule to Agonize A2A for Hematological, Immunology and Neurological Disorders - Drug Profile 155
Small Molecules for Sickle Cell Disease - Drug Profile 156
Small Molecules for Sickle Cell Disease - Drug Profile 157
Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease - Drug Profile 158
Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 159
sotatercept - Drug Profile 160
Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 165
Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 166
Stem Cell Therapy for Sickle Cell Disease - Drug Profile 167
Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile 169
Trichosic - Drug Profile 170
vamorolone - Drug Profile 171
Sickle Cell Disease - Dormant Projects 173
Sickle Cell Disease - Discontinued Products 175
Sickle Cell Disease - Product Development Milestones 176
Featured News & Press Releases 176
Appendix 186
Methodology 186
Coverage 186
Secondary Research 186
Primary Research 186
Expert Panel Validation 186
Contact Us 186
Disclaimer 187

List of Tables
Number of Products under Development for Sickle Cell Disease, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017
Number of Products under Development by Companies, H1 2017 (Contd..2), H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Products under Development by Companies, H1 2017
Products under Development by Companies, H1 2017 (Contd..1), H1 2017
Products under Development by Companies, H1 2017 (Contd..2), H1 2017
Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Target, H1 2017 (Contd..1), H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017 (Contd..1), H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Sickle Cell Disease - Pipeline by Acceleron Pharma Inc, H1 2017
Sickle Cell Disease - Pipeline by Addex Therapeutics Ltd, H1 2017
Sickle Cell Disease - Pipeline by Addmedica SAS, H1 2017
Sickle Cell Disease - Pipeline by Advinus Therapeutics Ltd, H1 2017
Sickle Cell Disease - Pipeline by Alnylam Pharmaceuticals Inc, H1 2017
Sickle Cell Disease - Pipeline by Angiocrine Bioscience Inc, H1 2017
Sickle Cell Disease - Pipeline by ArQule Inc, H1 2017
Sickle Cell Disease - Pipeline by Bio Products Laboratory Ltd, H1 2017
Sickle Cell Disease - Pipeline by Bioverativ Inc, H1 2017
Sickle Cell Disease - Pipeline by bluebird bio Inc, H1 2017
Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Company, H1 2017
Sickle Cell Disease - Pipeline by Calimmune Inc, H1 2017
Sickle Cell Disease - Pipeline by CRISPR Therapeutics, H1 2017
Sickle Cell Disease - Pipeline by CSL Ltd, H1 2017
Sickle Cell Disease - Pipeline by Editas Medicine Inc, H1 2017
Sickle Cell Disease - Pipeline by Errant Gene Therapeutics LLC, H1 2017
Sickle Cell Disease - Pipeline by Gamida Cell Ltd, H1 2017
Sickle Cell Disease - Pipeline by Genethon SA, H1 2017
Sickle Cell Disease - Pipeline by Gilead Sciences Inc, H1 2017
Sickle Cell Disease - Pipeline by Global Blood Therapeutics Inc, H1 2017
Sickle Cell Disease - Pipeline by Incyte Corp, H1 2017
Sickle Cell Disease - Pipeline by Johnson & Johnson, H1 2017
Sickle Cell Disease - Pipeline by La Jolla Pharmaceutical Company, H1 2017
Sickle Cell Disease - Pipeline by MaxCyte Inc, H1 2017
Sickle Cell Disease - Pipeline by Merck & Co Inc, H1 2017
Sickle Cell Disease - Pipeline by Modus Therapeutics Holding AB, H1 2017
Sickle Cell Disease - Pipeline by Morphogenesis Inc, H1 2017
Sickle Cell Disease - Pipeline by NKT Therapeutics Inc, H1 2017
Sickle Cell Disease - Pipeline by Novartis AG, H1 2017
Sickle Cell Disease - Pipeline by Orphagen Pharmaceuticals Inc, H1 2017
Sickle Cell Disease - Pipeline by Pfizer Inc, H1 2017
Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals LLC, H1 2017
Sickle Cell Disease - Pipeline by Protagonist Therapeutics Inc, H1 2017
Sickle Cell Disease - Pipeline by ReveraGen BioPharma Inc, H1 2017
Sickle Cell Disease - Pipeline by Sancilio & Company Inc, H1 2017
Sickle Cell Disease - Pipeline by Sangamo Therapeutics Inc, H1 2017
Sickle Cell Disease - Dormant Projects, H1 2017
Sickle Cell Disease - Dormant Projects, H1 2017 (Contd..1), H1 2017
Sickle Cell Disease - Discontinued Products, H1 2017

List of Figures
Number of Products under Development for Sickle Cell Disease, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Number of Products by Top 10 Targets, H1 2017
Number of Products by Stage and Top 10 Targets, H1 2017
Number of Products by Top 10 Mechanism of Actions, H1 2017
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017
Number of Products by Routes of Administration, H1 2017
Number of Products by Stage and Top 10 Routes of Administration, H1 2017
Number of Products by Top 10 Molecule Types, H1 2017
Number of Products by Stage and Top 10 Molecule Types, H1 2017

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