Sickle Cell Disease – Pipeline Review, H1 2012

Published : April 2012
No. of Pages : 141

Price:

Sickle Cell Disease – Pipeline Review, H1 2012

Summary

Global Markets Direct’s, 'Sickle Cell Disease - Pipeline Review, H1 2012', provides an overview of the Sickle Cell Disease therapeutic pipeline. This report provides information on the therapeutic development for Sickle Cell Disease, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Sickle Cell Disease. 'Sickle Cell Disease - Pipeline Review, H1 2012' is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- A snapshot of the global therapeutic scenario for Sickle Cell Disease.
- A review of the Sickle Cell Disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Sickle Cell Disease pipeline on the basis of route of administration and molecule type.
- Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Sickle Cell Disease.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players with the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Sickle Cell Disease pipeline depth and focus of Sickle Cell Disease 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.
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.

Table of Contents
Table of Contents 2
List of Tables 9
List of Figures 10
Introduction 11
Global Markets Direct Report Coverage 11
Sickle Cell Disease Overview 12
Therapeutics Development 13
An Overview of Pipeline Products for Sickle Cell Disease 13
Sickle Cell Disease Therapeutics under Development by Companies 15
Sickle Cell Disease Therapeutics under Investigation by Universities/Institutes 17
Late Stage Products 19
Comparative Analysis 19
Mid Clinical Stage Products 20
Comparative Analysis 20
Early Clinical Stage Products 21
Comparative Analysis 21
Discovery and Pre-Clinical Stage Products 22
Comparative Analysis 22
Sickle Cell Disease Therapeutics – Products under Development by Companies 23
Sickle Cell Disease Therapeutics – Products under Investigation by Universities/Institutes 24
Companies Involved in Sickle Cell Disease Therapeutics Development 26
Sangart, Inc. 26
Daiichi Sankyo Company, Ltd 27
Sangamo BioSciences, Inc. 28
Gamida Cell Ltd. 29
Novartis AG 30
Pfizer Inc. 31
ADVENTRX Pharmaceuticals 32
Advinus Therapeutics Pvt. Ltd. 33
ERYtech Pharma 34
Archemix Corp. 35
HemaQuest Pharmaceuticals, Inc. 36
GENETIX Pharmaceuticals, Inc. 37
Selexys Pharmaceuticals Corporation 38
Morphogenesis, Inc. 39
Emmaus Medical, Inc. 40
AesRx, LLC. 41
Errant Gene Therapeutics, LLC 42
Sickle Cell Disease – Therapeutics Assessment 43
Assessment by Monotherapy Products 43
Assessment by Combination Products 44
Assessment by Route of Administration 45
Assessment by Molecule Type 47
Drug Profiles 50
LBH589 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Aptamers for Sicke Cell Disease - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Soluble P-selectin - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
LentiGlobin - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Warfarin - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
PselmabG1 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Enhoxy - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
NiCord - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Trichosic - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Zocor - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Hydroxyurea - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Humanized antibodies to P-selectin - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Aes-103 - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
PF-06460031 - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Hydroxyurea + L-Arginine - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Ketamine - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Vorinostat - Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
Glutamine - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Tadalafil - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
Efient - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
Lexiscan - Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 78
Apha Lipoic Acid + Acetyl L Carnitine - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
Nitric Oxide - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
Propranolol - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
HQK-1001 - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
Fludarabine + Mycophenolate Mofetil + Cyclosporine + Allogeneic Bone Marrow Transplantation + Radiation Therapy - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 85
Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Stem Cell Transplantation + Campath + Rapamune + Cytoxan - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 87
Campath + Rapamune + Radiation Therapy + Non-Myeloablative Allogeneic Peripheral Hematopoietic Stem Cell Transplantation - Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
Busulfex + Fludarabine + FK506 + Prednisone + Allogeneic Stem Cell Transplantation - Drug Profile 90
Product Description 90
Mechanism of Action 90
R&D Progress 91
Filgrastim + Cyclophosphamide + Fludarabine + Mycophenolate + Tacrolimus + Radiation Therapy + Allogeneic Bone Marrow Transplantation - Drug Profile 92
Product Description 92
Mechanism of Action 92
R&D Progress 93
Sodium Nitrite - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
L-Glutamine - Drug Profile 95
Product Description 95
Mechanism of Action 95
R&D Progress 95
Fludarabine + Busulfan + Alemtuzumab + Allogeneic Stem Cell Transplantation - Drug Profile 96
Product Description 96
Mechanism of Action 96
R&D Progress 96
Busulfan + Cyclophosphamide + Horse ATG + Stem Cell Transplantation - Drug Profile 98
Product Description 98
Mechanism of Action 98
R&D Progress 98
Cyclosporine + Fludarabine + Mycophenolate + Allogeneic Bone Marrow Transplantation + Radiation Therapy - Drug Profile 100
Product Description 100
Mechanism of Action 100
R&D Progress 100
Magnesium Pidolate + Hydroxyurea - Drug Profile 102
Product Description 102
Mechanism of Action 102
R&D Progress 102
L-Arginine - Drug Profile 103
Product Description 103
Mechanism of Action 103
R&D Progress 103
Alemtuzumab + Sirolimus + Irradiation + Allogeneic Non-Myeloablative Stem Cell Transplantation - Drug Profile 104
Product Description 104
Mechanism of Action 104
R&D Progress 104
Campath-1H - Drug Profile 106
Product Description 106
Mechanism of Action 106
R&D Progress 106
Decitabine - Drug Profile 107
Product Description 107
Mechanism of Action 107
R&D Progress 107
Nitric Oxide - Drug Profile 108
Product Description 114
Mechanism of Action 114
R&D Progress 114
Hematopoietic Stem Cell Transplantation - Drug Profile 115
Product Description 115
Mechanism of Action 115
R&D Progress 115
Fludarabine + Rituximab + Busulfan + Thymoglobulin + Cyclophosphamide + Mycophenolate Mofetil + Tacrolimus + Allogeneic Hematopoietic Stem Cell Transplantation - Drug Profile 117
Product Description 117
Mechanism of Action 117
R&D Progress 118
Alemtuzumab + Fludarabine + Melphalan + Hematopoietic Stem Cell Transplantation - Drug Profile 119
Product Description 125
Mechanism of Action 125
R&D Progress 125
Sickle Cell Disease Therapeutics – Drug Profile Updates 126
Sickle Cell Disease Therapeutics – Discontinued Products 133
Sickle Cell Disease Therapeutics - Dormant Products 134
Sickle Cell Disease – Product Development Milestones 135
Featured News & Press Releases 135
Feb 22, 2012: ERYTECH Pharma To Present Two Posters At Emirates Hematology Conference 135
Jan 12, 2012: Sangart Initiates Phase Ib Study Of MP4CO In Patients With Sickle Cell Disease 135
Dec 19, 2011: Selexys Pharmaceuticals Completes Phase I Clinical Study For SelG1 136
Dec 06, 2011: AesRx Begins Clinical Trial Of Aes-103 For Sickle Cell Disease 136
Dec 01, 2011: ADVENTRX Meets FDA To Discuss Development Plans For ANX-188 137
Sep 19, 2011: FerroKin BioSciences Starts Phase II Clinical Trial Of FBS0701 As New Oral Treatment For Transfusional Iron Overload In Pediatric Population 137
Sep 12, 2011: HemaQuest Completes Enrollment In Phase II Clinical Study Of HQK-1001 In Patients With Sickle Cell Disease 138
Aug 30, 2011: ADVENTRX Provides Update On ANX-188 138
Apr 25, 2011: HemaQuest Announces First Patient Enrolled In Phase II Clinical Study Of HQK-1001 In Patients With Sickle Cell Disease 138
Mar 16, 2011: bluebird bio Receives Upto $4.2m From French Muscular Dystrophy Association For LentiGlobin 139
Appendix 140
Methodology 140
Coverage 140
Secondary Research 140
Primary Research 140
Expert Panel Validation 140
Contact Us 141
Disclaimer 141

List of Tables
Number of Products Under Development for Sickle Cell Disease, H1 2012 13
Products under Development for Sickle Cell Disease – Comparative Analysis, H1 2012 14
Number of Products under Development by Companies, H1 2012 16
Number of Products under Investigation by Universities/Institutes, H1 2012 18
Comparative Analysis by Late Stage Development, H1 2012 19
Comparative Analysis by Mid Clinical Stage Development, H1 2012 20
Comparative Analysis by Early Clinical Stage Development, H1 2012 21
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H1 2012 22
Products under Development by Companies, H1 2012 23
Products under Investigation by Universities/Institutes, H1 2012 24
Sangart, Inc., H1 2012 26
Daiichi Sankyo Company, Ltd, H1 2012 27
Sangamo BioSciences, Inc., H1 2012 28
Gamida Cell Ltd., H1 2012 29
Novartis AG, H1 2012 30
Pfizer Inc., H1 2012 31
ADVENTRX Pharmaceuticals, H1 2012 32
Advinus Therapeutics Pvt. Ltd., H1 2012 33
ERYtech Pharma, H1 2012 34
Archemix Corp., H1 2012 35
HemaQuest Pharmaceuticals, Inc., H1 2012 36
GENETIX Pharmaceuticals, Inc., H1 2012 37
Selexys Pharmaceuticals Corporation, H1 2012 38
Morphogenesis, Inc., H1 2012 39
Emmaus Medical, Inc., H1 2012 40
AesRx, LLC., H1 2012 41
Errant Gene Therapeutics, LLC, H1 2012 42
Assessment by Monotherapy Products, H1 2012 43
Assessment by Combination Products, H1 2012 44
Assessment by Stage and Route of Administration, H1 2012 46
Assessment by Stage and Molecule Type, H1 2012 49
Sickle Cell Disease Therapeutics – Drug Profile Updates 126
Sickle Cell Disease Therapeutics – Discontinued Products 133
Sickle Cell Disease Therapeutics – Dormant Products 134

List of Figures
Number of Products under Development for Sickle Cell Disease, H1 2012 13
Products under Development for Sickle Cell Disease – Comparative Analysis, H1 2012 14
Products under Development by Companies, H1 2012 15
Products under Investigation by Universities/Institutes, H1 2012 17
Late Stage Products, H1 2012 19
Mid Clinical Stage Products, H1 2012 20
Early Clinical Stage Products, H1 2012 21
Discovery and Pre-Clinical Stage Products, H1 2012 22
Assessment by Monotherapy Products, H1 2012 43
Assessment by Combination Products, H1 2012 44
Assessment by Route of Administration, H1 2012 45
Assessment by Stage and Route of Administration, H1 2012 46
Assessment by Molecule Type, H1 2012 47
Assessment by Stage and Molecule Type, H1 2012 48

Sickle Cell Disease – Pipeline Review, H1 2012

Published By: Global Markets Direct

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