Sections
Title | Starting Page | Number of Pages |
---|
Table of Contents | 2 | 10 |
List of Tables | 10 | 1 |
List of Figures | 11 | 1 |
Introduction | 12 | 1 |
Global Markets Direct Report Coverage | 12 | 1 |
Sickle Cell Disease Overview | 13 | 1 |
Therapeutics Development | 14 | 2 |
Pipeline Products for Sickle Cell Disease Overview | 14 | 1 |
Pipeline Products for Sickle Cell Disease Comparative Analysis | 15 | 1 |
Sickle Cell Disease Therapeutics under Development by Companies | 16 | 4 |
Sickle Cell Disease Therapeutics under Investigation by Universities/Institutes | 20 | 1 |
Sickle Cell Disease Pipeline Products Glance | 21 | 4 |
Late Stage Products | 21 | 1 |
Clinical Stage Products | 22 | 1 |
Early Stage Products | 23 | 1 |
Unknown Stage Products | 24 | 1 |
Sickle Cell Disease Products under Development by Companies | 25 | 3 |
Sickle Cell Disease Products under Investigation by Universities/Institutes | 28 | 1 |
Sickle Cell Disease Companies Involved in Therapeutics Development | 29 | 34 |
Acceleron Pharma, Inc. | 29 | 1 |
Acetylon Pharmaceuticals, Inc. | 30 | 1 |
Addex Therapeutics Ltd | 31 | 1 |
Advinus Therapeutics Ltd | 32 | 1 |
Alnylam Pharmaceuticals, Inc. | 33 | 1 |
Angiocrine Bioscience, Inc. | 34 | 1 |
Bio Products Laboratory Ltd | 35 | 1 |
bluebird bio, Inc. | 36 | 1 |
Bristol-Myers Squibb Company | 37 | 1 |
Complexa, Inc. | 38 | 1 |
CRISPR Therapeutics | 39 | 1 |
CSL Limited | 40 | 1 |
Editas Medicine, Inc. | 41 | 1 |
Emmaus Medical, Inc. | 42 | 1 |
Errant Gene Therapeutics, LLC | 43 | 1 |
Gamida Cell Ltd. | 44 | 1 |
Gilead Sciences, Inc. | 45 | 1 |
Global Blood Therapeutics, Inc. | 46 | 1 |
Johnson &Johnson | 47 | 1 |
La Jolla Pharmaceutical Company | 48 | 1 |
Merck &Co., Inc. | 49 | 1 |
Modus Therapeutics Holding AB | 50 | 1 |
Morphogenesis, Inc. | 51 | 1 |
NKT Therapeutics, Inc. | 52 | 1 |
Novartis AG | 53 | 1 |
Orphagen Pharmaceuticals, Inc. | 54 | 1 |
Pfizer Inc. | 55 | 1 |
PharmaEssentia Corporation | 56 | 1 |
Prolong Pharmaceuticals, LLC | 57 | 1 |
Protagonist Therapeutics Inc. | 58 | 1 |
ReveraGen BioPharma, Inc. | 59 | 1 |
Sancilio &Company, Inc. | 60 | 1 |
Sangamo BioSciences, Inc. | 61 | 1 |
Selexys Pharmaceuticals Corporation | 62 | 1 |
Sickle Cell Disease Therapeutics Assessment | 63 | 12 |
Assessment by Monotherapy Products | 63 | 1 |
Assessment by Combination Products | 64 | 1 |
Assessment by Target | 65 | 3 |
Assessment by Mechanism of Action | 68 | 3 |
Assessment by Route of Administration | 71 | 2 |
Assessment by Molecule Type | 73 | 2 |
Drug Profiles | 75 | 108 |
(decitabine + tetrahydrouridine) Drug Profile | 75 | 1 |
ACY-957 Drug Profile | 76 | 2 |
AIC-6020 Drug Profile | 78 | 1 |
ALN-TMP Drug Profile | 79 | 2 |
ambrisentan Drug Profile | 81 | 2 |
apixaban Drug Profile | 83 | 7 |
BB-305 Drug Profile | 90 | 7 |
benserazide Drug Profile | 97 | 1 |
CNTO-530 Drug Profile | 98 | 1 |
CordIn Drug Profile | 99 | 1 |
CXA-10 Drug Profile | 100 | 1 |
didox Drug Profile | 101 | 3 |
Drugs to Inhibit PRMT5 for Sickle Cell Anemia Drug Profile | 104 | 1 |
DRX-194 Drug Profile | 105 | 1 |
E-CEL-UVEC Drug Profile | 106 | 1 |
EdX-17 Drug Profile | 107 | 1 |
GBT-440 Drug Profile | 108 | 4 |
Gene Therapy for Sickle Cell Anemia Drug Profile | 112 | 1 |
Gene Therapy for Sickle Cell Disease and Thalassemia Drug Profile | 113 | 1 |
Gene Therapy to Activate Gamma Globin for Sickle Cell Disease Drug Profile | 114 | 1 |
glutamine Drug Profile | 115 | 3 |
haptoglobin (human) Drug Profile | 118 | 1 |
haptoglobin + hemopexin Drug Profile | 119 | 1 |
HBI-002 Drug Profile | 120 | 1 |
HBI-137 Drug Profile | 121 | 1 |
hydroxyurea Drug Profile | 122 | 1 |
IMR-687 Drug Profile | 123 | 1 |
LJPC-401 Drug Profile | 124 | 2 |
luspatercept Drug Profile | 126 | 5 |
M-012 Drug Profile | 131 | 2 |
NiCord Drug Profile | 133 | 3 |
NKTT-120 Drug Profile | 136 | 2 |
NM-96 Drug Profile | 138 | 1 |
panobinostat Drug Profile | 139 | 7 |
pentosan polysulfate sodium Drug Profile | 146 | 1 |
PF-04447943 Drug Profile | 147 | 1 |
PNQ-103 Drug Profile | 148 | 1 |
Protein for Sickle Cell Disease Drug Profile | 149 | 1 |
PTG-300 Drug Profile | 150 | 1 |
regadenoson Drug Profile | 151 | 1 |
RN-1 Drug Profile | 152 | 1 |
RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia Drug Profile | 153 | 1 |
Sanguinate Drug Profile | 154 | 2 |
SC-411 Drug Profile | 156 | 1 |
SCD-101 Drug Profile | 157 | 1 |
SCPF Drug Profile | 158 | 1 |
SelG-1 Drug Profile | 159 | 2 |
sevuparin sodium Drug Profile | 161 | 3 |
Small Molecule for Beta Thalassemia and Sickle Cell Anemia Drug Profile | 164 | 1 |
Small Molecule for Sickle Cell Disease Drug Profile | 165 | 1 |
Small Molecule to Agonize A2A for Immunology, Hematological and Neurological Disorders Drug Profile | 166 | 1 |
Small Molecules for Sickle Cell Anemia Drug Profile | 167 | 1 |
Small Molecules to Antagonize TNFR1 for Immunology, Gastrointestinal, Neurology and Hematological Disorders Drug Profile | 168 | 1 |
Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease Drug Profile | 169 | 1 |
Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours Drug Profile | 170 | 1 |
sotatercept Drug Profile | 171 | 5 |
Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease Drug Profile | 176 | 1 |
Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease Drug Profile | 177 | 1 |
Stem Cell Therapy for Sickle Cell Disease Drug Profile | 178 | 1 |
Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease Drug Profile | 179 | 1 |
Trichosic Drug Profile | 180 | 1 |
vamorolone Drug Profile | 181 | 2 |
Sickle Cell Disease Dormant Projects | 183 | 3 |
Sickle Cell Disease Discontinued Products | 186 | 1 |
Sickle Cell Disease Product Development Milestones | 187 | 11 |
Featured News &Press Releases | 187 | 1 |
Oct 07, 2016: Global Blood Therapeutics Presents GBT440 Data that Continue to Support Durability, Safety and Mechanism of Action in Sickle Cell Disease | 187 | 1 |
Sep 08, 2016: Emmaus Life Sciences Submits New Drug Application for Sickle Cell Disease Treatment | 188 | 1 |
Sep 07, 2016: La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401 | 189 | 1 |
Aug 22, 2016: Emmaus Life Sciences to Seek Marketing Approval for First New Sickle Cell Disease Treatment in Nearly 20 Years; Expects to Submit New Drug Application to FDA in September | 189 | 1 |
Aug 05, 2016: CDSCO requests further data for granting additional indication for Ambrisentan tablet 5mg/10mg | 190 | 1 |
Jul 08, 2016: Global Blood Therapeutics Announces Publication of Preclinical GBT440 Results in British Journal of Haematology that Support Sickle Cell Disease (SCD) Program | 190 | 1 |
Jun 29, 2016: Global Blood Therapeutics Announces Initiation of Phase 2a Study of GBT440 in Adolescents with Sickle Cell Disease | 191 | 1 |
Jun 10, 2016: Global Blood Therapeutics Announces New Clinical Data that Continue to Support Safety and Efficacy Profile of GBT440 as a Potentially Disease-Modifying Therapy for Sickle Cell Disease | 191 | 2 |
Jun 02, 2016: Global Blood Therapeutics to Host Investor Event on Friday, June 10 to Review GBT440 Data in Sickle Cell Disease Being Presented at the European Hematology Associations 21st Congress | 193 | 1 |
May 24, 2016: Hillhurst Biopharmaceuticals Receives SBIR Funding For Development Of HBI-002 For Prevention Of Vaso-Occlusive Crises In Sickle Cell Disease | 193 | 1 |
May 19, 2016: Global Blood Therapeutics Announces Presentation of New GBT440 Data in Sickle Cell Disease at the European Hematology Associations 21st Congress | 194 | 1 |
May 09, 2016: Merganser Biotech Announces Issuance of U.S. Patent 9,315,545 Covering Development Compound M012 | 194 | 1 |
May 04, 2016: Acetylon Pharmaceuticals Publishes New Mechanism for the Treatment of Sickle Cell Disease/Beta-Thalassemia with Selective HDAC1/2 Inhibition in PLoS One | 195 | 1 |
Jan 25, 2016: Emmaus Life Sciences Announces Reappointment of Dr. Yutaka Niihara as CEO | 196 | 1 |
Dec 31, 2015: Global Blood Therapeutics Receives FDA Orphan Drug Designation for GBT440 in Sickle Cell Disease | 196 | 2 |
Appendix | 198 | 2 |
Methodology | 198 | 1 |
Coverage | 198 | 1 |
Secondary Research | 198 | 1 |
Primary Research | 198 | 1 |
Expert Panel Validation | 198 | 1 |
Contact Us | 198 | 1 |
Disclaimer | 199 | 1 |