Sickle Cell Anaemia. ADNM Drug Originator Originator Now Co. Code MoA MoA Code Molecular formula CompoundType Route Dec_01 Jun_02 Dec_02
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1 ADNM Originator Originator Now Co. Code MoA MoA Code Molecular formula CompoundType Route Dec_01 Jun_02 Dec_02 Nonindustrial Bar-Ilan University AI Fetal haemoglobin stimulants Angiogenix Inc Angiogenix Inc BioPharm Nitric oxide agonists, Nitric oxide donors Nonindustrial Nonindustrial Celgene Cellerant Questcor Childrens Hospital of Oakland MI Gene transcription activators, Tyrosine Oklahoma Medical Research Foundation RI Haemoglobin modifiers, Undefined mechanism Celgene SBT Interleukin 1-beta antagonists, Interleukin NT NT 3C6 H14 N4 O2. 4C4 H8 O2 C C C IV II II II NT C14 H18 N2 O5 C PO NT C13 H11 N3 O4 C PO Cellerant SBT Cell-replacements NT B Parenteral Questcor SMP Immunosuppressants, Undefined mechanism Pharmachemie SuperGen SMP DNA methylation GlaxoSmithKline, NeuroSearch Biopure Bristol-Myers Barr Laboratories, Bristol- Squibb, Duramed Myers Squibb Nonindustrial NeuroSearch BioPharm Chloride channel antagonists NT C6 H14 O12 P2 C IV Phase-III Phase-III Phase-III NT C8 H12 N4 O4. H2 O C IV/IV- infusion/iv- Phase-II Phase-II Phase-II NT C PO I I I Biopure SMP Haemoglobin agonists NT B IV/IV-infusion Phase-II Phase-II Phase-II BP Ribonucleotide reductase Childrens Hospital of Oakland MI Gene transcription activators NT C H4 N2 O2 C PO Launched Launched Launched MT C4 H9 N O C PO Suspended-II Suspended-II Suspended-II Medinox Medinox SMP Undefined mechanism C PO CytRx therapeutics - Sangamo therapy - Virginia Selexys OSI CytRx SMP Undefined mechanism C IV Phase-III Phase-III Phase-III Selexys SMP Selectin antagonists MT B OSI SBT Fetal haemoglobin stimulants FibroGen FibroGen SBT Hydroxylase NT C PO Nonindustrial Sangamo BioSciences Virginia Commonwealth SCD 1 - Tapestry NaPro Bio NT C NDR- NDR- NDR- Nonindustrial Ion channel modulators MT C 0 NDR- NDR- Sangamo BioSciences SBT Gene repair NT B Virginia Commonwealth University AI Undefined mechanism C IV/PO Tapestry SBT MT B Icagen Icagen BioPharm Potassium channel antagonists gene therapy gene therapy - - Celgene Albert Einstein College of Albert Einstein College of Medicine, NT C20 H15 F2 N O C PO Phase-I Phase-I Phase-II AI NT B SBT NT B Celgene Transcend Vertex Celgene SBT Cell-replacements NT B Transcend [CEASED] Vertex SBT Histone deacetylase Undefined mechanism C NT C Wyeth Wyeth BP Selectin antagonists NT B IV/Topical 1 of 5
2 Jun_03 Dec_03 Jun_04 Dec_04 Jun_05 Dec_05 Jun_06 Dec_06 Apr_07 PC-I Fail-I I-II Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II II II II II II II II II II NDR-III NDR-III NDR-III NDR-III NDR-III III III III III Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II I I I I I I I I I 1 Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II Phase-II NDR-II Launched Launched Launched Launched Launched Launched Launched Launched Launched Suspended-II Suspended-II Suspended-II Suspended-II Suspended-II Suspended-II Suspended-II Suspended-II Suspended-II 1 Phase-III Phase-III Phase-III Phase-III Phase-III Phase-III Phase-III Phase-III Phase-III therapeutics - Sangamo therapy - Virginia SCD 1 - Tapestry gene therapy gene therapy - - Celgene NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- NDR- Phase-II Phase-II Phase-II Phase-II Phase-III Phase-III Phase-III Phase-III Phase-III 1 NDR- NDR- NDR- NDR- NDR- I I I I I I I I of 5
3 ADNM Fail-II II-III Fail-III III-PR Fail-PR PR-R Fail-R PR/R-L Fail-L Success Indication Group Indication Licensee License Date therapeutics - Sangamo therapy - Virginia SCD 1 - Tapestry gene therapy gene therapy - - Celgene Discovery Laboratories Vertex Pharmaceutical SuperGen GlaxoSmithKlin e, TopoTarget Abazali Bio Ventures, Vertex Pharmaceutical SynthRx Icagen Xechem International Childrens Hospital Ortho-McNeil Ys 04-Oct Aug Jan Oct Jan Aug Jun Jun-05 3 of 5
4 License Phase Country Lead Indication(s) Lead Indication Months PC Month PI Months PII Months PIII Months PR Months R Date_PC Date_PI USA 20-Feb-97 USA USA, Thalassaemia USA, Osteoarthritis, Rheumatoid arthritis, Pain USA Multiple myeloma, Prostate cancer No 31-Dec-05 USA Cancer No 30-Aug-05 USA USA Myelodysplastic syndromes No USA Cancer No 24-Sep-01 USA Anaemia No USA Cancer No 9 USA, Thalassaemia Nov-95 USA 27-Oct-00 Phase-III USA therapeutics - Sangamo therapy - Virginia USA, Inflammation 14-Dec-05 USA, Thalassaemia 28-May-98 USA 29-Aug-06 USA 31-Mar-00 USA 23-Jul-02 USA 13-Dec SCD 1 - Tapestry USA 16-Jun gene therapy gene therapy - - Celgene Phase-II USA Dec-99 USA 05-Mar-02 USA 29-Apr-97 USA 31-Dec-04 USA, Thalassaemia, Anaemia 10-Sep-97 USA Apr Oct-03 USA Delayed graft function No 08-Jan-04 4 of 5
5 Date_PII Date_PIII Date_PR Date_R Date_L Date_Fail Phase Fail Reason Fail Time In Failed Phase 12-Jul Nov-97 9 months 20-Oct Jul-96 Phase-II II 9 months 03-Nov Jun Oct-05 Phase-III III 88 months 14-Aug May Feb May-02 8 months 06-Oct Apr-98 therapeutics - Sangamo therapy - Virginia SCD 1 - Tapestry 25-Sep Feb Aug months gene therapy gene therapy - - Celgene 18-Jun months 19-Nov-03 Phase-I I 1 month 13-Dec months 5 of 5
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