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Published: Mar, 2015 | Pages:
100 | Publisher: Global Markets Direct
Industry: Pharmaceutical | Report Format: Electronic (PDF)
Thalassemia - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Thalassemia - Pipeline Review, H1 2015', provides an overview of the Thalassemia's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Thalassemia, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Thalassemia and special features on late-stage and discontinued projects. Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of Thalassemia - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Thalassemia and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Thalassemia products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Thalassemia pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Thalassemia - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Thalassemia 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 - 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 table 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Thalassemia Overview 8 Therapeutics Development 9 Pipeline Products for Thalassemia - Overview 9 Pipeline Products for Thalassemia - Comparative Analysis 10 Thalassemia - Therapeutics under Development by Companies 11 Thalassemia - Therapeutics under Investigation by Universities/Institutes 12 Thalassemia - Pipeline Products Glance 13 Late Stage Products 13 Clinical Stage Products 14 Early Stage Products 15 Thalassemia - Products under Development by Companies 16 Thalassemia - Products under Investigation by Universities/Institutes 18 Thalassemia - Companies Involved in Therapeutics Development 19 Acceleron Pharma, Inc. 19 Acetylon Pharmaceuticals, Inc. 20 Acino Pharma AG 21 Alnylam Pharmaceuticals, Inc. 22 Bluebird bio, Inc. 23 Emmaus Medical, Inc. 24 Errant Gene Therapeutics, LLC 25 Gamida Cell Ltd. 26 Incyte Corporation 27 IRBM Science Park SpA 28 Isis Pharmaceuticals, Inc. 29 Johnson & Johnson 30 Protagonist Therapeutics Inc. 31 Sangamo BioSciences, Inc. 32 Thalassemia - Therapeutics Assessment 33 Assessment by Monotherapy Products 33 Assessment by Target 34 Assessment by Mechanism of Action 36 Assessment by Route of Administration 38 Assessment by Molecule Type 40 Drug Profiles 42 ACY-957 - Drug Profile 42 Product Description 42 Mechanism of Action 42 R&D Progress 42 ALN-TMP - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 BB-305 - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 BtX-13 - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 CNTO-530 - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 glutamine - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 luspatercept - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 NiCord - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 PB-04 - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 Peptide Agonists of Hepcidin for Beta-Thalassemia and Juvenile Hemochromatosis - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Project x-1450 - Drug Profile 57 Product Description 57 Mechanism of Action 57 R&D Progress 57 ruxolitinib phosphate - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 Small Molecule to Inhibit Histone Deacetylase for Beta-Thalassemia - Drug Profile 63 Product Description 63 Mechanism of Action 63 R&D Progress 63 sotatercept - Drug Profile 64 Product Description 64 Mechanism of Action 64 R&D Progress 64 Stem Cell Therapy for Beta Thalassemia - Drug Profile 66 Product Description 66 Mechanism of Action 66 R&D Progress 66 Synthetic Peptides Hematological and Genetic Disorders - Drug Profile 67 Product Description 67 Mechanism of Action 67 R&D Progress 67 Thalagen - Drug Profile 69 Product Description 69 Mechanism of Action 69 R&D Progress 69 Thalassemia - Recent Pipeline Updates 70 Thalassemia - Dormant Projects 90 Thalassemia - Product Development Milestones 91 Featured News & Press Releases 91 Feb 04, 2015: Sangamo BioSciences Announces FDA Acceptance of IND to Initiate Clinical Trial of its Novel ZFP Therapeutic for Beta-Thalassemia 91 Dec 08, 2014: Acetylon Pharmaceuticals Presents Positive Preclinical Data on Selective HDAC1/2 Inhibition for the Treatment of Sickle Cell Disease thalassemia 92 Dec 07, 2014: New Data from Luspatercept Phase 2 Clinical Trial in Beta-Thalassemia Presented at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition 92 Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on luspatercept at the 2014 American Society of Hematology Annual Meeting 94 Nov 06, 2014: Celgene To Present Phase II trial Results Update On Luspatercept at American Society of Hematology Annual Meeting 95 Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on sotatercept at the 2014 American Society of Hematology Annual Meeting 95 Oct 01, 2014: Acceleron to Host Educational Webcast on Luspatercept 96 Oct 01, 2014: Acceleron to Host Educational Webcast on Beta-thalassemia 96 Jun 14, 2014: Celgene and Acceleron announces interim data from ACE-536 at the 19th European Hematology Association annual congress 96 Jun 14, 2014: Studies Presented at 19th European Hematology Association Annual Congress Evaluate Activin Receptor Ligand Trap Programs in Beta-Thalassemia 97 Appendix 99 Methodology 99 Coverage 99 Secondary Research 99 Primary Research 99 Expert Panel Validation 99 Contact Us 99 Disclaimer 100
List of Tables Number of Products under Development for Thalassemia, H1 2015 9 Number of Products under Development for Thalassemia - Comparative Analysis, H1 2015 10 Number of Products under Development by Companies, H1 2015 11 Number of Products under Investigation by Universities/Institutes, H1 2015 12 Comparative Analysis by Late Stage Development, H1 2015 13 Comparative Analysis by Clinical Stage Development, H1 2015 14 Comparative Analysis by Early Stage Development, H1 2015 15 Products under Development by Companies, H1 2015 16 Products under Development by Companies, H1 2015 (Contd..1) 17 Products under Investigation by Universities/Institutes, H1 2015 18 Thalassemia - Pipeline by Acceleron Pharma, Inc., H1 2015 19 Thalassemia - Pipeline by Acetylon Pharmaceuticals, Inc., H1 2015 20 Thalassemia - Pipeline by Acino Pharma AG, H1 2015 21 Thalassemia - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 22 Thalassemia - Pipeline by Bluebird bio, Inc., H1 2015 23 Thalassemia - Pipeline by Emmaus Medical, Inc., H1 2015 24 Thalassemia - Pipeline by Errant Gene Therapeutics, LLC, H1 2015 25 Thalassemia - Pipeline by Gamida Cell Ltd., H1 2015 26 Thalassemia - Pipeline by Incyte Corporation, H1 2015 27 Thalassemia - Pipeline by IRBM Science Park SpA, H1 2015 28 Thalassemia - Pipeline by Isis Pharmaceuticals, Inc., H1 2015 29 Thalassemia - Pipeline by Johnson & Johnson, H1 2015 30 Thalassemia - Pipeline by Protagonist Therapeutics Inc., H1 2015 31 Thalassemia - Pipeline by Sangamo BioSciences, Inc., H1 2015 32 Assessment by Monotherapy Products, H1 2015 33 Number of Products by Stage and Target, H1 2015 35 Number of Products by Stage and Mechanism of Action, H1 2015 37 Number of Products by Stage and Route of Administration, H1 2015 39 Number of Products by Stage and Molecule Type, H1 2015 41 Thalassemia Therapeutics - Recent Pipeline Updates, H1 2015 70 Thalassemia - Dormant Projects, H1 2015 90
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