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Thalassemia - Pipeline Review, H2 2015

Thalassemia - Pipeline Review, H2 2015

Summary

Global Markets Direct’s, ‘Thalassemia - Pipeline Review, H2 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


Introduction
Global Markets Direct Report Coverage
Thalassemia Overview
Therapeutics Development
Pipeline Products for Thalassemia - Overview
Pipeline Products for Thalassemia - Comparative Analysis
Thalassemia - Therapeutics under Development by Companies
Thalassemia - Therapeutics under Investigation by Universities/Institutes
Thalassemia - Pipeline Products Glance
Late Stage Products
Clinical Stage Products
Early Stage Products
Thalassemia - Products under Development by Companies
Thalassemia - Products under Investigation by Universities/Institutes
Thalassemia - Companies Involved in Therapeutics Development
Acceleron Pharma, Inc.
Acetylon Pharmaceuticals, Inc.
Acino Pharma AG
Alnylam Pharmaceuticals, Inc.
Bluebird bio, Inc.
Emmaus Medical, Inc.
Errant Gene Therapeutics, LLC
Gamida Cell Ltd.
Incyte Corporation
IRBM Science Park SpA
Isis Pharmaceuticals, Inc.
Johnson & Johnson
Kiadis Pharma B.V.
La Jolla Pharmaceutical Company
PharmaEssentia Corporation
Protagonist Therapeutics Inc.
Sangamo BioSciences, Inc.
Thalassemia - Therapeutics Assessment
Assessment by Monotherapy Products
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Drug Profiles
ACY-957 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ALN-TMP - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ATIR-201 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
BB-305 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
BtX-13 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
CNTO-530 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
CordIn - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
glutamine - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ISIS-TMPRSS6LRx - Drug Profile
Product Description
Mechanism of Action
R&D Progress
LJPC-401 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
luspatercept - Drug Profile
Product Description
Mechanism of Action
R&D Progress
M-012 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
NiCord - Drug Profile
Product Description
Mechanism of Action
R&D Progress
PB-04 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Peptide Agonists of Hepcidin for Beta-Thalassemia and Juvenile Hemochromatosis - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Project x-1450 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ruxolitinib phosphate - Drug Profile
Product Description
Mechanism of Action
R&D Progress
SCD-101 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecule to Inhibit Histone Deacetylase 1 for Beta-Thalassemia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile
Product Description
Mechanism of Action
R&D Progress
sotatercept - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Stem Cell Therapy for Beta Thalassemia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Stem Cell Therapy to Activate Beta Globin for Thalassemia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Thalagen - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Thalassemia - Recent Pipeline Updates
Thalassemia - Dormant Projects
Thalassemia - Product Development Milestones
Featured News & Press Releases
Aug 11, 2015: La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401
Jun 12, 2015: Acceleron Announces New Luspatercept Phase 2 Clinical Results at the 20th Congress of the European Hematology Association
Jun 09, 2015: Acceleron Pharma to Host Conference Call and Webcast to Review New Luspatercept Data Presented at the 20th Congress of the European Hematology Association
May 21, 2015: Acceleron to Present Luspatercept Clinical Trials at the 20th Congress of the European Hematology Association
May 18, 2015: FDA Fast Track Designation Granted to Luspatercept for the Treatment of Patients with Beta-Thalassemia
May 13, 2015: Sangamo BioSciences Announces Joint Steering Committee Decision To Consolidate ZFP Therapeutic Strategy For Hemoglobinopathies
Mar 31, 2015: Emmaus Life Sciences to Present Phase 3 Sickle Cell Disease Clinical Trial Data
Feb 04, 2015: Sangamo BioSciences Announces FDA Acceptance of IND to Initiate Clinical Trial of its Novel ZFP Therapeutic for Beta-Thalassemia
Dec 08, 2014: Acetylon Pharmaceuticals Presents Positive Preclinical Data on Selective HDAC1/2 Inhibition for the Treatment of Sickle Cell Disease/ß-thalassemia
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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development for Thalassemia, H2 2015
Number of Products under Development for Thalassemia - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Late Stage Development, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Development, H2 2015
Products under Development by Companies, H2 2015
Products under Development by Companies, H2 2015 (Contd..1)
Products under Investigation by Universities/Institutes, H2 2015
Thalassemia - Pipeline by Acceleron Pharma, Inc., H2 2015
Thalassemia - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2015
Thalassemia - Pipeline by Acino Pharma AG, H2 2015
Thalassemia - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2015
Thalassemia - Pipeline by Bluebird bio, Inc., H2 2015
Thalassemia - Pipeline by Emmaus Medical, Inc., H2 2015
Thalassemia - Pipeline by Errant Gene Therapeutics, LLC, H2 2015
Thalassemia - Pipeline by Gamida Cell Ltd., H2 2015
Thalassemia - Pipeline by Incyte Corporation, H2 2015
Thalassemia - Pipeline by IRBM Science Park SpA, H2 2015
Thalassemia - Pipeline by Isis Pharmaceuticals, Inc., H2 2015
Thalassemia - Pipeline by Johnson & Johnson, H2 2015
Thalassemia - Pipeline by Kiadis Pharma B.V., H2 2015
Thalassemia - Pipeline by La Jolla Pharmaceutical Company, H2 2015
Thalassemia - Pipeline by PharmaEssentia Corporation, H2 2015
Thalassemia - Pipeline by Protagonist Therapeutics Inc., H2 2015
Thalassemia - Pipeline by Sangamo BioSciences, Inc., H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Stage and Target, H2 2015
Number of Products by Stage and Mechanism of Action, H2 2015
Number of Products by Stage and Route of Administration, H2 2015
Number of Products by Stage and Molecule Type, H2 2015
Thalassemia Therapeutics - Recent Pipeline Updates, H2 2015
Thalassemia - Dormant Projects, H2 2015
List of Figures
Number of Products under Development for Thalassemia, H2 2015
Number of Products under Development for Thalassemia - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Products, H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Top 10 Targets, H2 2015
Number of Products by Stage and Top 10 Targets, H2 2015
Number of Products by Top 10 Mechanism of Actions, H2 2015
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015
Number of Products by Top 10 Routes of Administration, H2 2015
Number of Products by Stage and Top 10 Routes of Administration, H2 2015
Number of Products by Top 10 Molecule Types, H2 2015
Number of Products by Stage and Top 10 Molecule Types, H2 2015

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