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Thalassemia - Pipeline Review, H1 2017
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Published by Global Markets Direct: 22 Mar 2017 | 50192 | In Stock
Related Topics: Healthcare

Introduction

Thalassemia - Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Thalassemia - Pipeline Review, H1 2017, provides an overview of the Thalassemia (Hematological Disorders) pipeline landscape.

Thalassemia is a group of inherited blood disorders that affect the body's ability to produce hemoglobin and red blood cells. Symptoms include fussiness, paleness, frequent infections, failure to thrive, poor appetite and jaundice. Predisposing factors include family history. Treatment includes blood transfusions and bone marrow transplant.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Thalassemia - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Thalassemia (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Thalassemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Thalassemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 5, 6, 17 and 4 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 2 and 1 molecules, respectively.

Thalassemia (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Thalassemia (Hematological Disorders).

- The pipeline guide reviews pipeline therapeutics for Thalassemia (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

- The pipeline guide reviews key companies involved in Thalassemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Thalassemia (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

- The pipeline guide reviews latest news related to pipeline therapeutics for Thalassemia (Hematological Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Find and recognize significant and varied types of therapeutics under development for Thalassemia (Hematological Disorders).

- Classify potential new clients or partners in the target demographic.

- Develop tactical initiatives by understanding the focus areas of leading companies.

- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.

- Formulate corrective measures for pipeline projects by understanding Thalassemia (Hematological Disorders) 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.

- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents
for Thalassemia - Pipeline Review, H1 2017

  • Table of Contents

    List of Tables

    List of Figures

    Introduction

    Global Markets Direct Report Coverage

    Thalassemia - Overview

    Thalassemia - Therapeutics Development

    Pipeline Overview

    Pipeline by Companies

    Pipeline by Universities/Institutes

    Products under Development by Companies

    Products under Development by Universities/Institutes

    Thalassemia - Therapeutics Assessment

    Assessment by Target

    Assessment by Mechanism of Action

    Assessment by Route of Administration

    Assessment by Molecule Type

    Thalassemia - Companies Involved in Therapeutics Development

    Acceleron Pharma Inc

    Agios Pharmaceuticals Inc

    Alnylam Pharmaceuticals Inc

    bluebird bio Inc

    Calimmune Inc

    CRISPR Therapeutics

    Editas Medicine Inc

    Errant Gene Therapeutics LLC

    Gamida Cell Ltd

    Gilead Sciences Inc

    Incyte Corp

    Ionis Pharmaceuticals Inc

    IRBM Science Park SpA

    Johnson & Johnson

    Kiadis Pharma NV

    La Jolla Pharmaceutical Company

    Merck & Co Inc

    PharmaEssentia Corp

    Protagonist Therapeutics Inc

    Sangamo Therapeutics Inc

    Zydus Cadila Healthcare Ltd

    Thalassemia - Drug Profiles

    (decitabine + tetrahydrouridine) - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    ACY-957 - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    AG-348 - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    ALN-TMP - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    ambrisentan - 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

    benserazide - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    CAL-H - 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

    Drugs to Chelate Iron for Bone Degeneration, Postmenopausal Osteoporosis and 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

    Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    GSK-2696277 - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    IMR-687 - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    IONISTMPRSS-6LRx - 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-009 - 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

    PEG-EPO - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    PHBB-101 - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    PTG-300 - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    RCY-1497 - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - 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

    sirolimus - 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 for Beta Thalassemia and Sickle Cell Disease - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    Thalagen - Drug Profile

    Product Description

    Mechanism Of Action

    R&D Progress

    Thalassemia - Dormant Projects

    Thalassemia - Product Development Milestones

    Featured News & Press Releases

    Feb 06, 2017: Kiadis Pharma Provides Update on ATIR201

    Dec 15, 2016: Kiadis Pharma announces initiation of Phase I/II clinical trial with ATIR201 for thalassemia

    Dec 14, 2016: bluebird bio Announces First Patient Treated with LentiGlobin Drug Product in Northstar-2 (HGB-207) Phase 3 Trial of Patients with Transfusion-Dependent ß-Thalassemia

    Dec 06, 2016: bluebird bio Provides Updates on HSC Gene Therapy Programs

    Dec 05, 2016: Acceleron and Celgene Announce Updated Results from Phase 2 Studies of Luspatercept in Beta-Thalassemia Presented at the 58th Annual Meeting of the American Society of Hematology

    Dec 04, 2016: Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Gamma Globin (HBG) Protein Expression for the Treatment of Sickle Cell Disease and Beta-Thalassemia

    Dec 03, 2016: bluebird bio Presents New Data from HGB-205 Study of LentiGlobin Drug Product in Patients with Transfusion-Dependent ß-Thalassemia and Severe Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting

    Dec 02, 2016: Acetylon to present data on ACY-957 at the American Society of Hematology Annual Meeting

    Nov 21, 2016: CRISPR Therapeutics Announces Two Presentations Demonstrating the Potential for CRISPR Gene Editing To Treat Sickle Cell Disease and ß-Thalassemia

    Nov 03, 2016: bluebird bio to Present New Data from Three LentiGlobin Clinical Studies at American Society of Hematology (ASH) Annual Meeting

    Oct 13, 2016: bluebird bio Provides Update on LentiGlobin Programs and Research and Development Strategy at Gene Therapy Day

    Sep 21, 2016: LentiGlobin Investigational Gene Therapy for Transfusion-Dependent Beta-Thalassemia Accepted into European Medicines Agency’s PRIME Program

    Sep 08, 2016: bluebird bio Opens Phase 3 Study of LentiGlobin Drug Product in Patients with Transfusion-Dependent Beta-Thalassemia

    Sep 07, 2016: La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401

    Appendix

    Methodology

    Coverage

    Secondary Research

    Primary Research

    Expert Panel Validation

    Contact Us

    Disclaimer

Additional Details

Publisher

Global Markets Direct

Publisher Information

Reference

50192 | GMDHC9080IDB

Number of Pages

140

Report Format

PDF

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