Thalassemia – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Thalassemia – Pipeline Review, H2 2017’, provides an overview of the Thalassemia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Thalassemia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Thalassemia and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

The report provides a snapshot of the global therapeutic landscape of Thalassemia

The report reviews pipeline therapeutics for Thalassemia by companies and universities/research institutes based on information derived from company and industry-specific sources

The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

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

The report reviews key players involved Thalassemia therapeutics and enlists all their major and minor projects

The report assesses Thalassemia therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

The report summarizes all the dormant and discontinued pipeline projects

The report reviews latest news related to pipeline therapeutics for Thalassemia

Reasons to buy

Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

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

Identify and understand important and diverse types of therapeutics under development for Thalassemia

Identify potential new clients or partners in the target demographic

Develop strategic initiatives by understanding the focus areas of leading companies

Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

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

Companies mentioned

Acceleron Pharma Inc

Agios Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

bluebird bio Inc

Cadila Healthcare Ltd

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

Kiadis Pharma NV

La Jolla Pharmaceutical Company

Merck & Co Inc

Protagonist Therapeutics Inc

Sangamo Therapeutics Inc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Thalassemia - Overview 6

Thalassemia - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 11

Products under Development by Companies 12

Products under Development by Universities/Institutes 14

Thalassemia - Therapeutics Assessment 15

Assessment by Target 15

Assessment by Mechanism of Action 17

Assessment by Route of Administration 19

Assessment by Molecule Type 21

Thalassemia - Companies Involved in Therapeutics Development 23

Acceleron Pharma Inc 23

Agios Pharmaceuticals Inc 23

Alnylam Pharmaceuticals Inc 24

bluebird bio Inc 24

Cadila Healthcare Ltd 25

Calimmune Inc 25

CRISPR Therapeutics 26

Editas Medicine Inc 26

Errant Gene Therapeutics LLC 27

Gamida Cell Ltd 27

Gilead Sciences Inc 28

Incyte Corp 28

Ionis Pharmaceuticals Inc 29

IRBM Science Park SpA 29

Kiadis Pharma NV 30

La Jolla Pharmaceutical Company 30

Merck & Co Inc 31

Protagonist Therapeutics Inc 31

Sangamo Therapeutics Inc 32

Thalassemia - Drug Profiles 33

(decitabine + tetrahydrouridine) - Drug Profile 33

ACY-957 - Drug Profile 34

AG-348 - Drug Profile 37

ALN-TMP - Drug Profile 42

ambrisentan - Drug Profile 44

ATIR-201 - Drug Profile 47

BB-305 - Drug Profile 48

benserazide - Drug Profile 57

CAL-H - Drug Profile 59

CordIn - Drug Profile 60

Drugs to Chelate Iron for Bone Degeneration, Postmenopausal Osteoporosis and Thalassemia - Drug Profile 61

Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile 62

Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia - Drug Profile 63

GSK-2696277 - Drug Profile 64

IMR-687 - Drug Profile 65

IONISTMPRSS-6LRx - Drug Profile 66

LJPC-401 - Drug Profile 67

luspatercept - Drug Profile 69

M-009 - Drug Profile 78

M-012 - Drug Profile 79

NiCord - Drug Profile 81

PEG-EPO - Drug Profile 85

PHBB-101 - Drug Profile 86

PTG-300 - Drug Profile 87

RCY-1497 - Drug Profile 88

RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile 89

ruxolitinib phosphate - Drug Profile 90

SCD-101 - Drug Profile 105

sirolimus - Drug Profile 106

Small Molecule to Inhibit Histone Deacetylase 1 for Beta Thalassemia - Drug Profile 107

Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile 108

sotatercept - Drug Profile 109

ST-400 - Drug Profile 114

Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 116

Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile 117

Stem Cell Therapy to Activate Hemoglobin Beta for Beta-Thalassemia - Drug Profile 119

Thalagen - Drug Profile 120

Thalassemia - Dormant Projects 121

Thalassemia - Product Development Milestones 122

Featured News & Press Releases 122

Appendix 137

Methodology 137

Coverage 137

Secondary Research 137

Primary Research 137

Expert Panel Validation 137

Contact Us 137

Disclaimer 138

List of Tables

List of Tables

Number of Products under Development for Thalassemia, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Thalassemia – Pipeline by Acceleron Pharma Inc, H2 2017

Thalassemia – Pipeline by Agios Pharmaceuticals Inc, H2 2017

Thalassemia – Pipeline by Alnylam Pharmaceuticals Inc, H2 2017

Thalassemia – Pipeline by bluebird bio Inc, H2 2017

Thalassemia – Pipeline by Cadila Healthcare Ltd, H2 2017

Thalassemia – Pipeline by Calimmune Inc, H2 2017

Thalassemia – Pipeline by CRISPR Therapeutics, H2 2017

Thalassemia – Pipeline by Editas Medicine Inc, H2 2017

Thalassemia – Pipeline by Errant Gene Therapeutics LLC, H2 2017

Thalassemia – Pipeline by Gamida Cell Ltd, H2 2017

Thalassemia – Pipeline by Gilead Sciences Inc, H2 2017

Thalassemia – Pipeline by Incyte Corp, H2 2017

Thalassemia – Pipeline by Ionis Pharmaceuticals Inc, H2 2017

Thalassemia – Pipeline by IRBM Science Park SpA, H2 2017

Thalassemia – Pipeline by Kiadis Pharma NV, H2 2017

Thalassemia – Pipeline by La Jolla Pharmaceutical Company, H2 2017

Thalassemia – Pipeline by Merck & Co Inc, H2 2017

Thalassemia – Pipeline by Protagonist Therapeutics Inc, H2 2017

Thalassemia – Pipeline by Sangamo Therapeutics Inc, H2 2017

Thalassemia – Dormant Projects, H2 2017

List of Figures

List of Figures

Number of Products under Development for Thalassemia, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Top 10 Targets, H2 2017

Number of Products by Stage and Top 10 Targets, H2 2017

Number of Products by Top 10 Mechanism of Actions, H2 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Top 10 Molecule Types, H2 2017

Number of Products by Stage and Top 10 Molecule Types, H2 2017

    Pricing

Discounts available for multiple purchases.

reportstore@globalmarketsdirect.com
+44 20 7947 2745

Saved reports