Thalassemia-Pipeline Review, H1 2017

Thalassemia-Pipeline Review, H1 2017


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Executive Summary

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.



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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 -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 -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 -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

List of Figures

Number of Products under Development for Thalassemia, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Top 10 Targets, H1 2017

Number of Products by Stage and Top 10 Targets, H1 2017

Number of Products by Top 10 Mechanism of Actions, H1 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Top 10 Molecule Types, H1 2017

Number of Products by Stage and Top 10 Molecule Types, H1 2017

List of Tables

Number of Products under Development for Thalassemia, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Thalassemia-Pipeline by Acceleron Pharma Inc, H1 2017

Thalassemia-Pipeline by Agios Pharmaceuticals Inc, H1 2017

Thalassemia-Pipeline by Alnylam Pharmaceuticals Inc, H1 2017

Thalassemia-Pipeline by bluebird bio Inc, H1 2017

Thalassemia-Pipeline by Calimmune Inc, H1 2017

Thalassemia-Pipeline by CRISPR Therapeutics, H1 2017

Thalassemia-Pipeline by Editas Medicine Inc, H1 2017

Thalassemia-Pipeline by Errant Gene Therapeutics LLC, H1 2017

Thalassemia-Pipeline by Gamida Cell Ltd, H1 2017

Thalassemia-Pipeline by Gilead Sciences Inc, H1 2017

Thalassemia-Pipeline by Incyte Corp, H1 2017

Thalassemia-Pipeline by Ionis Pharmaceuticals Inc, H1 2017

Thalassemia-Pipeline by IRBM Science Park SpA, H1 2017

Thalassemia-Pipeline by Johnson & Johnson, H1 2017

Thalassemia-Pipeline by Kiadis Pharma NV, H1 2017

Thalassemia-Pipeline by La Jolla Pharmaceutical Company, H1 2017

Thalassemia-Pipeline by Merck & Co Inc, H1 2017

Thalassemia-Pipeline by PharmaEssentia Corp, H1 2017

Thalassemia-Pipeline by Protagonist Therapeutics Inc, H1 2017

Thalassemia-Pipeline by Sangamo Therapeutics Inc, H1 2017

Thalassemia-Pipeline by Zydus Cadila Healthcare Ltd, H1 2017

Thalassemia-Dormant Projects, H1 2017

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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 Therapeutic Products under Development, Key Players in Thalassemia Therapeutics, Thalassemia Pipeline Overview, Thalassemia Pipeline, Thalassemia Pipeline Assessment

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