Thalassemia – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Thalassemia – Pipeline Review, H1 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

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

Table of Contents

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

List of Tables

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

List of Figures

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

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