Thalassemia - Pipeline Review, H1 2017

Published: Mar, 2017 | Pages: 140 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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

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 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