Bharat Book

Sickle Cell Disease - Pipeline Review, H2 2013

Press Release   •   Dec 04, 2013 09:45 IST


Dec 04, 2013 : Bharat Book Bureau presents the new report, on 'Sickle Cell Disease - Pipeline Review, H2 2013'  provides an overview of the indication’s therapeutic pipeline It also reviews key players involved in the therapeutic development for Sickle Cell Disease. Sickle Cell Disease - Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team.

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


- A snapshot of the global therapeutic scenario for Sickle Cell Disease.
- A review of the Sickle Cell Disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Sickle Cell Disease pipeline on the basis of route of administration and molecule type.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Sickle Cell Disease.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Sickle Cell Disease 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

Johnson & Johnson
Medinox, Inc.
Sangart, Inc.
Daiichi Sankyo Company, Ltd
Sangamo BioSciences, Inc.
Gamida Cell Ltd.
Novartis AG
Astellas Pharma Inc.
ADVENTRX Pharmaceuticals
Alnylam Pharmaceuticals, Inc
Cellerant Therapeutics, Inc.
Advinus Therapeutics Pvt. Ltd.
Prolong Pharmaceuticals
SynZyme Technologies, LLC
ERYtech Pharma
Archemix Corp.
HemaQuest Pharmaceuticals, Inc.
GENETIX Pharmaceuticals, Inc.
Morphogenesis, Inc.
NKT Therapeutics, Inc.
Acetylon Pharmaceuticals, Inc.
Emmaus Medical, Inc.
AesRx, LLC.
Errant Gene Therapeutics, LLC

Table of contents :

Table of Contents 2
List of Tables 7
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Sickle Cell Disease Overview 10
Therapeutics Development 11
An Overview of Pipeline Products for Sickle Cell Disease 11
Sickle Cell Disease Therapeutics under Development by Companies 13
Sickle Cell Disease Therapeutics under Investigation by Universities/Institutes 16
Late Stage Products 17
Comparative Analysis 17
Mid Clinical Stage Products 18
Comparative Analysis 18
Early Clinical Stage Products 19
Comparative Analysis 19
Discovery and Pre-Clinical Stage Products 20
Comparative Analysis 20
Sickle Cell Disease Therapeutics - Products under Development by Companies 21
Sickle Cell Disease Therapeutics - Products under Investigation by Universities/Institutes 22
Companies Involved in Sickle Cell Disease Therapeutics Development 23
Johnson & Johnson 23
Medinox, Inc. 24
Sangart, Inc. 25
Daiichi Sankyo Company, Ltd 26
Sangamo BioSciences, Inc. 27
Gamida Cell Ltd. 28
Novartis AG 29
Astellas Pharma Inc. 30
ADVENTRX Pharmaceuticals 31
Alnylam Pharmaceuticals, Inc 32
Cellerant Therapeutics, Inc. 33
Advinus Therapeutics Pvt. Ltd. 34
Prolong Pharmaceuticals 35
SynZyme Technologies, LLC 36
ERYtech Pharma 37
Archemix Corp. 38
HemaQuest Pharmaceuticals, Inc. 39
GENETIX Pharmaceuticals, Inc. 40
Morphogenesis, Inc. 41
NKT Therapeutics, Inc. 42
Acetylon Pharmaceuticals, Inc. 43
Emmaus Medical, Inc. 44
AesRx, LLC. 45
Errant Gene Therapeutics, LLC 46
Sickle Cell Disease - Therapeutics Assessment 47
Assessment by Monotherapy Products 47
Assessment by Combination Products 48
Assessment by Route of Administration 49
Assessment by Molecule Type 51
Drug Profiles 53
panobinostat - Drug Profile 53
Product Description 53

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