Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fanconi Anemia - Pipeline Review, H2 2016, provides an overview of the Fanconi Anemia (Hematological Disorders) pipeline landscape.
Fanconi anemia (fan-KO-nee uh-NEE-me-uh), or FA, is a rare, inherited blood disorder that leads to bone marrow failure. The disorder also is called Fanconi’s anemia. FA is a type of aplastic anemia. In aplastic anemia, the bone marrow stops making or doesn't make enough of all three types of blood cells. Low levels of the three types of blood cells can harm many of the body's organs, tissues, and systems. Treatment is recommended for significant cytopenias, such as hemoglobin less than 8 g/dL, platelets fewer than 500/μL.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fanconi Anemia - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Fanconi Anemia (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 Fanconi Anemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fanconi Anemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 2, 1, 2 and 1 respectively. Similarly, the Universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 3 molecules, respectively.Fanconi Anemia.
Fanconi Anemia (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 Fanconi Anemia (Hematological Disorders).
- The pipeline guide reviews pipeline therapeutics for Fanconi Anemia (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 Fanconi Anemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Fanconi Anemia (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 Fanconi Anemia (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 Fanconi Anemia (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 Fanconi Anemia (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.
Companies Mentioned
Abeona Therapeutics Inc
Bellicum Pharmaceuticals Inc
Genethon SA
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Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Fanconi Anemia Overview 6
Therapeutics Development 7
Pipeline Products for Fanconi Anemia - Overview 7
Pipeline Products for Fanconi Anemia - Comparative Analysis 8
Fanconi Anemia - Therapeutics under Development by Companies 9
Fanconi Anemia - Therapeutics under Investigation by Universities/Institutes 10
Fanconi Anemia - Pipeline Products Glance 11
Clinical Stage Products 11
Early Stage Products 12
Fanconi Anemia - Products under Development by Companies 13
Fanconi Anemia - Products under Investigation by Universities/Institutes 14
Fanconi Anemia - Companies Involved in Therapeutics Development 15
Abeona Therapeutics Inc 15
Bellicum Pharmaceuticals Inc 16
Genethon SA 17
Fanconi Anemia - Therapeutics Assessment 18
Assessment by Monotherapy Products 18
Assessment by Target 19
Assessment by Mechanism of Action 21
Assessment by Route of Administration 23
Assessment by Molecule Type 25
Drug Profiles 27
ABO-301 - Drug Profile 27
Product Description 27
Mechanism Of Action 27
R&D Progress 27
AD-6626 - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
Cell Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
JP4-039 - Drug Profile 30
Product Description 30
Mechanism Of Action 30
R&D Progress 30
rimiducid - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Small Molecules for Fanconi Anemia - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
Stem Cell Therapy for Fanconi Anemia - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
Stem Cell Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Fanconi Anemia - Dormant Projects 42
Fanconi Anemia - Product Development Milestones 43
Featured News & Press Releases 43
Feb 24, 2016: Abeona Therapeutics to Present on CRISPR/CAS9 Technology Platform Progress at CRISPR Precision Gene Editing Congress 43
Oct 03, 2011: Experimental Drug Could Help Protect Some Cancer Patients From Radiation Side Effects, UPCI Researchers Find 44
Appendix 45
Methodology 45
Coverage 45
Secondary Research 45
Primary Research 45
Expert Panel Validation 45
Contact Us 45
Disclaimer 46
List of Tables
Number of Products under Development for Fanconi Anemia, H2 2016 7
Number of Products under Development for Fanconi Anemia - Comparative Analysis, H2 2016 8
Number of Products under Development by Companies, H2 2016 9
Number of Products under Investigation by Universities/Institutes, H2 2016 10
Comparative Analysis by Clinical Stage Development, H2 2016 11
Comparative Analysis by Early Stage Development, H2 2016 12
Products under Development by Companies, H2 2016 13
Products under Investigation by Universities/Institutes, H2 2016 14
Fanconi Anemia - Pipeline by Abeona Therapeutics Inc, H2 2016 15
Fanconi Anemia - Pipeline by Bellicum Pharmaceuticals Inc, H2 2016 16
Fanconi Anemia - Pipeline by Genethon SA, H2 2016 17
Assessment by Monotherapy Products, H2 2016 18
Number of Products by Stage and Target, H2 2016 20
Number of Products by Stage and Mechanism of Action, H2 2016 22
Number of Products by Stage and Route of Administration, H2 2016 24
Number of Products by Stage and Molecule Type, H2 2016 26
Fanconi Anemia - Dormant Projects, H2 2016 42
List of Figures
Number of Products under Development for Fanconi Anemia, H2 2016 7
Number of Products under Development for Fanconi Anemia - Comparative Analysis, H2 2016 8
Number of Products under Development by Companies, H2 2016 9
Number of Products under Investigation by Universities/Institutes, H2 2016 10
Comparative Analysis by Clinical Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Assessment by Monotherapy Products, H2 2016 18
Number of Products by Targets, H2 2016 19
Number of Products by Stage and Targets, H2 2016 19
Number of Products by Mechanism of Actions, H2 2016 21
Number of Products by Stage and Mechanism of Actions, H2 2016 21
Number of Products by Routes of Administration, H2 2016 23
Number of Products by Stage and Routes of Administration, H2 2016 23
Number of Products by Molecule Types, H2 2016 25
Number of Products by Stage and Molecule Types, H2 2016 25
