Fanconi Anemia – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Fanconi Anemia – Pipeline Review, H2 2017’, provides an overview of the Fanconi Anemia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Fanconi Anemia, 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 Fanconi Anemia 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 Fanconi Anemia

The report reviews pipeline therapeutics for Fanconi Anemia 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 Fanconi Anemia therapeutics and enlists all their major and minor projects

The report assesses Fanconi Anemia 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 Fanconi Anemia

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 Fanconi Anemia

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 Fanconi Anemia 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

Abeona Therapeutics Inc

Genethon SA

Novartis AG

Table of Contents

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

Fanconi Anemia - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 9

Products under Development by Companies 10

Products under Development by Universities/Institutes 11

Fanconi Anemia - Therapeutics Assessment 12

Assessment by Target 12

Assessment by Mechanism of Action 14

Assessment by Route of Administration 16

Assessment by Molecule Type 18

Fanconi Anemia - Companies Involved in Therapeutics Development 20

Abeona Therapeutics Inc 20

Genethon SA 20

Novartis AG 20

Fanconi Anemia - Drug Profiles 22

ABO-301 - Drug Profile 22

Product Description 22

Mechanism Of Action 22

R&D Progress 22

AD-6626 - Drug Profile 23

Product Description 23

Mechanism Of Action 23

R&D Progress 23

eltrombopag olamine - Drug Profile 24

Product Description 24

Mechanism Of Action 24

R&D Progress 24

EXG-34217 - Drug Profile 31

Product Description 31

Mechanism Of Action 31

R&D Progress 31

Fancalen - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

JP4-039 - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Small Molecules for Fanconi Anemia - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

Stem Cell Therapy for Fanconi Anemia - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

Stem Cell Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

Stem Cell Therapy to Activate FANCA Protein 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

Fanconi Anemia - Dormant Projects 39

Fanconi Anemia - Product Development Milestones 40

Featured News & Press Releases 40

Feb 24, 2016: Abeona Therapeutics to Present on CRISPR/CAS9 Technology Platform Progress at CRISPR Precision Gene Editing Congress 40

Oct 03, 2011: Experimental Drug Could Help Protect Some Cancer Patients From Radiation Side Effects, UPCI Researchers Find 40

Appendix 42

Methodology 42

Coverage 42

Secondary Research 42

Primary Research 42

Expert Panel Validation 42

Contact Us 42

Disclaimer 43

List of Tables

List of Tables

Number of Products under Development for Fanconi Anemia, H2 2017 7

Number of Products under Development by Companies, H2 2017 8

Number of Products under Development by Universities/Institutes, H2 2017 9

Products under Development by Companies, H2 2017 10

Products under Development by Universities/Institutes, H2 2017 11

Number of Products by Stage and Target, H2 2017 13

Number of Products by Stage and Mechanism of Action, H2 2017 15

Number of Products by Stage and Route of Administration, H2 2017 17

Number of Products by Stage and Molecule Type, H2 2017 19

Fanconi Anemia – Pipeline by Abeona Therapeutics Inc, H2 2017 20

Fanconi Anemia – Pipeline by Genethon SA, H2 2017 20

Fanconi Anemia – Pipeline by Novartis AG, H2 2017 21

Fanconi Anemia – Dormant Projects, H2 2017 39

List of Figures

List of Figures

Number of Products under Development for Fanconi Anemia, H2 2017 7

Number of Products under Development by Companies, H2 2017 8

Number of Products under Development by Universities/Institutes, H2 2017 9

Number of Products by Targets, H2 2017 12

Number of Products by Stage and Targets, H2 2017 12

Number of Products by Mechanism of Actions, H2 2017 14

Number of Products by Stage and Mechanism of Actions, H2 2017 14

Number of Products by Routes of Administration, H2 2017 16

Number of Products by Stage and Routes of Administration, H2 2017 16

Number of Products by Molecule Types, H2 2017 18

Number of Products by Stage and Molecule Types, H2 2017 18

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