Fanconi Anemia – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Fanconi Anemia – Pipeline Review, H2 2016’, 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

Bellicum Pharmaceuticals Inc

Genethon SA

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

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

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

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

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