Genethon SA - Product Pipeline Review

Global Markets Direct’s, ‘Genethon SA - Product Pipeline Review - 2016’, provides an overview of the Genethon SA’s pharmaceutical research and development focus.

The report provides comprehensive information on the therapeutics under development by Genethon SA, 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 the 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 pipeline therapeutic landscape of Genethon SA

The report provides overview of Genethon SA including its business description, key facts, and locations and subsidiaries

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 covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

The report assesses Genethon SA’s pipeline therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

The report features Genethon SA’s out-licensed and partnered product portfolio and summarizes its dormant and discontinued projects

Reasons to buy

Evaluate Genethon SA’s strategic position with total access to detailed information on its product pipeline

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 Genethon SA

Identify potential new clients or partners in the target demographic

Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

Devise corrective measures for pipeline projects by understanding Genethon SA’s pipeline depth and focus of pipeline 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

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

Genethon SA ...

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Genethon SA Snapshot 6

Genethon SA Overview 6

Key Facts 6

Genethon SA - Research and Development Overview 7

Key Therapeutic Areas 7

Genethon SA - Pipeline Review 10

Pipeline Products by Stage of Development 10

Pipeline Products - Monotherapy 11

Pipeline Products - Partnered Products 12

Partnered Products/Combination Treatment Modalities 13

Genethon SA - Pipeline Products Glance 14

Genethon SA - Clinical Stage Pipeline Products 14

Phase II Products/Combination Treatment Modalities 14

Genethon SA - Early Stage Pipeline Products 15

Preclinical Products/Combination Treatment Modalities 15

Discovery Products/Combination Treatment Modalities 16

Genethon SA - Drug Profiles 17

Cell Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile 17

Product Description 17

Mechanism Of Action 17

R&D Progress 17

CPK-850 - Drug Profile 18

Product Description 18

Mechanism Of Action 18

R&D Progress 18

G-1XCGD - Drug Profile 19

Product Description 19

Mechanism Of Action 19

R&D Progress 19

Gene Therapy for Duchenne Muscular Dystrophy - Drug Profile 21

Product Description 21

Mechanism Of Action 21

R&D Progress 21

Gene Therapy for Sickle Cell Disease - Drug Profile 22

Product Description 22

Mechanism Of Action 22

R&D Progress 22

Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 23

Product Description 23

Mechanism Of Action 23

R&D Progress 23

Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 24

Product Description 24

Mechanism Of Action 24

R&D Progress 24

Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile 25

Product Description 25

Mechanism Of Action 25

R&D Progress 25

Gene Therapy to Activate Gamma-Sarcoglycan for Gamma Sarcoglycanopathy - Drug Profile 26

Product Description 26

Mechanism Of Action 26

R&D Progress 26

Gene Therapy to Activate IL2RG for SCID-X1 - Drug Profile 28

Product Description 28

Mechanism Of Action 28

R&D Progress 28

Gene Therapy to Activate SGCA for Alpha-Sarcoglycanopathy - Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

Gene Therapy to Activate UGT1A1 for Crigler-Najjar Syndrome - Drug Profile 31

Product Description 31

Mechanism Of Action 31

R&D Progress 31

Stem Cell Therapy to Activate Artemis for Radiosensitive Severe Combined Immunodeficiency - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

Stem Cell Therapy to Activate WASP for Wiskott-Aldrich Syndrome - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Genethon SA - Pipeline Analysis 35

Genethon SA - Pipeline Products by Target 35

Genethon SA - Pipeline Products by Route of Administration 36

Genethon SA - Pipeline Products by Molecule Type 37

Genethon SA - Pipeline Products by Mechanism of Action 38

Genethon SA - Dormant Projects 39

Genethon SA - Locations And Subsidiaries 40

Head Office 40

Appendix 41

Methodology 41

Coverage 41

Secondary Research 41

Primary Research 41

Expert Panel Validation 41

Contact Us 41

Disclaimer 42

List of Tables

List of Tables

Genethon SA, Key Facts 6

Genethon SA - Pipeline by Indication, 2016 9

Genethon SA - Pipeline by Stage of Development, 2016 10

Genethon ...

List of Tables

Genethon SA, Key Facts 6

Genethon SA - Pipeline by Indication, 2016 9

Genethon SA - Pipeline by Stage of Development, 2016 10

Genethon SA - Monotherapy Products in Pipeline, 2016 11

Genethon SA - Partnered Products in Pipeline, 2016 12

Genethon SA - Partnered Products/ Combination Treatment Modalities, 2016 13

Genethon SA - Phase II, 2016 14

Genethon SA - Preclinical, 2016 15

Genethon SA - Discovery, 2016 16

Genethon SA - Pipeline by Target, 2016 35

Genethon SA - Pipeline by Route of Administration, 2016 36

Genethon SA - Pipeline by Molecule Type, 2016 37

Genethon SA - Pipeline Products by Mechanism of Action, 2016 38

Genethon SA - Dormant Developmental Projects,2016 39

List of Figures

List of Figures

Genethon SA - Pipeline by Top 10 Indication, 2016 8

Genethon SA - Pipeline by Stage of Development, 2016 10

Genethon SA - Monotherapy Products ...

List of Figures

Genethon SA - Pipeline by Top 10 Indication, 2016 8

Genethon SA - Pipeline by Stage of Development, 2016 10

Genethon SA - Monotherapy Products in Pipeline, 2016 11

Genethon SA - Partnered Products in Pipeline, 2016 12

Genethon SA - Pipeline by Top 10 Target, 2016 35

Genethon SA - Pipeline by Route of Administration, 2016 36

Genethon SA - Pipeline by Molecule Type, 2016 37

Genethon SA - Pipeline Products by Top 10 Mechanism of Action, 2016 38

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