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Genethon - Product Pipeline Review - 2015

Published: Feb-2015 | Format: PDF | Global Markets Direct | Number of pages: 34 | Code: MRS - 7032


Global Markets Direct’s, ‘Genethon Product Pipeline Review 2015’, provides an overview of the Genethon’s pharmaceutical research and development focus.


This report provides comprehensive information on the current therapeutic developmental pipeline of Genethon’s, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews latest updates, and featured news and press releases, along with special features on late-stage 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, 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. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.


The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.




The report provides brief overview of Genethon including business description, key information and facts, and its locations and subsidiaries
The report reviews current pipeline of Genethon’s human therapeutic division and enlists all their major and minor projects
The report features product description and descriptive mechanism of action for key pipeline products along with the product’s developmental history and major milestones 
Special feature on out-licensed and partnered product portfolio
The report summarizes all the dormant and discontinued pipeline projects
Latest company statement 
Latest news and deals relating to the Genethon’s pipeline products


Reasons to buy


Evaluate Genethon’s strategic position with total access to detailed information on its product pipeline
Assess the growth potential of Genethon in its therapy areas of focus
Identify new drug targets and therapeutic classes in the Genethon’s R&D portfolio and develop key strategic initiatives to reinforce pipeline in those areas
Exploit in-licensing opportunities by identifying windows of opportunity to fill portfolio gaps
Develop strategic initiatives by understanding the focus areas of Genethon and exploit collaboration and partnership opportunities
Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
Plan mergers and acquisitions effectively by identifying the most promising pipeline of Genethon
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
Explore the dormant and discontinued projects of Genethon and identify potential opportunities in those areas
Avoid Intellectual Property Rights related issues


Table of Contents


Table of Contents 2
List of Tables 4
List of Figures 4
Genethon Snapshot 5
Genethon Overview 5
Key Information 5
Key Facts 5
Genethon - Research and Development Overview 6
Key Therapeutic Areas 6
Genethon - Pipeline Review 8
Pipeline Products by Stage of Development 8
Pipeline Products - Monotherapy 9
Pipeline Products - Partnered Products 10
Partnered Products/Combination Treatment Modalities 11
Genethon - Pipeline Products Glance 12
Genethon - Clinical Stage Pipeline Products 12
Phase I Products/Combination Treatment Modalities 12
Genethon - Early Stage Pipeline Products 13
Preclinical Products/Combination Treatment Modalities 13
Genethon - Drug Profiles 14
G-1XCGD 14
Product Description 14
Mechanism of Action 14
R&D Progress 14
Gene Therapy to Activate WASP for Wiskott-Aldrich Syndrome 15
Product Description 15
Mechanism of Action 15
R&D Progress 15
Gene Therapy for Duchenne Muscular Dystrophy 17
Product Description 17
Mechanism of Action 17
R&D Progress 17
Gene Therapy to Activate Artemis for Radiosensitive Severe Combined Immunodeficiency 18
Product Description 18
Mechanism of Action 18
R&D Progress 18
Gene Therapy to Activate CNTF for Huntington’s Disease 19
Product Description 19
Mechanism of Action 19
R&D Progress 19
Gene Therapy to Activate Dysferlin for Dysferlinopathies 20
Product Description 20
Mechanism of Action 20
R&D Progress 20
Gene Therapy to Activate FANC-A for Fanconi Anemia 21
Product Description 21
Mechanism of Action 21
R&D Progress 21
Gene Therapy to Activate Gamma-Sarcoglycan for Gamma-sarcoglycanopathy 22
Product Description 22
Mechanism of Action 22
R&D Progress 22
Gene Therapy to Activate SGCA for Alpha-sarcoglycanopathy 23
Product Description 23
Mechanism of Action 23
R&D Progress 23
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy 24
Product Description 24
Mechanism of Action 24
R&D Progress 24
Gene Therapy to Activate UGT1A1 for Crigler-Najjar Syndrome 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
Genethon - Pipeline Analysis 26
Genethon - Pipeline Products by Target 26
Genethon - Pipeline Products by Route of Administration 27
Genethon - Pipeline Products by Molecule Type 28
Genethon - Pipeline Products by Mechanism of Action 29
Genethon - Recent Pipeline Updates 30
Genethon - Dormant Projects 31
Genethon - Locations And Subsidiaries 32
Head Office 32


Appendix 33
Methodology 33
Coverage 33
Secondary Research 33
Primary Research 33
Expert Panel Validation 33
Contact Us 33
Disclaimer 34

List of Tables

Genethon, Key Information 5
Genethon, Key Facts 5
Genethon - Pipeline by Indication, 2015 7
Genethon - Pipeline by Stage of Development, 2015 8
Genethon - Monotherapy Products in Pipeline, 2015 9
Genethon - Partnered Products in Pipeline, 2015 10
Genethon - Partnered Products/ Combination Treatment Modalities, 2015 11
Genethon - Phase I, 2015 12
Genethon - Preclinical, 2015 13
Genethon - Pipeline by Target, 2015 26
Genethon - Pipeline by Route of Administration, 2015 27
Genethon - Pipeline by Molecule Type, 2015 28
Genethon - Pipeline Products by Mechanism of Action, 2015 29
Genethon - Recent Pipeline Updates, 2015 30
Genethon - Dormant Developmental Projects,2015 31

List of Figures

Genethon - Pipeline by Top 10 Indication, 2015 7
Genethon - Pipeline by Stage of Development, 2015 8
Genethon - Monotherapy Products in Pipeline, 2015 9
Genethon - Partnered Products in Pipeline, 2015 10
Genethon - Pipeline by Top 10 Target, 2015 26
Genethon - Pipeline by Top 10 Route of Administration, 2015 27
Genethon - Pipeline by Top 10 Molecule Type, 2015 28
Genethon - Pipeline Products by Top 10 Mechanism of Action, 2015 29

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