JSB Market Research: Friedreich Ataxia - Pipeline Review, H2 2014

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Friedreich Ataxia - Pipeline Review, H2 2014 th

On 16 December 2014

Summary Friedreich Ataxia - Pipeline Review, H2 2014, provides an overview of the Friedreich Ataxias therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Friedreich Ataxia, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Friedreich Ataxia and special features on late-stage and discontinued projects.

You can buy this Report @ http://www.jsbmarketresearch.com/healthcaremedical/r-friedreich-ataxia-pipeline-review-h2-2014-140031 Global Markets Directs 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 Directs 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 Directs 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 Email ID- contact@jsbmarketresearch.com Tel No- 91 2241236650 Published by- http:/www.jsbmarketresearch.com/


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. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope - The report provides a snapshot of the global therapeutic landscape of Friedreich Ataxia - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Friedreich Ataxia and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Friedreich Ataxia products under development by companies and universities/research institutes based on information derived from company and industryspecific sources - Pipeline products coverage based on various stages of development ranging from preregistration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Friedreich Ataxia pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products

Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage Email ID- contact@jsbmarketresearch.com Tel No- 91 2241236650 Published by- http:/www.jsbmarketresearch.com/


- Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Friedreich Ataxia - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Friedreich Ataxia 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

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Discounts on Market Research Reports till 31st December, 2014 For more inquiries, contact at +91 - 998 729 5242 / contact@jsbmarketresearch.com Table of Content List of Tables 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Email ID- contact@jsbmarketresearch.com Tel No- 91 2241236650 Published by- http:/www.jsbmarketresearch.com/


Friedreich Ataxia Overview 8 Therapeutics Development 9 Pipeline Products for Friedreich Ataxia - Overview 9 Pipeline Products for Friedreich Ataxia - Comparative Analysis 10 Friedreich Ataxia - Therapeutics under Development by Companies 11 Friedreich Ataxia - Therapeutics under Investigation by Universities/Institutes 13 Friedreich Ataxia - Pipeline Products Glance 14 Clinical Stage Products 14 Early Stage Products 15 Friedreich Ataxia - Products under Development by Companies 16 Friedreich Ataxia - Products under Investigation by Universities/Institutes 18 Friedreich Ataxia - Companies Involved in Therapeutics Development 19 AAVLife 19 BioMarin Pharmaceutical Inc. 20 Biovista Inc. 21 Edison Pharmaceuticals, Inc. 22 Horizon Pharma plc 23 MI.TO. Technology S.r.L. 24 Reata Pharmaceuticals, Inc. 25 Shire Plc 26 STATegics, Inc. 27 Varinel, Inc. 28 Friedreich Ataxia - Therapeutics Assessment 29 Assessment by Monotherapy Products 29 Assessment by Target 30 Assessment by Mechanism of Action 32 Assessment by Route of Administration 34 Assessment by Molecule Type 36 Drug Profiles 38 BB-FA - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 BVA-202 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 BVA-203 - Drug Profile 40 Product Description 40 Email ID- contact@jsbmarketresearch.com Tel No- 91 2241236650 Published by- http:/www.jsbmarketresearch.com/


Mechanism of Action 40 R&D Progress 40 Cell Therapy for Friedreichs Ataxia - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 diazoxide - Drug Profile 42 Product Description 42 Mechanism of Action 42 R&D Progress 42 Gene Therapy for Friedreichs ataxia - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 Gene Therapy for Friedreichs Ataxia - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 Gene Therapy to Activate Frataxin for Friedreichs Ataxia - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 Gene Therapy to Activate Frataxin for Friedrichs Ataxia - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 interferon gamma-1b (recombinant) - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 Oligonucleotide to Activate Frataxin for Friedreichs Ataxia - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 RG-2833 - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 RG-3250 - Drug Profile 52 Email ID- contact@jsbmarketresearch.com Tel No- 91 2241236650 Published by- http:/www.jsbmarketresearch.com/


Product Description 52 Mechanism of Action 52 R&D Progress 52 RTA-408 - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 SHP-622 - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 STSE-15 - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 VAR-10100 - Drug Profile 57 Product Description 57 Mechanism of Action 57 R&D Progress 57 VAR-10300 - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 vatiquinone - Drug Profile 60 Product Description 60 Mechanism of Action 60 R&D Progress 60 VYFXNO-1 - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 Friedreich Ataxia - Recent Pipeline Updates 63 Friedreich Ataxia - Dormant Projects 70 Friedreich Ataxia - Discontinued Products 71 Friedreich Ataxia - Product Development Milestones 72 Featured News & Press Releases 72 Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreichs Ataxia 72 Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon Email ID- contact@jsbmarketresearch.com Tel No- 91 2241236650 Published by- http:/www.jsbmarketresearch.com/


gamma-1b) in Friedreichs Ataxia 73 Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreichs Ataxia and Mitochondrial Myopathies 73 Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals EPI-743 for Friedreichs Ataxia 74 Feb 04, 2014: FDA Grants Edison Pharmaceuticals EPI-743 Orphan Status for Friedreichs Ataxia 75 Nov 04, 2013: Edison Pharmaceuticals, FARA, and USF Announce Initiation of EPI-743 Phase 2 Friedreichs Ataxia Clinical Trial in Adults with Point Mutations 75 Jan 07, 2013: Edison Pharma Initiates Phase IIb Clinical Trial Of EPI-743 In Friedreich Ataxia 76 Mar 15, 2012: Repligen Initiates Phase I Clinical Trial Of RG2833 In Patients With Friedreichs Ataxia 76 May 24, 2010: Repligen Receives FDA Orphan Drug Designation For RG2833 For Treatment Of Friedreichs Ataxia 78 May 13, 2010: Repligen Files Investigational New Drug Application with FDA for First Drug Targeting The Core Genetic Defect Of Friedreichs Ataxia 78 Appendix 80 Methodology 80 Coverage 80 Secondary Research 80 Primary Research 80 Expert Panel Validation 80 Contact Us 81 Disclaimer 81 List Of Tables Number of Products under Development for Friedreich Ataxia, H2 2014 9 Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2014 10 Number of Products under Development by Companies, H2 2014 12 Number of Products under Investigation by Universities/Institutes, H2 2014 13 Comparative Analysis by Clinical Stage Development, H2 2014 14 Comparative Analysis by Early Stage Development, H2 2014 15 Products under Development by Companies, H2 2014 16 Products under Development by Companies, H2 2014 (Contd..1) 17 Products under Investigation by Universities/Institutes, H2 2014 18 Friedreich Ataxia - Pipeline by AAVLife, H2 2014 19 Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H2 2014 20 Email ID- contact@jsbmarketresearch.com Tel No- 91 2241236650 Published by- http:/www.jsbmarketresearch.com/


Friedreich Ataxia - Pipeline by Biovista Inc., H2 2014 21 Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H2 2014 22 Friedreich Ataxia - Pipeline by Horizon Pharma plc, H2 2014 23 Friedreich Ataxia - Pipeline by MI.TO. Technology S.r.L., H2 2014 24 Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H2 2014 25 Friedreich Ataxia - Pipeline by Shire Plc, H2 2014 26 Friedreich Ataxia - Pipeline by STATegics, Inc., H2 2014 27 Friedreich Ataxia - Pipeline by Varinel, Inc., H2 2014 28 Assessment by Monotherapy Products, H2 2014 29 Number of Products by Stage and Target, H2 2014 31 Number of Products by Stage and Mechanism of Action, H2 2014 33 Number of Products by Stage and Route of Administration, H2 2014 35 Number of Products by Stage and Molecule Type, H2 2014 37 Friedreich Ataxia Therapeutics - Recent Pipeline Updates, H2 2014 63 Friedreich Ataxia - Dormant Projects, H2 2014 70 Friedreich Ataxia - Discontinued Products, H2 2014 71 List Of Figures Number of Products under Development for Friedreich Ataxia, H2 2014 9 Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2014 10 Number of Products under Development by Companies, H2 2014 11 Comparative Analysis by Clinical Stage Development, H2 2014 14 Comparative Analysis by Early Stage Products, H2 2014 15 Assessment by Monotherapy Products, H2 2014 29 Number of Products by Top 10 Targets, H2 2014 30 Number of Products by Stage and Top 10 Targets, H2 2014 31 Number of Products by Top 10 Mechanism of Actions, H2 2014 32 Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014 33 Number of Products by Top 10 Routes of Administration, H2 2014 34 Number of Products by Stage and Top 10 Routes of Administration, H2 2014 35 Number of Products by Top 10 Molecule Types, H2 2014 36 Number of Products by Stage and Top 10 Molecule Types, H2 2014 37

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