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Friedreich Ataxia - Pipeline Review, H2 2016

Friedreich Ataxia - Pipeline Review, H2 2016

Summary

Global Markets Direct’s, ‘Friedreich Ataxia - Pipeline Review, H2 2016’, provides an overview of the Friedreich Ataxia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Friedreich Ataxia, 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 Friedreich Ataxia 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 Friedreich Ataxia
    • The report reviews pipeline therapeutics for Friedreich Ataxia 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 Friedreich Ataxia therapeutics and enlists all their major and minor projects
    • The report assesses Friedreich Ataxia 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 Friedreich Ataxia
    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 Friedreich Ataxia
    • 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 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


    Introduction
    Global Markets Direct Report Coverage
    Friedreich Ataxia Overview
    Therapeutics Development
    Pipeline Products for Friedreich Ataxia - Overview
    Pipeline Products for Friedreich Ataxia - Comparative Analysis
    Friedreich Ataxia - Therapeutics under Development by Companies
    Friedreich Ataxia - Therapeutics under Investigation by Universities/Institutes
    Friedreich Ataxia - Pipeline Products Glance
    Late Stage Products
    Clinical Stage Products
    Early Stage Products
    Friedreich Ataxia - Products under Development by Companies
    Friedreich Ataxia - Products under Investigation by Universities/Institutes
    Friedreich Ataxia - Companies Involved in Therapeutics Development
    Adverum Biotechnologies, Inc.
    Almirall, S.A.
    BioMarin Pharmaceutical Inc.
    Biovista Inc.
    Catabasis Pharmaceuticals, Inc.
    Edison Pharmaceuticals, Inc.
    Horizon Pharma Plc
    Ixchel Pharma, LLC
    ProQR Therapeutics N.V.
    RaNA Therapeutics, Inc.
    Reata Pharmaceuticals, Inc.
    Retrotope, Inc
    Shire Plc
    STATegics, Inc.
    Voyager Therapeutics, Inc.
    Friedreich Ataxia - Therapeutics Assessment
    Assessment by Monotherapy Products
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Drug Profiles
    AGIL-FA - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ANN-003 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    BVA-202 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    BVA-203 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    CAT-4001 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    dimethyl fumarate - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    interferon gamma-1b - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    interferon gamma-1b - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    IXC-101 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    JOT-101 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    omaveloxolone - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    QRX-604 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Protein for Friedreich Ataxia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RG-3250 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RT-001 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RT-002 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SHP-622 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    STSE-15 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    TAT-FXN - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    vatiquinone - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    VYFXN-01 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Friedreich Ataxia - Dormant Projects
    Friedreich Ataxia - Discontinued Products
    Friedreich Ataxia - Product Development Milestones
    Featured News & Press Releases
    Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia
    May 05, 2016: Horizon Pharma Completes Target Enrollment of 90 Patients for Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich Ataxia
    Jan 19, 2016: FARA Announces Catabasis Pharmaceuticals as the Recipient of the Kyle Bryant Translational Research Award to Evaluate CAT-4001 as a Potential Therapy for Friedreich’s Ataxia
    Aug 05, 2015: Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
    Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia
    Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia
    Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference
    Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia
    Jan 29, 2015: Reata Enrolls First Patient in the MOXIe Study, a Phase 2/3 Study Examining RTA 408 in Friedreich's Ataxia Patients
    Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia
    Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia
    Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies
    Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia
    Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia
    Nov 04, 2013: Edison Pharmaceuticals, FARA, and USF Announce Initiation of EPI-743 Phase 2 Friedreich's Ataxia Clinical Trial in Adults with Point Mutations
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer
    List of Tables
    Number of Products under Development for Friedreich Ataxia, H2 2016
    Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2016
    Number of Products under Development by Companies, H2 2016
    Number of Products under Investigation by Universities/Institutes, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Development, H2 2016
    Products under Development by Companies, H2 2016
    Products under Investigation by Universities/Institutes, H2 2016
    Friedreich Ataxia - Pipeline by Adverum Biotechnologies, Inc., H2 2016
    Friedreich Ataxia - Pipeline by Almirall, S.A., H2 2016
    Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H2 2016
    Friedreich Ataxia - Pipeline by Biovista Inc., H2 2016
    Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H2 2016
    Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H2 2016
    Friedreich Ataxia - Pipeline by Horizon Pharma Plc, H2 2016
    Friedreich Ataxia - Pipeline by Ixchel Pharma, LLC, H2 2016
    Friedreich Ataxia - Pipeline by ProQR Therapeutics N.V., H2 2016
    Friedreich Ataxia - Pipeline by RaNA Therapeutics, Inc., H2 2016
    Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H2 2016
    Friedreich Ataxia - Pipeline by Retrotope, Inc, H2 2016
    Friedreich Ataxia - Pipeline by Shire Plc, H2 2016
    Friedreich Ataxia - Pipeline by STATegics, Inc., H2 2016
    Friedreich Ataxia - Pipeline by Voyager Therapeutics, Inc., H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Stage and Target, H2 2016
    Number of Products by Stage and Mechanism of Action, H2 2016
    Number of Products by Stage and Route of Administration, H2 2016
    Number of Products by Stage and Molecule Type, H2 2016
    Friedreich Ataxia - Dormant Projects, H2 2016
    Friedreich Ataxia - Discontinued Products, H2 2016
    List of Figures
    Number of Products under Development for Friedreich Ataxia, H2 2016
    Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2016
    Number of Products under Development by Companies, H2 2016
    Number of Products under Investigation by Universities/Institutes, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Products, H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Targets, H2 2016
    Number of Products by Stage and Targets, H2 2016
    Number of Products by Top 10 Mechanism of Actions, H2 2016
    Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016
    Number of Products by Routes of Administration, H2 2016
    Number of Products by Stage and Routes of Administration, H2 2016
    Number of Products by Molecule Types, H2 2016
    Number of Products by Stage and Molecule Types, H2 2016

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