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Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Pipeline Review, H2 2016

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Pipeline Review, H2 2016

Summary

Global Markets Direct’s, ‘Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Pipeline Review, H2 2016’, provides in depth analysis on Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted pipeline therapeutics.

The report provides comprehensive information on the Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1), targeted therapeutics, complete with analysis by indications, stage of development, 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 Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics development 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 for Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)
    • The report reviews Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics under development 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 in Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics and enlists all their major and minor projects
    • The report assesses Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics based on 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 and deals related to Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics
    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 the targeted therapy areas and indications for Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)
    • Identify the use of drugs for target identification and drug repurposing
    • 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 Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) development landscape
    • 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


    Introduction
    Global Markets Direct Report Coverage
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) Overview
    Therapeutics Development
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Products under Development by Stage of Development
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Products under Development by Therapy Area
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Products under Development by Indication
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Pipeline Products Glance
    Late Stage Products
    Early Stage Products
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Products under Development by Companies
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Products under Development by Universities/Institutes
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Therapeutics Assessment
    Assessment by Monotherapy/Combination Products
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Companies Involved in Therapeutics Development
    Adverum Biotechnologies, Inc.
    Bio Blast Pharma Ltd.
    ProQR Therapeutics N.V.
    RaNA Therapeutics, Inc.
    Voyager Therapeutics, Inc.
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - 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
    BB-FA - 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
    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
    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
    TAT-FXN - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    VYFXN-01 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Featured News & Press Releases
    Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia
    Aug 05, 2015: Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
    Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Friedreich’s Ataxia at Two Leading Scientific Meetings in June
    Mar 26, 2015: BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in Friedreich's Ataxia
    Dec 17, 2014: BioBlast Receives Notice of Patent Issuance From USPTO for Its Novel Fusion Protein-Based Platform for the Treatment of Friedrich's Ataxia
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer
    List of Tables
    Number of Products under Development for, H2 2016
    Number of Products under Development by Therapy Area, H2 2016
    Number of Products under Development by Indication, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Early Stage Products, H2 2016
    Number of Products under Development by Companies, H2 2016
    Products under Development by Companies, H2 2016
    Number of Products under Investigation by Universities/Institutes, H2 2016
    Products under Investigation by Universities/Institutes, H2 2016
    Assessment by Monotherapy/Combination Products, 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
    Pipeline by Adverum Biotechnologies, Inc., H2 2016
    Pipeline by Bio Blast Pharma Ltd., H2 2016
    Pipeline by ProQR Therapeutics N.V., H2 2016
    Pipeline by RaNA Therapeutics, Inc., H2 2016
    Pipeline by Voyager Therapeutics, Inc., H2 2016
    List of Figures
    Number of Products under Development for, H2 2016
    Number of Products under Development by Therapy Area, H2 2016
    Number of Products under Development by Indication, H2 2016
    Comparative Analysis by Early Stage Products, H2 2016
    Assessment by Monotherapy/Combination Products, H2 2016
    Number of Products by Mechanism of Actions, H2 2016
    Number of Products by Stage and 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 Type, H2 2016

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