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Friedreich Ataxia - Pipeline Review, H2 2015

Friedreich Ataxia - Pipeline Review, H2 2015

Summary

Global Markets Direct’s, ‘Friedreich Ataxia - Pipeline Review, H2 2015’, provides an overview of the Friedreich Ataxia’s 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.

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.

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 industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration 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
  • 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


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
AAVLife
Bioblast Pharma Ltd.
BioMarin Pharmaceutical Inc.
Biovista Inc.
Catabasis Pharmaceuticals, Inc.
Edison Pharmaceuticals, Inc.
Horizon Pharma Plc
MI.TO. Technology S.r.L.
Reata Pharmaceuticals, Inc.
Retrotope, Inc
Shire Plc
STATegics, Inc.
Varinel, 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
BB-FA - Drug Profile
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
Cell Therapy for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
diazoxide - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Gene Therapy for Friedreich's ataxia - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Gene Therapy 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
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
RG-2833 - 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
SHP-622 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
STSE-15 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
VAR-10100 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
VAR-10300 - 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 - Recent Pipeline Updates
Friedreich Ataxia - Dormant Projects
Friedreich Ataxia - Discontinued Products
Friedreich Ataxia - Product Development Milestones
Featured News & Press Releases
Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Friedreich’s Ataxia at Two Leading Scientific Meetings in June
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 26, 2015: BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in 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
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
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
Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia
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 2015
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Late Stage Development, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Development, H2 2015
Products under Development by Companies, H2 2015
Products under Investigation by Universities/Institutes, H2 2015
Friedreich Ataxia - Pipeline by AAVLife, H2 2015
Friedreich Ataxia - Pipeline by Bioblast Pharma Ltd., H2 2015
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H2 2015
Friedreich Ataxia - Pipeline by Biovista Inc., H2 2015
Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H2 2015
Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H2 2015
Friedreich Ataxia - Pipeline by Horizon Pharma Plc, H2 2015
Friedreich Ataxia - Pipeline by MI.TO. Technology S.r.L., H2 2015
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H2 2015
Friedreich Ataxia - Pipeline by Retrotope, Inc, H2 2015
Friedreich Ataxia - Pipeline by Shire Plc, H2 2015
Friedreich Ataxia - Pipeline by STATegics, Inc., H2 2015
Friedreich Ataxia - Pipeline by Varinel, Inc., H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Stage and Target, H2 2015
Number of Products by Stage and Mechanism of Action, H2 2015
Number of Products by Stage and Route of Administration, H2 2015
Number of Products by Stage and Molecule Type, H2 2015
Friedreich Ataxia Therapeutics - Recent Pipeline Updates, H2 2015
Friedreich Ataxia - Dormant Projects, H2 2015
Friedreich Ataxia - Discontinued Products, H2 2015
List of Figures
Number of Products under Development for Friedreich Ataxia, H2 2015
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Products, H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Top 10 Targets, H2 2015
Number of Products by Stage and Top 10 Targets, H2 2015
Number of Products by Top 10 Mechanism of Actions, H2 2015
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015
Number of Products by Routes of Administration, H2 2015
Number of Products by Stage and Routes of Administration, H2 2015
Number of Products by Molecule Types, H2 2015
Number of Products by Stage and Molecule Types, H2 2015

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