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Friedreich Ataxia - Pipeline Review, H1 2015

  • ID: 3266293
  • April 2015
  • 85 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • AAVLife
  • Bioblast Pharma Ltd.
  • Biovista Inc.
  • Horizon Pharma plc
  • MI.TO. Technology S.r.L.
  • Shire Plc
  • MORE

Friedreich Ataxia - Pipeline Review, H1 2015

Summary

This, ‘Friedreich Ataxia - Pipeline Review, H1 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.

This 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 This 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 This team. Drug profiles/records featured in the report undergoes periodic updation following a stringent READ MORE >

Note: Product cover images may vary from those shown
2 of 4

FEATURED COMPANIES

  • AAVLife
  • Bioblast Pharma Ltd.
  • Biovista Inc.
  • Horizon Pharma plc
  • MI.TO. Technology S.r.L.
  • Shire Plc
  • MORE

List of Tables
List of Figures
Introduction
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
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.
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
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
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 (recombinant) - 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
RG-2833 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RG-3250 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RTA-408 - 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
VYFXNO-1 - 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
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
Jan 29, 2015: Reata Enrolls First Patient in the MOXIe Study, a Phase 2/3 Study Examining RTA 408 in Friedreich's Ataxia Patients
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
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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Friedreich Ataxia, H1 2015
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H1 2015
Number of Products under Development by Companies, H1 2015
Number of Products under Investigation by Universities/Institutes, H1 2015
Comparative Analysis by Clinical Stage Development, H1 2015
Comparative Analysis by Early Stage Development, H1 2015
Products under Development by Companies, H1 2015
Products under Development by Companies, H1 2015 (Contd..1)
Products under Investigation by Universities/Institutes, H1 2015
Friedreich Ataxia - Pipeline by AAVLife, H1 2015
Friedreich Ataxia - Pipeline by Bioblast Pharma Ltd., H1 2015
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H1 2015
Friedreich Ataxia - Pipeline by Biovista Inc., H1 2015
Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H1 2015
Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H1 2015
Friedreich Ataxia - Pipeline by Horizon Pharma plc, H1 2015
Friedreich Ataxia - Pipeline by MI.TO. Technology S.r.L., H1 2015
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H1 2015
Friedreich Ataxia - Pipeline by Shire Plc, H1 2015
Friedreich Ataxia - Pipeline by STATegics, Inc., H1 2015
Friedreich Ataxia - Pipeline by Varinel, Inc., H1 2015
Assessment by Monotherapy Products, H1 2015
Number of Products by Stage and Target, H1 2015
Number of Products by Stage and Mechanism of Action, H1 2015
Number of Products by Stage and Route of Administration, H1 2015
Number of Products by Stage and Molecule Type, H1 2015
Friedreich Ataxia Therapeutics - Recent Pipeline Updates, H1 2015
Friedreich Ataxia - Dormant Projects, H1 2015
Friedreich Ataxia - Discontinued Products, H1 2015

List of Figures
Number of Products under Development for Friedreich Ataxia, H1 2015
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H1 2015
Number of Products under Development by Companies, H1 2015
Comparative Analysis by Clinical Stage Development, H1 2015
Comparative Analysis by Early Stage Products, H1 2015
Assessment by Monotherapy Products, H1 2015
Number of Products by Top 10 Targets, H1 2015
Number of Products by Stage and Top 10 Targets, H1 2015
Number of Products by Top 10 Mechanism of Actions, H1 2015
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015
Number of Products by Top 10 Routes of Administration, H1 2015
Number of Products by Stage and Top 10 Routes of Administration, H1 2015
Number of Products by Top 10 Molecule Types, H1 2015
Number of Products by Stage and Top 10 Molecule Types, H1 2015

Note: Product cover images may vary from those shown
3 of 4

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.
Shire Plc
STATegics, Inc.
Varinel, Inc.

Note: Product cover images may vary from those shown
4 of 4
Note: Product cover images may vary from those shown

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