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2020 Friedreich Ataxia Drug Pipeline Report- Current Status, Phase, Mechanism, Route of Administration, and Companies, of Pre-Clinical And Clinical Drugs

February 2020 | 102 pages | ID: 2C67BE6E4598EN
VPAResearch

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The 2020 Friedreich Ataxia pipeline report presents a comprehensive overview of the research and development of Friedreich Ataxia drug candidates. It presents drugs in development that could potentially reach the market in the next 5 to 10 years: Five drugs in Research phase, 10 drugs in pre-clinical phase, four drugs in Phase 1, three drugs in Phase 2, and two drugs in Phase 3

As of February 2020, the Friedreich Ataxia pipeline remains robust with 25 therapeutic candidates under development. An increasing number of companies are actively participating in the development of Friedreich Ataxia treatment. Diverse types of targeted therapies are being explored through clinical trials including Antioxidants; Erythropoietin receptor agonists; Frataxin expression stimulants; Frataxin protein replacements; Histone deacetylase inhibitors; Lipid peroxidation inhibitors; Mitochondrial protein modulators; NF E2 related factor 2 stimulants; NQO1 modulators; Oxidoreductase modulators; Peroxisome proliferator-activated receptor gamma agonists; Protein aggregation inhibitors; RNF126 Inhibitor; tyrosine protein kinase Src Inhibitor.

The report provides complete details of pipeline drugs including the development phase, mechanism of action, companies involved, clinical trial developments, molecule type, and other details. Further, the report also provides Friedreich Ataxia drug development history, latest news, and other developments.

It assists companies, governments, investors and research organizations to understand the current status in 2020 and possible development in the next 10 years. Further, it enables readers to track new companies in the market and their developments. The product portfolio of different companies and their growth strategies are also detailed in the report.

PUBLISHER EXPERTISE

VPAResearch online databases analyze pipeline drugs and developments for over 2,000 diseases worldwide. All our reports and databases are developed through intensive primary and secondary research methods. The insights and data presented in the databases are validated through industry experts and represent completely unbiased opinions.

SCOPE:
  • The report scope comprises of both pre-clinical phase and clinical phase development drugs for Friedreich Ataxia development
  • Friedreich Ataxia pipeline compounds and molecules under study by both large scale and small companies are included in the report
  • Friedreich Ataxia pipeline across different phases including discovery, research, and pre-clinical stage, phase 1, phase 2, phase 3 and pre-registration phases are covered
  • Drug profile comprising of current development status, regulatory progress, companies, sponsors, mechanism of action, route of administration, molecule, and discovery details are covered
  • Further, orphan drug status, fast track designation, different grants awarded and special status for Friedreich Ataxia pipeline candidates included
  • Business overview and snapshot of all companies involved in Friedreich Ataxia pipeline are included
  • Latest market and pipeline developments are provided in the report
Friedreich Ataxia pipeline companies included in the report are- Agios Pharmaceuticals Inc, AmorChem Holdings Inc, Amylyx Pharmaceutical Corp, BioMarin Pharmaceutical Inc, Biovista Inc, Cardero Therapeutics Inc, Chondrial Therapeutics LLC, Exicure Inc, Fratagene Therapeutics Srl, Fulcrum Therapeutics Inc, Ixchel Pharma LLC, Jupiter Orphan Therapeutics Inc, MetroBiotech LLC, Minoryx Therapeutics sl, PTC Therapeutics, PTC Therapeutics Inc, Reata Pharmaceuticals Inc, Retrotope Inc, Seelos Therapeutics Inc, STATegics Inc, Voyager Therapeutics Inc

Friedreich Ataxia pipeline drugs profiled in the report include- Small Molecules for Friedreich Ataxia, AMX0035 (Sodium phenylbutyrate and tauroursodeoxycholic acid ), vatiquinone (EPI-743), BMN290, BVA202, BVA203, Epicatechin, CTI-1601, Oligonucleotides for Neurology, Src Inhibitors for Friedreich Ataxia, etravirine, RNF126 Inhibitors for Friedreich Ataxia, Frataxin for Friedreich Ataxia, IXC-103, IXC-109, JOT101, TALENs, MIB-626, leriglitazone, GT-FA, omaveloxolone, RT001, trehalose, STS-E15, VY-FXN01
1. TABLE OF CONTENTS

1.1 List of Tables
1.2 List of Figures

2. EXECUTIVE SUMMARY

2.1 Report Scope and Research Methodology
2.2 Introduction to Friedreich Ataxia Condition
2.3 Friedreich Ataxia Pipeline Snapshot, 2020
2.4 Companies investing in Friedreich Ataxia pipeline therapeutics
2.5 Phase wise Friedreich Ataxia Pipeline Candidates
2.6 Most Researched Mechanism of Action of Friedreich Ataxia Pipeline Products
2.7 Route of Administration of Friedreich Ataxia Pipeline Drugs

3. COMPANIES ACTIVE IN PIPELINE DEVELOPMENT

3.1 Agios Pharmaceuticals Inc Overview, Contacts and ASD Pipeline Drugs
3.2 AmorChem Holdings Inc Overview, Contacts and ASD Pipeline Drugs
3.3 Amylyx Pharmaceutical Corp Overview, Contacts and ASD Pipeline Drugs
3.4 BioMarin Pharmaceutical Inc Overview, Contacts and ASD Pipeline Drugs
3.5 Biovista Inc Overview, Contacts and ASD Pipeline Drugs
3.6 Cardero Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.7 Chondrial Therapeutics LLC Overview, Contacts and ASD Pipeline Drugs
3.8 Exicure Inc Overview, Contacts and ASD Pipeline Drugs
3.9 Fratagene Therapeutics Srl Overview, Contacts and ASD Pipeline Drugs
3.10 Fulcrum Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.11 Ixchel Pharma LLC Overview, Contacts and ASD Pipeline Drugs
3.12 Jupiter Orphan Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.13 MetroBiotech LLC Overview, Contacts and ASD Pipeline Drugs
3.14 Minoryx Therapeutics sl Overview, Contacts and ASD Pipeline Drugs
3.15 PTC Therapeutics Overview, Contacts and ASD Pipeline Drugs
3.16 PTC Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.17 Reata Pharmaceuticals Inc Overview, Contacts and ASD Pipeline Drugs
3.18 Retrotope Inc Overview, Contacts and ASD Pipeline Drugs
3.19 Seelos Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs
3.20 STATegics Inc Overview, Contacts and ASD Pipeline Drugs
3.21 Voyager Therapeutics Inc Overview, Contacts and ASD Pipeline Drugs

4. ACTIVE PIPELINE DRUG DETAILS, 2020

4.1 Small Molecules for Friedreich Ataxia Drug Details
  4.1.1 Small Molecules for Friedreich Ataxia Current Status
  4.1.2 Small Molecules for Friedreich Ataxia Drug Overview
  4.1.3 Small Molecules for Friedreich Ataxia Mechanism of Action
  4.1.4 Small Molecules for Friedreich Ataxia Licensing/Collaboration Companies
  4.1.5 Small Molecules for Friedreich Ataxia Clinical Trials
4.2 AMX0035 (Sodium phenylbutyrate and tauroursodeoxycholic acid ) Drug Details
  4.2.1 AMX0035 (Sodium phenylbutyrate and tauroursodeoxycholic acid ) Current Status
  4.2.2 AMX0035 (Sodium phenylbutyrate and tauroursodeoxycholic acid ) Drug Overview
  4.2.3 AMX0035 (Sodium phenylbutyrate and tauroursodeoxycholic acid ) Mechanism of Action
  4.2.4 AMX0035 (Sodium phenylbutyrate and tauroursodeoxycholic acid ) Licensing/Collaboration Companies
  4.2.5 AMX0035 (Sodium phenylbutyrate and tauroursodeoxycholic acid ) Clinical Trials
4.3 vatiquinone (EPI-743) Drug Details
  4.3.1 vatiquinone (EPI-743) Current Status
  4.3.2 vatiquinone (EPI-743) Drug Overview
  4.3.3 vatiquinone (EPI-743) Mechanism of Action
  4.3.4 vatiquinone (EPI-743) Licensing/Collaboration Companies
  4.3.5 vatiquinone (EPI-743) Clinical Trials
4.4 BMN290 Drug Details
  4.4.1 BMN290 Current Status
  4.4.2 BMN290 Drug Overview
  4.4.3 BMN290 Mechanism of Action
  4.4.4 BMN290 Licensing/Collaboration Companies
  4.4.5 BMN290 Clinical Trials
4.5 BVA202 Drug Details
  4.5.1 BVA202 Current Status
  4.5.2 BVA202 Drug Overview
  4.5.3 BVA202 Mechanism of Action
  4.5.4 BVA202 Licensing/Collaboration Companies
  4.5.5 BVA202 Clinical Trials
4.6 BVA203 Drug Details
  4.6.1 BVA203 Current Status
  4.6.2 BVA203 Drug Overview
  4.6.3 BVA203 Mechanism of Action
  4.6.4 BVA203 Licensing/Collaboration Companies
  4.6.5 BVA203 Clinical Trials
4.7 Epicatechin Drug Details
  4.7.1 Epicatechin Current Status
  4.7.2 Epicatechin Drug Overview
  4.7.3 Epicatechin Mechanism of Action
  4.7.4 Epicatechin Licensing/Collaboration Companies
  4.7.5 Epicatechin Clinical Trials
4.8 CTI-1601 Drug Details
  4.8.1 CTI-1601 Current Status
  4.8.2 CTI-1601 Drug Overview
  4.8.3 CTI-1601 Mechanism of Action
  4.8.4 CTI-1601 Licensing/Collaboration Companies
  4.8.5 CTI-1601 Clinical Trials
4.9 Oligonucleotides for Neurology Drug Details
  4.9.1 Oligonucleotides for Neurology Current Status
  4.9.2 Oligonucleotides for Neurology Drug Overview
  4.9.3 Oligonucleotides for Neurology Mechanism of Action
  4.9.4 Oligonucleotides for Neurology Licensing/Collaboration Companies
  4.9.5 Oligonucleotides for Neurology Clinical Trials
4.10 Src Inhibitors for Friedreich Ataxia Drug Details
  4.10.1 Src Inhibitors for Friedreich Ataxia Current Status
  4.10.2 Src Inhibitors for Friedreich Ataxia Drug Overview
  4.10.3 Src Inhibitors for Friedreich Ataxia Mechanism of Action
  4.10.4 Src Inhibitors for Friedreich Ataxia Licensing/Collaboration Companies
  4.10.5 Src Inhibitors for Friedreich Ataxia Clinical Trials
4.11 etravirine Drug Details
  4.11.1 etravirine Current Status
  4.11.2 etravirine Drug Overview
  4.11.3 etravirine Mechanism of Action
  4.11.4 etravirine Licensing/Collaboration Companies
  4.11.5 etravirine Clinical Trials
4.12 RNF126 Inhibitors for Friedreich Ataxia Drug Details
  4.12.1 RNF126 Inhibitors for Friedreich Ataxia Current Status
  4.12.2 RNF126 Inhibitors for Friedreich Ataxia Drug Overview
  4.12.3 RNF126 Inhibitors for Friedreich Ataxia Mechanism of Action
  4.12.4 RNF126 Inhibitors for Friedreich Ataxia Licensing/Collaboration Companies
  4.12.5 RNF126 Inhibitors for Friedreich Ataxia Clinical Trials
4.13 Frataxin for Friedreich Ataxia Drug Details
  4.13.1 Frataxin for Friedreich Ataxia Current Status
  4.13.2 Frataxin for Friedreich Ataxia Drug Overview
  4.13.3 Frataxin for Friedreich Ataxia Mechanism of Action
  4.13.4 Frataxin for Friedreich Ataxia Licensing/Collaboration Companies
  4.13.5 Frataxin for Friedreich Ataxia Clinical Trials
4.14 IXC-103 Drug Details
  4.14.1 IXC-103 Current Status
  4.14.2 IXC-103 Drug Overview
  4.14.3 IXC-103 Mechanism of Action
  4.14.4 IXC-103 Licensing/Collaboration Companies
  4.14.5 IXC-103 Clinical Trials
4.15 IXC-109 Drug Details
  4.15.1 IXC-109 Current Status
  4.15.2 IXC-109 Drug Overview
  4.15.3 IXC-109 Mechanism of Action
  4.15.4 IXC-109 Licensing/Collaboration Companies
  4.15.5 IXC-109 Clinical Trials
4.16 JOT101 Drug Details
  4.16.1 JOT101 Current Status
  4.16.2 JOT101 Drug Overview
  4.16.3 JOT101 Mechanism of Action
  4.16.4 JOT101 Licensing/Collaboration Companies
  4.16.5 JOT101 Clinical Trials
4.17 TALENs Drug Details
  4.17.1 TALENs Current Status
  4.17.2 TALENs Drug Overview
  4.17.3 TALENs Mechanism of Action
  4.17.4 TALENs Licensing/Collaboration Companies
  4.17.5 TALENs Clinical Trials
4.18 MIB-626 Drug Details
  4.18.1 MIB-626 Current Status
  4.18.2 MIB-626 Drug Overview
  4.18.3 MIB-626 Mechanism of Action
  4.18.4 MIB-626 Licensing/Collaboration Companies
  4.18.5 MIB-626 Clinical Trials
4.19 leriglitazone Drug Details
  4.19.1 leriglitazone Current Status
  4.19.2 leriglitazone Drug Overview
  4.19.3 leriglitazone Mechanism of Action
  4.19.4 leriglitazone Licensing/Collaboration Companies
  4.19.5 leriglitazone Clinical Trials
4.20 GT-FA Drug Details
  4.20.1 GT-FA Current Status
  4.20.2 GT-FA Drug Overview
  4.20.3 GT-FA Mechanism of Action
  4.20.4 GT-FA Licensing/Collaboration Companies
  4.20.5 GT-FA Clinical Trials
4.21 omaveloxolone Drug Details
  4.21.1 omaveloxolone Current Status
  4.21.2 omaveloxolone Drug Overview
  4.21.3 omaveloxolone Mechanism of Action
  4.21.4 omaveloxolone Licensing/Collaboration Companies
  4.21.5 omaveloxolone Clinical Trials
4.22 RT001 Drug Details
  4.22.1 RT001 Current Status
  4.22.2 RT001 Drug Overview
  4.22.3 RT001 Mechanism of Action
  4.22.4 RT001 Licensing/Collaboration Companies
  4.22.5 RT001 Clinical Trials
4.23 trehalose Drug Details
  4.23.1 trehalose Current Status
  4.23.2 trehalose Drug Overview
  4.23.3 trehalose Mechanism of Action
  4.23.4 trehalose Licensing/Collaboration Companies
  4.23.5 trehalose Clinical Trials
4.24 STS-E15 Drug Details
  4.24.1 STS-E15 Current Status
  4.24.2 STS-E15 Drug Overview
  4.24.3 STS-E15 Mechanism of Action
  4.24.4 STS-E15 Licensing/Collaboration Companies
  4.24.5 STS-E15 Clinical Trials
4.25 VY-FXN01 Drug Details
  4.25.1 VY-FXN01 Current Status
  4.25.2 VY-FXN01 Drug Overview
  4.25.3 VY-FXN01 Mechanism of Action
  4.25.4 VY-FXN01 Licensing/Collaboration Companies
  4.25.5 VY-FXN01 Clinical Trials

5. LATEST FRIEDREICH ATAXIA PIPELINE NEWS AND DEALS

6. APPENDIX

6.1 Our Databases and Reports
6.2 Research Methodolgy


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