Friedreich Ataxia Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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Friedreich Ataxia Drugs in Development Report Overview

Friedreich’s ataxia (FA) is a neuromuscular disease that mainly affects the nervous system and the heart. FA is a hereditary disease, caused by a defective gene that can be passed down through a family. Its signs and symptoms include ataxia, weakness and spasticity, sensory impairment, skeletal abnormalities, cardiac difficulties, and diabetes.

The Friedreich Ataxia pipeline drugs market research 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 the 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.

Key Targets Frataxin Mitochondrial, Nuclear Factor Erythroid 2 Related Factor 2, Adenosine Monophosphate Activated Protein Kinase Hydroxymethylglutaryl CoA Reductase, Arachidonate 15 Lipoxygenase, Cardiolipin, E3 Ubiquitin Protein Ligase RNF126, Erythropoietin Receptor, NAD Dependent Protein Deacetylase Sirtuin 1, NAD(P)H Dehydrogenase [Quinone] 1 (Azoreductase or DT Diaphorase or DTD or Menadione Reductase or NAD, Peroxisome Proliferator Activated Receptor Gamma, and Others
Key Mechanisms of Action Frataxin Mitochondrial Activator, Frataxin Mitochondrial Replacement, Nuclear Factor Erythroid 2 Related Factor 2 Activator, Adenosine Monophosphate Activated Protein Kinase Hydroxymethylglutaryl CoA Reductase Activator, Arachidonate 15 Lipoxygenase Inhibitor, E3 Ubiquitin Protein Ligase RNF126 Inhibitor, Erythropoietin Receptor Agonist, NAD Dependent Protein Deacetylase Sirtuin 1 Activator, NAD(P)H Dehydrogenase [Quinone] 1 Azoreductase or DT Diaphorase or DTD or Menadione Reductase or NADActivator, and Peroxisome Proliferator Activated Receptor Gamma Agonist, and Others
Key Routes of Administration Oral, Intravenous, Ophthalmic, Parenteral, Topical, and Subcutaneous
Key Molecule Type Small Molecule, Gene Therapy, Gene-Modified Cell Therapy, Recombinant Protein, Synthetic Peptide, and Cell Therapy
Major Companies Fratagene Therapeutics Srl, Biovista Inc, Ixchel Pharma LLC, PTC Therapeutics Inc, Reata Pharmaceuticals Inc, Retrotope Inc, Stealth BioTherapeutics Corp, AavantiBio Inc, Amylyx Pharmaceuticals Inc, and Anima Biotech Inc

For more insights on the Friedreich Ataxia pipeline drugs market, download a free report sample

Key Targets in the Friedreich Ataxia Pipeline Products Market

The key targets in the Friedreich Ataxia pipeline products market are Frataxin Mitochondrial, Nuclear Factor Erythroid 2 Related Factor 2, Adenosine Monophosphate Activated Protein Kinase Hydroxymethylglutaryl CoA Reductase, Arachidonate 15 Lipoxygenase, Cardiolipin, E3 Ubiquitin Protein Ligase RNF126, Erythropoietin Receptor, NAD Dependent Protein Deacetylase Sirtuin 1,  NAD(P)H Dehydrogenase [Quinone] 1 (Azoreductase or DT Diaphorase or DTD or Menadione Reductase or NAD, Peroxisome Proliferator Activated Receptor Gamma, and others.

Friedreich Ataxia Pipeline Products Analysis Market by Targets

Friedreich Ataxia Pipeline Products Analysis Market by Targets

For more target insights, download a free report sample

Key Mechanisms of Action in the Friedreich Ataxia Pipeline Products Market

The key mechanisms of action in the Friedreich Ataxia pipeline products market are Frataxin Mitochondrial Activator, Frataxin Mitochondrial Replacement, Nuclear Factor Erythroid 2 Related Factor 2 Activator, Adenosine Monophosphate Activated Protein Kinase Hydroxymethylglutaryl CoA Reductase Activator, Arachidonate 15 Lipoxygenase Inhibitor, E3 Ubiquitin Protein Ligase RNF126 Inhibitor, Erythropoietin Receptor Agonist, NAD Dependent Protein Deacetylase Sirtuin 1 Activator, NAD(P)H Dehydrogenase [Quinone] 1 Azoreductase or DT Diaphorase or DTD or Menadione Reductase or NADActivator, and Peroxisome Proliferator Activated Receptor Gamma Agonist, and others.

Friedreich Ataxia Pipeline Products Market Analysis by Mechanisms of Action

Friedreich Ataxia Pipeline Products Market Analysis by Mechanisms of Action

For more mechanisms of action insights, download a free report sample

Key Routes of Administration in the Friedreich Ataxia Pipeline Products Market

The key routes of administration in the Friedreich Ataxia pipeline products market are oral, intravenous, ophthalmic, parenteral, topical, and subcutaneous.

Friedreich Ataxia Pipeline Products Market Analysis by Routes of Administration

Friedreich Ataxia Pipeline Products Market Analysis by Routes of Administration

For more routes of administration insights, download a free report sample

Key Molecule Types in the Friedreich Ataxia Pipeline Products Market

The key molecule types in the Friedreich Ataxia pipeline products market are small molecule, gene therapy, gene-modified cell therapy, recombinant protein, synthetic peptide, and cell therapy.

Friedreich Ataxia Pipeline Products Market Analysis by Molecule Types

Friedreich Ataxia Pipeline Products Market Analysis by Molecule Types

For more molecule type insights, download a free report sample

Major Companies in the Friedreich Ataxia Pipeline Products Market

Some of the major companies in the Friedreich Ataxia pipeline products market are Fratagene Therapeutics Srl, Biovista Inc, Ixchel Pharma LLC, PTC Therapeutics Inc, Reata Pharmaceuticals Inc, Retrotope Inc, Stealth BioTherapeutics Corp, AavantiBio Inc, Amylyx Pharmaceuticals Inc, and Anima Biotech Inc.

Fratagene Therapeutics Srl: Fulcrum Therapeutics Inc (Fulcrum Therapeutics) is a drug development company. The company is involved in the discovery and development of small molecule therapies to unlock gene regulation in disease. Its pipeline products include Losmapimod for facioscapulohumeral muscular dystrophy (FSHD) and FTX-6058 for sickle cell disease and ß Thalassemia. Fulcrum Therapeutics products intend to develop disease-modifying therapies for various diseases in the therapeutic areas of neuromuscular, muscular, hematologic, muscle disorders, blood disorders, and central nervous system, or CNS, disorders. The company partners with other biopharmaceutical companies. Fulcrum Therapeutics is headquartered in Cambridge, Massachusetts, the US.

Friedreich Ataxia Pipeline Products Market Analysis by Companies

Friedreich Ataxia Pipeline Products Market Analysis by Companies

To know more about major companies, download a free report sample

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Friedreich Ataxia (Central Nervous System).

  • The pipeline guide reviews pipeline therapeutics for Friedreich Ataxia (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Friedreich Ataxia (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Friedreich Ataxia (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA), and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews the latest news related to pipeline therapeutics for Friedreich Ataxia (Central Nervous System)

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolios and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Friedreich Ataxia (Central Nervous System).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and their most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Friedreich Ataxia (Central Nervous System) 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from the pipeline.

AavantiBio Inc
Amylyx Pharmaceuticals Inc
Anima Biotech Inc
Astellas Pharma Inc
Biointaxis SL
Biovista Inc
Cellivery Therapeutics Inc
CRISPR Therapeutics AG
Cytonus Therapeutics Inc
Design Therapeutics Inc
Epirium Bio Inc
Fratagene Therapeutics Srl
Fulcrum Therapeutics Inc
Healx Ltd
Ixchel Pharma LLC
Jupiter Neurosciences Inc
Lacerta Therapeutics Inc
LEXEO Therapeutics LLC
Metro International Biotech LLC
Minoryx Therapeutics sl
Novartis Gene Therapies
OMEICOS Therapeutics GmbH
PTC Therapeutics Inc
Reata Pharmaceuticals Inc
Retrotope Inc
Scriptr Global Inc
Seelos Therapeutics, Inc.
STATegics Inc
Stealth BioTherapeutics Corp
StrideBio Inc
Voyager Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Friedreich Ataxia – Overview

Friedreich Ataxia – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Friedreich Ataxia – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Friedreich Ataxia – Companies Involved in Therapeutics Development

Friedreich Ataxia – Drug Profiles

Friedreich Ataxia – Dormant Projects

Friedreich Ataxia – Discontinued Products

Friedreich Ataxia – Product Development Milestones

Featured News & Press Releases

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development for Friedreich Ataxia, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Companies, 2022 (Contd..1)

Number of Products under Development by Universities/Institutes, 2022

Products under Development by Companies, 2022

Products under Development by Companies, 2022 (Contd..1)

Products under Development by Universities/Institutes, 2022

Number of Products by Stage and Target, 2022

Number of Products by Stage and Mechanism of Action, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Friedreich Ataxia – Dormant Projects, 2022

Friedreich Ataxia – Dormant Projects, 2022 (Contd..1)

Friedreich Ataxia – Discontinued Products, 2022

Figures

List of Figures

Number of Products under Development for Friedreich Ataxia, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Number of Products by Top 10 Targets, 2022

Number of Products by Stage and Top 10 Targets, 2022

Number of Products by Top 10 Mechanism of Actions, 2022

Number of Products by Stage and Top 10 Mechanism of Actions, 2022

Number of Products by Routes of Administration, 2022

Number of Products by Stage and Routes of Administration, 2022

Number of Products by Molecule Types, 2022

Number of Products by Stage and Molecule Types, 2022

Frequently asked questions

  • What are the key targets in the Friedreich Ataxia products market?

    The key targets in the Friedreich Ataxia pipeline products market are Frataxin Mitochondrial,  Nuclear Factor Erythroid 2 Related Factor 2, Adenosine Monophosphate Activated Protein Kinase Hydroxymethylglutaryl CoA Reductase, Arachidonate 15 Lipoxygenase, Cardiolipin, E3 Ubiquitin Protein Ligase RNF126, Erythropoietin Receptor, NAD Dependent Protein Deacetylase Sirtuin 1,  NAD(P)H Dehydrogenase [Quinone] 1 (Azoreductase or DT Diaphorase or DTD or Menadione Reductase or NAD, Peroxisome Proliferator Activated Receptor Gamma, and others.

  • What are the key mechanisms of action in the Friedreich Ataxia pipeline products market?

    The key mechanisms of action in the Friedreich Ataxia pipeline products market are Frataxin Mitochondrial Activator, Frataxin Mitochondrial Replacement, Nuclear Factor Erythroid 2 Related Factor 2 Activator, Adenosine Monophosphate Activated Protein Kinase Hydroxymethylglutaryl CoA Reductase Activator, Arachidonate 15 Lipoxygenase Inhibitor, E3 Ubiquitin Protein Ligase RNF126 Inhibitor, Erythropoietin Receptor Agonist, NAD Dependent Protein Deacetylase Sirtuin 1 Activator, NAD(P)H Dehydrogenase [Quinone] 1 Azoreductase or DT Diaphorase or DTD or Menadione Reductase or NADActivator, and Peroxisome Proliferator Activated Receptor Gamma Agonist, and others.

  • What are the key routes of administration in the Friedreich Ataxia pipeline products market?

    The key routes of administration in the Friedreich Ataxia pipeline products market are oral, intravenous, ophthalmic, parenteral, topical, and subcutaneous.

  • What are the key molecule types in the Friedreich Ataxia pipeline products market?

    The key molecule types in the Friedreich Ataxia pipeline products market are small molecule, gene therapy, gene-modified cell therapy, recombinant protein, synthetic peptide, and cell therapy.

  • Which are the major companies in the Friedreich Ataxia pipeline products market?

    Some of the major companies in the Friedreich Ataxia pipeline products market are Fratagene Therapeutics Srl, Biovista Inc, Ixchel Pharma LLC, PTC Therapeutics Inc, Reata Pharmaceuticals Inc, Retrotope Inc, Stealth BioTherapeutics Corp, AavantiBio Inc, Amylyx Pharmaceuticals Inc, and Anima Biotech Inc.

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