Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

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Frataxin is a protein encoded by the FXN gene. It promotes the biosynthesis of heme and assembly and repair of iron-sulfur clusters by delivering Fe2+ to proteins involved in these pathways. It plays a role in the protection against iron-catalyzed oxidative stress through its ability to catalyze the oxidation of Fe2+ to Fe3.

The Frataxin Mitochondrial – Drugs in Development research report provides a comprehensive overview on the therapeutics under development for Frataxin Mitochondrial, 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 latest news and press releases.  Additionally, the report provides an overview of key players involved in therapeutic development for Frataxin Mitochondrial and features dormant and discontinued projects.

Key Therapy Areas in the Frataxin Mitochondrial Pipeline Drugs Market

In the Frataxin Mitochondrial pipeline drugs market the key therapy areas are Central Nervous System, and Cardiovascular. Central Nervous System has the highest number of pipeline products.

Frataxin Mitochondrial Drugs Market, by Therapy Areas

Frataxin Mitochondrial Drugs Market, by Therapy Areas

For more therapy area insights, download a free report sample

Key MoA in the Frataxin Mitochondrial Pipeline Drugs Market

Frataxin Mitochondrial Activator has the highest number of pipeline products followed by Frataxin Mitochondrial Replacement.

Frataxin Mitochondrial Pipeline Drugs Market, by MoA

Frataxin Mitochondrial Pipeline Drugs Market, by MoA

To get more insights on key MoA, download a free sample report

Frataxin Mitochondrial Pipeline Drugs Market Segmentation by RoA

The key routes of administration in the Frataxin Mitochondrial pipeline drugs market are intravenous, oral, and parenteral.

Key Molecule Types in the Frataxin Mitochondrial Pipeline Drugs Market

The key molecule types in the Frataxin Mitochondrial pipeline drugs market are Gene Therapy, Small Molecule, Recombinant Protein, Gene-Modified Cell Therapy, and Synthetic Peptide.

Frataxin Mitochondrial Pipeline Drugs Market, by Molecule Type

Frataxin Mitochondrial Pipeline Drugs Market, by Molecule Type

To get more insights on key molecule types, download a free sample report

Major Companies in the Frataxin Mitochondrial Pipeline Drugs Market

The major companies in the Frataxin Mitochondrial pipeline drugs market are Jupiter Neurosciences Inc, AavantiBio Inc, Anima Biotech Inc, Astellas Pharma Inc, Biointaxis SL, Cellivery Therapeutics Inc, CRISPR Therapeutics AG, Design Therapeutics Inc, Fratagene Therapeutics Srl, and Fulcrum Therapeutics Inc.

Frataxin Mitochondrial Pipeline Drugs Market, by Major Companies

Frataxin Mitochondrial Pipeline Drugs Market, by Major Companies

For more company insights, download a free sample report

Frataxin Mitochondrial Pipeline Drugs Market Overview

Key Therapy Areas Central Nervous System, and Cardiovascular
Key Mechanisms of action Frataxin Mitochondrial Activator, Frataxin Mitochondrial Replacement
Key Routes of Administration Intravenous, Oral, and Parenteral
Key molecule types Gene Therapy, Small Molecule, Recombinant Protein, Gene-Modified Cell Therapy, and Synthetic Peptide
Major companies Jupiter Neurosciences Inc, AavantiBio Inc, Anima Biotech Inc, Astellas Pharma Inc, Biointaxis SL, Cellivery Therapeutics Inc, CRISPR Therapeutics AG, Design Therapeutics Inc, Fratagene Therapeutics Srl, and Fulcrum Therapeutics Inc

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Frataxin Mitochondrial
  • The pipeline guide reviews pipeline therapeutics for Frataxin Mitochondrial 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 Frataxin Mitochondrial therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Frataxin Mitochondrial 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 Frataxin Mitochondrial

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with a potentially strong product portfolio and create effective counter strategies to gain a competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Frataxin Mitochondrial
  • 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 Frataxin Mitochondrial 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
Anima Biotech Inc
Astellas Pharma Inc
Biointaxis SL
Cellivery Therapeutics Inc
CRISPR Therapeutics AG
Design Therapeutics Inc
Fratagene Therapeutics Srl
Fulcrum Therapeutics Inc
Jupiter Neurosciences Inc
Lacerta Therapeutics Inc
LEXEO Therapeutics LLC
Novartis Gene Therapies
PTC Therapeutics Inc
Voyager Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) – Overview

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) – Therapeutics Development

Products under Development by Stage of Development

Products under Development by Therapy Area

Products under Development by Indication

Products under Development by Companies

Products under Development by Universities/Institutes

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) – Therapeutics Assessment

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

AavantiBio Inc

Anima Biotech Inc

Astellas Pharma Inc

Biointaxis SL

Cellivery Therapeutics Inc

CRISPR Therapeutics AG

Design Therapeutics Inc

Fratagene Therapeutics Srl

Fulcrum Therapeutics Inc

Jupiter Neurosciences Inc

Lacerta Therapeutics Inc

LEXEO Therapeutics LLC

Novartis Gene Therapies

PTC Therapeutics Inc

Voyager Therapeutics Inc

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) – Drug Profiles

AT-808 – Drug Profile

Product Description

Mechanism Of Action

AVXS-401 – Drug Profile

Product Description

Mechanism Of Action

History of Events

BTX-101 – Drug Profile

Product Description

Mechanism Of Action

History of Events

CV-14 – Drug Profile

Product Description

Mechanism Of Action

History of Events

DT-216 – Drug Profile

Product Description

Mechanism Of Action

History of Events

etravirine – Drug Profile

Product Description

Mechanism Of Action

History of Events

Gene Therapy to Activate Frataxin for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate Frataxin for Friedreich’s Ataxia – Drug Profile

Product Description

Mechanism Of Action

History of Events

Gene Therapy to Activate Frataxin Mitochondrial for Friedreich’s Ataxia – Drug Profile

Product Description

Mechanism Of Action

History of Events

Gene Therapy to Activate FXN for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

History of Events

Gene Therapy to Activate FXN for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate FXN for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

History of Events

Gene Therapy to Activate FXN for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

JNS-101 – Drug Profile

Product Description

Mechanism Of Action

History of Events

LX-2006 – Drug Profile

Product Description

Mechanism Of Action

History of Events

PTC-FA – Drug Profile

Product Description

Mechanism Of Action

History of Events

Recombinant Proteins to Replace Frataxin Mitochondrial for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

Small Molecule to Activate Frataxin for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

Small Molecules to Activate Frataxin for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

Small Molecules to Activate Frataxin for Friedreich Ataxia – Drug Profile

Product Description

Mechanism Of Action

History of Events

VYFXN-01 – Drug Profile

Product Description

Mechanism Of Action

History of Events

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) – Dormant Products

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) – Product Development Milestones

Featured News & Press Releases

Mar 30, 2022: Design Therapeutics completes dosing in first patient cohort of phase 1 trial of DT-216 GeneTAC molecule for the treatment of Friedreich Ataxia

Feb 28, 2022: Design Therapeutics announces FDA clearance of investigational New Drug Application for first GeneTAC molecule for Friedreich Ataxia

Feb 16, 2022: LEXEO Therapeutics announces FDA clearance of investigational new drug application for LX2006, an AAV-based gene therapy candidate for Friedreich’s Ataxia Cardiomyopathy

Jun 30, 2021: LEXEO Therapeutics receives rare pediatric disease designation and orphan drug designation for LX2006 for the treatment of Friedreich’s Ataxia

Apr 28, 2021: LEXEO Therapeutics announces upcoming data presentations at American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting, provides update on LX2006

Jan 13, 2020: MDA awards venture philanthropy funding of more than $1M to AavantiBio to develop gene-targeting therapy for Friedreich’s Ataxia

Nov 30, 2019: GoFAR partners with AavantiBio to advance gene therapy for Friedreich’s Ataxia

Nov 30, 2019: GoFAR partners with AavantiBio to advance gene therapy for Friedreich’s Ataxia

Aug 13, 2019: Ataxia UK funds new FA research project developing new FA animal models to test gene therapy

May 18, 2018: Voyager Therapeutics Announces Additional Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting

Oct 31, 2017: Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich Ataxia

Oct 25, 2017: Transplanted hematopoietic stem cells reverse damage caused by neuromuscular disorder

Aug 22, 2017: Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich’s Ataxia

Mar 14, 2017: New research front to tackle Friedreich’s Ataxia

Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development by Stage of Development, 2022

Number of Products under Development by Therapy Areas, 2022

Number of Products under Development by Indication, 2022

Number of Products under Development by Companies, 2022

Products under Development by Companies, 2022

Number of Products under Investigation by Universities/Institutes, 2022

Products under Investigation by Universities/Institutes, 2022

Number of Products by Stage and Mechanism of Actions, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Pipeline by AavantiBio Inc, 2022

Pipeline by Anima Biotech Inc, 2022

Pipeline by Astellas Pharma Inc, 2022

Pipeline by Biointaxis SL, 2022

Pipeline by Cellivery Therapeutics Inc, 2022

Pipeline by CRISPR Therapeutics AG, 2022

Pipeline by Design Therapeutics Inc, 2022

Pipeline by Fratagene Therapeutics Srl, 2022

Pipeline by Fulcrum Therapeutics Inc, 2022

Pipeline by Jupiter Neurosciences Inc, 2022

Pipeline by Lacerta Therapeutics Inc, 2022

Pipeline by LEXEO Therapeutics LLC, 2022

Pipeline by Novartis Gene Therapies, 2022

Pipeline by PTC Therapeutics Inc, 2022

Pipeline by Voyager Therapeutics Inc, 2022

Dormant Projects, 2022

Figures

List of Figures

Number of Products under Development by Stage of Development, 2022

Number of Products under Development by Therapy Areas, 2022

Number of Products under Development by Top 10 Indications, 2022

Number of Products by Mechanism of Actions, 2022

Number of Products by Stage and 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

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