Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) Drugs Development Report 2022: 21 Molecules with Approximately 15 Molecules Developed by Companies, Remaining by Universities – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “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” report has been added to ResearchAndMarkets.com’s offering.

Frataxin Mitochondrial – Drugs In Development 2022 outlays comprehensive information on the Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) – 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 molecules developed by companies in Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 8 and 4 respectively.

Similarly, the universities portfolio in Preclinical and Discovery stages comprises 3 and 3 molecules, respectively. Report covers products from therapy areas Central Nervous System and Cardiovascular which include indications Friedreich Ataxia, Cardiomyopathy and Mild Cognitive Impairment.

Furthermore, this report also reviews key players involved in Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics development with respective active and dormant or discontinued projects.

Driven by data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.

Scope

• The report provides a snapshot of the global therapeutic landscape for Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)
• The report reviews Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
• The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
• The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
• The report reviews key players involved in Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics and enlists all their major and minor projects
• The report assesses Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
• The report summarizes all the dormant and discontinued pipeline projects
• The report reviews latest news and deals related to Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics

Key Topics Covered:

Introduction

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
• BTX-101 – Drug Profile
• CV-14 – Drug Profile
• DT-216 – Drug Profile
• etravirine – Drug Profile
• Gene Therapy to Activate Frataxin for Friedreich Ataxia – Drug Profile
• Gene Therapy to Activate Frataxin Mitochondrial for Friedreich’s Ataxia – Drug Profile
• Gene Therapy to Activate FXN for Friedreich Ataxia – Drug Profile
• JNS-101 – Drug Profile
• LX-2006 – Drug Profile
• PTC-FA – Drug Profile
• Recombinant Proteins to Replace Frataxin Mitochondrial for Friedreich Ataxia – Drug Profile
• Small Molecules to Activate Frataxin for Friedreich Ataxia – Drug Profile
• VYFXN-01 – Drug Profile

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

For more information about this report visit https://www.researchandmarkets.com/r/b1xi86

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