Table of Content


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
Recombint 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 investigatiol New Drug Application for first GeneTAC molecule for Friedreich Ataxia
Feb 16, 2022: LEXEO Therapeutics announces FDA clearance of investigatiol 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 desigtion and orphan drug desigtion 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 Additiol Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting
Oct 31, 2017: Agilis Biotherapeutics Announces Orphan Product Desigtion 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 Desigtion 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 Desigtion for the Treatment of Friedreich’s Ataxia
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of 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

List of Tables

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