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
Adverum Biotechnologies Inc
Biointaxis SL
Chondrial Therapeutics LLC
Design Therapeutics Inc
Fratagene Therapeutics Srl
Fulcrum Therapeutics Inc
Jupiter Orphan Therapeutics Inc
Panorama Research Inc
Pfizer Inc
PTC Therapeutics Inc
Voyager Therapeutics Inc
Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Drug Profiles
ADVM-063 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CTI-1601 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
etravirine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedrich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate FXN for Friedreich Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JOT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PTC-FA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Activate Frataxin for Friedreich Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate Frataxin for Friedreich Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Synthetic Peptide to Activate Frataxin for Friedreich Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VYFXN-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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
Jan 13, 2020: MDA awards venture philanthropy funding of more than $1M to AavantiBio to develop gene-targeting therapy for Friedreich’s Ataxia
Dec 18, 2019: Chondrial Therapeutics announces dosing of first patients in phase 1 clinical program of CTI-1601 for treatment of Friedreich’s ataxia and CTI-1601 granted rare pediatric disease desigtion and fast track desigtion by U.S. FDA
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
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
Aug 22, 2017: Jupiter Orphan Therapeutics Receives Orphan Drug Desigtion for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich’s Ataxia
Aug 03, 2017: Chondrial Announces FDA Orphan Drug Desigtion for CTI-1601, a Novel Investigatiol Technology for the Treatment of Friedreich’s Ataxia
Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Desigtion for the Treatment of Friedreich’s Ataxia
Feb 18, 2016: Agilis Biotherapeutics and Waisman Biomanufacturing enter into exclusive manufacturing agreement for Friedreich’s Ataxia gene therapy
Aug 05, 2015: Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development by Stage of Development, H1 2020
Number of Products under Development by Therapy Areas, H1 2020
Number of Products under Development by Top 10 Indications, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020


List of Tables


Number of Products under Development by Stage of Development, H1 2020
Number of Products under Development by Therapy Areas, H1 2020
Number of Products under Development by Indication, H1 2020
Number of Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020
Number of Products under Investigation by Universities/Institutes, H1 2020
Products under Investigation by Universities/Institutes, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Pipeline by AavantiBio Inc, H1 2020
Pipeline by Adverum Biotechnologies Inc, H1 2020
Pipeline by Biointaxis SL, H1 2020
Pipeline by Chondrial Therapeutics LLC, H1 2020
Pipeline by Design Therapeutics Inc, H1 2020
Pipeline by Fratagene Therapeutics Srl, H1 2020
Pipeline by Fulcrum Therapeutics Inc, H1 2020
Pipeline by Jupiter Orphan Therapeutics Inc, H1 2020
Pipeline by Panorama Research Inc, H1 2020
Pipeline by Pfizer Inc, H1 2020
Pipeline by PTC Therapeutics Inc, H1 2020
Pipeline by Voyager Therapeutics Inc, H1 2020
Dormant Projects, H1 2020