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

April 2022 | 64 pages | ID: F3E4BADBB584EN
Global Markets Direct

Abstracts

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

SUMMARY

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) pipeline Target constitutes close to 21 molecules. Out of which approximately 15 molecules are developed by companies and remaining by the universities/institutes. The latest report 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.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

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

REASONS TO BUY

• Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
• Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
• Identify and understand the targeted therapy areas and indications for Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)
• Identify the use of drugs for target identification and drug repurposing
• Identify potential new clients or partners in the target demographic
• Develop strategic initiatives by understanding the focus areas of leading companies
• Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
• Devise corrective measures for pipeline projects by understanding Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) development landscape
• 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

Contents

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

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

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