Frataxin Mitochondrial - Pipeline Review, H1 2020
Frataxin Mitochondrial - Pipeline Review, H1 2020
SUMMARY
Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) pipeline Target constitutes close to 14 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Frataxin Mitochondrial - Pipeline Review, H1 2020, 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 I, Preclinical and Discovery stages are 2, 6 and 4 respectively. Similarly, the universities portfolio in Discovery stages comprises 2 molecules, respectively. Report covers products from therapy areas Central Nervous System and Cardiovascular which include indications Friedreich Ataxia and Cardiomyopathy.
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
SUMMARY
Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) pipeline Target constitutes close to 14 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Frataxin Mitochondrial - Pipeline Review, H1 2020, 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 I, Preclinical and Discovery stages are 2, 6 and 4 respectively. Similarly, the universities portfolio in Discovery stages comprises 2 molecules, respectively. Report covers products from therapy areas Central Nervous System and Cardiovascular which include indications Friedreich Ataxia and Cardiomyopathy.
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
- 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
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 designation and fast track designation 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 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
Aug 22, 2017: Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich's Ataxia
Aug 03, 2017: Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich's Ataxia
Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation 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
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 designation and fast track designation 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 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
Aug 22, 2017: Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich's Ataxia
Aug 03, 2017: Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich's Ataxia
Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation 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 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
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
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
COMPANIES MENTIONED
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
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
COMPANIES MENTIONED
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
