Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Pipeline Review, H2 2017
Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Pipeline Review, H2 2017
SUMMARY
According to the recently published report 'Frataxin Mitochondrial - Pipeline Review, H2 2017'; Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) pipeline Target constitutes close to 11 molecules. Out of which approximately 9 molecules are developed by companies and remaining by the universities/institutes.
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 report 'Frataxin Mitochondrial - Pipeline Review, H2 2017' 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; that are being developed by Companies/Universities.
It 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. Currently, The molecules developed by companies in Phase II, Preclinical and Discovery stages are 1, 6 and 2 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.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
According to the recently published report 'Frataxin Mitochondrial - Pipeline Review, H2 2017'; Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) pipeline Target constitutes close to 11 molecules. Out of which approximately 9 molecules are developed by companies and remaining by the universities/institutes.
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 report 'Frataxin Mitochondrial - Pipeline Review, H2 2017' 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; that are being developed by Companies/Universities.
It 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. Currently, The molecules developed by companies in Phase II, Preclinical and Discovery stages are 1, 6 and 2 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.
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
Adverum Biotechnologies Inc
Bio Blast Pharma Ltd
Pfizer Inc
ProQR Therapeutics NV
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
AGIL-FA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BB-FA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CTI-1601 - 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
JOT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QRX-604 - 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
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
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
Aug 05, 2015: Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Friedreich’s Ataxia at Two Leading Scientific Meetings in June
Mar 26, 2015: BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in Friedreich's Ataxia
Dec 17, 2014: BioBlast Receives Notice of Patent Issuance From USPTO for Its Novel Fusion Protein-Based Platform for the Treatment of Friedrich'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
Adverum Biotechnologies Inc
Bio Blast Pharma Ltd
Pfizer Inc
ProQR Therapeutics NV
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
AGIL-FA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BB-FA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CTI-1601 - 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
JOT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QRX-604 - 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
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
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
Aug 05, 2015: Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Friedreich’s Ataxia at Two Leading Scientific Meetings in June
Mar 26, 2015: BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in Friedreich's Ataxia
Dec 17, 2014: BioBlast Receives Notice of Patent Issuance From USPTO for Its Novel Fusion Protein-Based Platform for the Treatment of Friedrich'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, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indication, H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Number of Products under Investigation by Universities/Institutes, H2 2017
Products under Investigation by Universities/Institutes, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Pipeline by Adverum Biotechnologies Inc, H2 2017
Pipeline by Bio Blast Pharma Ltd, H2 2017
Pipeline by Pfizer Inc, H2 2017
Pipeline by ProQR Therapeutics NV, H2 2017
Pipeline by Voyager Therapeutics Inc, H2 2017
Dormant Projects, H2 2017
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indication, H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Number of Products under Investigation by Universities/Institutes, H2 2017
Products under Investigation by Universities/Institutes, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Pipeline by Adverum Biotechnologies Inc, H2 2017
Pipeline by Bio Blast Pharma Ltd, H2 2017
Pipeline by Pfizer Inc, H2 2017
Pipeline by ProQR Therapeutics NV, H2 2017
Pipeline by Voyager Therapeutics Inc, H2 2017
Dormant Projects, H2 2017
LIST OF FIGURES
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indications, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017
COMPANIES MENTIONED
Adverum Biotechnologies Inc
Bio Blast Pharma Ltd
Pfizer Inc
ProQR Therapeutics NV
Voyager Therapeutics Inc
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indications, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017
COMPANIES MENTIONED
Adverum Biotechnologies Inc
Bio Blast Pharma Ltd
Pfizer Inc
ProQR Therapeutics NV
Voyager Therapeutics Inc
