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Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Drugs In Development, 2021

  • ID: 5359566
  • Drug Pipelines
  • June 2021
  • Region: Global
  • 77 pages
  • Global Markets Direct

FEATURED COMPANIES

  • AavantiBio Inc
  • Anima Biotech Inc
  • Biointaxis SL
  • CRISPR Therapeutics AG
  • Design Therapeutics Inc
  • Fulcrum Therapeutics Inc
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Summary

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.

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 molecules developed by companies in Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 9 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.

The latest report Frataxin Mitochondrial - Drugs In Development, 2021, 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. 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.

The report is built using 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
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • AavantiBio Inc
  • Anima Biotech Inc
  • Biointaxis SL
  • CRISPR Therapeutics AG
  • Design Therapeutics Inc
  • Fulcrum Therapeutics Inc
Introduction
  • 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
  • Anima Biotech Inc
  • Biointaxis SL
  • Cellivery Therapeutics Inc
  • CRISPR Therapeutics AG
  • Design Therapeutics Inc
  • Fratagene Therapeutics Srl
  • Fulcrum Therapeutics Inc
  • Jupiter Orphan Therapeutics Inc
  • Lacerta Therapeutics Inc
  • Larimar Therapeutics Inc
  • Novartis Gene Therapies
  • 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
AVXS-401 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
CTI-1601 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
CV-14 - 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 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 Mitochondrial for Friedreich’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
Gene Therapy to Activate FXN for Friedreich 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
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
Recombinant Proteins to Replace Frataxin Mitochondrial for Friedreich Ataxia - 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
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
  • May 25, 2021: Larimar Therapeutics reports FDA clinical hold on CTI-1601 and termination of recently announced private placement financing
  • May 20, 2021: Larimar Therapeutics receives European Medicines Agency Priority Medicines (PRIME) designation for CTI-1601 in Friedreich’s ataxia
  • May 11, 2021: Larimar Therapeutics reports positive topline phase 1 clinical trial data showing dose-dependent increases in frataxin levels in patients with Friedreich’s Ataxia
  • Dec 08, 2020: Larimar Therapeutics announces completion of dosing of the single ascending dose clinical trial in Friedreich’s Ataxia patients and provides program update
  • Jul 28, 2020: Larimar Therapeutics announces positive opinion on Orphan Drug Designation received from the European Medicines Agency for CTI-1601 for the treatment of Friedreich’s Ataxia
  • Jul 20, 2020: Larimar Therapeutics announces dosing of patients in third cohort of phase 1 SAD trial of CTI-1601 for treatment of Friedreich’s ataxia
  • 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
  • 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
Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact the Publisher
  • Disclaimer
List of Tables
  • Number of Products under Development by Stage of Development, 2021
  • Number of Products under Development by Therapy Areas, 2021
  • Number of Products under Development by Indication, 2021
  • Number of Products under Development by Companies, 2021
  • Products under Development by Companies, 2021
  • Number of Products under Investigation by Universities/Institutes, 2021
  • Products under Investigation by Universities/Institutes, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Stage and Route of Administration, 2021
  • Number of Products by Stage and Molecule Type, 2021
  • Pipeline by AavantiBio Inc, 2021
  • Pipeline by Adverum Biotechnologies Inc, 2021
  • Pipeline by Anima Biotech Inc, 2021
  • Pipeline by Biointaxis SL, 2021
  • Pipeline by Cellivery Therapeutics Inc, 2021
  • Pipeline by CRISPR Therapeutics AG, 2021
  • Pipeline by Design Therapeutics Inc, 2021
  • Pipeline by Fratagene Therapeutics Srl, 2021
  • Pipeline by Fulcrum Therapeutics Inc, 2021
  • Pipeline by Jupiter Orphan Therapeutics Inc, 2021
  • Pipeline by Lacerta Therapeutics Inc, 2021
  • Pipeline by Larimar Therapeutics Inc, 2021
  • Pipeline by Novartis Gene Therapies, 2021
  • Pipeline by PTC Therapeutics Inc, 2021
  • Pipeline by Voyager Therapeutics Inc, 2021
  • Dormant Projects, 2021
List of Figures
  • Number of Products under Development by Stage of Development, 2021
  • Number of Products under Development by Therapy Areas, 2021
  • Number of Products under Development by Top 10 Indications, 2021
  • Number of Products by Mechanism of Actions, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Routes of Administration, 2021
  • Number of Products by Stage and Routes of Administration, 2021
  • Number of Products by Molecule Types, 2021
  • Number of Products by Stage and Molecule Types, 2021
Note: Product cover images may vary from those shown
  • AavantiBio Inc
  • Adverum Biotechnologies Inc
  • Anima Biotech Inc
  • Biointaxis SL
  • Cellivery Therapeutics Inc
  • CRISPR Therapeutics AG
  • Design Therapeutics Inc
  • Fratagene Therapeutics Srl
  • Fulcrum Therapeutics Inc
  • Jupiter Orphan Therapeutics Inc
  • Lacerta Therapeutics Inc
  • Larimar Therapeutics Inc
  • Novartis Gene Therapies
  • PTC Therapeutics Inc
  • Voyager Therapeutics Inc
Note: Product cover images may vary from those shown
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