
Friedreich Ataxia Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Description
Friedreich Ataxia Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Friedreich Ataxia - Drugs In Development, 2022, provides an overview of the Friedreich Ataxia (Central Nervous System) pipeline landscape.
Friedreich’s ataxia (FA) is a neuromuscular disease that mainly affects the nervous system and the heart. FA is a hereditary disease, caused by a defective gene that can be passed down through a family. Signs and symptoms include ataxia, weakness and spasticity, sensory impairment, skeletal abnormalities, cardiac difficulties and diabetes.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Friedreich Ataxia - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Friedreich Ataxia (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Friedreich Ataxia (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Friedreich Ataxia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 2, 5, 3, 20, 6 and 1 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 5 and 3 molecules, respectively.
Friedreich Ataxia (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note:Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Friedreich Ataxia - Drugs In Development, 2022, provides an overview of the Friedreich Ataxia (Central Nervous System) pipeline landscape.
Friedreich’s ataxia (FA) is a neuromuscular disease that mainly affects the nervous system and the heart. FA is a hereditary disease, caused by a defective gene that can be passed down through a family. Signs and symptoms include ataxia, weakness and spasticity, sensory impairment, skeletal abnormalities, cardiac difficulties and diabetes.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Friedreich Ataxia - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Friedreich Ataxia (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Friedreich Ataxia (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Friedreich Ataxia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 2, 5, 3, 20, 6 and 1 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 5 and 3 molecules, respectively.
Friedreich Ataxia (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note:Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Friedreich Ataxia (Central Nervous System).
- The pipeline guide reviews pipeline therapeutics for Friedreich Ataxia (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Friedreich Ataxia (Central Nervous System) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Friedreich Ataxia (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Friedreich Ataxia (Central Nervous System)
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Friedreich Ataxia (Central Nervous System).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Friedreich Ataxia (Central Nervous System) pipeline depth and focus of Indication therapeutics.
- 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
98 Pages
- Introduction
- Global Markets Direct Report Coverage
- Friedreich Ataxia – Overview
- Friedreich Ataxia – Therapeutics Development
- Pipeline Overview
- Pipeline by Companies
- Pipeline by Universities/Institutes
- Products under Development by Companies
- Products under Development by Universities/Institutes
- Friedreich Ataxia – Therapeutics Assessment
- Assessment by Target
- Assessment by Mechanism of Action
- Assessment by Route of Administration
- Assessment by Molecule Type
- Friedreich Ataxia – Companies Involved in Therapeutics Development
- AavantiBio Inc
- Amylyx Pharmaceuticals Inc
- Anima Biotech Inc
- Astellas Pharma Inc
- Biointaxis SL
- Biovista Inc
- Cellivery Therapeutics Inc
- CRISPR Therapeutics AG
- Cytonus Therapeutics Inc
- Design Therapeutics Inc
- Epirium Bio Inc
- Fratagene Therapeutics Srl
- Fulcrum Therapeutics Inc
- Healx Ltd
- Ixchel Pharma LLC
- Jupiter Neurosciences Inc
- Lacerta Therapeutics Inc
- LEXEO Therapeutics LLC
- Metro International Biotech LLC
- Minoryx Therapeutics sl
- Novartis Gene Therapies
- OMEICOS Therapeutics GmbH
- PTC Therapeutics Inc
- Reata Pharmaceuticals Inc
- Retrotope Inc
- Scriptr Global Inc
- Seelos Therapeutics, Inc.
- STATegics Inc
- Stealth BioTherapeutics Corp
- StrideBio Inc
- Voyager Therapeutics Inc
- Friedreich Ataxia – Drug Profiles
- (sodium phenylbutyrate + taurursodiol) – Drug Profile
- Product Description
- Mechanism Of Action
- AT-808 – Drug Profile
- Product Description
- Mechanism Of Action
- AVXS-401 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- BDTg-332 – Drug Profile
- Product Description
- Mechanism Of Action
- BTX-101 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- BVA-202 – Drug Profile
- Product Description
- Mechanism Of Action
- BVA-203 – Drug Profile
- Product Description
- Mechanism Of Action
- Cell Therapy for Central Nervous System and Neurodegenerative Diseases – Drug Profile
- Product Description
- Mechanism Of Action
- CV-14 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- deulinoleate ethyl – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- dextro epicatechin – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Drug for Friedreich Ataxia – Drug Profile
- Product Description
- Mechanism Of Action
- Drugs to Inhibit SRC for Friedreich Ataxia – Drug Profile
- Product Description
- Mechanism Of Action
- DT-216 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- elamipretide hydrochloride – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- etravirine – Drug Profile
- Product Description
- Mechanism Of Action
- Friedreich Ataxia – Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapies for Friedreich Ataxia and Myotonic Dystrophy – Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy for Friedreichs Ataxia – Drug Profile
- Product Description
- Mechanism Of Action
- 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
- Gene Therapy to Activate Frataxin Mitochondrial for Friedreich’s Ataxia – Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy to Activate FXN for Friedreich Ataxia – Drug Profile
- Product Description
- Mechanism Of Action
- IXC-103 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- IXC-109 – Drug Profile
- Product Description
- Mechanism Of Action
- JNS-101 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- leriglitazone hydrochloride – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- LX-2006 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- MIB-626 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- omaveloxolone – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- OMT-28 – Drug Profile
- Product Description
- Mechanism Of Action
- 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
- RT-002 – Drug Profile
- Product Description
- Mechanism Of Action
- SBT-550 – 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
- History of Events
- Small Molecules to Inhibit RNF126 for Friedreich Ataxia – Drug Profile
- Product Description
- Mechanism Of Action
- STRX-110 – Drug Profile
- Product Description
- Mechanism Of Action
- STSE-15 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- trehalose – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- vatiquinone – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- VYFXN-01 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Friedreich Ataxia – Dormant Projects
- Friedreich Ataxia – Discontinued Products
- Friedreich Ataxia – Product Development Milestones
- Featured News & Press Releases
- Mar 31, 2022: Reata Pharmaceuticals completes rolling submission of new drug application for Omaveloxolone for the treatment of patients with Friedreich’s Ataxia
- 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
- Mar 28, 2022: Stealth BioTherapeutics receives Orphan Drug Designation from FDA for Elamipretide for the treatment of Friedreich's 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
- Jan 31, 2022: Reata Pharmaceuticals initiates rolling submission of new drug application with U.S. FDA for Omaveloxolone for the treatment of patients with Friedreich’s ataxia
- Nov 18, 2021: Reata Pharmaceuticals receives Fast Track Designation from the FDA for Omaveloxolone for the treatment of Friedreich’s Ataxia
- Sep 30, 2021: Reata Pharmaceuticals plans NDA submission for Omaveloxolone in first quarter of 2022 following completion of pre-NDA meeting with FDA
- Jun 30, 2021: LEXEO Therapeutics receives rare pediatric disease designation and orphan drug designation for LX2006 for the treatment of Friedreich’s Ataxia
- May 19, 2021: Reata announces that the FDA has asked the company to request a pre-NDA meeting for omaveloxolone 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
- Dec 21, 2020: Seelos Therapeutics announces issuance of a patent for Trehalose (SLS-005) in Israel
- Dec 15, 2020: Minoryx’s clinical candidate leriglitazone shows clinical benefit in a proof of concept phase 2 study in Friedreich´s ataxia
- Nov 30, 2020: PTC Therapeutics announces initiation of global phase 3 clinical trial to evaluate vatiquinone in Friedreich Ataxia
- Nov 24, 2020: Reata provides update on Omaveloxolone program for patients with friedreich’s ataxia
- Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact Us
- Disclaimer
- List of Tables
- Table 1: Number of Products under Development for Friedreich Ataxia, 2022
- Table 2: Number of Products under Development by Companies, 2022
- Table 3: Number of Products under Development by Companies, 2022 (Contd..1)
- Table 4: Number of Products under Development by Universities/Institutes, 2022
- Table 5: Products under Development by Companies, 2022
- Table 6: Products under Development by Companies, 2022 (Contd..1)
- Table 7: Products under Development by Universities/Institutes, 2022
- Table 8: Number of Products by Stage and Target, 2022
- Table 9: Number of Products by Stage and Mechanism of Action, 2022
- Table 10: Number of Products by Stage and Route of Administration, 2022
- Table 11: Number of Products by Stage and Molecule Type, 2022
- Table 12: Friedreich Ataxia – Pipeline by AavantiBio Inc, 2022
- Table 13: Friedreich Ataxia – Pipeline by Amylyx Pharmaceuticals Inc, 2022
- Table 14: Friedreich Ataxia – Pipeline by Anima Biotech Inc, 2022
- Table 15: Friedreich Ataxia – Pipeline by Astellas Pharma Inc, 2022
- Table 16: Friedreich Ataxia – Pipeline by Biointaxis SL, 2022
- Table 17: Friedreich Ataxia – Pipeline by Biovista Inc, 2022
- Table 18: Friedreich Ataxia – Pipeline by Cellivery Therapeutics Inc, 2022
- Table 19: Friedreich Ataxia – Pipeline by CRISPR Therapeutics AG, 2022
- Table 20: Friedreich Ataxia – Pipeline by Cytonus Therapeutics Inc, 2022
- Table 21: Friedreich Ataxia – Pipeline by Design Therapeutics Inc, 2022
- Table 22: Friedreich Ataxia – Pipeline by Epirium Bio Inc, 2022
- Table 23: Friedreich Ataxia – Pipeline by Fratagene Therapeutics Srl, 2022
- Table 24: Friedreich Ataxia – Pipeline by Fulcrum Therapeutics Inc, 2022
- Table 25: Friedreich Ataxia – Pipeline by Healx Ltd, 2022
- Table 26: Friedreich Ataxia – Pipeline by Ixchel Pharma LLC, 2022
- Table 27: Friedreich Ataxia – Pipeline by Jupiter Neurosciences Inc, 2022
- Table 28: Friedreich Ataxia – Pipeline by Lacerta Therapeutics Inc, 2022
- Table 29: Friedreich Ataxia – Pipeline by LEXEO Therapeutics LLC, 2022
- Table 30: Friedreich Ataxia – Pipeline by Metro International Biotech LLC, 2022
- Table 31: Friedreich Ataxia – Pipeline by Minoryx Therapeutics sl, 2022
- Table 32: Friedreich Ataxia – Pipeline by Novartis Gene Therapies, 2022
- Table 33: Friedreich Ataxia – Pipeline by OMEICOS Therapeutics GmbH, 2022
- Table 34: Friedreich Ataxia – Pipeline by PTC Therapeutics Inc, 2022
- Table 35: Friedreich Ataxia – Pipeline by Reata Pharmaceuticals Inc, 2022
- Table 36: Friedreich Ataxia – Pipeline by Retrotope Inc, 2022
- Table 37: Friedreich Ataxia – Pipeline by Scriptr Global Inc, 2022
- Table 38: Friedreich Ataxia – Pipeline by Seelos Therapeutics, Inc., 2022
- Table 39: Friedreich Ataxia – Pipeline by STATegics Inc, 2022
- Table 40: Friedreich Ataxia – Pipeline by Stealth BioTherapeutics Corp, 2022
- Table 41: Friedreich Ataxia – Pipeline by StrideBio Inc, 2022
- Table 42: Friedreich Ataxia – Pipeline by Voyager Therapeutics Inc, 2022
- Table 43: Friedreich Ataxia – Dormant Projects, 2022
- Table 44: Friedreich Ataxia – Dormant Projects, 2022 (Contd..1)
- Table 45: Table 46: Friedreich Ataxia – Discontinued Products, 2022
- List of Figures
- Figure 1: Number of Products under Development for Friedreich Ataxia, 2022
- Figure 2: Number of Products under Development by Companies, 2022
- Figure 3: Number of Products under Development by Universities/Institutes, 2022
- Figure 4: Number of Products by Top 10 Targets, 2022
- Figure 5: Number of Products by Stage and Top 10 Targets, 2022
- Figure 6: Number of Products by Top 10 Mechanism of Actions, 2022
- Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, 2022
- Figure 8: Number of Products by Routes of Administration, 2022
- Figure 9: Number of Products by Stage and Routes of Administration, 2022
- Figure 10: Number of Products by Molecule Types, 2022
- Figure 11: Number of Products by Stage and Molecule Types, 2022
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