Friedreich Ataxia Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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

  • 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)
Reasons to Buy
  • 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.


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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