Friedreich Ataxia (Central Nervous System) – Drugs In Development, 2021

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Friedreich Ataxia – Drugs In Development, 2021, 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, 2021, 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 Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 3, 4, 3, 21, 5 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.

Companies mentioned

AavantiBio Inc

Adverum Biotechnologies Inc

Amylyx Pharmaceuticals Inc

Anima Biotech Inc

Biointaxis SL

Biovista Inc

Cellivery Therapeutics Inc

CRISPR Therapeutics AG

Design Therapeutics Inc

Epirium Bio Inc

Exicure Inc

Fratagene Therapeutics Srl

Fulcrum Therapeutics Inc

Healx Ltd

Ixchel Pharma LLC

Jupiter Orphan Therapeutics Inc

Lacerta Therapeutics Inc

Larimar Therapeutics Inc

LEXEO Therapeutics LLC

Metro International Biotech LLC

Minoryx Therapeutics sl

Novartis Gene Therapies

PTC Therapeutics Inc

Reata Pharmaceuticals Inc

Retrotope Inc

Seelos Therapeutics, Inc.

STATegics Inc

Stealth BioTherapeutics Corp

StrideBio Inc

Voyager Therapeutics Inc

Table of Contents

Table of Contents

Introduction

Friedreich Ataxia - Overview

Friedreich Ataxia - Therapeutics Development

Friedreich Ataxia - Therapeutics Assessment

Friedreich Ataxia - Companies Involved in Therapeutics Development

Friedreich Ataxia - Drug Profiles

Friedreich Ataxia - Dormant Projects

Friedreich Ataxia - Discontinued Products

Friedreich Ataxia - Product Development Milestones

Appendix

List of Tables

List of Tables

Number of Products under Development for Friedreich Ataxia, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Companies, 2021 (Contd..1)

Number of Products under Development by Universities/Institutes, 2021

Products under Development by Companies, 2021

Products under Development by Companies, 2021 (Contd..1)

Products under Development by Companies, 2021 (Contd..2)

Products under Development by Universities/Institutes, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Friedreich Ataxia – Pipeline by AavantiBio Inc, 2021

Friedreich Ataxia – Pipeline by Adverum Biotechnologies Inc, 2021

Friedreich Ataxia – Pipeline by Amylyx Pharmaceuticals Inc, 2021

Friedreich Ataxia – Pipeline by Anima Biotech Inc, 2021

Friedreich Ataxia – Pipeline by Biointaxis SL, 2021

Friedreich Ataxia – Pipeline by Biovista Inc, 2021

Friedreich Ataxia – Pipeline by Cellivery Therapeutics Inc, 2021

Friedreich Ataxia – Pipeline by CRISPR Therapeutics AG, 2021

Friedreich Ataxia – Pipeline by Design Therapeutics Inc, 2021

Friedreich Ataxia – Pipeline by Epirium Bio Inc, 2021

Friedreich Ataxia – Pipeline by Exicure Inc, 2021

Friedreich Ataxia – Pipeline by Fratagene Therapeutics Srl, 2021

Friedreich Ataxia – Pipeline by Fulcrum Therapeutics Inc, 2021

Friedreich Ataxia – Pipeline by Healx Ltd, 2021

Friedreich Ataxia – Pipeline by Ixchel Pharma LLC, 2021

Friedreich Ataxia – Pipeline by Jupiter Orphan Therapeutics Inc, 2021

Friedreich Ataxia – Pipeline by Lacerta Therapeutics Inc, 2021

Friedreich Ataxia – Pipeline by Larimar Therapeutics Inc, 2021

Friedreich Ataxia – Pipeline by LEXEO Therapeutics LLC, 2021

Friedreich Ataxia – Pipeline by Metro International Biotech LLC, 2021

Friedreich Ataxia – Pipeline by Minoryx Therapeutics sl, 2021

Friedreich Ataxia – Pipeline by Novartis Gene Therapies, 2021

Friedreich Ataxia – Pipeline by PTC Therapeutics Inc, 2021

Friedreich Ataxia – Pipeline by Reata Pharmaceuticals Inc, 2021

Friedreich Ataxia – Pipeline by Retrotope Inc, 2021

Friedreich Ataxia – Pipeline by Seelos Therapeutics, Inc., 2021

Friedreich Ataxia – Pipeline by STATegics Inc, 2021

Friedreich Ataxia – Pipeline by Stealth BioTherapeutics Corp, 2021

Friedreich Ataxia – Pipeline by StrideBio Inc, 2021

Friedreich Ataxia – Pipeline by Voyager Therapeutics Inc, 2021

Friedreich Ataxia – Dormant Projects, 2021

Friedreich Ataxia – Dormant Projects, 2021 (Contd..1)

Friedreich Ataxia – Discontinued Products, 2021

List of Figures

List of Figures

Number of Products under Development for Friedreich Ataxia, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Number of Products by Top 10 Targets, 2021

Number of Products by Stage and Top 10 Targets, 2021

Number of Products by Top 10 Mechanism of Actions, 2021

Number of Products by Stage and Top 10 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

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