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Friedreich Ataxia-Pipeline Review, H1 2018

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Friedreich Ataxia-Pipeline Review, H1 2018



Executive Summary

Friedreich Ataxia-Pipeline Review, H1 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Friedreich Ataxia-Pipeline Review, H1 2018, 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-Pipeline Review, H1 2018, 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 and Discovery stages are 1, 5, 1, 14 and 2 respectively. Similarly, the Universities portfolio in Phase III, Preclinical and Discovery stages comprises 1, 1 and 2 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.

Table of Contents

List of Tables

List of Figures

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

Adverum Biotechnologies Inc

Bio Blast Pharma Ltd

BioElectron Technology Corp

BioMarin Pharmaceutical Inc

Biovista Inc

Catabasis Pharmaceuticals Inc

Ixchel Pharma LLC

Pfizer Inc

ProQR Therapeutics NV

Reata Pharmaceuticals Inc

STATegics Inc

Takeda Pharmaceutical Co Ltd

Voyager Therapeutics Inc

Friedreich Ataxia-Drug Profiles

(sodium phenylbutyrate + tauroursodeoxycholic acid)-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

BMN-290-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BVA-202-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BVA-203-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CAT-4001-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Cell Therapy for Central Nervous System and Neurodegenerative Diseases-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CTI-1601-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

dextro epicatechin-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

interferon gamma-1b-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

IXC-103-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JOT-101-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

omaveloxolone-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QRX-604-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RG-3250-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RT-001-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RT-002-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

STSE-15-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

TAK-831-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

vatiquinone-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

VYFXN-01-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Friedreich Ataxia-Dormant Projects

Friedreich Ataxia-Discontinued Products

Friedreich Ataxia-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

Nov 17, 2016: Catabasis Pharmaceuticals Provides Update on Rare Disease Program CAT-4001 at Investor Day

Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich's Ataxia

Jan 19, 2016: FARA Announces Catabasis Pharmaceuticals as the Recipient of the Kyle Bryant Translational Research Award to Evaluate CAT-4001 as a Potential Therapy for 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

Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference

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

Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia

Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia

Nov 04, 2013: Edison Pharmaceuticals, FARA, and USF Announce Initiation of EPI-743 Phase 2 Friedreich's Ataxia Clinical Trial in Adults with Point Mutations

Jan 07, 2013: Edison Pharma Initiates Phase IIb Clinical Trial Of EPI-743 In Friedreich Ataxia

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

To know more information on Purchase by Section, please send a mail to sales@kenresearch.com

List of Figures

Number of Products under Development for Friedreich Ataxia, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Targets, H1 2018

Number of Products by Stage and Targets, H1 2018

Number of Products by Mechanism of Actions, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

List of Tables

Number of Products under Development for Friedreich Ataxia, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Friedreich Ataxia-Pipeline by Adverum Biotechnologies Inc, H1 2018

Friedreich Ataxia-Pipeline by Bio Blast Pharma Ltd, H1 2018

Friedreich Ataxia-Pipeline by BioElectron Technology Corp, H1 2018

Friedreich Ataxia-Pipeline by BioMarin Pharmaceutical Inc, H1 2018

Friedreich Ataxia-Pipeline by Biovista Inc, H1 2018

Friedreich Ataxia-Pipeline by Catabasis Pharmaceuticals Inc, H1 2018

Friedreich Ataxia-Pipeline by Ixchel Pharma LLC, H1 2018

Friedreich Ataxia-Pipeline by Pfizer Inc, H1 2018

Friedreich Ataxia-Pipeline by ProQR Therapeutics NV, H1 2018

Friedreich Ataxia-Pipeline by Reata Pharmaceuticals Inc, H1 2018

Friedreich Ataxia-Pipeline by STATegics Inc, H1 2018

Friedreich Ataxia-Pipeline by Takeda Pharmaceutical Co Ltd, H1 2018

Friedreich Ataxia-Pipeline by Voyager Therapeutics Inc, H1 2018

Friedreich Ataxia-Dormant Projects, H1 2018

Friedreich Ataxia-Discontinued Products, H1 2018

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Adverum Biotechnologies Inc

Bio Blast Pharma Ltd

BioElectron Technology Corp

BioMarin Pharmaceutical Inc

Biovista Inc

Catabasis Pharmaceuticals Inc

Ixchel Pharma LLC

Pfizer Inc

ProQR Therapeutics NV

Reata Pharmaceuticals Inc

STATegics Inc

Takeda Pharmaceutical Co Ltd

Voyager Therapeutics Inc


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