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Published: Sep, 2017 | Pages:
84 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Friedreich Ataxia - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Friedreich Ataxia - Pipeline Review, H2 2017, 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, H2 2017, 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, 4, 3, 13 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 BioElectron Technology Corp Biohaven Pharmaceutical Holding Company Ltd BioMarin Pharmaceutical Inc Biovista Inc Catabasis Pharmaceuticals Inc Pfizer Inc ProQR Therapeutics NV Reata Pharmaceuticals Inc Shire Plc STATegics Inc Takeda Pharmaceutical Company Ltd Translate Bio Inc 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 BHV-4157 - 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 JOT-101 - Drug Profile Product Description Mechanism Of Action R&D Progress Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - 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 SHP-622 - 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 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 Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference 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 Aug 01, 2012: Intellect Neurosciences Receives New Patent Allowance For OX1 Aug 02, 2011: STATegics, Inc. Announces a Grant from Friedreich's Ataxia Research Alliance to Support Its Program to Develop Small Molecule Mimetics of Erythropoietin for the Treatment of Friedreich's Ataxia Apr 12, 2011: Intellect Neurosciences Files Orphan Drug Application In US For OX1 In Friedreich's Ataxia Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer
List of Tables
Number of Products under Development for Friedreich Ataxia, H2 2017 Number of Products under Development by Companies, H2 2017 Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017 Number of Products under Development by Universities/Institutes, H2 2017 Products under Development by Companies, H2 2017 Products under Development by Companies, H2 2017 (Contd..1), H2 2017 Products under Development by Universities/Institutes, H2 2017 Number of Products by Stage and Target, H2 2017 Number of Products by Stage and Mechanism of Action, H2 2017 Number of Products by Stage and Route of Administration, H2 2017 Number of Products by Stage and Molecule Type, H2 2017 Friedreich Ataxia - Pipeline by Adverum Biotechnologies Inc, H2 2017 Friedreich Ataxia - Pipeline by BioElectron Technology Corp, H2 2017 Friedreich Ataxia - Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H2 2017 Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc, H2 2017 Friedreich Ataxia - Pipeline by Biovista Inc, H2 2017 Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals Inc, H2 2017 Friedreich Ataxia - Pipeline by Pfizer Inc, H2 2017 Friedreich Ataxia - Pipeline by ProQR Therapeutics NV, H2 2017 Friedreich Ataxia - Pipeline by Reata Pharmaceuticals Inc, H2 2017 Friedreich Ataxia - Pipeline by Shire Plc, H2 2017 Friedreich Ataxia - Pipeline by STATegics Inc, H2 2017 Friedreich Ataxia - Pipeline by Takeda Pharmaceutical Company Ltd, H2 2017 Friedreich Ataxia - Pipeline by Translate Bio Inc, H2 2017 Friedreich Ataxia - Pipeline by Voyager Therapeutics Inc, H2 2017 Friedreich Ataxia - Dormant Projects, H2 2017 Friedreich Ataxia - Discontinued Products, H2 2017
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