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市場調查報告書

Frataxin線粒體(代謝鐵的粒腺體蛋白):開發中產品分析

Frataxin Mitochondrial - Pipeline Review, H1 2020

出版商 Global Markets Direct 商品編碼 371023
出版日期 內容資訊 英文 55 Pages
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Frataxin線粒體(代謝鐵的粒腺體蛋白):開發中產品分析 Frataxin Mitochondrial - Pipeline Review, H1 2020
出版日期: 2020年02月28日內容資訊: 英文 55 Pages
簡介

本報告提供以Frataxin為標的之治療藥開發平台現狀及最新更新的各開發階段比較分析,提供您加上最新的新聞和發表之企業和研究機關開發中的治療藥,治療藥評估,後期階段及中止的計劃等相關資訊。

簡介

  • 調查範圍

Frataxin線粒體(代謝鐵的粒腺體蛋白)概要

治療藥的開發

  • 開發中的產品:各開發階段
  • 開發中的產品:各治療領域
  • 開發中的產品:不同症狀

開發中產品概況

  • 後期階段的產品
  • 初期階段的產品

企業開發中的產品

大學/機關開發中的產品

治療藥的評估

  • 單劑/並用治療藥的情況
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

治療藥的開發企業

  • Adverum Biotechnologies, Inc.
  • Bio Blast Pharma Ltd.
  • ProQR Therapeutics N.V.
  • RaNA Therapeutics, Inc.
  • Voyager Therapeutics, Inc.

藥物簡介

主要消息及新聞稿

附錄

圖表

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目錄
Product Code: GMDHC2290TDB

Summary

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) pipeline Target constitutes close to 14 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Frataxin Mitochondrial - Pipeline Review, H1 2020, outlays comprehensive information on the Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Frataxin is a protein encoded by the FXN gene. It promotes the biosynthesis of heme and assembly and repair of iron-sulfur clusters by delivering Fe2+ to proteins involved in these pathways. It plays a role in the protection against iron-catalyzed oxidative stress through its ability to catalyze the oxidation of Fe2+ to Fe3. The molecules developed by companies in Phase I, Preclinical and Discovery stages are 2, 6 and 4 respectively. Similarly, the universities portfolio in Discovery stages comprises 2 molecules, respectively. Report covers products from therapy areas Central Nervous System and Cardiovascular which include indications Friedreich Ataxia and Cardiomyopathy.

Furthermore, this report also reviews key players involved in Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by data and information sourced from 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.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The report provides a snapshot of the global therapeutic landscape for Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)
  • The report reviews Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews latest news and deals related to Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics

Reasons to buy

  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Identify and understand the targeted therapy areas and indications for Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)
  • Identify the use of drugs for target identification and drug repurposing
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
  • Devise corrective measures for pipeline projects by understanding Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) development landscape
  • 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

Table of Contents

List of Tables

List of Figures

Introduction

  • Global Markets Direct Report Coverage
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Overview
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Therapeutics Development
  • Products under Development by Stage of Development
  • Products under Development by Therapy Area
  • Products under Development by Indication
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Therapeutics Assessment
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Companies Involved in Therapeutics Development
  • AavantiBio Inc
  • Adverum Biotechnologies Inc
  • Biointaxis SL
  • Chondrial Therapeutics LLC
  • Design Therapeutics Inc
  • Fratagene Therapeutics Srl
  • Fulcrum Therapeutics Inc
  • Jupiter Orphan Therapeutics Inc
  • Panorama Research Inc
  • Pfizer Inc
  • PTC Therapeutics Inc
  • Voyager Therapeutics Inc
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Drug Profiles
  • ADVM-063 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • CTI-1601 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • etravirine - 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 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
  • Gene Therapy to Activate FXN for Friedreich Ataxia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • JOT-101 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • PTC-FA - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecule to Activate Frataxin for Friedreich Ataxia - 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
  • Synthetic Peptide to Activate Frataxin for Friedreich Ataxia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • VYFXN-01 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Dormant Products
  • Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) - Product Development Milestones
  • Featured News & Press Releases
  • Jan 13, 2020: MDA awards venture philanthropy funding of more than $1M to AavantiBio to develop gene-targeting therapy for Friedreich's Ataxia
  • Dec 18, 2019: Chondrial Therapeutics announces dosing of first patients in phase 1 clinical program of CTI-1601 for treatment of Friedreich's ataxia and CTI-1601 granted rare pediatric disease designation and fast track designation by U.S. FDA
  • Nov 30, 2019: GoFAR partners with AavantiBio to advance gene therapy for Friedreich's Ataxia
  • Nov 30, 2019: GoFAR partners with AavantiBio to advance gene therapy for Friedreich's Ataxia
  • May 18, 2018: Voyager Therapeutics Announces Additional Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting
  • 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
  • Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich's Ataxia
  • Feb 18, 2016: Agilis Biotherapeutics and Waisman Biomanufacturing enter into exclusive manufacturing agreement for Friedreich's Ataxia gene therapy
  • Aug 05, 2015: Key Patent Granted For AAVLife's Gene-Therapy Program to Treat Cardiomyopathy 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 by Stage of Development, H1 2020
  • Number of Products under Development by Therapy Areas, H1 2020
  • Number of Products under Development by Indication, H1 2020
  • Number of Products under Development by Companies, H1 2020
  • Products under Development by Companies, H1 2020
  • Number of Products under Investigation by Universities/Institutes, H1 2020
  • Products under Investigation by Universities/Institutes, H1 2020
  • Number of Products by Stage and Mechanism of Actions, H1 2020
  • Number of Products by Stage and Route of Administration, H1 2020
  • Number of Products by Stage and Molecule Type, H1 2020
  • Pipeline by AavantiBio Inc, H1 2020
  • Pipeline by Adverum Biotechnologies Inc, H1 2020
  • Pipeline by Biointaxis SL, H1 2020
  • Pipeline by Chondrial Therapeutics LLC, H1 2020
  • Pipeline by Design Therapeutics Inc, H1 2020
  • Pipeline by Fratagene Therapeutics Srl, H1 2020
  • Pipeline by Fulcrum Therapeutics Inc, H1 2020
  • Pipeline by Jupiter Orphan Therapeutics Inc, H1 2020
  • Pipeline by Panorama Research Inc, H1 2020
  • Pipeline by Pfizer Inc, H1 2020
  • Pipeline by PTC Therapeutics Inc, H1 2020
  • Pipeline by Voyager Therapeutics Inc, H1 2020
  • Dormant Projects, H1 2020

List of Figures

  • Number of Products under Development by Stage of Development, H1 2020
  • Number of Products under Development by Therapy Areas, H1 2020
  • Number of Products under Development by Top 10 Indications, H1 2020
  • Number of Products by Mechanism of Actions, H1 2020
  • Number of Products by Stage and Mechanism of Actions, H1 2020
  • Number of Products by Routes of Administration, H1 2020
  • Number of Products by Stage and Routes of Administration, H1 2020
  • Number of Products by Molecule Types, H1 2020
  • Number of Products by Stage and Molecule Types, H1 2020