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亨丁頓蛋白(Huntingtin):開發中產品分析

Huntingtin (Huntington Disease Protein or HTT) - Pipeline Review, H2 2017

出版商 Global Markets Direct 商品編碼 358692
出版日期 內容資訊 英文 60 Pages
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亨丁頓蛋白(Huntingtin):開發中產品分析 Huntingtin (Huntington Disease Protein or HTT) - Pipeline Review, H2 2017
出版日期: 2017年10月31日 內容資訊: 英文 60 Pages
簡介

本報告提供全球各國治療亨丁頓蛋白(Huntingtin) 所用的開發中產品之開發情形相關分析,提供您產品開發·上市的最新趨勢,及臨床實驗的各階段產品一覽,主要企業簡介,主要藥物概要,最新的產業趨勢等資訊。

目錄

  • 簡介
    • 分析範圍
  • 亨丁頓蛋白(Huntingtin)的概要
  • 治療藥的開發
    • 臨床實驗中的產品:臨床實驗的各階段
    • 臨床實驗中的產品:各治療領域
    • 臨床實驗中的產品:不同症狀
  • 開發中產品的概要
    • 後期階段的產品
    • 初期階段的產品
  • 各企業開發中的產品
  • 治療藥的評估
    • 單劑治療藥/並用治療藥的情況
    • 各作用機制
    • 各投藥途徑
    • 各分子類型
  • 亨丁頓蛋白(Huntingtin)的治療藥開發企業
    • AFFiRiS AG
    • Ionis Pharmaceuticals, Inc.
    • Neurimmune Holding AG
    • nLife Therapeutics, S.L.
    • reMYND NV
    • Sangamo BioSciences, Inc.
    • UniQure N.V.
    • Voyager Therapeutics, Inc.
    • Vybion, Inc.
    • WAVE Life Sciences Ltd.
  • 藥物簡介
    • AMT-130
      • 產品概要
      • 作用機制
      • 研究開發 (R&D)的進展
    • 亨丁頓舞蹈症蛋白質抑制用的反義寡核甘酸1
    • 亨丁頓舞蹈症蛋白質抑制用的反義寡核甘酸2
    • 亨丁頓舞蹈症HTT抑制用的反義寡核甘酸
    • 亨丁頓舞蹈症蛋白質抑制用的反義寡核甘酸
    • 亨丁頓舞蹈症蛋白質抑制用的基因治療
    • INT-41
    • IONIS-HTTRx
    • NI-302
    • 亨丁頓舞蹈症蛋白質抑制用的小分子
    • 亨丁頓舞蹈症蛋白質抑制用疫苗
    • VYHTT-01
  • 開發暫停的計劃
  • 值得注意的最新趨勢·新聞稿 (全7件)
  • 附錄

圖表一覽

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目錄
Product Code: GMDHC1076TDB

Summary:

According to the recently published report 'Huntingtin - Pipeline Review, H2 2017'; Huntingtin (Huntington Disease Protein or HTT) pipeline Target constitutes close to 16 molecules. Out of which approximately 14 molecules are developed by companies and remaining by the universities/institutes.

Huntingtin (Huntington Disease Protein or HTT) - Huntingtin protein is an encoded by the huntingtin gene, also called the HTT or HD (Huntington disease) gene. Huntingtin up regulates the expression of brain derived neurotrophic factor (BDNF) at the transcription level. Huntingtin is primarily associated with vesicles and microtubules. These indicate an important role in cytoskeletal anchoring or transport of mitochondria. The Htt protein is involved in vesicle trafficking as it interacts with HIP1 to mediate endocytosis. Huntingtin also plays an important role in the establishment in epithelial polarity through its interaction with RAB11A.

The report 'Huntingtin - Pipeline Review, H2 2017' outlays comprehensive information on the Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities.

It also reviews key players involved in Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Phase II, Preclinical and Discovery stages are 3, 7 and 4 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Genetic Disorders which include indications Huntington Disease, Alzheimer's Disease, Kennedy's Disease (Spinal and Bulbar Muscular Atrophy) and Spinocerebellar Ataxia (SCA).

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 Huntingtin (Huntington Disease Protein or HTT)
  • The report reviews Huntingtin (Huntington Disease Protein or HTT) 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 Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Huntingtin (Huntington Disease Protein or HTT) 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 Huntingtin (Huntington Disease Protein or HTT) 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 Huntingtin (Huntington Disease Protein or HTT)
  • 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 Huntingtin (Huntington Disease Protein or HTT) 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

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Huntingtin (Huntington Disease Protein or HTT) - Overview
    • Huntingtin (Huntington Disease Protein or HTT) - 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
  • Huntingtin (Huntington Disease Protein or HTT) - Therapeutics Assessment
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Huntingtin (Huntington Disease Protein or HTT) - Companies Involved in Therapeutics Development
    • AFFiRiS AG
    • Ionis Pharmaceuticals Inc
    • Neurimmune Holding AG
    • nLife Therapeutics SL
    • PTC Therapeutics Inc
    • reMYND NV
    • Shire Plc
    • UniQure NV
    • Voyager Therapeutics Inc
    • Vybion Inc
    • WAVE Life Sciences Ltd
  • Huntingtin (Huntington Disease Protein or HTT) - Drug Profiles
    • AMT-130 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Antisense Oligonucleotide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Antisense Oligonucleotides to Inhibit HTT for Huntington's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Antisense Oligonucleotides to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • INT-41 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IONIS-HTTRx - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • NI-302 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • NLF-HD XXXX - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • P-301905 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Synthetic Peptide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Vaccine to Target Huntingtin for Huntington's Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VYHTT-01 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • WVE-120101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • WVE-120102 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Huntingtin (Huntington Disease Protein or HTT) - Dormant Products
  • Huntingtin (Huntington Disease Protein or HTT) - Product Development Milestones
    • Featured News & Press Releases
      • Oct 18, 2017: uniQure Presents New Preclinical Data on AMT-130 in Huntington's Disease at the ESGCT 25th Anniversary Congress in Berlin
      • Oct 06, 2017: UniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntingtons disease
      • Jul 17, 2017: Wave Life Sciences Initiates Phase 1b/2a Clinical Trial: PRECISION-HD1 in Patients with Huntington's Disease
      • Jul 17, 2017: Wave Life Sciences Announces Initiation of Phase 1b/2a Clinical Trial: PRECISION-HD2 in Patients with Huntington's Disease
      • Jun 22, 2017: Enrollment in Phase 1/2a Study of IONIS-HTT Rx in Patients with Huntingtons Disease Completed and Open-Label Extension Study to Open in 2H 2017
      • Jun 01, 2017: Voyager Therapeutics Selects Lead Clinical Candidate for Huntington's Disease
      • Apr 26, 2017: uniQure Presents New Preclinical Data on AMT-130 in Huntington's Disease at CHDI's 12th Annual Huntington's Disease Therapeutics Conference
      • Apr 24, 2017: uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy
      • Apr 04, 2017: uniQure Publishes Data Demonstrating Wide Distribution of AAV5 in the Central Nervous System
      • Jan 30, 2017: Vybion Huntington's Disease Drug Patent receives Track I Status
      • Jan 06, 2017: WAVE Life Sciences Provides Update on Lead Huntington's Disease Program WVE-120101
      • Jan 06, 2017: WAVE Life Sciences Provides Update on Lead Huntington's Disease Program WVE-120102
      • Aug 24, 2016: Novel Gene Therapy Drug for Huntington Disease
      • Jun 21, 2016: WAVE Life Sciences Receives Orphan Drug Designation from FDA for its Lead Candidate Designed to Treat Huntington's Disease
      • Mar 22, 2016: uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington's Disease
  • 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, H2 2017
  • Number of Products under Development by Therapy Areas, H2 2017
  • Number of Products under Development by Indication, H2 2017
  • Number of Products under Development by Companies, H2 2017
  • Products under Development by Companies, H2 2017
  • Number of Products under Investigation by Universities/Institutes, H2 2017
  • Products under Investigation by Universities/Institutes, H2 2017
  • Number of Products by Stage and Mechanism of Actions, H2 2017
  • Number of Products by Stage and Route of Administration, H2 2017
  • Number of Products by Stage and Molecule Type, H2 2017
  • Pipeline by AFFiRiS AG, H2 2017
  • Pipeline by Ionis Pharmaceuticals Inc, H2 2017
  • Pipeline by Neurimmune Holding AG, H2 2017
  • Pipeline by nLife Therapeutics SL, H2 2017
  • Pipeline by PTC Therapeutics Inc, H2 2017
  • Pipeline by reMYND NV, H2 2017
  • Pipeline by Shire Plc, H2 2017
  • Pipeline by UniQure NV, H2 2017
  • Pipeline by Voyager Therapeutics Inc, H2 2017
  • Pipeline by Vybion Inc, H2 2017
  • Pipeline by WAVE Life Sciences Ltd, H2 2017
  • Dormant Projects, H2 2017

List of Figures

  • Number of Products under Development by Stage of Development, H2 2017
  • Number of Products under Development by Therapy Areas, H2 2017
  • Number of Products under Development by Top 10 Indications, H2 2017
  • Number of Products by Mechanism of Actions, H2 2017
  • Number of Products by Stage and Mechanism of Actions, H2 2017
  • Number of Products by Routes of Administration, H2 2017
  • Number of Products by Stage and Routes of Administration, H2 2017
  • Number of Products by Molecule Types, H2 2017
  • Number of Products by Stage and Molecule Types, H2 2017
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