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市場調查報告書

運動神經元存活蛋白:開發中產品分析

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H1 2018

出版商 Global Markets Direct 商品編碼 365803
出版日期 內容資訊 英文 70 Pages
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運動神經元存活蛋白:開發中產品分析 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H1 2018
出版日期: 2018年01月31日 內容資訊: 英文 70 Pages
簡介

本報告提供以運動神經元存活蛋白為標的治療藥之開發平台現狀及最新更新的各開發階段比較分析,提供您附最新的新聞和發表之企業和研究機關正在開發的治療藥,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

運動神經元存活蛋白 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

  • AveXis, Inc.
  • Bioblast Pharma Ltd.
  • F. Hoffmann-La Roche Ltd.
  • Genethon
  • Genzyme Corporation
  • Ionis Pharmaceuticals, Inc.
  • Novartis AG
  • Sarepta Therapeutics, Inc.
  • Voyager Therapeutics, Inc.
  • WAVE Life Sciences Ltd.

藥物簡介

暫停中的計劃

主要消息及新聞稿

附錄

圖表

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目錄
Product Code: GMDHC1280TDB

Summary:

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 16 molecules. Out of which approximately 11 molecules are developed by companies and remaining by the universities/institutes. The latest report Survival Motor Neuron Protein - Pipeline Review, H1 2018, outlays comprehensive information on the Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Survival of motor neuron or survival motor neuron (SMN) is a protein encoded by the SMN1 and SMN2 genes. The SMN complex plays a catalyst role in the assembly of small nuclear ribonucleoproteins (snRNPs). They play an important role in the splicing of cellular pre-mRNAs. They ensure the correct splicing of U12 intron-containing genes that is important for normal motor and proprioceptive neurons development. They are required for resolving RNA-DNA hybrids created by RNA polymerase II. They play a role in the metabolism of small nucleolar ribonucleoprotein. The molecules developed by companies in Phase III, Phase II and Preclinical stages are 2, 1 and 8 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 4 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Respiratory which include indications Spinal Muscular Atrophy (SMA) and Cystic Fibrosis.

Furthermore, this report also reviews key players involved in Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)
  • The report reviews Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)
  • 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) 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
  • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Overview
    • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - 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
  • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Assessment
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development
    • AveXis Inc
    • F. Hoffmann-La Roche Ltd
    • Genethon SA
    • Genzyme Corp
    • Ionis Pharmaceuticals Inc
    • Novartis AG
    • Sarepta Therapeutics Inc
    • Spotlight Innovation Inc
    • Voyager Therapeutics Inc
    • WAVE Life Sciences Ltd
  • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Drug Profiles
    • ALB-111 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AVXS-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • branaplam - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IONISENAC-2.5Rx - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PMO-25 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RG-7916 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • STL-182 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VYSMN-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Dormant Products
  • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Discontinued Products
  • Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Product Development Milestones
    • Featured News & Press Releases
      • Jan 27, 2018: Preliminary Data from FIREFISH trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
      • Jan 23, 2018: Data from RG7916 Programs in SMA to be Presented at the International Scientific Congress on Spinal Muscular Atrophy
      • Jan 16, 2018: AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
      • Jan 04, 2018: AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1
      • Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
      • Nov 01, 2017: AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1
      • Oct 12, 2017: Spinal Muscular Atrophy Program Advances into Pivotal Study in SMA Patients
      • Oct 03, 2017: AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society
      • Oct 03, 2017: RG7916 Increased SMN Protein Production in SUNFISH Clinical Trial in Patients with Type 2/3 Spinal Muscular Atrophy
      • Sep 29, 2017: AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
      • Aug 24, 2017: Iowa State University biomedical researcher conducts promising trial of potential therapy for spinal muscular atrophy
      • Jul 01, 2017: Preliminary Phase 2 Data from Spinal Muscular Atrophy Program Presented at CureSMA Conference
      • Jun 22, 2017: Spotlight Innovation to Present Poster at the Cure SMA 2017 Annual SMA Conference
      • Jun 14, 2017: AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101
      • Apr 25, 2017: AveXis Presents Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology
  • 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 2018
  • Number of Products under Development by Therapy Areas, H1 2018
  • Number of Products under Development by Indication, H1 2018
  • Number of Products under Development by Companies, H1 2018
  • Products under Development by Companies, H1 2018
  • Number of Products under Investigation by Universities/Institutes, H1 2018
  • Products under Investigation by Universities/Institutes, H1 2018
  • Number of Products by Stage and Mechanism of Actions, H1 2018
  • Number of Products by Stage and Route of Administration, H1 2018
  • Number of Products by Stage and Molecule Type, H1 2018
  • Pipeline by AveXis Inc, H1 2018
  • Pipeline by F. Hoffmann-La Roche Ltd, H1 2018
  • Pipeline by Genethon SA, H1 2018
  • Pipeline by Genzyme Corp, H1 2018
  • Pipeline by Ionis Pharmaceuticals Inc, H1 2018
  • Pipeline by Novartis AG, H1 2018
  • Pipeline by Sarepta Therapeutics Inc, H1 2018
  • Pipeline by Spotlight Innovation Inc, H1 2018
  • Pipeline by Voyager Therapeutics Inc, H1 2018
  • Pipeline by WAVE Life Sciences Ltd, H1 2018
  • Dormant Projects, H1 2018
  • Discontinued Products, H1 2018

List of Figures

  • Number of Products under Development by Stage of Development, H1 2018
  • Number of Products under Development by Therapy Areas, H1 2018
  • Number of Products under Development by Indications, 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
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