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市場調査レポート

脊髄性筋萎縮症(SMA):パイプライン分析

Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2016

発行 Global Markets Direct 商品コード 199802
出版日 ページ情報 英文 116 Pages
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脊髄性筋萎縮症(SMA):パイプライン分析 Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2016
出版日: 2016年08月10日 ページ情報: 英文 116 Pages
概要

脊髄性筋萎縮症(SMA)は、脊髄にある神経細胞(運動ニューロン)を攻撃する遺伝性疾患です。この神経細胞は、電気・化学的メッセージを筋肉細胞へ供給する、極めて重要な役割を担っています。運動ニューロンから適切な刺激を受けなければ、筋肉細胞は正常に機能することができず、萎縮して、筋力低下を引き起こします。歩行、匍匐前進、嚥下、頭部・頸部制御(首のすわり)などに影響を及ぼします。

当レポートでは、脊髄性筋萎縮症(SMA)に対する治療薬の開発状況について調査分析し、パイプライン製品の概要、治験の段階別の製品の概要、主要企業プロファイル、薬剤のプロファイル、パイプライン製品の最新動向、最新ニュースとプレスリリースなどについて、体系的な情報を提供しています。

目次

イントロダクション

脊髄性筋萎縮症(SMA)の概要

治療薬の開発

  • パイプライン製品:概要
  • パイプライン製品:比較分析

開発中の治療薬:企業別

調査中の治療薬:大学/研究機関別

パイプライン製品の概要

  • 後期段階の製品
  • 治験段階の製品
  • 初期段階の製品

開発中の製品:企業別

調査中の製品:大学/研究機関別

治療薬の開発に従事している企業

  • アステラス製薬
  • AveXis, Inc.
  • Bioblast Pharma Ltd.
  • Cytokinetics, Inc. 24
  • F. Hoffmann-La Roche Ltd.
  • Genethon
  • Genzyme Corporation
  • GMP-Orphan SAS
  • Ionis Pharmaceuticals, Inc.
  • Longevity Biotech, Inc
  • Neurodyn Inc.
  • Neurotune AG
  • Novartis AG
  • Sarepta Therapeutics, Inc.
  • Voyager Therapeutics, Inc.
  • Vybion, Inc.
  • WAVE Life Sciences Ltd.

治療薬の評価

  • 単独療法の場合
  • 標的別
  • 作用機序別
  • 投与経路別
  • 分子タイプ別

薬剤のプロファイル

  • ALB-111
  • 脊髄性筋萎縮症治療用 SMN2遺伝子阻害アンチセンス・オリゴヌクレオチド
  • 脊髄性筋萎縮症治療用アンチセンス・オリゴヌクレオチド
  • ARM-210
  • AVXS-101
  • azithromycin
  • CK-2127107
  • 脊髄性筋萎縮症治療用 SMN活性遺伝子療法
  • INT-41
  • LBT-3627
  • LMI-070
  • ND-602
  • NT-1654
  • nusinersen
  • NXD-30001
  • olesoxime
  • 脊髄性筋萎縮症治療用 SMN2活性オリゴヌクレオチド
  • PMO-25
  • RG-7800
  • RG-7916
  • 中枢神経系障害治療用小分子
  • 脊髄性筋萎縮症治療用 SMN2活性小分子
  • 非神経筋疾患神経筋疾患・治療用トロポニン活性小分子
  • フェニル酪酸ナトリウム
  • tirasemtiv
  • VYSMN-101

パイプライン製品の最新動向

休止状態のプロジェクト

開発が中止された製品

製品開発のマイルストーン

  • 最新ニュースとプレスリリース

付録

図表

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
Product Code: GMDHC8336IDB

Summary

Global Markets Direct's, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2016', provides an overview of the Spinal Muscular Atrophy (SMA) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Spinal Muscular Atrophy (SMA) and features dormant and discontinued projects.

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

  • The report provides a snapshot of the global therapeutic landscape of Spinal Muscular Atrophy (SMA)
  • The report reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) 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 Spinal Muscular Atrophy (SMA) therapeutics and enlists all their major and minor projects
  • The report assesses Spinal Muscular Atrophy (SMA) therapeutics based on drug target, 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 related to pipeline therapeutics for Spinal Muscular Atrophy (SMA)

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 important and diverse types of therapeutics under development for Spinal Muscular Atrophy (SMA)
  • 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 Spinal Muscular Atrophy (SMA) 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
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Spinal Muscular Atrophy (SMA) Overview
  • Therapeutics Development
    • Pipeline Products for Spinal Muscular Atrophy (SMA) - Overview
    • Pipeline Products for Spinal Muscular Atrophy (SMA) - Comparative Analysis
  • Spinal Muscular Atrophy (SMA) - Therapeutics under Development by Companies
  • Spinal Muscular Atrophy (SMA) - Therapeutics under Investigation by Universities/Institutes
  • Spinal Muscular Atrophy (SMA) - Pipeline Products Glance
    • Late Stage Products
    • Clinical Stage Products
    • Early Stage Products
  • Spinal Muscular Atrophy (SMA) - Products under Development by Companies
  • Spinal Muscular Atrophy (SMA) - Products under Investigation by Universities/Institutes
  • Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development
    • Astellas Pharma Inc.
    • AveXis, Inc.
    • Bio Blast Pharma Ltd.
    • Cytokinetics, Inc.
    • F. Hoffmann-La Roche Ltd.
    • Genethon
    • Genzyme Corporation
    • Ionis Pharmaceuticals, Inc.
    • Neurodyn Inc.
    • Neurotune AG
    • Novartis AG
    • Paratek Pharmaceuticals, Inc.
    • RaNA Therapeutics, Inc.
    • Sarepta Therapeutics, Inc.
    • Voyager Therapeutics, Inc.
    • WAVE Life Sciences Ltd.
  • Spinal Muscular Atrophy (SMA) - Therapeutics Assessment
    • Assessment by Monotherapy Products
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Drug Profiles
    • AAD-2004 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • 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
    • Antisense Oligonucleotides 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
    • azithromycin - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CK-2127107 - 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
    • LMI-070 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ND-602 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • NT-1654 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • nusinersen - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • olesoxime - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PMO-25 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PTKSMA-1 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RG-7800 - 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 Central Nervous System Disorders - 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
    • Small Molecules to Activate Survival Motor Neuron Protein for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Activate Troponin for Non-Neuromuscular and Neuromuscular Disorders - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit GSK-3 for Spinal Muscular Atrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • tirasemtiv - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VYSMN-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Spinal Muscular Atrophy (SMA) - Dormant Projects
  • Spinal Muscular Atrophy (SMA) - Discontinued Products
  • Spinal Muscular Atrophy (SMA) - Product Development Milestones
    • Featured News & Press Releases
      • Apr 21, 2016: Ionis Pharmaceuticals Reports Data Update from Nusinersen Phase 2 Study in Infants with Spinal Muscular Atrophy and Reviews Neurological Disease Franchise
      • Mar 31, 2016: Ionis Pharmaceuticals Advances Nusinersen in the Ongoing SHINE Study in Patients with SMA
      • Mar 23, 2016: RaNA Therapeutics Presents Data on Advances in RNA Therapy Targeting Spinal Muscular Atrophy
      • Jan 12, 2016: Ionis Pharmaceuticals Completes Target Enrollment for Nusinersen Phase 3 Study, CHERISH, in Children with Spinal Muscular Atrophy
      • Jan 07, 2016: Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development
      • Jan 04, 2016: Cytokinetics Announces Start Of Phase 2 Clinical Trial Of Ck-2127107 In Patients With Spinal Muscular Atrophy
      • Oct 15, 2015: Isis Pharmaceuticals Initiates SHINE Study to Provide ISIS-SMN for Patients With SMA Who Have Completed the Phase 3 ENDEAR and CHERISH Studies
      • Oct 04, 2015: Phase 2 Data from Spinal Muscular Atrophy Program Presented at the 20th International Annual Congress of the World Muscle Society
      • Jul 14, 2015: Isis Pharmaceuticals Earns $2.15M for Advancing ISIS-SMN in Children With Spinal Muscular Atrophy
      • Jun 22, 2015: BioBlast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy
      • Jun 22, 2015: Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Spinal Muscular Atrophy
      • Jun 19, 2015: Cytokinetics Presents Results From Phase I Studies Of Ck-2127107
      • Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Spinal Muscular Atrophy at Two Leading Scientific Meetings in June
      • Jun 11, 2015: Isis Pharmaceuticals Reports Data From ISIS-SMN Rx Phase 2 Study in Infants With Spinal Muscular Atrophy
      • Apr 20, 2015: Phase 1 Data from Spinal Muscular Atrophy Program to be Presented at the 2015 American Academy of Neurology Annual Meeting
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2016
  • Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Development, H2 2016
  • Products under Development by Companies, H2 2016
  • Products under Investigation by Universities/Institutes, H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Astellas Pharma Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by AveXis, Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Bio Blast Pharma Ltd., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals, Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by RaNA Therapeutics, Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics, Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics, Inc., H2 2016
  • Spinal Muscular Atrophy (SMA) - Pipeline by WAVE Life Sciences Ltd., H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Stage and Target, H2 2016
  • Number of Products by Stage and Mechanism of Action, H2 2016
  • Number of Products by Stage and Route of Administration, H2 2016
  • Number of Products by Stage and Molecule Type, H2 2016
  • Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2016
  • Spinal Muscular Atrophy (SMA) - Dormant Projects (Contd..1), H2 2016
  • Spinal Muscular Atrophy (SMA) - Discontinued Products, H2 2016

List of Figures

  • Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2016
  • Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Products, H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Targets, H2 2016
  • Number of Products by Stage and Targets, H2 2016
  • Number of Products by Mechanism of Actions, H2 2016
  • Number of Products by Stage and Mechanism of Actions, H2 2016
  • Number of Products by Routes of Administration, H2 2016
  • Number of Products by Stage and Routes of Administration, H2 2016
  • Number of Products by Molecule Types, H2 2016
  • Number of Products by Stage and Molecule Types, H2 2016
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