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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, H1 2017

発行 Global Markets Direct 商品コード 199802
出版日 ページ情報 英文 111 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, H1 2017
出版日: 2017年03月15日 ページ情報: 英文 111 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: GMDHC9065IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2017, provides an overview of the Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline landscape.

Spinal muscular atrophy (SMA) is a genetic disease that attacks nerve cells, called motor neurons, in the spinal cord. These critically important cells are responsible for supplying electrical and chemical messages to muscle cells. Without the proper input from the motor neurons, muscle cells cannot function properly. The muscle cells will, therefore, become much smaller (atrophy) and will produce symptoms of muscle weakness. This can affect walking, crawling, breathing, swallowing, and head and neck control.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA) (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 Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Spinal Muscular Atrophy (SMA) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 4, 2, 9 and 2 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 6 and 2 molecules, respectively.

Spinal Muscular Atrophy (SMA) (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 Spinal Muscular Atrophy (SMA) (Central Nervous System).
  • The pipeline guide reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) (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 Spinal Muscular Atrophy (SMA) (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Spinal Muscular Atrophy (SMA) (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 Spinal Muscular Atrophy (SMA) (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 Spinal Muscular Atrophy (SMA) (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 Spinal Muscular Atrophy (SMA) (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

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Spinal Muscular Atrophy (SMA) - Overview
  • Spinal Muscular Atrophy (SMA) - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Pipeline by Universities/Institutes
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Spinal Muscular Atrophy (SMA) - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development
    • AveXis Inc
    • Cytokinetics Inc
    • F. Hoffmann-La Roche Ltd
    • Genethon SA
    • Genzyme Corp
    • Ionis Pharmaceuticals Inc
    • Kowa Company Ltd
    • Neurotune AG
    • Novartis AG
    • Paratek Pharmaceuticals Inc
    • RaNA Therapeutics Inc
    • Sarepta Therapeutics Inc
    • Spotlight Innovation Inc
    • WAVE Life Sciences Ltd
  • Spinal Muscular Atrophy (SMA) - 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
    • 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
    • K-828SP - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • LMI-070 - 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 Type 1 - 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 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 Inhibit GSK-3 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
    • tirasemtiv - 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
      • Feb 08, 2017: Spotlight Innovation Engages Top-Tier Contract Research Organization to Conduct Preclinical Studies of STL-182
      • Jan 13, 2017: New Data Show SPINRAZA (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation in Infantile-Onset Spinal Muscular Atrophy
      • Jan 06, 2017: RG7916 Granted Orphan Drug Designation in the U.S. for the Treatment of Spinal Muscular Atrophy
      • Jan 05, 2017: PTC Therapeutics Announces Initiation of FIREFISH Study in Infant (Type I) SMA Patients
      • Dec 28, 2016: Biogen's atrophy drug SPINRAZA to cost $750,000 per patient
      • Dec 23, 2016: FDA approves first drug for spinal muscular atrophy
      • Dec 06, 2016: Study Finds New Treatment For Spinal Muscular Atrophy Safe For Infants
      • Nov 07, 2016: Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study in Later-Onset Spinal Muscular Atrophy
      • Oct 28, 2016: Biogen's Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA
      • Oct 20, 2016: Spinal Muscular Atrophy Program Advances into Phase 2 Clinical Studies in SMA Patients with RG7916
      • Oct 08, 2016: New Data Presented at World Muscle Society Congress Support Potential Benefit of Investigational Treatment Nusinersen in Spinal Muscular Atrophy
      • Sep 26, 2016: Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy
      • Sep 23, 2016: CHMP of the EMA has Granted Nusinersen Accelerated Assessment
      • Aug 04, 2016: Candidate Drug for Spinal Muscular Atrophy, with Roots in CSHL Research, Passes Major Hurdle
      • Aug 01, 2016: Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy
  • 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), H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Products under Development by Companies, H1 2017
  • Products under Development by Companies, H1 2017 (Contd..1), H1 2017
  • Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Stage and Target, H1 2017
  • Number of Products by Stage and Mechanism of Action, H1 2017
  • Number of Products by Stage and Route of Administration, H1 2017
  • Number of Products by Stage and Molecule Type, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by AveXis Inc, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics Inc, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Genethon SA, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corp, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Ionis Pharmaceuticals Inc, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Company Ltd, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals Inc, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by RaNA Therapeutics Inc, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, H1 2017
  • Spinal Muscular Atrophy (SMA) - Pipeline by WAVE Life Sciences Ltd, H1 2017
  • Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2017
  • Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2017 (Contd..1), H1 2017
  • Spinal Muscular Atrophy (SMA) - Discontinued Products, H1 2017

List of Figures

  • Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Targets, H1 2017
  • Number of Products by Stage and Targets, H1 2017
  • Number of Products by Mechanism of Actions, H1 2017
  • Number of Products by Stage and Mechanism of Actions, H1 2017
  • Number of Products by Routes of Administration, H1 2017
  • Number of Products by Stage and Routes of Administration, H1 2017
  • Number of Products by Molecule Types, H1 2017
  • Number of Products by Stage and Molecule Types, H1 2017
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