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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 2018

発行 Global Markets Direct 商品コード 199802
出版日 ページ情報 英文 113 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 2018
出版日: 2018年02月20日 ページ情報: 英文 113 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

目次
Product Code: GMDHC10158IDB

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018, 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 2018, 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 Phase III, Phase II, Phase I, Preclinical and Discovery stages are 4, 3, 1, 13 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 7 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
    • BioMarin Pharmaceutical Inc
    • Cytokinetics Inc
    • Exicure Inc
    • F. Hoffmann-La Roche Ltd
    • Genethon SA
    • Genzyme Corp
    • Kowa Co Ltd
    • Neurotune AG
    • Novartis AG
    • Paratek Pharmaceuticals Inc
    • Recursion Pharmaceuticals Inc
    • Sarepta Therapeutics Inc
    • Scholar Rock Inc
    • Spotlight Innovation Inc
    • Voyager Therapeutics Inc
  • 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
    • amifampridine phosphate - 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
    • branaplam - 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
    • NT-1654 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • olesoxime - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotides for Spinal Muscular Atrophy and Huntington Disease - 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
    • REC-0000716 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • REC-0001202 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RG-7916 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule to Inhibit GSK-3 for Spinal Muscular Atrophy - 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 for X-Linked Infantile 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 SRSF10 and BCLAF1 for HIV Infection, Spinal Muscular Atrophy and Colon Cancer - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SRK-105 - 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
    • Xcel-hNu - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Xcel-hNuP - 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 02, 2018: Scholar Rock Announces Publication of Comprehensive Pharmacological Study of SRK-015, a Novel Inhibitor of Myostatin Activation, Supporting the Treatment of Muscle Atrophy Disorders
      • Jan 30, 2018: AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients
      • 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 23, 2018: Scholar Rock and Collaborators at Harvard Medical School and Northeastern University Elucidate Molecular Basis of Myostatin Activation - a Key Physiological Process in Muscle Health
      • 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
      • Jan 03, 2018: Cytokinetics Provides on CK-2127107
      • Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
      • Dec 11, 2017: Cytokinetics Publishes Clinical Trial Data for CK-2127107
      • Nov 21, 2017: Catalyst Pharmaceuticals Announces Phase 2 Study of Firdapse in Ambulatory Patients with Spinal Muscular Atrophy (SMA)
      • 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
  • 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 2018
  • Number of Products under Development by Companies, H1 2018
  • Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018
  • Number of Products under Development by Universities/Institutes, H1 2018
  • Products under Development by Companies, H1 2018
  • Products under Development by Companies, H1 2018 (Contd..1), H1 2018
  • Products under Development by Universities/Institutes, H1 2018
  • Number of Products by Stage and Target, H1 2018
  • Number of Products by Stage and Mechanism of Action, H1 2018
  • Number of Products by Stage and Route of Administration, H1 2018
  • Number of Products by Stage and Molecule Type, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by AveXis Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by BioMarin Pharmaceutical Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Exicure Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Genethon SA, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corp, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Co Ltd, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Recursion Pharmaceuticals Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Scholar Rock Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics Inc, H1 2018
  • Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2018
  • Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2018 (Contd..1), H1 2018
  • Spinal Muscular Atrophy (SMA) - Discontinued Products, H1 2018

List of Figures

  • Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2018
  • Number of Products under Development by Companies, H1 2018
  • Number of Products under Development by Universities/Institutes, H1 2018
  • Number of Products by Top 10 Targets, H1 2018
  • Number of Products by Stage and Top 10 Targets, H1 2018
  • Number of Products by Top 10 Mechanism of Actions, H1 2018
  • Number of Products by Stage and Top 10 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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