Cover Image
市場調查報告書

脊髓性肌萎縮症(SMA):開發平台分析

Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018

出版商 Global Markets Direct 商品編碼 199802
出版日期 內容資訊 英文 113 Pages
訂單完成後即時交付
價格
Back to Top
脊髓性肌萎縮症(SMA):開發平台分析 Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018
出版日期: 2018年02月20日 內容資訊: 英文 113 Pages
簡介

脊髓性肌萎縮症(SMA),是攻擊脊髓的神經細胞(運動神經原)的遺傳性疾病。這個神經細胞,供給肌肉細胞電力、化學的訊息,肩負重要任務。如果無法由運動神經接受恰當的刺激,肌肉細胞便無法正常作用,而引起萎縮、肌肉無力。影響步行、匍匐前進、吞嚥、頭部、頸椎控制。

本報告提供脊髓性肌萎縮症(SMA)的治療藥的開發情形調查分析,開發中產品的概要,臨床實驗的各階段的產品概要,主要企業簡介,藥物簡介,開發中產品的最新趨勢,最新消息和新聞稿等相關的系統性資訊。

目錄

簡介

脊髓性肌萎縮症(SMA)概要

治療藥的開發

  • 開發中產品:概要
  • 開發中產品:比較分析

正在開發的治療藥:各企業

調查中的治療藥:各大學/研究機關

開發中產品的概要

  • 後期階段的產品
  • 臨床實驗階段的產品
  • 初期階段的產品

開發中的產品:各企業

調查中的產品:各大學/研究機關

開發治療藥的企業

  • Astellas Pharma Inc.
  • 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.

治療藥的評估

  • 單獨療法的情況
  • 各標的
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

  • ALB-111
  • 脊髓性肌萎縮症治療用 SMN2遺傳基因阻礙反義寡核甘酸
  • 脊髓性肌萎縮症治療用反義寡核甘酸
  • ARM-210
  • AVXS-101
  • azithromycin
  • CK-2127107
  • 脊髓性肌萎縮症治療用 SMN活性遺傳基因療法
  • INT-41
  • LBT-3627
  • LMI-070
  • ND-602
  • NT-1654
  • nusinersen
  • NXD-30001
  • olesoxime
  • 脊髓性肌萎縮症治療用 SMN2活性寡核甘酸
  • PMO-25
  • RG-7800
  • RG-7916
  • 中樞神經系統障礙治療用小分子
  • 脊髓性肌萎縮症治療用 SMN2活性小分子
  • 非神經肌肉疾病神經肌肉疾病、治療用肌鈣蛋白活性小分子
  • 苯丁酸鈉
  • tirasemtiv
  • VYSMN-101

開發中產品的最新趨勢

暫停的計劃

開發中止的產品

產品開發的里程碑

  • 最新消息和新聞稿

附錄

圖表

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
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
Back to Top