脊髄性筋萎縮症 (SMA) (中枢神経系) - 開発中の薬剤：2021年

脊髄性筋萎縮症 (SMA) は、脊髄の運動ニューロンと呼ばれる神経細胞が攻撃される遺伝性疾患です。これらの重要な細胞は、筋細胞に電気的および化学的メッセージを供給する責任があります。運動ニューロンからの適切な入力を受けなければ、筋細胞は適切に機能することができません。そのため、筋細胞は小さくなり（萎縮）、筋力低下の症状を引き起こします。歩行、這うこと、呼吸、嚥下、頭頸部の制御に影響を与える可能性があります。

当レポートでは、脊髄性筋萎縮症 (SMA) (中枢神経系) に対する開発中の薬剤について調査分析し、パイプライン情勢の概要を提供します。

目次

目次

• イントロダクション
• 脊髄性筋萎縮症 (SMA) - 概要
• 脊髄性筋萎縮症 (SMA) - 治療薬の開発
• 脊髄性筋萎縮症 (SMA) - 治療薬の評価
• 脊髄性筋萎縮症 (SMA) - 治療薬開発に従事している企業
• 脊髄性筋萎縮症 (SMA) - 薬剤プロファイル
• 脊髄性筋萎縮症 (SMA) - 休止中のプロジェクト
• 脊髄性筋萎縮症 (SMA) - 開発が中止された製品
• 脊髄性筋萎縮症 (SMA) - 製品開発のマイルストーン
• 付録

List of Tables

List of Tables

• Number of Products under Development for Spinal Muscular Atrophy (SMA), 2021
• Number of Products under Development by Companies, 2021
• Number of Products under Development by Universities/Institutes, 2021
• Products under Development by Companies, 2021
• Products under Development by Universities/Institutes, 2021
• Number of Products by Stage and Target, 2021
• Number of Products by Stage and Mechanism of Action, 2021
• Number of Products by Stage and Route of Administration, 2021
• Number of Products by Stage and Molecule Type, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Alcyone Therapeutics Inc, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Allianz Pharmascience Ltd (Inactive), 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by AlphaCognition Inc, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Anima Biotech Inc, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Apteeus, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Biogen Inc, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Bioleaders Corp, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by BioMarin Pharmaceutical Inc, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Biophytis SA, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Cell Tech Pharmed Co, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Enzerna Biosciences LLC, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by GNT Pharma Co Ltd, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Imago Pharmaceuticals Inc, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Co Ltd, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Myocea Inc, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by NMD Pharma AS, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Novartis Gene Therapies, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by Ono Pharmaceutical Co Ltd, 2021
• Spinal Muscular Atrophy (SMA) - Pipeline by PTC Therapeutics Inc, 2021
• Spinal Muscular Atrophy (SMA) - Dormant Projects, 2021
• Spinal Muscular Atrophy (SMA) - Discontinued Products, 2021

List of Figures

List of Figures

• Number of Products under Development for Spinal Muscular Atrophy (SMA), 2021
• Number of Products under Development by Companies, 2021
• Number of Products under Development by Universities/Institutes, 2021
• Number of Products by Top 10 Targets, 2021
• Number of Products by Stage and Top 10 Targets, 2021
• Number of Products by Top 10 Mechanism of Actions, 2021
• Number of Products by Stage and Top 10 Mechanism of Actions, 2021
• Number of Products by Routes of Administration, 2021
• Number of Products by Stage and Routes of Administration, 2021
• Number of Products by Molecule Types, 2021
• Number of Products by Stage and Molecule Types, 2021

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Spinal Muscular Atrophy - Drugs In Development, 2021, provides an overview of the Spinal Muscular Atrophy (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 - Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (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 (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, IND/CTA Filed, Preclinical and Discovery stages are 2, 1, 4, 4, 1, 20 and 5 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 8 and 2 molecules, respectively.

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

• Introduction
• Spinal Muscular Atrophy (SMA) - Overview
• Spinal Muscular Atrophy (SMA) - Therapeutics Development
• Spinal Muscular Atrophy (SMA) - Therapeutics Assessment
• Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development
• Spinal Muscular Atrophy (SMA) - Drug Profiles
• Spinal Muscular Atrophy (SMA) - Dormant Projects
• Spinal Muscular Atrophy (SMA) - Discontinued Products
• Spinal Muscular Atrophy (SMA) - Product Development Milestones
• Appendix