Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2019

Global Markets Direct’s, ‘Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2019’, 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

Companies mentioned

AveXis Inc

Biogen Inc

BioMarin Pharmaceutical Inc

Biophytis SA

Cytokinetics Inc

Exicure Inc

F. Hoffmann-La Roche Ltd

Ionis Pharmaceuticals Inc

Kowa Co Ltd

Neurodyn Life ...

AveXis Inc

Biogen Inc

BioMarin Pharmaceutical Inc

Biophytis SA

Cytokinetics Inc

Exicure Inc

F. Hoffmann-La Roche Ltd

Ionis Pharmaceuticals Inc

Kowa Co Ltd

Neurodyn Life Sciences Inc

Neurotune AG

Novartis AG

Ono Pharmaceutical Co Ltd

Recursion Pharmaceuticals Inc

Sarepta Therapeutics Inc

Scholar Rock Inc

Spotlight Innovation Inc

Vybion Inc

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 ...

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

List of Tables

List of Tables

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2019

Number of Products under Development by Companies, H1 2019

Number ...

List of Tables

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products under Development by Universities/Institutes, H1 2019

Products under Development by Companies, H1 2019

Products under Development by Companies, H1 2019 (Contd..1), H1 2019

Products under Development by Companies, H1 2019 (Contd..2), H1 2019

Products under Development by Universities/Institutes, H1 2019

Number of Products by Stage and Target, H1 2019

Number of Products by Stage and Mechanism of Action, H1 2019

Number of Products by Stage and Route of Administration, H1 2019

Number of Products by Stage and Molecule Type, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by AveXis Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Biogen Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by BioMarin Pharmaceutical Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Biophytis SA, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Cytokinetics Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Exicure Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by F. Hoffmann-La Roche Ltd, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Ionis Pharmaceuticals Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Kowa Co Ltd, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Neurodyn Life Sciences Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Neurotune AG, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Novartis AG, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Ono Pharmaceutical Co Ltd, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Recursion Pharmaceuticals Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Sarepta Therapeutics Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Scholar Rock Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Spotlight Innovation Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Pipeline by Vybion Inc, H1 2019

Spinal Muscular Atrophy (SMA) – Dormant Projects, H1 2019

Spinal Muscular Atrophy (SMA) – Dormant Projects, H1 2019 (Contd..1), H1 2019

Spinal Muscular Atrophy (SMA) – Discontinued Products, H1 2019

List of Figures

List of Figures

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2019

Number of Products under Development by Companies, H1 2019

Number ...

List of Figures

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products under Development by Universities/Institutes, H1 2019

Number of Products by Top 10 Targets, H1 2019

Number of Products by Stage and Top 10 Targets, H1 2019

Number of Products by Top 10 Mechanism of Actions, H1 2019

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2019

Number of Products by Routes of Administration, H1 2019

Number of Products by Stage and Routes of Administration, H1 2019

Number of Products by Molecule Types, H1 2019

Number of Products by Stage and Molecule Types, H1 2019

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