Spinal Muscular Atrophy (SMA) – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Spinal Muscular Atrophy (SMA) – Pipeline Review, H2 2017’, 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

Cytokinetics Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Kowa Company Ltd

Neurotune AG

Novartis AG

Paratek Pharmaceuticals Inc

Recursion Pharmaceuticals Inc

Sarepta Therapeutics Inc

Scholar Rock Inc

Spotlight Innovation Inc

Translate Bio Inc

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Spinal Muscular Atrophy (SMA) - Overview 6

Spinal Muscular Atrophy (SMA) - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 11

Products under Development by Companies 12

Products under Development by Universities/Institutes 14

Spinal Muscular Atrophy (SMA) - Therapeutics Assessment 15

Assessment by Target 15

Assessment by Mechanism of Action 17

Assessment by Route of Administration 19

Assessment by Molecule Type 21

Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development 23

AveXis Inc 23

Biogen Inc 23

Cytokinetics Inc 24

F. Hoffmann-La Roche Ltd 24

Genethon SA 25

Genzyme Corp 25

Kowa Company Ltd 26

Neurotune AG 26

Novartis AG 27

Paratek Pharmaceuticals Inc 27

Recursion Pharmaceuticals Inc 28

Sarepta Therapeutics Inc 28

Scholar Rock Inc 29

Spotlight Innovation Inc 29

Translate Bio Inc 30

WAVE Life Sciences Ltd 30

Spinal Muscular Atrophy (SMA) - Drug Profiles 31

AAD-2004 - Drug Profile 31

ALB-111 - Drug Profile 32

Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 33

Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 34

AVXS-101 - Drug Profile 35

branaplam - Drug Profile 41

CK-2127107 - Drug Profile 42

Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 48

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 49

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 50

K-828SP - Drug Profile 51

NT-1654 - Drug Profile 52

nusinersen - Drug Profile 53

olesoxime - Drug Profile 62

Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 67

Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - Drug Profile 68

PMO-25 - Drug Profile 69

PTKSMA-1 - Drug Profile 70

RG-7916 - Drug Profile 71

Small Molecules 1 for Spinal Muscular Atrophy - Drug Profile 73

Small Molecules 2 for Spinal Muscular Atrophy - Drug Profile 74

Small Molecules for Central Nervous System Disorders - Drug Profile 75

Small Molecules for Spinal Muscular Atrophy - Drug Profile 76

Small Molecules for Spinal Muscular Atrophy - Drug Profile 77

Small Molecules for Spinal Muscular Atrophy - Drug Profile 78

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 79

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 80

Small Molecules to Inhibit GSK-3 for Spinal Muscular Atrophy - Drug Profile 81

SRK-015 - Drug Profile 82

STL-182 - Drug Profile 84

tirasemtiv - Drug Profile 85

Xcel-hNu - Drug Profile 96

Xcel-hNuP - Drug Profile 97

Spinal Muscular Atrophy (SMA) - Dormant Projects 98

Spinal Muscular Atrophy (SMA) - Discontinued Products 100

Spinal Muscular Atrophy (SMA) - Product Development Milestones 101

Featured News & Press Releases 101

Appendix 115

Methodology 115

Coverage 115

Secondary Research 115

Primary Research 115

Expert Panel Validation 115

Contact Us 115

Disclaimer 116

List of Tables

List of Tables

Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by AveXis Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Biogen Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Cytokinetics Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by F. Hoffmann-La Roche Ltd, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Genethon SA, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Genzyme Corp, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Kowa Company Ltd, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Neurotune AG, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Novartis AG, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Paratek Pharmaceuticals Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Recursion Pharmaceuticals Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Sarepta Therapeutics Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Scholar Rock Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Spotlight Innovation Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by Translate Bio Inc, H2 2017

Spinal Muscular Atrophy (SMA) – Pipeline by WAVE Life Sciences Ltd, H2 2017

Spinal Muscular Atrophy (SMA) – Dormant Projects, H2 2017

Spinal Muscular Atrophy (SMA) – Dormant Projects, H2 2017 (Contd..1), H2 2017

Spinal Muscular Atrophy (SMA) – Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Top 10 Targets, H2 2017

Number of Products by Stage and Top 10 Targets, H2 2017

Number of Products by Top 10 Mechanism of Actions, H2 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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