Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) – Pipeline Review, H2 2017’, provides in depth analysis on Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Central Nervous System and Respiratory under development targeting Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2).

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.

*Updated report will be delivered in 48 hours of order confirmation.

Scope

The report provides a snapshot of the global therapeutic landscape for Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)

The report reviews Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics under development 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 in Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics and enlists all their major and minor projects

The report assesses Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics based on 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 and deals related to Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics

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 the targeted therapy areas and indications for Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)

Identify the use of drugs for target identification and drug repurposing

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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) development landscape

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

Companies mentioned

AveXis Inc

Biogen Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Ionis Pharmaceuticals Inc

Novartis AG

Sarepta Therapeutics 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

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Overview 6

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Development 7

Products under Development by Stage of Development 7

Products under Development by Therapy Area 8

Products under Development by Indication 9

Products under Development by Companies 10

Products under Development by Universities/Institutes 12

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Assessment 14

Assessment by Mechanism of Action 14

Assessment by Route of Administration 15

Assessment by Molecule Type 17

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development 19

AveXis Inc 19

Biogen Inc 19

F. Hoffmann-La Roche Ltd 20

Genethon SA 20

Genzyme Corp 21

Ionis Pharmaceuticals Inc 21

Novartis AG 22

Sarepta Therapeutics Inc 22

Spotlight Innovation Inc 23

Translate Bio Inc 23

WAVE Life Sciences Ltd 24

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Drug Profiles 25

ALB-111 - Drug Profile 25

Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 26

AVXS-101 - Drug Profile 27

Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 33

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 34

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 35

IONISENAC-2.5Rx - Drug Profile 36

LMI-070 - Drug Profile 37

nusinersen - Drug Profile 38

Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 47

Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - Drug Profile 48

PMO-25 - Drug Profile 49

RG-7916 - Drug Profile 50

Small Molecules for Spinal Muscular Atrophy - Drug Profile 52

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 53

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 54

STL-182 - Drug Profile 55

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Dormant Products 56

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Discontinued Products 57

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Product Development Milestones 58

Featured News & Press Releases 58

Appendix 75

Methodology 75

Coverage 75

Secondary Research 75

Primary Research 75

Expert Panel Validation 75

Contact Us 75

Disclaimer 76

List of Tables

List of Tables

Number of Products under Development by Stage of Development, H2 2017

Number of Products under Development by Therapy Areas, H2 2017

Number of Products under Development by Indication, H2 2017

Number of Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017

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

Products under Investigation by Universities/Institutes, H2 2017

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

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

Number of Products by Stage and Molecule Type, H2 2017

Pipeline by AveXis Inc, H2 2017

Pipeline by Biogen Inc, H2 2017

Pipeline by F. Hoffmann-La Roche Ltd, H2 2017

Pipeline by Genethon SA, H2 2017

Pipeline by Genzyme Corp, H2 2017

Pipeline by Ionis Pharmaceuticals Inc, H2 2017

Pipeline by Novartis AG, H2 2017

Pipeline by Sarepta Therapeutics Inc, H2 2017

Pipeline by Spotlight Innovation Inc, H2 2017

Pipeline by Translate Bio Inc, H2 2017

Pipeline by WAVE Life Sciences Ltd, H2 2017

Dormant Projects, H2 2017

Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development by Stage of Development, H2 2017

Number of Products under Development by Therapy Areas, H2 2017

Number of Products under Development by Indications, H2 2017

Number of Products by Stage and 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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