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

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TABLE OF CONTENTS

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DESCRIPTION

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

Summary

According to the recently published report Survival Motor Neuron Protein - Pipeline Review, H2 2017; Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 17 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes.

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Survival of motor neuron or survival motor neuron (SMN) is a protein encoded by the SMN1 and SMN2 genes. The SMN complex plays a catalyst role in the assembly of small nuclear ribonucleoproteins (snRNPs). They play an important role in the splicing of cellular pre-mRNAs. They ensure the correct splicing of U12 intron-containing genes that is important for normal motor and proprioceptive neurons development. They are required for resolving RNA-DNA hybrids created by RNA polymerase II. They play a role in the metabolism of small nucleolar ribonucleoprotein.

The report Survival Motor Neuron Protein - Pipeline Review, H2 2017 outlays comprehensive information on the Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities.

It also reviews key players involved in Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Pre-Registration, Phase II and Preclinical stages are 1, 3 and 8 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 4 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Respiratory which include indications Spinal Muscular Atrophy (SMA) and Cystic Fibrosis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

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 its 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

TABLE OF CONTENTS

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

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

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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