Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Pipeline Review, H1 2016

Published: Jun, 2016 | Pages: 76 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Pipeline Review, H1 2016', 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 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. 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.

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 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

 Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) Overview 8
Therapeutics Development 9
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Products under Development by Stage of Development 9
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Products under Development by Therapy Area 10
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Products under Development by Indication 11
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Pipeline Products Glance 12
Late Stage Products 12
Early Stage Products 13
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Products under Development by Companies 14
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Products under Development by Universities/Institutes 16
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Therapeutics Assessment 18
Assessment by Monotherapy/Combination Products 18
Assessment by Mechanism of Action 19
Assessment by Route of Administration 20
Assessment by Molecule Type 22
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development 24
AveXis, Inc. 24
Bioblast Pharma Ltd. 25
F. Hoffmann-La Roche Ltd. 26
Genethon 27
Genzyme Corporation 28
Ionis Pharmaceuticals, Inc. 29
Novartis AG 30
Sarepta Therapeutics, Inc. 31
Voyager Therapeutics, Inc. 32
WAVE Life Sciences Ltd. 33
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Drug Profiles 34
ALB-111 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
AVXS-101 - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
azithromycin - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
LMI-070 - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
nusinersen - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
PMO-25 - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
RG-7800 - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
RG-7916 - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Small Molecules for Spinal Muscular Atrophy - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
Small Molecules to Activate Survival Motor Neuron Protein for Spinal Muscular Atrophy - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
VYSMN-101 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Dormant Projects 60
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin-1 or SMN1 or SMN2) - Featured News & Press Releases 61
May 16, 2016: AveXis Presents Pulmonary Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 61
May 06, 2016: AveXis Reports Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 62
Apr 25, 2016: AveXis to Report Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 64
Apr 21, 2016: Ionis Pharmaceuticals Reports Data Update from Nusinersen Phase 2 Study in Infants with Spinal Muscular Atrophy and Reviews Neurological Disease Franchise 64
Mar 31, 2016: Ionis Pharmaceuticals Advances Nusinersen in the Ongoing SHINE Study in Patients with SMA 65
Jan 12, 2016: Ionis Pharmaceuticals Completes Target Enrollment for Nusinersen Phase 3 Study, CHERISH, in Children with Spinal Muscular Atrophy 66
Jan 07, 2016: Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development 67
Jan 04, 2016: AveXis Announces Completed Enrollment of Phase 1 Clinical Trial for AVXS-101 in Spinal Muscular Atrophy Type 1 67
Oct 15, 2015: Isis Pharmaceuticals Initiates SHINE Study to Provide ISIS-SMN for Patients With SMA Who Have Completed the Phase 3 ENDEAR and CHERISH Studies 68
Oct 05, 2015: Data from Ongoing Study of AVXS-101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress 69
Oct 04, 2015: Phase 2 Data from Spinal Muscular Atrophy Program Presented at the 20th International Annual Congress of the World Muscle Society 70
Jul 14, 2015: Isis Pharmaceuticals Earns $2.15M for Advancing ISIS-SMN in Children With Spinal Muscular Atrophy 71
Jun 23, 2015: AveXis Gains Orphan Drug Designation From the European Medicines Agency for Gene Therapy Treatment in Spinal Muscular Atrophy 72
Jun 22, 2015: Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Spinal Muscular Atrophy 72
Jun 22, 2015: BioBlast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy 73
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 for, H1 2016 9
Number of Products under Development by Therapy Area, H1 2016 10
Number of Products under Development by Indication, H1 2016 11
Comparative Analysis by Late Stage Development, H1 2016 12
Comparative Analysis by Early Stage Products, H1 2016 13
Number of Products under Development by Companies, H1 2016 14
Products under Development by Companies, H1 2016 15
Number of Products under Investigation by Universities/Institutes, H1 2016 16
Products under Investigation by Universities/Institutes, H1 2016 17
Assessment by Monotherapy/Combination Products, H1 2016 18
Number of Products by Stage and Mechanism of Action, H1 2016 19
Number of Products by Stage and Route of Administration, H1 2016 21
Number of Products by Stage and Molecule Type, H1 2016 23
Pipeline by AveXis, Inc., H1 2016 24
Pipeline by Bioblast Pharma Ltd., H1 2016 25
Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 26
Pipeline by Genethon, H1 2016 27
Pipeline by Genzyme Corporation, H1 2016 28
Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 29
Pipeline by Novartis AG, H1 2016 30
Pipeline by Sarepta Therapeutics, Inc., H1 2016 31
Pipeline by Voyager Therapeutics, Inc., H1 2016 32
Pipeline by WAVE Life Sciences Ltd., H1 2016 33
Dormant Projects, H1 2016 60