Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2016

Published: Dec, 2016 | Pages: 82 | 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, H2 2016

Summary

Global Markets Direct's, 'Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 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.
*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 ands scope

 Table of Contents


Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Overview 7
Therapeutics Development 8
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Stage of Development 8
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Therapy Area 9
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Indication 10
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Products Glance 11
Late Stage Products 11
Early Stage Products 12
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Companies 13
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Universities/Institutes 15
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Assessment 17
Assessment by Monotherapy/Combination Products 17
Assessment by Mechanism of Action 18
Assessment by Route of Administration 19
Assessment by Molecule Type 21
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development 23
AveXis Inc 23
Biogen Inc 24
F. Hoffmann-La Roche Ltd 25
Genethon SA 26
Genzyme Corp 27
Novartis AG 28
Sarepta Therapeutics Inc 29
Spotlight Innovation Inc 30
Voyager Therapeutics Inc 31
WAVE Life Sciences Ltd 32
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Drug Profiles 33
ALB-111 - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
AVXS-101 - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Gene Therapy to Activate SMN1 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
LMI-070 - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
nusinersen - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - 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 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 SMN2 for Spinal Muscular Atrophy - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
STL-182 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
VYSMN-101 - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Dormant Projects 61
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Featured News & Press Releases 62
Dec 06, 2016: Study Finds New Treatment For Spinal Muscular Atrophy Safe For Infants 62
Nov 07, 2016: Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study in Later-Onset Spinal Muscular Atrophy 63
Nov 01, 2016: AveXis Announces Single-Arm Design for U.S. Pivotal Study of AVXS-101 in SMA Type 1 Patients 64
Oct 28, 2016: Biogen's Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA 65
Oct 20, 2016: Spinal Muscular Atrophy Program Advances into Phase 2 Clinical Studies in SMA Patients with RG7916 66
Oct 08, 2016: New Data Presented at World Muscle Society Congress Support Potential Benefit of Investigational Treatment Nusinersen in Spinal Muscular Atrophy 67
Oct 08, 2016: AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 as Presented at the International Annual Congress of the World Muscle Society 69
Sep 26, 2016: Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy 71
Sep 23, 2016: CHMP of the EMA has Granted Nusinersen Accelerated Assessment 72
Aug 11, 2016: AveXis Reports Interim Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 72
Aug 04, 2016: Candidate Drug for Spinal Muscular Atrophy, with Roots in CSHL Research, Passes Major Hurdle 75
Aug 01, 2016: Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy 76
Jul 25, 2016: Update on LMI070 for the Treatment of Type 1 SMA 77
Jul 20, 2016: AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1 78
May 16, 2016: AveXis Presents Pulmonary Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 78

Appendix 81
Methodology 81
Coverage 81
Secondary Research 81
Primary Research 81
Expert Panel Validation 81
Contact Us 81
Disclaimer 82

List of Tables

Number of Products under Development for, H2 2016 8
Number of Products under Development by Therapy Area, H2 2016 9
Number of Products under Development by Indication, H2 2016 10
Comparative Analysis by Late Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Number of Products under Development by Companies, H2 2016 13
Products under Development by Companies, H2 2016 14
Number of Products under Investigation by Universities/Institutes, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Assessment by Monotherapy/Combination Products, H2 2016 17
Number of Products by Stage and Mechanism of Action, H2 2016 18
Number of Products by Stage and Route of Administration, H2 2016 20
Number of Products by Stage and Molecule Type, H2 2016 22
Pipeline by AveXis Inc, H2 2016 23
Pipeline by Biogen Inc, H2 2016 24
Pipeline by F. Hoffmann-La Roche Ltd, H2 2016 25
Pipeline by Genethon SA, H2 2016 26
Pipeline by Genzyme Corp, H2 2016 27
Pipeline by Novartis AG, H2 2016 28
Pipeline by Sarepta Therapeutics Inc, H2 2016 29
Pipeline by Spotlight Innovation Inc, H2 2016 30
Pipeline by Voyager Therapeutics Inc, H2 2016 31
Pipeline by WAVE Life Sciences Ltd, H2 2016 32
Dormant Projects, H2 2016 61