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

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 and scope

Companies mentioned

AveXis Inc

Biogen Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Novartis AG

Sarepta Therapeutics Inc

Spotlight Innovation Inc

Voyager Therapeutics Inc

WAVE Life Sciences Ltd

Table of Contents

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

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

List of Figures

List of Figures

Number of Products under Development for, H2 2016 8

Comparative Analysis by Late Stage Development, H2 2016 11

Comparative Analysis by Early Stage Products, H2 2016 12

Assessment by Monotherapy/Combination Products, H2 2016 17

Number of Products by Stage and Mechanism of Actions, H2 2016 18

Number of Products by Routes of Administration, H2 2016 19

Number of Products by Stage and Routes of Administration, H2 2016 19

Number of Products by Molecule Types, H2 2016 21

Number of Products by Stage and Molecule Type, H2 2016 21

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