Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) – Pipeline Review, H1 2018’, 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

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Ionis Pharmaceuticals Inc

Novartis AG

Sarepta Therapeutics Inc

Spotlight Innovation Inc

Voyager Therapeutics Inc

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

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

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

Products under Development by Stage of Development

Products under Development by Therapy Area

Products under Development by Indication

Products under Development by Companies

Products under Development by Universities/Institutes

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

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

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

AveXis Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Ionis Pharmaceuticals Inc

Novartis AG

Sarepta Therapeutics Inc

Spotlight Innovation Inc

Voyager Therapeutics Inc

WAVE Life Sciences Ltd

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

ALB-111 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AVXS-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

branaplam Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate SMN for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

IONISENAC-2.5Rx Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

PMO-25 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RG-7916 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

STL-182 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

VYSMN-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

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

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

Featured News & Press Releases

Jan 27, 2018: Preliminary Data from FIREFISH trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy

Jan 23, 2018: Data from RG7916 Programs in SMA to be Presented at the International Scientific Congress on Spinal Muscular Atrophy

Jan 16, 2018: AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

Jan 04, 2018: AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1

Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

Nov 01, 2017: AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1

Oct 12, 2017: Spinal Muscular Atrophy Program Advances into Pivotal Study in SMA Patients

Oct 03, 2017: AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society

Oct 03, 2017: RG7916 Increased SMN Protein Production in SUNFISH Clinical Trial in Patients with Type 2/3 Spinal Muscular Atrophy

Sep 29, 2017: AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

Aug 24, 2017: Iowa State University biomedical researcher conducts promising trial of potential therapy for spinal muscular atrophy

Jul 01, 2017: Preliminary Phase 2 Data from Spinal Muscular Atrophy Program Presented at CureSMA Conference

Jun 22, 2017: Spotlight Innovation to Present Poster at the Cure SMA 2017 Annual SMA Conference

Jun 14, 2017: AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101

Apr 25, 2017: AveXis Presents Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development by Stage of Development, H1 2018

Number of Products under Development by Therapy Areas, H1 2018

Number of Products under Development by Indication, H1 2018

Number of Products under Development by Companies, H1 2018

Products under Development by Companies, H1 2018

Number of Products under Investigation by Universities/Institutes, H1 2018

Products under Investigation by Universities/Institutes, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Pipeline by AveXis Inc, H1 2018

Pipeline by F. Hoffmann-La Roche Ltd, H1 2018

Pipeline by Genethon SA, H1 2018

Pipeline by Genzyme Corp, H1 2018

Pipeline by Ionis Pharmaceuticals Inc, H1 2018

Pipeline by Novartis AG, H1 2018

Pipeline by Sarepta Therapeutics Inc, H1 2018

Pipeline by Spotlight Innovation Inc, H1 2018

Pipeline by Voyager Therapeutics Inc, H1 2018

Pipeline by WAVE Life Sciences Ltd, H1 2018

Dormant Projects, H1 2018

Discontinued Products, H1 2018

List of Figures

List of Figures

Number of Products under Development by Stage of Development, H1 2018

Number of Products under Development by Therapy Areas, H1 2018

Number of Products under Development by Indications, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

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