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Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H1 2019

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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

                                      Biogen Inc

                                        Exicure Inc

                                          F. Hoffmann-La Roche Ltd

                                            Ionis Pharmaceuticals Inc

                                              Novartis AG

                                                Sarepta Therapeutics Inc

                                                  Shift Pharmaceuticals

                                                    Spotlight Innovation Inc

                                                      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

                                                                        BIIB-089 - Drug Profile

                                                                          Product Description

                                                                            Mechanism Of Action

                                                                              R&D Progress

                                                                                branaplam - Drug Profile

                                                                                  Product Description

                                                                                    Mechanism Of Action

                                                                                      R&D Progress

                                                                                        E-1V111 - 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

                                                                                                                GTP-201 - Drug Profile

                                                                                                                  Product Description

                                                                                                                    Mechanism Of Action

                                                                                                                      R&D Progress

                                                                                                                        IONISENAC-2.5Rx - Drug Profile

                                                                                                                          Product Description

                                                                                                                            Mechanism Of Action

                                                                                                                              R&D Progress

                                                                                                                                LDN-5178 - Drug Profile

                                                                                                                                  Product Description

                                                                                                                                    Mechanism Of Action

                                                                                                                                      R&D Progress

                                                                                                                                        Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile

                                                                                                                                          Product Description

                                                                                                                                            Mechanism Of Action

                                                                                                                                              R&D Progress

                                                                                                                                                onasemnogene abeparvovec - Drug Profile

                                                                                                                                                  Product Description

                                                                                                                                                    Mechanism Of Action

                                                                                                                                                      R&D Progress

                                                                                                                                                        PMO-25 - 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

                                                                                                                                                                        Appendix

                                                                                                                                                                          Methodology

                                                                                                                                                                            Coverage

                                                                                                                                                                              Secondary Research

                                                                                                                                                                                Primary Research

                                                                                                                                                                                  Expert Panel Validation

                                                                                                                                                                                    Contact Us

                                                                                                                                                                                      Disclaimer

                                                                                                                                                                                      Survival Motor Neuron Protein - Pipeline Review, H1 2020

                                                                                                                                                                                      Summary

                                                                                                                                                                                      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.

                                                                                                                                                                                      Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 18 molecules. Out of which approximately 10 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase III, Phase II and Preclinical stages are 1, 1, 2 and 6 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 4 and 2 molecules, respectively. Report covers products from therapy areas Central Nervous System and Respiratory which include indications Spinal Muscular Atrophy (SMA), Chronic Bronchitis, Chronic Obstructive Pulmonary Disease (COPD) and Cystic Fibrosis.

                                                                                                                                                                                      The latest report Survival Motor Neuron Protein – Pipeline Review, H1 2020, 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. 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.

                                                                                                                                                                                      The report is built using data and information sourced from 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.

                                                                                                                                                                                      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 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

                                                                                                                                                                                      Buy now