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
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- 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
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- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
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