Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2018

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2018


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Executive Summary

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)-Pipeline Review, H2 2018

Summary

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 15 molecules. Out of which approximately 9 molecules are developed by companies and remaining by the universities/institutes. The latest report Survival Motor Neuron Protein-Pipeline Review, H2 2018, 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.

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. The molecules developed by companies in Phase III and Preclinical stages are 3 and 6 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 4 and 2 molecules, respectively. Report covers products from therapy areas Central Nervous System and Respiratory which include indications Spinal Muscular Atrophy (SMA) and Cystic Fibrosis.

Furthermore, this report 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. Driven by 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



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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

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Ionis Pharmaceuticals Inc

Novartis AG

Sarepta Therapeutics Inc

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

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

onasemnogene abeparvovec-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 2 to Activate SMN2 for Spinal Muscular Atrophy-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

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

Jun 16, 2018: Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies Presented at the CureSMA Conference

May 08, 2018: AveXis Issues Community Statement on the SPR1NT Trial

May 08, 2018: Scientists investigate new strategy to treat spinal muscular atrophy in infants

Apr 25, 2018: AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3

Apr 24, 2018: AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology

Apr 24, 2018: Preliminary Clinical Data from FIREFISH Trial in Type 1 SMA Patients Presented at the American Academy of Neurology Annual Meeting

Apr 19, 2018: AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology

Apr 18, 2018: Preliminary Study Suggests Drug May Help Babies with Spinal Muscular Atrophy

Apr 15, 2018: Genentech Provided Update on RG-7916

Mar 27, 2018: AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1

Mar 15, 2018: FIREFISH Transitions into Pivotal Phase

Mar 13, 2018: Newly Issued U.S. Patent Covers Spotlight Innovations Spinal Muscular Atrophy Drug Candidates

Jan 30, 2018: AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Figures

Number of Products under Development by Stage of Development, H2 2018

Number of Products under Development by Therapy Areas, H2 2018

Number of Products under Development by Top 10 Indications, H2 2018

Number of Products by Stage and Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Molecule Types, H2 2018

Number of Products by Stage and Molecule Types, H2 2018

List of Tables

Number of Products under Development by Stage of Development, H2 2018

Number of Products under Development by Therapy Areas, H2 2018

Number of Products under Development by Indication, H2 2018

Number of Products under Development by Companies, H2 2018

Products under Development by Companies, H2 2018

Number of Products under Investigation by Universities/Institutes, H2 2018

Products under Investigation by Universities/Institutes, H2 2018

Number of Products by Stage and Mechanism of Actions, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Pipeline by AveXis Inc, H2 2018

Pipeline by F. Hoffmann-La Roche Ltd, H2 2018

Pipeline by Genethon SA, H2 2018

Pipeline by Genzyme Corp, H2 2018

Pipeline by Ionis Pharmaceuticals Inc, H2 2018

Pipeline by Novartis AG, H2 2018

Pipeline by Sarepta Therapeutics Inc, H2 2018

Pipeline by Spotlight Innovation Inc, H2 2018

Dormant Projects, H2 2018

Discontinued Products, H2 2018

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AveXis Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Ionis Pharmaceuticals Inc

Novartis AG

Sarepta Therapeutics Inc

Spotlight Innovation Inc

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Therapeutic Products under Development, Key Players in Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Therapeutics, Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Pipeline Overview, Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Pipeline, Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Pipeline Assessment

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