Table of Content


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
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 Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotides to Activate SMN2 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BIIB-089 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
braplam - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate SMN for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate SMN1 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
IONISEC-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 Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
osemnogene abeparvovec - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PMO-25 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
risdiplam - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules 2 to Activate SMN2 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate SMN2 for Spil Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate SMN2 for Spil 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
Apr 03, 2019: ICER Issues Fil Report on Zolgensma, provides Policy recommendations related to Pricing and Coverage of treatments for Spil Muscular Atrophy
Apr 01, 2019: AveXis expands world-leading gene therapy manufacturing capacity with purchase of advanced biologics therapy manufacturing campus in Longmont, Colorado
Mar 27, 2019: Chugai receives Orphan Drug Desigtion for Risdiplam in Spil Muscular Atrophy
Feb 18, 2019: Gene Therapy Firm AveXis to expand in Durham County adding 200 jobs and $60 Million in investment
Feb 01, 2019: First phase 3 gene therapy trial in spil muscular atrophy at The John Walton Muscular Dystrophy Centre
Jan 08, 2019: Genentech, Roche and PTC Therapeutics provide update on Regulatory Filing for Risdiplam
Dec 19, 2018: Roche gets PRIME desigtion for SMA drug in Europe
Dec 03, 2018: Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1
Nov 05, 2018: Novartis provides update on AVXS101
Oct 03, 2018: Roche announces new data for Risdiplam in spil muscular atrophy(SMA) at the World Muscle Society Congress
Oct 03, 2018: Risdiplam Demonstrates Prelimiry Evidence of Clinical Benefit in Type 1, 2, & 3 Spil Muscular Atrophy Patients
Jul 24, 2018: Spotlight Innovation spil muscular atrophy research collaborator professor Kevin Hodgetts receives $300,000 grant from Cure SMA
Jun 16, 2018: Updated Prelimiry Data from SMA FIREFISH Program in Type 1 Babies Presented at the CureSMA Conference
Jun 14, 2018: Exicure to Present Data at the Cure Spil Muscular Atrophy Annual Conference in Dallas
May 08, 2018: AveXis Issues Community Statement on the SPR1NT Trial
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, H1 2019
Number of Products under Development by Therapy Areas, H1 2019
Number of Products under Development by Top 10 Indications, H1 2019
Number of Products by Stage and Mechanism of Actions, H1 2019
Number of Products by Routes of Administration, H1 2019
Number of Products by Stage and Routes of Administration, H1 2019
Number of Products by Molecule Types, H1 2019
Number of Products by Stage and Molecule Types, H1 2019


List of Tables


Number of Products under Development by Stage of Development, H1 2019
Number of Products under Development by Therapy Areas, H1 2019
Number of Products under Development by Indication, H1 2019
Number of Products under Development by Companies, H1 2019
Products under Development by Companies, H1 2019
Number of Products under Investigation by Universities/Institutes, H1 2019
Products under Investigation by Universities/Institutes, H1 2019
Number of Products by Stage and Mechanism of Actions, H1 2019
Number of Products by Stage and Route of Administration, H1 2019
Number of Products by Stage and Molecule Type, H1 2019
Pipeline by AveXis Inc, H1 2019
Pipeline by Biogen Inc, H1 2019
Pipeline by Exicure Inc, H1 2019
Pipeline by F. Hoffmann-La Roche Ltd, H1 2019
Pipeline by Ionis Pharmaceuticals Inc, H1 2019
Pipeline by Novartis AG, H1 2019
Pipeline by Sarepta Therapeutics Inc, H1 2019
Pipeline by Spotlight Innovation Inc, H1 2019
Dormant Projects, H1 2019
Discontinued Products, H1 2019