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Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Publisher Global Markets Direct
Published Dec 28, 2022
Length 82 Pages
SKU # GMD17708785

Description

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 20 molecules. Out of which approximately 13 molecules are developed by companies and remaining by the universities/institutes. The latest report Survival Motor Neuron Protein - Drugs In Development, 2022, 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 Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 1, 1, 7 and 2 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 4 and 2 molecules, respectively. Report covers products from therapy areas Central Nervous System which include indications Spinal Muscular Atrophy (SMA) and Huntington Disease.

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

Table of Contents

82 Pages
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
Amogen Pharma Pvt Ltd
Beijing GeneCradle Technology Co Ltd
Biocad
CANbridge Life Sciences Ltd
F. Hoffmann-La Roche Ltd
Huida (Shanghai) Biotechnology Co Ltd
Novartis AG
Novartis Gene Therapies
Sarepta Therapeutics Inc
Shift Pharmaceuticals
Spotlight Innovation Inc (Inactive)
Suzhou GenAssist Therapeutics Co Ltd
Voyager Therapeutics Inc
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) – Drug Profiles
ALB-111 – Drug Profile
ANB-4 – Drug Profile
Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy – Drug Profile
branaplam hydrochloride – Drug Profile
E-1V111 – Drug Profile
GC-101 – Drug Profile
GEN-8010 – Drug Profile
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy – Drug Profile
HG-001 – Drug Profile
onasemnogene abeparvovec – Drug Profile
PMO-25 – Drug Profile
Resagen – Drug Profile
risdiplam – Drug Profile
Second-Generation scAAV9 Gene Therapy – Drug Profile
Small Molecules 2 to Activate SMN2 for Spinal Muscular Atrophy – Drug Profile
Small Molecules for Spinal Muscular Atrophy – Drug Profile
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy – Drug Profile
Spinal Muscular Atrophy – Drug Profile
STL-182 – Drug Profile
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
Oct 13, 2022: CANbridge-UMass Chan Medical School gene therapy research in oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress
Oct 12, 2022: Positive new data for Roche’s Evrysdi in largest trial ever undertaken in patients with previously-treated spinal muscular atrophy (SMA)
Jun 17, 2022: Novartis announces Nature Medicine publication of Zolgensma data demonstrating age-appropriate milestones when treating children with SMA presymptomatically
May 31, 2022: Roche’s Evrysdi gets FDA approval for spinal muscular atrophy in babies
May 02, 2022: Roche's Evrysdi offers survival benefits in babies with SMA
Apr 05, 2022: Novartis expands Zolgensma manufacturing capacity with approval of multi-product North Carolina facility
Mar 16, 2022: New data for Roche’s Evrysdi (risdiplam) demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophy (SMA)
Mar 16, 2022: New data for Genentech’s Evrysdi (risdiplam) demonstrate long-term efficacy and safety in a broad population of people with Spinal Muscular Atrophy (SMA)
Mar 14, 2022: Novartis data again demonstrate age-appropriate development when Zolgensma is used presymptomatically, and post-hoc data reveal SMA Type 1 patients could speak, swallow and maintain airway protection
Mar 13, 2022: Novartis data again demonstrate age-appropriate development when Zolgensma is used presymptomatically, and post-hoc data reveal SMA Type 1 patients could speak, swallow and maintain airway protection
Mar 08, 2022: Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio
Feb 03, 2022: Roche Canada and the pan-Canadian Pharmaceutical Alliance (pCPA) successfully complete negotiations for EVRYSDI (risdiplam) for the treatment of adults and children with spinal muscular atrophy (SMA)
Jan 31, 2022: Cost-effectiveness analysis temporarily halted for Zolgensma to collect long-term data
Jan 27, 2022: 1st RWE data comes out confirming efficacy of Zolgensma in Korean SMA patients
Jan 25, 2022: Roche’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under 2 months of age with spinal muscular atrophy (SMA)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development by Stage of Development, 2022
Table 2: Number of Products under Development by Therapy Areas, 2022
Table 3: Number of Products under Development by Indication, 2022
Table 4: Number of Products under Development by Companies, 2022
Table 5: Products under Development by Companies, 2022
Table 6: Number of Products under Investigation by Universities/Institutes, 2022
Table 7: Products under Investigation by Universities/Institutes, 2022
Table 8: Number of Products by Stage and Mechanism of Actions, 2022
Table 9: Number of Products by Stage and Route of Administration, 2022
Table 10: Number of Products by Stage and Molecule Type, 2022
Table 11: Pipeline by Amogen Pharma Pvt Ltd, 2022
Table 12: Pipeline by Beijing GeneCradle Technology Co Ltd, 2022
Table 13: Pipeline by Biocad, 2022
Table 14: Pipeline by CANbridge Life Sciences Ltd, 2022
Table 15: Pipeline by F. Hoffmann-La Roche Ltd, 2022
Table 16: Pipeline by Huida (Shanghai) Biotechnology Co Ltd, 2022
Table 17: Pipeline by Novartis AG, 2022
Table 18: Pipeline by Novartis Gene Therapies, 2022
Table 19: Pipeline by Sarepta Therapeutics Inc, 2022
Table 20: Pipeline by Shift Pharmaceuticals, 2022
Table 21: Pipeline by Spotlight Innovation Inc (Inactive), 2022
Table 22: Pipeline by Suzhou GenAssist Therapeutics Co Ltd, 2022
Table 23: Pipeline by Voyager Therapeutics Inc, 2022
Table 24: Dormant Projects, 2022
Table 25: Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development by Stage of Development, 2022
Figure 2: Number of Products under Development by Top 10 Indications, 2022
Figure 3: Number of Products by Stage and Mechanism of Actions, 2022
Figure 4: Number of Products by Routes of Administration, 2022
Figure 5: Number of Products by Stage and Routes of Administration, 2022
Figure 6: Number of Products by Molecule Types, 2022
Figure 7: Number of Products by Stage and Molecule Types, 2022

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