Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drugs In Development, 2022, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) pipeline landscape.

Niemann-Pick C disease is one of a group of lysosomal storage diseases that affect metabolism and that are caused by genetic mutations. It involves the accumulation of sphingolipids in cells throughout the body, particularly reticuloendothelial cells (the mononuclear phagocyte system). Symptoms include enlarged liver, brain damage, difficulty walking and swallowing, increased sensitivity to touch, difficulty speaking, loss of muscle tone, learning difficulties.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase I, Preclinical, Discovery and Unknown stages are 1, 3, 2, 14, 5 and 1 respectively. Similarly, the Universities portfolio in Phase 0, Preclinical and Discovery stages comprises 1, 2 and 2 molecules, respectively.

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note:Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders)
Reasons to Buy
  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
  • 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.


Introduction
Global Markets Direct Report Coverage
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Overview
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Companies Involved in Therapeutics Development
Amicus Therapeutics Inc
Azafaros BV
Chameleon Biosciences Inc
Cyclo Therapeutics Inc
CycloLab Cyclodextrin Research and Development Laboratory Ltd
Deep Genomics Inc
E-scape Bio Inc
Endece LLC
Evox Therapeutics Ltd
Gene Therapy Research Institution Co Ltd
IntraBio Ltd
Jewell Laboratories LLC
Mandos LLC
Merck & Co Inc
Okklo Life Sciences BV
Oraxion Therapeutics Inc
Orphazyme A/S
Perlara PBC
Prevacus Inc
Scenic Biotech BV
Shanghai Pharmaceuticals Holding Co Ltd
SOM Biotech SL
StrideBio Inc
Synaptogenix Inc
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Drug Profiles
acetylleucine – Drug Profile
adrabetadex – Drug Profile
arimoclomol citrate – Drug Profile
AZ-3102 – Drug Profile
Bryostatin-1 – Drug Profile
Drugs to Activate NPC1 for Niemann-Pick Disease Type C – Drug Profile
ESB-1609 – Drug Profile
EVX-101 – Drug Profile
Gene Therapy for Acid Sphingomyelinase Deficiency Type C – Drug Profile
Gene Therapy to Activate NPC1 for Niemann-Pick Type C1 Disease – Drug Profile
GT-0009X – Drug Profile
HPGCD – Drug Profile
IB-4000 – Drug Profile
MLSA-1 – Drug Profile
Monoclonal Antibodies to Inhibit oxLDL for Metabolic Disorders – Drug Profile
NDC-1308 – Drug Profile
Niemann-Pick Disease Type C – Drug Profile
OKL-1014 – Drug Profile
ORX-301 – Drug Profile
PERL-101 – Drug Profile
PRV-001 – Drug Profile
Small Molecule for Atherosclerosis and Niemann-Pick Type C Disease – Drug Profile
Small Molecules for Cardiovascular, Oncology, Poisoning, Viral Infections and Neurodegenerative Diseases – Drug Profile
Small Molecules for Niemann-Pick Type C Disease – Drug Profile
Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease – Drug Profile
SOM-0208 – Drug Profile
SRD-4415 – Drug Profile
STRX-210 – Drug Profile
Trappsol Cyclo – Drug Profile
ursodiol – Drug Profile
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Dormant Projects
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Product Development Milestones
Featured News & Press Releases
Jul 13, 2022: Cyclo Therapeutics to present at the World Orphan Drug Congress USA 2022
Jun 24, 2022: Cyclo Therapeutics to present at the 2022 NPC Patient and Family Conference hosted by the Australian NPC Disease Foundation
May 09, 2022: Cyclo Therapeutics to participate in the virtual investor Niemann-Pick Disease Type C spotlight event
Mar 24, 2022: Azafaros announces FDA Grant of Orphan Drug Designation for AZ-3102 in the treatment of Niemann-Pick Disease
Feb 14, 2022: Azafaros presents positive clinical and preclinical data supporting development of lead compound AZ-3102 in lysosomal storage disorders at the 18th Annual WORLDSymposium conference
Feb 09, 2022: Cyclo therapeutics selected to present overview of pivotal phase 3 study for lead candidate, Trappsol Cyclo, at WORLDSymposium 2022
Feb 03, 2022: Cyclo Therapeutics announces formation of global steering committee comprised of leading experts to Advise on the global phase 3 clinical development program for Trappsol Cyclo in Niemann-Pick Disease Type C
Oct 18, 2021: Cyclo Therapeutics announces abstract accepted for poster presentation at the 14th International Congress of Inborn Errors of Metabolism (ICIEM)
Oct 06, 2021: Cyclo Therapeutics to participate in the 2021 Virtual Cyclodextrin Conference Hosted by CycloLab
Jul 30, 2021: Cyclo Therapeutics announces new positive safety and efficacy data from ongoing phase 1 open-label extension study of Trappsol Cyclo for the treatment of Niemann-Pick disease type C1
Jul 27, 2021: Cyclo Therapeutics commences commercial-scale manufacturing for Trappsol Cyclo
Jun 21, 2021: Cyclo Therapeutics to present at the 2021 NPC patient and family conference hosted by the Australian NPC Disease Foundation
Jun 18, 2021: Cyclo Therapeutics starts enrolment in Phase III NPC1 treatment trial
Apr 27, 2021: Cyclo Therapeutics announces design of pivotal phase 3 study evaluating Trappsol Cyclo in Niemann-Pick Type C1
Mar 25, 2021: Cyclo Therapeutics meets primary efficacy endpoint in phase 1/2 trial from intravenous Trappsol Cyclo in rare disease Niemann-Pick Type C1 (NPC1)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Number of Products under Development by Universities/Institutes, 2022
Table 4: Products under Development by Companies, 2022
Table 5: Products under Development by Companies, 2022 (Contd..1)
Table 6: Products under Development by Universities/Institutes, 2022
Table 7: Number of Products by Stage and Target, 2022
Table 8: Number of Products by Stage and Mechanism of Action, 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: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Amicus Therapeutics Inc, 2022
Table 12: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Azafaros BV, 2022
Table 13: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Chameleon Biosciences Inc, 2022
Table 14: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Cyclo Therapeutics Inc, 2022
Table 15: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by CycloLab Cyclodextrin Research and Development Laboratory Ltd, 2022
Table 16: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Deep Genomics Inc, 2022
Table 17: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by E-scape Bio Inc, 2022
Table 18: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Endece LLC, 2022
Table 19: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Evox Therapeutics Ltd, 2022
Table 20: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Gene Therapy Research Institution Co Ltd, 2022
Table 21: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by IntraBio Ltd, 2022
Table 22: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Jewell Laboratories LLC, 2022
Table 23: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Mandos LLC, 2022
Table 24: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Merck & Co Inc, 2022
Table 25: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Okklo Life Sciences BV, 2022
Table 26: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Oraxion Therapeutics Inc, 2022
Table 27: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Orphazyme A/S, 2022
Table 28: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Perlara PBC, 2022
Table 29: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Prevacus Inc, 2022
Table 30: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Scenic Biotech BV, 2022
Table 31: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Shanghai Pharmaceuticals Holding Co Ltd, 2022
Table 32: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by SOM Biotech SL, 2022
Table 33: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by StrideBio Inc, 2022
Table 34: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Synaptogenix Inc, 2022
Table 35: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Dormant Projects, 2022
List of Figures
Figure 1: Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products under Development by Universities/Institutes, 2022
Figure 4: Number of Products by Top 10 Targets, 2022
Figure 5: Number of Products by Stage and Top 10 Targets, 2022
Figure 6: Number of Products by Top 10 Mechanism of Actions, 2022
Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Figure 8: Number of Products by Routes of Administration, 2022
Figure 9: Number of Products by Stage and Routes of Administration, 2022
Figure 10: Number of Products by Molecule Types, 2022
Figure 11: Number of Products by Stage and Top 10 Molecule Types, 2022

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