Acid Sphingomyelinase Deficiency Type C (Genitourinary Disorders) – Drugs In Development, 2021

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency Type C – Drugs In Development, 2021, provides an overview of the Acid Sphingomyelinase Deficiency 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 Type C – Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency 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 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 Pre-Registration, Phase III, Phase I, Preclinical, Discovery and Unknown stages are 1, 3, 2, 13, 3 and 1 respectively. Similarly, the Universities portfolio in Phase 0, Preclinical and Discovery stages comprises 1, 2 and 2 molecules, respectively.

Acid Sphingomyelinase Deficiency 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 Type C (Genetic Disorders).

– The pipeline guide reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency 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 Type C (Genetic Disorders) therapeutics and enlists all their major and minor projects.

– The pipeline guide evaluates Acid Sphingomyelinase Deficiency 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 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 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 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.

Companies mentioned

Amicus Therapeutics Inc

Chameleon Biosciences Inc

Cyclo Therapeutics Inc

CycloLab Cyclodextrin Research and Development Laboratory Ltd

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

SOM Biotech SL

StrideBio Inc

Table of Contents

Table of Contents

Introduction

Acid Sphingomyelinase Deficiency (Niemann-Pick - Overview

Acid Sphingomyelinase Deficiency (Niemann-Pick - Therapeutics Development

Acid Sphingomyelinase Deficiency (Niemann-Pick - Therapeutics Assessment

Acid Sphingomyelinase Deficiency (Niemann-Pick - Companies Involved in Therapeutics Development

Acid Sphingomyelinase Deficiency (Niemann-Pick - Drug Profiles

Acid Sphingomyelinase Deficiency (Niemann-Pick - Dormant Projects

Acid Sphingomyelinase Deficiency (Niemann-Pick - Discontinued Products

Acid Sphingomyelinase Deficiency (Niemann-Pick - Product Development Milestones

Appendix

List of Tables

List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Companies, 2021 (Contd..1)

Number of Products under Development by Universities/Institutes, 2021

Products under Development by Companies, 2021

Products under Development by Companies, 2021 (Contd..1)

Products under Development by Universities/Institutes, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Amicus Therapeutics Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Chameleon Biosciences Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Cyclo Therapeutics Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by CycloLab Cyclodextrin Research and Development Laboratory Ltd, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by E-scape Bio Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Endece LLC, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Evox Therapeutics Ltd, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Gene Therapy Research Institution Co Ltd, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by IntraBio Ltd, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Jewell Laboratories LLC, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Mandos LLC, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Merck & Co Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Okklo Life Sciences BV, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Oraxion Therapeutics Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Orphazyme A/S, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Perlara PBC, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Prevacus Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Scenic Biotech BV, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by SOM Biotech SL, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by StrideBio Inc, 2021

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Dormant Projects, 2021

List of Figures

List of Figures

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Number of Products by Top 10 Targets, 2021

Number of Products by Stage and Top 10 Targets, 2021

Number of Products by Top 10 Mechanism of Actions, 2021

Number of Products by Stage and Top 10 Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

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