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산성 스핑고미엘리나제 결핍증(니만피크병) C형 : 파이프라인 리뷰

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2020

리서치사 Global Markets Direct
발행일 2020년 04월 상품 코드 332892
페이지 정보 영문 109 Pages
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산성 스핑고미엘리나제 결핍증(니만피크병) C형 : 파이프라인 리뷰 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2020
발행일 : 2020년 04월 페이지 정보 : 영문 109 Pages

산성 스핑고미엘리나제 결핍증(니만피크병)은 리소좀 축적증의 하나로, 대사에 영향을 미치고 유전자 돌연변이에 기인하는 질환입니다. 체내 세포, 특히 망내계 세포(단핵식세포계)에 스핑고지질이 축적됩니다. C형(NPC)은 특히 A형 및 B형(ASMD)과는 크게 다릅니다. 환자는 콜레스테롤 및 기타 지질을 세포내에서 적절히 대사할 수 없습니다. 이에 따라 간 및 비장에 콜레스테롤, 뇌내에 지질이 과도하게 축적됩니다. 증상은 간 비대, 뇌장애, 보행 곤란, 연하장애, 접촉 과민, 발화 장애, 근긴장 소실, 학습 장애 등을 들 수 있습니다.

세계 산성 스핑고미엘리나제 결핍증(니만피크병) C형 파이프라인 제품의 개발 상황에 대해 분석했으며, 파이프라인 제품의 최신 동향, 주요 기업의 개요, 주요 약제 개요(제품 개요, 기능 메커니즘, 연구개발의 진전 상황), 최신 업계 동향 등을 조사한 결과를 정리하여 전해드립니다.

서론

  • 조사 범위
  • 산성 스핑고미엘리나제 결핍증(니만피크병) C형 개요

치료제 개발

  • 파이프라인 제품;개요
  • 기업에서 개발중인 치료제
  • 대학/기관에서 연구중인 치료제
  • 기업에서 개발중인 제품
  • 대학/기관에서 연구중인 제품

치료제 평가

  • 표적별
  • 작용기서별
  • 투여 경로별
  • 분자 종류별

치료제 개발에 참여하고 있는 기업

  • CTD Holdings Inc
  • Merck & Co., Inc.
  • Okklo Life Sciences BV
  • Orphazyme ApS
  • Sucampo Pharmaceuticals Inc

약제 개요

휴지중인 프로젝트

산성 스핑고미엘리나제 결핍증(니만피크병) C형 치료제 개발 마일스톤

  • 주목해야 할 최신 동향·프레스 릴리스

부록

도표

KSA 17.05.30

이 페이지에 게재되어 있는 내용은 최신판과 약간 차이가 있을 수 있으므로 영문목차를 함께 참조하여 주시기 바랍니다. 기타 자세한 사항은 문의 바랍니다.

List of Tables

  • Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2020
  • Number of Products under Development by Companies, H1 2020
  • Number of Products under Development by Universities/Institutes, H1 2020
  • Products under Development by Companies, H1 2020
  • Products under Development by Companies, H1 2020 (Contd..1), H1 2020
  • Products under Development by Universities/Institutes, H1 2020
  • Number of Products by Stage and Target, H1 2020
  • Number of Products by Stage and Mechanism of Action, H1 2020
  • Number of Products by Stage and Route of Administration, H1 2020
  • Number of Products by Stage and Molecule Type, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Amicus Therapeutics Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Cyclo Therapeutics Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by E-scape Bio Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Endece LLC, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Evox Therapeutics Ltd, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by IntraBio Ltd, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Jewell Laboratories LLC, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Lauren Sciences LLC, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Mallinckrodt Plc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Neurotrope Bioscience Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Oraxion Therapeutics Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme A/S, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Perlara PBC, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Prevacus Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by SOM Biotech SL, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by StrideBio Inc, H1 2020
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H1 2020

List of Figures

  • Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2020
  • Number of Products under Development by Companies, H1 2020
  • Number of Products under Development by Universities/Institutes, H1 2020
  • Number of Products by Top 10 Targets, H1 2020
  • Number of Products by Stage and Top 10 Targets, H1 2020
  • Number of Products by Top 10 Mechanism of Actions, H1 2020
  • Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
  • Number of Products by Routes of Administration, H1 2020
  • Number of Products by Stage and Routes of Administration, H1 2020
  • Number of Products by Top 10 Molecule Types, H1 2020
  • Number of Products by Stage and Top 10 Molecule Types, H1 2020

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency Type C - Pipeline Review, H1 2020, 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 - Pipeline Review, H1 2020, 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 Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 3, 1, 2, 10, 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.

Table of Contents

  • Introduction
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Development
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drug Profiles
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones
  • Appendix
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