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폼페병 : 파이프라인 리뷰

Pompe Disease - Pipeline Review, H1 2020

리서치사 Global Markets Direct
발행일 2020년 04월 상품 코드 200991
페이지 정보 영문 102 Pages
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폼페병 : 파이프라인 리뷰 Pompe Disease - Pipeline Review, H1 2020
발행일 : 2020년 04월 페이지 정보 : 영문 102 Pages

폼페병(II형 당원병)은 유전성 질환의 하나로, 초기 증상으로 보행·호흡에 이용하는 근육의 단계적인 쇠약을 들 수 있습니다. 특히 유아에게 발병할 경우, 때때로 심근에 심각한 영향을 미칩니다. α글루코시다아제(말타아제)라는 글리코겐 분해 기능이 있는 효소의 돌연변이체가 이 질환을 일으키는 원인입니다. 발증의 결과, 글리코겐이 인체(특히 근육)에 축적되어 개별 세포에 타격을 줍니다.

세계 각국에서의 폼페병 치료용 파이프라인 제품 개발 상황에 대해 분석했으며, 파이프라인 제품의 최신 동향, 임상시험 각 단계의 제품 리스트, 주요 기업 개요, 주요 약제 개요, 최신 업계 동향 등을 조사하여 전해드립니다.

서론

  • 조사 범위

폼페병 - 개요

폼페병 - 치료제 개발

  • 파이프라인 개요
  • 기업별 파이프라인 동향
  • 대학·연구기관별 파이프라인 동향
  • 기업에서 개발중인 제품
  • 대학·연구기관에서 개발중인 제품

폼페병 - 치료제 평가

  • 표적별
  • 작용기서별
  • 투여 경로별
  • 분자 종류별

폼페병 치료제 개발에 참여하고 있는 기업

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • EpiVax Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Huons Co Ltd
  • JCR Pharmaceuticals Co Ltd
  • Oxyrane Belgium NV
  • Pharming Group NV
  • Sarepta Therapeutics Inc

약제 개요

폼페병 - 휴지중인 프로젝트

폼페병 - 개발이 중지된 제품

폼페병 - 제품 개발 마일스톤

  • 주요 뉴스와 프레스 릴리스

부록

KSA 17.06.13

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 Pompe Disease, 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
  • Pompe Disease - Pipeline by Abeona Therapeutics Inc, H1 2020
  • Pompe Disease - Pipeline by Actus Therapeutics Inc, H1 2020
  • Pompe Disease - Pipeline by Amicus Therapeutics Inc, H1 2020
  • Pompe Disease - Pipeline by Audentes Therapeutics Inc, H1 2020
  • Pompe Disease - Pipeline by Avidity Biosciences LLC, H1 2020
  • Pompe Disease - Pipeline by AVROBIO Inc, H1 2020
  • Pompe Disease - Pipeline by EpiVax Inc, H1 2020
  • Pompe Disease - Pipeline by Etubics Corp, H1 2020
  • Pompe Disease - Pipeline by Genzyme Corp, H1 2020
  • Pompe Disease - Pipeline by Greenovation Biotech GmbH, H1 2020
  • Pompe Disease - Pipeline by Icagen Inc, H1 2020
  • Pompe Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020
  • Pompe Disease - Pipeline by NanoMedSyn SAS, H1 2020
  • Pompe Disease - Pipeline by Oxyrane Belgium NV, H1 2020
  • Pompe Disease - Pipeline by Pharming Group NV, H1 2020
  • Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc, H1 2020
  • Pompe Disease - Pipeline by Sarepta Therapeutics Inc, H1 2020
  • Pompe Disease - Pipeline by Spark Therapeutics Inc, H1 2020
  • Pompe Disease - Pipeline by Valerion Therapeutics LLC, H1 2020
  • Pompe Disease - Dormant Projects, H1 2020
  • Pompe Disease - Discontinued Products, H1 2020

List of Figures

  • Number of Products under Development for Pompe Disease, 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 Targets, H1 2020
  • Number of Products by Stage and Targets, H1 2020
  • Number of Products by Mechanism of Actions, H1 2020
  • Number of Products by Stage and 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 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 Pompe Disease - Pipeline Review, H1 2020, provides an overview of the Pompe Disease (Metabolic Disorders) pipeline landscape.

Pompe disease is an inherited disorder caused by defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). This enzyme is needed to break down glycogen, a form of sugar stored in muscle cells. When too much glycogen builds up in the muscle cells, the cells become damaged and the muscles cannot function properly. Symptoms include respiratory failure, muscle pain, proximal muscle weakness, respiratory tract infections, headache and difficulty chewing or jaw muscle fatigue.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Pompe Disease - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Pompe Disease (Metabolic 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 Pompe Disease (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Pompe Disease 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, IND/CTA Filed, Preclinical and Discovery stages are 2, 2, 1, 14 and 2 respectively. Similarly, the Universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 1 molecules, respectively.

Pompe Disease (Metabolic 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 Pompe Disease (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Pompe Disease (Metabolic 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 Pompe Disease (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Pompe Disease (Metabolic 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 Pompe Disease (Metabolic 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 Pompe Disease (Metabolic 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 Pompe Disease (Metabolic 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
  • Pompe Disease - Overview
  • Pompe Disease - Therapeutics Development
  • Pompe Disease - Therapeutics Assessment
  • Pompe Disease - Companies Involved in Therapeutics Development
  • Pompe Disease - Drug Profiles
  • Pompe Disease - Dormant Projects
  • Pompe Disease - Discontinued Products
  • Pompe Disease - Product Development Milestones
  • Appendix
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