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C型酸性神經磷脂酶缺乏症(尼曼匹克症):開發中產品分析

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018

出版商 Global Markets Direct 商品編碼 332892
出版日期 內容資訊 英文 78 Pages
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C型酸性神經磷脂酶缺乏症(尼曼匹克症):開發中產品分析 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018
出版日期: 2018年04月30日 內容資訊: 英文 78 Pages
簡介

酸性神經磷脂酶缺乏症(尼曼匹克症)是影響代謝並由遺傳突變引起的一種溶小體儲積症之一。它涉及鞘脂在整個身體的細胞內積蓄,特別是網狀內皮細胞(單核吞噬細胞系統)。C型(NPC)與A型和B型(ASMD)大不相同。患者無法在細胞內適當代謝膽固醇和其他脂質。因此造成肝臟和脾臟過度積蓄膽固醇,以及腦內過度積存脂質。症狀包括肝臟肥大,腦功能障礙,步行困難,吞嚥障礙,觸覺敏感度高,說話困難,肌肉緊張喪失,學習困難等。

本報告提供全球C型酸性神經磷脂酶缺乏症(尼曼匹克症)的開發中產品之開發情形相關分析,提供您開發中產品的最新趨勢,再加上主要企業簡介,主要藥物概要(產品概要,功能機制,研究開發的發展情形),最新的產業趨勢等調查結果。

簡介

  • 調查範圍
  • C型酸性神經磷脂酶缺乏症(尼曼匹克症) 概要

治療藥的開發

  • 開發中產品;概要
  • 企業開發中的治療藥
  • 大學/機關研究中的治療藥
  • 企業開發中的產品
  • 大學/機關研究中的產品

治療藥的評估

  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

開發治療藥的企業

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

藥物簡介

暫停中的計劃

酸性神經磷脂酶缺乏症 (尼曼匹克症)) C 型治療藥開發的里程碑

  • 值得注意的最新趨勢·新聞稿

附錄

圖表

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目錄
Product Code: GMDHC10401IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018, 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 - Pipeline Review, H1 2018, 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 Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 2, 1, 6 and 2 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.

Table of Contents

List of Tables

List of Figures

  • 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
  • CTD Holdings Inc
  • MallInckrodt Plc
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • Orphazyme A/S
  • Perlara PBC
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drug Profiles
  • acetylleucine - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • adrabetadex - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • arimoclomol - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Bryostatin-1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Delta-tocopherol - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate NPC1 for Niemann-Pick Type C1 Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • MLSA-1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Monoclonal Antibodies to Inhibit oxLDL for Metabolic Disorders - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • OKL-1014 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ORX-301 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • PERL-101 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • RND-001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecule for Atherosclerosis and Niemann-Pick Type C Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for Niemann-Pick Type C Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Trappsol Cyclo - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • vorinostat - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • 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
  • Mar 29, 2018: CTD Holdings Conducts Initial Review of Safety Data for its Phase I and Phase I/II Trials of Trappsol Cyclo to Treat Niemann-Pick Disease Type C
  • Jan 19, 2018: Arimoclomol For NPC Receives Rare Pediatric Disease Designation
  • Dec 06, 2017: CTD Holdings Receives Rare Pediatric Disease Designation for Trappsol Cyclo
  • Sep 28, 2017: CTD Holdings Enrolls First Patient in US Phase I Clinical Trial of Trappsol Cyclo for Treatment of Niemann-Pick Disease Type C
  • Sep 18, 2017: CTD Holdings Announces Approval of Clinical Trial Application to Conduct Phase I/II Clinical Study of Trappsol Cyclo in Patients with Niemann-Pick Type C Disease in Israel
  • Aug 10, 2017: Study Results Published in The Lancet Demonstrate Safety and Efficacy of Intrathecal VTS-270 in Niemann-Pick Disease Type C1
  • Jul 19, 2017: CTD Holdings Announces First Patient Dosed in Phase I/II Clinical Trial to Evaluate Trappsol Cyclo in Niemann-Pick Disease Type C
  • Jul 17, 2017: NIH scientists find rare disease clues in cell's recycling system
  • Jul 13, 2017: CTD Holdings to Present at 25th Annual Family Conference of the National Niemann-Pick Disease Foundation
  • Jun 27, 2017: CTD Holdings Ships Additional Trappsol Cyclo to Brazil
  • Jun 21, 2017: CTD Holdings Enrolls First Patient in European Phase I/II Clinical Trial of Trappsol Cyclo for Treatment of Niemann-Pick Disease Type C
  • Jun 14, 2017: CTD Holdings Announces Appointments of Two Patient Liaisons to Enhance Outreach to Niemann-Pick Disease Type C Families and Caregivers
  • May 16, 2017: CTD Holdings to Host Webinar to Provide Update on Trappsol Cyclo Drug Development Program for Treatment of Niemann-Pick Disease Type C
  • May 11, 2017: Orphazyme Completes Enrolment of Patients For Phase 3 Clinical Trial In Niemann-Pick Type C Disease
  • Apr 19, 2017: CTD Holdings Corrects Mis-Statements about Its Product Made in On-Line Publication
  • 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, H1 2018
  • Table 2: Number of Products under Development by Companies, H1 2018
  • Table 3: Number of Products under Development by Universities/Institutes, H1 2018
  • Table 4: Products under Development by Companies, H1 2018
  • Table 5: Products under Development by Universities/Institutes, H1 2018
  • Table 6: Number of Products by Stage and Target, H1 2018
  • Table 7: Number of Products by Stage and Mechanism of Action, H1 2018
  • Table 8: Number of Products by Stage and Route of Administration, H1 2018
  • Table 9: Number of Products by Stage and Molecule Type, H1 2018
  • Table 10: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by CTD Holdings Inc, H1 2018
  • Table 11: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by MallInckrodt Plc, H1 2018
  • Table 12: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co Inc, H1 2018
  • Table 13: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H1 2018
  • Table 14: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme A/S, H1 2018
  • Table 15: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Perlara PBC, H1 2018
  • Table 16: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H1 2018

List of Figures

  • Figure 1: Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2018
  • Figure 2: Number of Products under Development by Companies, H1 2018
  • Figure 3: Number of Products under Development by Universities/Institutes, H1 2018
  • Figure 4: Number of Products by Top 10 Targets, H1 2018
  • Figure 5: Number of Products by Stage and Top 10 Targets, H1 2018
  • Figure 6: Number of Products by Top 10 Mechanism of Actions, H1 2018
  • Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018
  • Figure 8: Number of Products by Routes of Administration, H1 2018
  • Figure 9: Number of Products by Stage and Routes of Administration, H1 2018
  • Figure 10: Number of Products by Molecule Types, H1 2018
  • Figure 11: Number of Products by Stage and Molecule Types, H1 2018
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