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이염성 백질 이영양증(MLD) : 파이프라인 리뷰

Metachromatic Leukodystrophy (MLD) (Central Nervous System) - Drugs in Development, 2021

발행일: | 리서치사: Global Markets Direct | 페이지 정보: 영문 51 Pages | 배송안내 : 즉시배송

※ 본 상품은 영문 자료로 한글과 영문 목차에 불일치하는 내용이 있을 경우 영문을 우선합니다. 정확한 검토를 위해 영문목차를 참고해주시기 바랍니다.

이염성 백질 이영양증(MLD : Metachromatic Leukodystrophy) 치료제 개발 상황에 대해 조사했으며, 파이프라인 제품 개요, 임상시험 단계별 제품 개요, 주요 기업 및 약제 개요, 파이프라인 제품의 최신 동향, 최신 뉴스 및 프레스 릴리스 등의 정보를 전해드립니다.

서론

  • 조사 범위

이염성 백질 이영양증(MLD) 개요

치료제 개발

  • 파이프라인 제품 개요
  • 파이프라인 제품 비교 분석

이염성 백질 이영양증(MLD) : 기업에서 개발중인 치료제

이염성 백질 이영양증(MLD) : 대학/기관에서 연구중인 치료제

이염성 백질 이영양증(MLD) : 파이프라인 제품 개요

  • 임상 단계 제품
  • 초기 단계 제품

이염성 백질 이영양증(MLD) : 기업에서 개발중인 제품

이염성 백질 이영양증(MLD) : 대학/기관에서 연구중인 제품

이염성 백질 이영양증(MLD) 치료제 개발에 참여하고 있는 기업

  • ArmaGen Inc.
  • GlaxoSmithKline Plc
  • RegenxBio Inc.
  • Shire Plc

이염성 백질 이영양증(MLD) : 치료제 평가

  • 단독치료 제품(Monotherapy Products)별
  • 표적별
  • 작용기서별
  • 투여 경로별
  • 분자 종류별

약제 개요

이염성 백질 이영양증(MLD) : 최근의 파이프라인 동향

이염성 백질 이영양증(MLD) : 휴지중인 프로젝트

이염성 백질 이영양증(MLD) : 개발이 중지된 제품

부록

KSM 16.08.01

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Metachromatic Leukodystrophy - Drugs In Development, 2021, provides an overview of the Metachromatic Leukodystrophy (Central Nervous System) pipeline landscape.

Metachromatic leukodystrophy is an inherited disorder characterized by the accumulation of fats called sulfatides in cells. Symptoms include vision problems leading to blindness, personality changes, and motor disturbances such as clumsiness, muscle weakness (hypotonia), rigidity, inability to coordinate movement (ataxia), and/or muscle spasms especially of the neck, spine, arms, and legs. Treatment for metachromatic leukodystrophy is symptomatic and supportive.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Metachromatic Leukodystrophy - Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Metachromatic Leukodystrophy (Central Nervous System), 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 Metachromatic Leukodystrophy (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Metachromatic Leukodystrophy (MLD) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Preclinical and Discovery stages are 2, 3 and 3 respectively. Similarly, the Universities portfolio in Phase II and Phase I stages comprises 2 and 1 molecules, respectively.

Metachromatic Leukodystrophy (Central Nervous System) 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 Metachromatic Leukodystrophy (Central Nervous System).
  • The pipeline guide reviews pipeline therapeutics for Metachromatic Leukodystrophy (Central Nervous System) 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 Metachromatic Leukodystrophy (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Metachromatic Leukodystrophy (Central Nervous System) 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 Metachromatic Leukodystrophy (Central Nervous System)

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 Metachromatic Leukodystrophy (Central Nervous System).
  • 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 Metachromatic Leukodystrophy (Central Nervous System) 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

Table of Contents

List of Tables

List of Figures

  • Introduction
  • Global Markets Direct Report Coverage
  • Metachromatic Leukodystrophy (MLD) - Overview
  • Metachromatic Leukodystrophy (MLD) - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
  • Metachromatic Leukodystrophy (MLD) - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Metachromatic Leukodystrophy (MLD) - Companies Involved in Therapeutics Development
  • Affinia Therapeutics
  • ArmaGen Inc
  • Denali Therapeutics Inc
  • Homology Medicines Inc
  • Orchard Therapeutics Plc
  • Passage Bio Inc
  • Recursion Pharmaceuticals Inc
  • Takeda Pharmaceutical Co Ltd
  • Metachromatic Leukodystrophy (MLD) - Drug Profiles
  • AGT-183 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • atidarsagene autotemcel - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • DUOC-01 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Fusion Protein to Replace Arylsulfatase A (ARSA) for Metachromatic Leukodystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy for Metachromatic Leukodystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Arylsulfatase A for Metachromatic Leukodystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • HMI-202 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • PBML-04 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules to Activate Arylsulfatase A for Metachromatic Leukodystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • TAK-611 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Metachromatic Leukodystrophy (MLD) - Dormant Projects
  • Metachromatic Leukodystrophy (MLD) - Discontinued Products
  • Metachromatic Leukodystrophy (MLD) - Product Development Milestones
  • Featured News & Press Releases
  • May 11, 2021: Passage Bio announces pre-clinical data presentation at Annual ASGCT Meeting
  • Feb 13, 2020: Homology Medicines presents new data characterizing AAVHSCs as potential gene therapies for nervous system disorders
  • Oct 21, 2019: Homology Medicines presents data from investigational MLD gene therapy program HMI-202 at the American Society of Human Genetics (ASHG) 2019 Meeting
  • Nov 07, 2018: Homology Medicines presents data on platform's ability to target the central and peripheral nervous system and therapeutic potential in the rare disease Metachromatic Leukodystrophy
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact Us
  • Disclaimer
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