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貝登氏症 - 開發中產品分析

Batten Disease - Pipeline Review, H1 2020

出版商 Global Markets Direct 商品編碼 277747
出版日期 內容資訊 英文 92 Pages
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貝登氏症 - 開發中產品分析 Batten Disease - Pipeline Review, H1 2020
出版日期: 2020年02月28日內容資訊: 英文 92 Pages
簡介

貝登氏症是惡化型代謝性神經退化性疾病的一種,又稱為神經元蠟樣脂褐質儲積症。症狀有從5歲到15歲之間的失明,肌肉的協調運動失調,精神遲緩或精神功能低,情緒障礙、抽筋、肌肉的痙攣、肌肉張力低落、異動症等。

本報告提供貝登氏症的治療藥開發情形調查分析,提供您開發中產品概要,臨床實驗的各階段產品概要,主要企業簡介,藥物簡介,開發中產品的最新趨勢,最新消息和新聞稿等相關的系統性資訊。

目錄

簡介

貝登氏症概要

治療藥的開發

  • 開發中產品:概要
  • 開發中產品:比較分析

貝登氏症 - 開發中的治療藥:各企業

貝登氏症 - 調查中的治療藥:大學/研究機關別

貝登氏症 - 開發中產品的概要

  • 臨床實驗階段的產品
  • 初期階段的產品
  • 開發階段不明的產品

貝登氏症 - 開發中的產品:各企業

貝登氏症 - 調查中的產品:大學/研究機關別

貝登氏症 - 開發治療藥的企業

  • Abeona Therapeutics, Inc.
  • BioMarin Pharmaceutical Inc.
  • CereSpir Incorporated
  • RegenxBio Inc.
  • Spark Therapeutics, Inc.

貝登氏症 -治療藥的評估

  • 單獨療法的情況
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

  • ABO-201
  • cerliponase alfa
  • CSP-1103
  • Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease
  • Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease
  • INI-0602
  • MP-101
  • Small Molecules to Inhibit CDC42 for Batten Disease
  • SNB-2401
  • SNB-4050
  • SPKTPP-1
  • XN-001

貝登氏症 - 開發中產品的最新趨勢

貝登氏症 - 開發暫停中的計劃

貝登氏症 - 開發中止的產品

貝登氏症 - 產品開發的里程碑

  • 最新消息和新聞稿

附錄

圖表

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: GMDHC11976IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Batten Disease - Pipeline Review, H1 2020, provides an overview of the Batten Disease (Central Nervous System) pipeline landscape.

Batten disease, a rare genetic disorder, belongs to a group of progressive degenerative neurometabolic disorders known as the neuronal ceroid lipofuscinoses. Symptoms of Batten disease usually become apparent between five and 15 years of age. Symptoms include vision loss, lack of muscle coordination, mental retardation or decreasing mental function, emotional disturbances, seizures, muscle spasms, deterioration of muscle tone and movement problems.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Batten Disease - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Batten Disease (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 Batten Disease (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Batten Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 3, 2, 16 and 1 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 4 and 2 molecules, respectively.

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

List of Tables

List of Figures

Introduction

  • Global Markets Direct Report Coverage
  • Batten Disease - Overview
  • Batten Disease - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
  • Batten Disease - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Batten Disease - Companies Involved in Therapeutics Development
  • Abeona Therapeutics Inc
  • Amicus Therapeutics Inc
  • Blue Turtle Bio Technologies Inc
  • Circumvent Pharmaceuticals Inc
  • Collaborations Pharmaceuticals Inc
  • Exicure Inc
  • Polaryx Therapeutics Inc
  • Recursion Pharmaceuticals Inc
  • RegenxBio Inc
  • Retrotope Inc
  • Seneb BioSciences Inc
  • Spark Therapeutics Inc
  • Theranexus SAS
  • Xonovo Inc
  • Batten Disease - Drug Profiles
  • (gemfibrozil + tretinoin) - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ABO-201 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ABO-202 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ATGTX-502 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • BBDF-101 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • DUOC-01 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • gemfibrozil - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate CLN1 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate CLN8 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate TPP1 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate TPP1 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • INI-0602 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotides for Neurology - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • PLX-300 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Recombinant Palmitoyl-Protein Thioesterase-1 Replacement for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • RGX-181 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • RT-001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • RT-002 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecule for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SNB-4050 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SPKTPP-1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • XN-001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Batten Disease - Dormant Projects
  • Batten Disease - Discontinued Products
  • Batten Disease - Product Development Milestones
  • Featured News & Press Releases
  • Jan 20, 2020: Polaryx Therapeutics receives IND approval for PLX-200 From the FDA for the treatment of late infantile neuronal ceroid lipofuscinosis
  • Oct 17, 2019: REGENXBIO Announces Presentations at the European Society of Gene & Cell Therapy 27th Annual Congress
  • Jun 18, 2019: Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease
  • May 21, 2019: Abeona Therapeutics announces FDA clearance of Investigational New Drug Application for ABO-202 Gene Therapy in CLN1 Disease
  • Apr 15, 2019: Abeona Therapeutics to present data for ABO-202 in CLN1 disease at the American Society of Gene and Cell Therapy Annual Meeting
  • Jan 31, 2019: Spark Therapeutics announces presentation of preclinical data in CLN2 disease at 15th annual WORLDSymposium
  • Jan 31, 2019: REGENXBIO receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy for the Treatment of CLN2 Form of Batten Disease
  • Dec 06, 2018: Abeona Therapeutics provides update on Infantile Batten Disease drug candidate ABO-202 at 2018 R&D Day
  • Nov 14, 2018: FDA grants Orphan Drug Designation to RGX-181 gene therapy for the treatment of CLN2 form of Batten disease
  • Sep 03, 2018: Regenxbio adds new neurodegenerative candidate to pipeline
  • Aug 30, 2018: REGENXBIO Announces Pipeline Expansion With RGX-181 Gene Therapy For CLN2 Form Of Batten Disease
  • Apr 20, 2018: Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease
  • Mar 15, 2018: Abeona Therapeutics Receives FDA Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease
  • Feb 12, 2018: Abeona Therapeutics Receives FDA Orphan Drug Designation for ABO-202 Gene Therapy Program in Infantile Batten Disease
  • Dec 11, 2017: Polaryx Therapeutics Receives Orphan Drug Designation From the US FDA for the Treatment of Neuronal Ceroid Lipofuscinoses With PLX-100
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development for Batten 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
  • Batten Disease - Pipeline by Abeona Therapeutics Inc, H1 2020
  • Batten Disease - Pipeline by Amicus Therapeutics Inc, H1 2020
  • Batten Disease - Pipeline by Blue Turtle Bio Technologies Inc, H1 2020
  • Batten Disease - Pipeline by Circumvent Pharmaceuticals Inc, H1 2020
  • Batten Disease - Pipeline by Collaborations Pharmaceuticals Inc, H1 2020
  • Batten Disease - Pipeline by Exicure Inc, H1 2020
  • Batten Disease - Pipeline by Polaryx Therapeutics Inc, H1 2020
  • Batten Disease - Pipeline by Recursion Pharmaceuticals Inc, H1 2020
  • Batten Disease - Pipeline by RegenxBio Inc, H1 2020
  • Batten Disease - Pipeline by Retrotope Inc, H1 2020
  • Batten Disease - Pipeline by Seneb BioSciences Inc, H1 2020
  • Batten Disease - Pipeline by Spark Therapeutics Inc, H1 2020
  • Batten Disease - Pipeline by Theranexus SAS, H1 2020
  • Batten Disease - Pipeline by Xonovo Inc, H1 2020
  • Batten Disease - Dormant Projects, H1 2020
  • Batten Disease - Discontinued Products, H1 2020

List of Figures

  • Number of Products under Development for Batten 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 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 Molecule Types, H1 2020
  • Number of Products by Stage and Molecule Types, H1 2020