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Dystrophin (DMD):開發中產品分析

Dystrophin (DMD) - Pipeline Review, H2 2019

出版商 Global Markets Direct 商品編碼 372677
出版日期 內容資訊 英文 117 Pages
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Dystrophin (DMD):開發中產品分析 Dystrophin (DMD) - Pipeline Review, H2 2019
出版日期: 2019年12月27日內容資訊: 英文 117 Pages
簡介

本報告提供以Dystrophin (DMD)為標的之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。

簡介

  • 調查範圍

Dystrophin (DMD) 概要

治療藥的開發

  • 開發中的產品:各開發階段
  • 開發中的產品:各治療領域
  • 開發中的產品:不同症狀

開發中產品概況

  • 後期階段的產品
  • 初期階段的產品

企業開發中的產品

大學/機關開發中的產品

治療藥的評估

  • 單劑/並用治療藥的情況
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

治療藥的開發企業

  • Asklepios BioPharmaceutical, Inc.
  • BioMarin Pharmaceutical Inc.
  • 第一三共
  • Genethon
  • 日本新藥
  • PTC Therapeutics, Inc.
  • Sarepta Therapeutics, Inc.
  • WAVE Life Sciences Ltd.

藥物簡介

暫停中的計劃

開發中止的產品

主要消息及新聞稿

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: GMDHC2157TDB

Summary

Dystrophin (DMD) - Dystrophin is a cytoplasmic protein. It anchors the extracellular matrix to the cytoskeleton via F-actin. It acts as ligand for dystroglycan. It act as component of the dystrophin-associated glycoprotein complex which accumulates at the neuromuscular junction and at a variety of synapses in the peripheral and central nervous systems and has a structural function in stabilizing the sarcolemma.

Dystrophin (DMD) pipeline Target constitutes close to 48 molecules. Out of which approximately 44 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 2, 4, 1, 23 and 12 respectively. Similarly, the universities portfolio in Preclinical stages comprises 4 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Musculoskeletal Disorders which include indications Duchenne Muscular Dystrophy and Muscular Dystrophy.

The latest report Dystrophin (DMD) - Pipeline Review, H2 2019, outlays comprehensive information on the Dystrophin (DMD) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews key players involved in Dystrophin (DMD) targeted therapeutics development with respective active and dormant or discontinued projects.

The report is built using data and information sourced from 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.

Scope

  • The report provides a snapshot of the global therapeutic landscape for Dystrophin (DMD)
  • The report reviews Dystrophin (DMD) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in Dystrophin (DMD) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Dystrophin (DMD) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews latest news and deals related to Dystrophin (DMD) targeted therapeutics

Reasons to buy

  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Identify and understand the targeted therapy areas and indications for Dystrophin (DMD)
  • Identify the use of drugs for target identification and drug repurposing
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
  • Devise corrective measures for pipeline projects by understanding Dystrophin (DMD) development landscape
  • 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

Table of Contents

List of Tables

List of Figures

  • Introduction
  • Global Markets Direct Report Coverage
  • Dystrophin (DMD) - Overview
  • Dystrophin (DMD) - Therapeutics Development
  • Products under Development by Stage of Development
  • Products under Development by Therapy Area
  • Products under Development by Indication
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
  • Dystrophin (DMD) - Therapeutics Assessment
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Dystrophin (DMD) - Companies Involved in Therapeutics Development
  • Alpha Anomeric
  • Audentes Therapeutics Inc
  • Daiichi Sankyo Co Ltd
  • Dystrogen Therapeutics SA
  • Editas Medicine Inc
  • Evox Therapeutics Ltd
  • MyoGene Bio LLC
  • Nippon Shinyaku Co Ltd
  • NS Pharma Inc
  • OliPass Corporation
  • Pepgen Ltd
  • Pfizer Inc
  • Sarepta Therapeutics Inc
  • Spinalcyte Llc
  • Sutura Therapeutics Ltd
  • Tolerion Inc
  • Vertex Pharmaceuticals Inc
  • Wave Life Sciences Ltd
  • Dystrophin (DMD) - Drug Profiles
  • Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Antisense Oligonucleotides to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • AT-702 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • AT-751 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • AT-753 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • casimersen - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • CYMD-201 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • DMD-1742 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • DS-5141 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • eteplirsen - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy 1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • golodirsen - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • NS-089 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotides 5 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotides 6 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotides to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • PF-06939926 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Protein to Target Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-4008 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-4044 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-4050 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-4052 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-4055 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-5044 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-5045 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-5050 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-5051 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-5052 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-5053 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRP-9001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Stem Cell Therapy 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Stem Cell Therapy 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SUT-001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • viltolarsen - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Dystrophin (DMD) - Dormant Products
  • Dystrophin (DMD) - Discontinued Products
  • Dystrophin (DMD) - Product Development Milestones
  • Featured News & Press Releases
  • Dec 13, 2019: Sarepta's DMD therapy Vyondys 53 secures FDA accelerated approval
  • Nov 05, 2019: Dystrogen Therapeutics announces that treatment with Dystrophin expressing Chimeric (DEC) Cells improves cardiac function in Preclinical Duchenne's Study
  • Aug 21, 2019: Sarepta fails to receive FDA approval for DMD drug golodirsen
  • Aug 08, 2019: Sarepta Therapeutics comments on erroneous submission to US FDA Adverse Event Reporting System (FAERS)
  • Jul 09, 2019: New analysis shows drug slows down respiratory decline
  • Jul 01, 2019: AskBio's Perspective on Pfizer's 1b clinical trial results on Duchenne MD Gene
  • Jun 28, 2019: Pfizer presents initial clinical data on phase 1b gene therapy study for Duchenne Muscular Dystrophy (DMD)
  • Apr 18, 2019: Parent Project Muscular Dystrophy awards $100,000 grant to Nationwide Children's Hospital to further explore GALGT2 gene therapy in duchenne muscular dystrophy
  • Mar 28, 2019: Sarepta Therapeutics announces positive expression results from the Casimersen (SRP-4045) arm of the ESSENCE study
  • Mar 25, 2019: Sarepta Therapeutics to provide update on Duchenne Muscular Dystrophy gene therapy program
  • Feb 15, 2019: FDA accepts Sarepta's NDA for precision medicine Golodirsen
  • Feb 06, 2019: NS Pharma begins rolling NDA submission to FDA for Viltolarsen (NS-065/NCNP-01)
  • Dec 20, 2018: Sarepta Therapeutics completes Submission of New Drug Application Seeking approval of Golodirsen (SRP-4053) in patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53
  • Nov 13, 2018: Evox Therapeutics wins funding from Duchenne UK to explore exosome-mediated delivery of dystrophin
  • Oct 03, 2018: NS-065/NCNP-01 (Viltolarsen) of Nippon Shinyaku' in-house product presentation on results of Phase I/II study in Japan
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development by Stage of Development, H2 2019
  • Number of Products under Development by Therapy Areas, H2 2019
  • Number of Products under Development by Indication, H2 2019
  • Number of Products under Development by Companies, H2 2019
  • Products under Development by Companies, H2 2019
  • Products under Development by Companies, H2 2019 (Contd..1), H2 2019
  • Products under Development by Companies, H2 2019 (Contd..2), H2 2019
  • Number of Products under Investigation by Universities/Institutes, H2 2019
  • Products under Investigation by Universities/Institutes, H2 2019
  • Number of Products by Stage and Mechanism of Actions, H2 2019
  • Number of Products by Stage and Route of Administration, H2 2019
  • Number of Products by Stage and Molecule Type, H2 2019
  • Pipeline by Alpha Anomeric, H2 2019
  • Pipeline by Audentes Therapeutics Inc, H2 2019
  • Pipeline by Daiichi Sankyo Co Ltd, H2 2019
  • Pipeline by Dystrogen Therapeutics SA, H2 2019
  • Pipeline by Editas Medicine Inc, H2 2019
  • Pipeline by Evox Therapeutics Ltd, H2 2019
  • Pipeline by MyoGene Bio LLC, H2 2019
  • Pipeline by Nippon Shinyaku Co Ltd, H2 2019
  • Pipeline by NS Pharma Inc, H2 2019
  • Pipeline by OliPass Corporation, H2 2019
  • Pipeline by Pepgen Ltd, H2 2019
  • Pipeline by Pfizer Inc, H2 2019
  • Pipeline by Sarepta Therapeutics Inc, H2 2019
  • Pipeline by Spinalcyte Llc, H2 2019
  • Pipeline by Sutura Therapeutics Ltd, H2 2019
  • Pipeline by Tolerion Inc, H2 2019
  • Pipeline by Vertex Pharmaceuticals Inc, H2 2019
  • Pipeline by Wave Life Sciences Ltd, H2 2019
  • Dormant Projects, H2 2019
  • Discontinued Products, H2 2019

List of Figures

  • Number of Products under Development by Stage of Development, H2 2019
  • Number of Products under Development by Therapy Areas, H2 2019
  • Number of Products under Development by Top 10 Indications, H2 2019
  • Number of Products by Stage and Mechanism of Actions, H2 2019
  • Number of Products by Routes of Administration, H2 2019
  • Number of Products by Stage and Routes of Administration, H2 2019
  • Number of Products by Molecule Types, H2 2019
  • Number of Products by Stage and Molecule Types, H2 2019
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