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市場調查報告書

α-L-iduronidase:開發中產品分析

Alpha L-Iduronidase - Pipeline Review, H1 2020

出版商 Global Markets Direct 商品編碼 371944
出版日期 內容資訊 英文 64 Pages
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α-L-iduronidase:開發中產品分析 Alpha L-Iduronidase - Pipeline Review, H1 2020
出版日期: 2020年02月28日內容資訊: 英文 64 Pages
簡介

本報告提供以α-L-iduronidase為標的之治療藥開發平台現狀及最新更新的各開發階段比較分析,提供您加上最新的新聞和發表之企業和研究機關開發中的治療藥,治療藥評估,後期階段及中止的計劃等相關資訊。

簡介

  • 調查範圍

α-L-iduronidase 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

  • AngioChem Inc.
  • ArmaGen Inc.
  • Bioasis Technologies Inc.
  • RegenxBio Inc.
  • Sangamo BioSciences, Inc.

藥物簡介

暫停中的計劃

主要消息及新聞稿

附錄

圖表

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目錄
Product Code: GMDHC2286TDB

Summary

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) pipeline Target constitutes close to 13 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Alpha L-Iduronidase - Pipeline Review, H1 2020, outlays comprehensive information on the Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Alpha L-iduronidase is an enzyme encoded by IDUA gene. It hydrolyzes the terminal alpha-L-iduronic acid residues of two glycosaminoglycans, dermatan sulfate and heparan sulfate. Mutations in this gene that result in enzymatic deficiency lead to the autosomal recessive disease mucopolysaccharidosis type I (MPS I). The molecules developed by companies in Phase II, Phase I, Preclinical and Discovery stages are 3, 1, 7 and 1 respectively. Similarly, the universities portfolio in Discovery stages comprises 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).

Furthermore, this report also reviews key players involved in Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by 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.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The report provides a snapshot of the global therapeutic landscape for Alpha L-Iduronidase (IDUA or EC 3.2.1.76)
  • The report reviews Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76)
  • 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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
  • Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Overview
  • Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - 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
  • Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Therapeutics Assessment
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Companies Involved in Therapeutics Development
  • ArmaGen Inc
  • BioStrategies LC
  • bluebird bio Inc
  • GT Gain Therapeutics SA
  • Immusoft Corp
  • JCR Pharmaceuticals Co Ltd
  • Orchard Therapeutics Plc
  • Ossianix Inc
  • RegenxBio Inc
  • Sangamo Therapeutics Inc
  • Tamid Bio Inc
  • Tega Therapeutics Inc
  • Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Drug Profiles
  • AGT-181 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate Alpha L-Iduronidase for Mucopolysaccharidosis I (MPS I) - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ISP-001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • laronidase - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • OTL-203 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Recombinant Alpha L-Iduronidase Replacement for Mucopolysaccharidosis I (MPS I) - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • RGX-111 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SB-318 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Tamid-001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • TXB-4LS1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • X-372 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Dormant Products
  • Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Product Development Milestones
  • Featured News & Press Releases
  • Feb 10, 2020: Orchard Therapeutics announces presentation on OTL-203 at 16th Annual WORLD Symposium
  • Dec 08, 2019: Orchard Therapeutics presents clinical data on OTL-203, at the 61st American Society of Hematology Annual Meeting
  • Nov 06, 2019: Orchard Therapeutics to present on its Hurler Syndrome drug candidate OTL-203 at the 61st American Society of Hematology Annual Meeting
  • Sep 04, 2019: Orchard Therapeutics announces encouraging update from proof-of-concept study of OTL-203 for the treatment of Mucopolysaccharidosis Type I (MPS-I)
  • Aug 27, 2019: Orchard Therapeutics announces presentation on OTL-203 at Upcoming Society for the Study of Inborn Errors of Metabolism (SSIEM) 2019 Annual Symposium
  • Jul 17, 2019: Immusoft announces the appointment of Robert Hayes, Ph.D. as Chief Scientific Officer
  • Apr 02, 2019: Sangamo provided an update on its in vivo genome editing program: SB-318
  • Feb 12, 2019: JCR announces presentation on JR-171 for hurler syndrome at the 15th Annual ORLDSymposium 2019
  • Feb 07, 2019: Sangamo Announces Interim Results Of Phase 1/2 EMPOWERS Study Evaluating SB-318 Zinc Finger Nuclease (ZFN) In Vivo Genome Editing Demonstrating Increased Leukocyte IDUA Activity In Patients With MPS I
  • Feb 01, 2019: Sangamo Therapeutics to host conference call to review interim results from phase 1/2 EMPOWERS study for Mucopolysaccharidosis type I
  • Jan 04, 2019: Sangamo announces upcoming clinical data Presentation on SB-318 At WORLDSymposium 2019
  • Oct 17, 2018: Immusoft receives rare pediatric disease designation for treatment of MPS I
  • Jul 23, 2018: Sangamo treats first patient in EMPOWERS Study of SB-318
  • Jul 10, 2018: ArmaGen's AGT-181 52-Week Phase 1/2 Proof-of-Concept Study Results Published in Orphanet Journal of Rare Diseases
  • Jun 12, 2018: REGENXBIO Receives FDA Fast Track Designation for RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
  • 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, H1 2020
  • Number of Products under Development by Therapy Areas, H1 2020
  • Number of Products under Development by Indication, H1 2020
  • Number of Products under Development by Companies, H1 2020
  • Products under Development by Companies, H1 2020
  • Number of Products under Investigation by Universities/Institutes, H1 2020
  • Products under Investigation by Universities/Institutes, H1 2020
  • Number of Products by Stage and Mechanism of Actions, H1 2020
  • Number of Products by Stage and Route of Administration, H1 2020
  • Number of Products by Stage and Molecule Type, H1 2020
  • Pipeline by ArmaGen Inc, H1 2020
  • Pipeline by BioStrategies LC, H1 2020
  • Pipeline by bluebird bio Inc, H1 2020
  • Pipeline by GT Gain Therapeutics SA, H1 2020
  • Pipeline by Immusoft Corp, H1 2020
  • Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020
  • Pipeline by Orchard Therapeutics Plc, H1 2020
  • Pipeline by Ossianix Inc, H1 2020
  • Pipeline by RegenxBio Inc, H1 2020
  • Pipeline by Sangamo Therapeutics Inc, H1 2020
  • Pipeline by Tamid Bio Inc, H1 2020
  • Pipeline by Tega Therapeutics Inc, H1 2020
  • Dormant Projects, H1 2020

List of Figures

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