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アルファ L-イズロニダーゼ:パイプライン製品の分析

Alpha L-Iduronidase - Pipeline Review, H1 2020

発行 Global Markets Direct 商品コード 371944
出版日 ページ情報 英文 64 Pages
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アルファ L-イズロニダーゼ:パイプライン製品の分析 Alpha L-Iduronidase - Pipeline Review, H1 2020
出版日: 2020年02月28日 ページ情報: 英文 64 Pages
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本レポートは最新情報反映のため適宜更新し、内容構成変更を行う場合があります。ご検討の際はお問い合わせください。
概要

当レポートでは、アルファ L-イズロニダーゼを標的とした治療薬開発パイプラインの現況と最新アップデートによる各開発段階の比較分析、企業や研究機関によって開発中の治療薬、治療薬の評価、後期段階および中止されたプロジェクトに関する情報などを最新のニュースや発表も交えて提供しています。

イントロダクション

  • 調査範囲

アルファ L-イズロニダーゼ 概要

治療薬の開発

  • 開発中の製品:開発段階別
  • 開発中の製品:治療領域別
  • 開発中の製品:症状別

パイプライン製品の概況

  • 後期段階の製品
  • 初期段階の製品

企業で開発中の製品

大学/機関で開発中の製品

治療薬の評価

  • 単剤/併用治療薬の場合
  • 作用機序別
  • 投与経路別
  • 分子タイプ別

治療薬開発に従事している企業

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

薬剤プロファイル

休止中のプロジェクト

主なニュースおよびプレスリリース

付録

図表

図表

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
目次
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