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Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Drugs In Development, 2021

  • ID: 5359586
  • Drug Pipelines
  • June 2021
  • Region: Global
  • 68 pages
  • Global Markets Direct

FEATURED COMPANIES

  • ArmaGen Inc
  • BioStrategies LC
  • Denali Therapeutics Inc
  • EdiGene Inc
  • Gain Therapeutics Inc
  • Immusoft Corp
1h Free Analyst Time

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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 - Drugs In Development, 2021, 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, Preclinical and Discovery stages are 4, 6 and 2 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
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • ArmaGen Inc
  • BioStrategies LC
  • Denali Therapeutics Inc
  • EdiGene Inc
  • Gain Therapeutics Inc
  • Immusoft Corp
Introduction
  • 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
  • Denali Therapeutics Inc
  • EdiGene Inc
  • Gain Therapeutics Inc
  • Immusoft Corp
  • JCR Pharmaceuticals Co Ltd
  • Orchard Therapeutics Plc
  • Ossianix Inc
  • Rain Bio Inc
  • RegenxBio 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
Fusion Protein to Replace IDUA for Mucopolysaccharidosis I - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
Gene Therapy to Activate Alpha-L-Iduronidase for Mucopolysaccharidosis 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
Oligonucleotide to Activate IDUA for Hurler Syndrome - 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
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) - Discontinued Products

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Product Development Milestones

Featured News & Press Releases
  • Mar 30, 2021: EMA grants Orphan Drug Designation to JR-171 for the treatment of Mucopolysaccaridosis Type I (MPS I)
  • Feb 12, 2021: US FDA grants orphan drug designation to JR-171 for the treatment of Mucopolysaccaridosis Type I (MPS I)
  • Feb 09, 2021: Orchard Therapeutics announces interim data for OTL-203 showing positive clinical results in multiple disease manifestations of mucopolysaccharidosis type I Hurler syndrome (MPS-IH)
  • Jan 28, 2021: Orchard Therapeutics to present abstact on OTL-203 at 2021 WORLDSymposium
  • Dec 01, 2020: RegenxBio announces dosing of first patient in phase I/II trial of RGX-111 for the treatment of Mucopolysaccharidosis type I
  • Sep 28, 2020: Orchard Therapeutics receives EMA PRIME designation for OTL-203 for the treatment of MPS-I
  • Sep 01, 2020: Orchard Therapeutics announces additional interim results from proof-of-concept study of OTL-203 for MPS-I
  • Aug 24, 2020: Orchard Therapeutics announces clinical data presentations at the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT)
  • Jul 20, 2020: Orchard Therapeutics announces orphan drug and rare pediatric disease designations for OTL-203 for the treatment of MPS-I
  • Jul 08, 2020: REGENXBIO provides update on progress of Clinical Programs including RGX-111 for Rare Genetic Neurodegenerative Diseases
  • May 15, 2020: Orchard Therapeutics presents new interim data from OTL-203 proof-of-concept study for MPS-I
  • 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)
Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact the Publisher
  • Disclaimer
List of Tables
  • Number of Products under Development by Stage of Development, 2021
  • Number of Products under Development by Therapy Areas, 2021
  • Number of Products under Development by Indication, 2021
  • Number of Products under Development by Companies, 2021
  • Products under Development by Companies, 2021
  • Number of Products under Investigation by Universities/Institutes, 2021
  • Products under Investigation by Universities/Institutes, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Stage and Route of Administration, 2021
  • Number of Products by Stage and Molecule Type, 2021
  • Pipeline by ArmaGen Inc, 2021
  • Pipeline by BioStrategies LC, 2021
  • Pipeline by Denali Therapeutics Inc, 2021
  • Pipeline by EdiGene Inc, 2021
  • Pipeline by Gain Therapeutics Inc, 2021
  • Pipeline by Immusoft Corp, 2021
  • Pipeline by JCR Pharmaceuticals Co Ltd, 2021
  • Pipeline by Orchard Therapeutics Plc, 2021
  • Pipeline by Ossianix Inc, 2021
  • Pipeline by Rain Bio Inc, 2021
  • Pipeline by RegenxBio Inc, 2021
  • Pipeline by Tega Therapeutics Inc, 2021
  • Dormant Projects, 2021
  • Discontinued Products, 2021
List of Figures
  • Number of Products under Development by Stage of Development, 2021
  • Number of Products by Mechanism of Actions, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Routes of Administration, 2021
  • Number of Products by Stage and Routes of Administration, 2021
  • Number of Products by Molecule Types, 2021
  • Number of Products by Stage and Molecule Types, 2021
Note: Product cover images may vary from those shown
  • ArmaGen Inc
  • BioStrategies LC
  • Denali Therapeutics Inc
  • EdiGene Inc
  • Gain Therapeutics Inc
  • Immusoft Corp
  • JCR Pharmaceuticals Co Ltd
  • Orchard Therapeutics Plc
  • Ossianix Inc
  • Rain Bio Inc
  • RegenxBio Inc
  • Tega Therapeutics Inc
Note: Product cover images may vary from those shown
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