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