Table of Content


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
Deli 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
Recombint Alpha L-Iduronidase Replacement for Mucopolysaccharidosis I (MPS I) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombint 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 Desigtion to JR-171 for the treatment of Mucopolysaccaridosis Type I (MPS I)
Feb 12, 2021: US FDA grants orphan drug desigtion 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 desigtion for OTL-203 for the treatment of MPS-I
Sep 01, 2020: Orchard Therapeutics announces additiol 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 desigtions 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 Figures

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

List of Tables

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