Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
ArmaGen Inc
BioStrategies LC
Chengdu Genevector Biotechnology Co Ltd
Cingen Co
Deli Therapeutics Inc
EdiGene Inc
Eloxx Pharmaceuticals Inc
Gain Therapeutics Inc
Immusoft Corp
JCR Pharmaceuticals Co Ltd
Jupiter Neurosciences Inc
Mi Therapeutics Ltd
Orchard Therapeutics Plc
Ossianix Inc
Paradigm Biopharmaceuticals Ltd
RegenxBio Inc
ReqMed Co Ltd
Sigilon Therapeutics Inc
Tega Therapeutics Inc
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
AGT-181 - Drug Profile
Product Description
Mechanism Of Action
Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
DNL-622 - Drug Profile
Product Description
Mechanism Of Action
Drugs for Hurler syndrome - Drug Profile
Product Description
Mechanism Of Action
ELX-02 - Drug Profile
Product Description
Mechanism Of Action
ISP-001 - Drug Profile
Product Description
Mechanism Of Action
JNS-102 - Drug Profile
Product Description
Mechanism Of Action
JWK-008 - Drug Profile
Product Description
Mechanism Of Action
laronidase - Drug Profile
Product Description
Mechanism Of Action
lepufusp alfa - Drug Profile
Product Description
Mechanism Of Action
MTL-CEBPA - Drug Profile
Product Description
Mechanism Of Action
Oligonucleotide to Activate IDUA for Hurler Syndrome, Unspecified Cancer, Unspecified Muscular Diseases, Unspecified Neurologic Disorders, and Unspecified Non-Genetic Diseases - Drug Profile
Product Description
Mechanism Of Action
OTL-203 - Drug Profile
Product Description
Mechanism Of Action
pentosan polysulfate sodium - Drug Profile
Product Description
Mechanism Of Action
pentosan polysulfate sodium - Drug Profile
Product Description
Mechanism Of Action
Recombint Alpha L-Iduronidase Replacement for Mucopolysaccharidosis I (MPS I) - Drug Profile
Product Description
Mechanism Of Action
Recombint Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
RGX-111 - Drug Profile
Product Description
Mechanism Of Action
SIG-005 - Drug Profile
Product Description
Mechanism Of Action
SIG-XXX: MPS-1 + CNS - Drug Profile
Product Description
Mechanism Of Action
TXB-4LS1 - Drug Profile
Product Description
Mechanism Of Action
X-372 - Drug Profile
Product Description
Mechanism Of Action
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
Featured News & Press Releases
Sep 26, 2022: Paradigm Reports Successful Safety Review in MPS VI Phase 2 Clinical Trial and update on timing of PARA_008 top-line data readout.
Sep 01, 2022: Immusoft announces FDA clearance of IND application for ISP-001 for MPS I, the first engineered B cell therapy to enter into clinical trials
Aug 08, 2022: MPS clinical program update and paradigm to present at the Intertiol Conference on lysosomal diseases
Feb 11, 2022: Intertiol researchers highlight the need for PPS as a new adjunct therapy treatment for unmet needs of patients with MPS VI
Feb 10, 2022: JCR pharmaceuticals to present posters on JR-171 at the 18th annual WORLDSymposium 2022
Feb 07, 2022: Immusoft to present at WORLDSymposium 2022 Conference
Jan 31, 2022: Sigilon Therapeutics to present preclinical data at the 18th Annual WORLDSymposium on Lysosomal Diseases, MPS-1 and MPS-6
Nov 23, 2021: Positive interim data from phase 2 rare disease trial presented at intertiol medical congress
Oct 05, 2021: JCR Pharmaceuticals: US FDA grants Fast Track Desigtion for JR-171 for the treatment of Mucopolysaccaridosis Type I (MPSI)
Sep 09, 2021: Sigilon Therapeutics announces acceptance of clinical trial application in the UK for SIG-005 for the treatment of mucopolysaccharidosis type I
Jul 23, 2021: Sigilon Therapeutics presents preclinical data at the 16th Intertiol Symposium on MPS and Related Diseases
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)
Dec 17, 2020: Sigilon Therapeutics receives Orphan Drug Desigtion for SIG-005 for the treatment of mucopolysaccharidosis type I
Nov 12, 2020: First patient dosed in Phase 2 clinical trial evaluating PPS in Mucopulysaccharidoses Type-1 (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 for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), 2022
Number of Products under Development by Companies, 2022
Number of Products by Top 10 Targets, 2022
Number of Products by Stage and Top 10 Targets, 2022
Number of Products by Top 10 Mechanism of Actions, 2022
Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Number of Products by Top 10 Routes of Administration, 2022
Number of Products by Stage and Top 10 Routes of Administration, 2022
Number of Products by Top 10 Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022

List of Tables

List of Tables
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by BioStrategies LC, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Chengdu Genevector Biotechnology Co Ltd, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Cinnagen Co, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Denali Therapeutics Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by EdiGene Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Gain Therapeutics Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Jupiter Neurosciences Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Mina Therapeutics Ltd, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Orchard Therapeutics Plc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Ossianix Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Paradigm Biopharmaceuticals Ltd, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ReqMed Co Ltd, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sigilon Therapeutics Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Tega Therapeutics Inc, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, 2022
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, 2022