Mucopolysaccharidosis - Drug Pipeline Landscape, 2023

Mucopolysaccharidosis is a group of metabolic inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). There are seven types of mucopolysaccharidosis : mps type I (hurler syndrome), mps type II (hunter syndrome), mps type III (sanfilippo syndrome), mps type IV (morquio syndrome), mps type VI (maroteaux-lamy syndrome), mps type VII (sly syndrome), and mps type IX (hyaluronidase deficiency).

Mucopolysaccharidosis is caused by the absence or malfunctioning of certain enzymes in the body needs to break down molecules called glycosaminoglycans.

Mucopolysaccharidosis main symptoms are abnormal bones in the spine, claw hands, cloudy corneas, deafness, heart valve problems, liver and spleen enlargement, and changing facial features.

Mucopolysaccharidosis is diagnosed by low iduronate 2-sulfatase enzyme activity and elevated plasma and or urine GAGs.

Mucopolysaccharidosis is treated by enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation.

Report Highlights

Global Insight Service’s, Mucopolysaccharidosis - Drug Pipeline Landscape, 2023 report provides an overview of the Mucopolysaccharidosis pipeline drugs. This report covers detailed insights on Mucopolysaccharidosis drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Mucopolysaccharidosis pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.


The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.


The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clinical trials, and key upcoming milestones are included.

Reasons to Buy

Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.

Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.

Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.

Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.

Please Note: This report will be delivered by publisher within 3-4 business days of order confirmation.

List of Tables
List of Figures
1. Introduction
1.1 Mucopolysaccharidosis – Pipeline Drugs, 2023 - Coverage
2. Disease Overview – Mucopolysaccharidosis
2.1 Causes
2.2 Signs and Symptoms
2.3 Diagnosis
2.4 Disease Management
3. Mucopolysaccharidosis – Pipeline Drugs Development - Overview
3.1 Comparative Analysis by Stage of Development
3.2 Number of Products Under Development by Companies, 2023
3.3 Products Under Development by Companies, 2023
4. Assessment of Therapeutics
4.1 Assessment by Target
4.2 Assessment by Mechanism of Action
4.3 Assessment by Molecule Type
4.3 Assessment by Route of Administration
5. Drug Profiles
5.1 Clinical Stage Drugs - Phase III
5.1.1 ABO-102
5.1.2 AX 250
5.1.3 DNL310
5.1.4 GNR-055
5.1.5 JR-141
5.1.6 Kineret
5.1.7 LYS-SAF302
5.1.8 RGX-121
5.2 Clinical Stage Drugs - Phase II
5.2.1 AAV2/8.TBG.hARSB
5.2.2 AGT-181
5.2.3 JR-171
5.2.4 Odiparcil
5.2.5 OTL-201
5.2.6 OTL-203
5.2.7 RGX-111
5.2.8 SOBI003
5.2.9 Sodium Pentosan Polysulfate
5.3 Clinical Stage Drugs - Phase I
5.2.1 AGT-182
5.2.2 EGT-101
5.2.3 HMI-203
5.2.4 JNS102
5.4 Early Stage Drugs – Preclinical
5.4.1 AGT-184
5.4.2 AGT-187
5.4.3 ALL-027
5.4.4 AX 313
5.4.5 BIOS: MPS I
5.4.6 CAP-001
5.4.7 DNL126
5.4.8 DNL622
5.4.9 Drug for MPS II
5.4.10 EGT-201
5.4.11 EGT-301
5.4.12 Gene Therapy for Mucopolysaccharidosis Type I (MPS I)
5.4.13 ISP-001
5.4.14 JLK-247
5.4.15 JR-441
5.4.16 JR-443
5.4.17 JR-446
5.4.18 MTL-CEBPA for MPS1 Hurler Syndrome
5.4.19 RBIO-1
5.5 Clinical Stage Drugs – Discovery
5.5.1 AAV9 gene replacement therapyAVP-6
5.5.3 BMRD-001
5.5.4 Drug for Mucopolysaccharidosis
5.5.5 GC1130A
5.5.6 GC1133A
5.5.7 IDUA
5.5.8 M043
5.5.9 M161
5.5.10 M201
5.5.11 Next Generation Program
5.5.12 SIG-018
5.5.13 SIG-020
5.5.14 SIG-XXX
6. Key Regulatory Designations
7. Key Deals
8. Key Upcoming Milestones
9. Key Companies Involved
9.1 Allievex Corporation
9.2 Amicus Therapeutics Inc
9.4 Apteeus SAS
9.5 ArmaGen Inc
9.6 Biorchestra Co., Ltd
9.7 BioStrategies LC
9.8 Bluebird bio Inc
9.9 Capsida Biotherapeutics Inc
9.10 Denali Therapeutics Inc
9.11 Esteve Pharmaceuticals SA
9.12 Fondazione Telethon
9.13 Gain Therapeutics Inc
9.14 GC Biopharma Corp
9.15 Homology Medicines Inc
9.16 Immusoft Corp
9.17 Inventiva Pharma
9.18 JCR Pharmaceuticals Co Ltd
9.19 Jupiter Neurosciences Inc
9.21 M6P Therapeutics
9.22 Mina Therapeutics Ltd
9.24 Orchard Therapeutics
9.25 Phoenix Nest
9.26 RainBIO Inc
9.28 ReqMed Co Ltd
9.29 Sigilon Therapeutics Inc
9.30 Talaris Therapeutics Inc
9.31 Swedish Orphan Biovitrum
9.32 Ultragenyx Pharmaceutical Inc
10. Dormant Drugs
10.1 Inactive Drugs
10.2 Discontinued Drugs
11. Appendix
List of Tables
Table 1.1 Number of Products Under Development for Mucopolysaccharidosis
Table 1.2 Number of Products Under Development by Companies
Table 1.3 Products Under Development by Companies
Table 1.4 Products by Targets
Table 1.5 Products by Mechanism of Action
Table 1.6 Products by Molecule Type
Table 1.7 Products by Route of Administration
Table 1.2 Number of Products Under Development by Companies
Table 1.3 Products Under Development by Companies
Table 1.4 Products by Targets
Table 1.5 Products by Mechanism of Action
Table 1.6 Products by Molecule Type
Table 1.7 Products by Route of Administration
Table 2.1 C Clinical Trial Details – ABO-102/Ultragenyx Pharmaceutical Inc
Table 2.2 Clinical Trial Details – AX 250/Allievex Corporation
Table 2.3 Clinical Trial Details – DNL310/Denali Therapeutics Inc
Table 2.4 Clinical Trial Details – GNR-055/AO GENERIUM
Table 2.5 Clinical Trial Details – JR-141/JCR Pharmaceuticals Co Ltd
Table 2.6 Clinical Trial Details – Kineret/Swedish Orphan Biovitrum
Table 2.7 Clinical Trial Details – LYS-SAF302/LYSOGENE
Table 2.8 Clinical Trial Details – RGX-121/REGENXBIO Inc
Table 2.9 Clinical Trial Details – AAV2/8.TBG.hARSB/Fondazione Telethon
Table 2.10 Clinical Trial Details – AGT-181/ArmaGen Inc
Table 2.11 Clinical Trial Details – Hematopoietic Stem Cell Infusion/Talaris Therapeutics Inc
Table 2.12 Clinical Trial Details – JR-171 /JCR Pharmaceuticals Co Ltd
Table 2.13 Clinical Trial Details – Odiparcil/Inventiva Pharma
Table 2.14 Clinical Trial Details – OTL-201/Orchard Therapeutics
Table 2.15 Clinical Trial Details – RGX-111/REGENXBIO Inc
Table 2.16 Clinical Trial Details – SOBI003/Swedish Orphan Biovitrum
Table 2.17 Clinical Trial Details – AGT-182/ArmaGen Inc
Table 2.18 Clinical Trial Details – HMI-203/Homology Medicines Inc
Table 3.1 Regulatory Designations
Table 4.1 Inactive Drugs
Table 4.2 Discontinued Drugs
List of Figures
Figure 1.1 Number of Products Under Development for Mucopolysaccharidosis, 2023
Figure 1.2 Products by Top 5 Targets and Stage of Development for Mucopolysaccharidosis, 2023
Figure 1.3 Products by Top 5 Mechanism of Action for Mucopolysaccharidosis, 2023
Figure 1.4 Products by Top 5 Molecule Type for Mucopolysaccharidosis, 2023
Figure 1.5 Products by Top 5 Route of Administration for Mucopolysaccharidosis, 2023

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