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Report Overview
Mucopolysaccharidosis (MPS) is a group of inherited lysosomal storage disorders caused by deficiencies in enzymes that degrade glycosaminoglycans. According to Assel Tulebayeva et al., 2025, the total MPS incidence ranges from 1.35 to 16.9 per 100,000 live births. Therapies include enzyme replacement, hematopoietic stem cell transplantation, and emerging gene and substrate reduction approaches. According to the mucopolysaccharidosis pipeline analysis by Expert Market Research, accelerating clinical programs, gene therapy advances, and regulatory incentives are driving pipeline activity. Topic-specific growth factors include improved diagnostics, orphan-drug designations, and rising R&D investment, supporting robust market growth in the coming years.
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Major companies involved in the mucopolysaccharidosis pipeline analysis include JCR Pharmaceuticals Co., Ltd., Generium, and others.
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Leading drugs currently in the pipeline include JR-141, GNR-055, DNL126, and others.
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Advancing gene therapies, expanding enzyme replacement candidates, and increasing regulatory designations are poised to accelerate clinical progress and attract greater investment, thereby strengthening overall pipeline growth in the coming years.
Report Coverage
The Mucopolysaccharidosis Pipeline Analysis Report by Expert Market Research gives comprehensive insights into mucopolysaccharidosis therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for mucopolysaccharidosis. The mucopolysaccharidosis report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The mucopolysaccharidosis pipeline landscape will include an analysis based on efficacy and safety measures outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with mucopolysaccharidosis treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to mucopolysaccharidosis.
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Mucopolysaccharidosis Pipeline Outlook
Mucopolysaccharidosis is a group of rare inherited metabolic disorders caused by enzyme deficiencies that hinder the breakdown of glycosaminoglycans, resulting in their harmful buildup within cells. This accumulation gradually damages the organs, skeletal system, and central nervous system, leading to progressive physical and neurological complications that significantly impact overall health and lifespan.
Mucopolysaccharidosis treatments include enzyme replacement therapy, gene therapy, and supportive interventions to slow disease progression and enhance patient quality of life. In October 2024, JCR Pharmaceuticals strengthened the mucopolysaccharidosis drug pipeline by initiating first patient dosing in a Phase I trial of JR-441, a recombinant enzyme engineered to cross the blood–brain barrier and specifically target MPS IIIA.
Mucopolysaccharidosis Epidemiology
Mucopolysaccharidosis epidemiology varies significantly, shaping focused drug development. According to Assel Tulebayeva et al., 2025, incidence ranges from 1.35 to 16.9 per 100,000 live births globally. Mucopolysaccharidosis II shows the highest prevalence in East Asia, accounting for up to 54.6% of cases, while mucopolysaccharidosis I is most common in Canada and Northern Europe. Mucopolysaccharidosis VI is highly prevalent in Saudi Arabia and India, whereas mucopolysaccharidosis VII remains ultra-rare, with a peak birth prevalence of 0.23 per 100,000 in Mexico. According to available literature, several regions still lack data, highlighting unmet needs that impact drug pipeline planning.
Mucopolysaccharidosis – Pipeline Therapeutic Assessment
This section of the report covers the analysis of mucopolysaccharidosis drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
- Late-Stage Products (Phase 3 and Phase 4)
- Mid-Stage Products (Phase 2)
- Early-Stage Products (Phase I)
- Preclinical and Discovery Stage Products
By Drug Class
The mucopolysaccharidosis pipeline analysis report covers 50+ drug analyses based on drug classes:
- Small Molecules
- Gene Therapies
- Cell-based Therapies
- Biologics
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration.
- Oral
- Parenteral
- Others
Mucopolysaccharidosis Pipeline Assessment Segmentation, By Phases
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II, with 42%, covers a major share of the total mucopolysaccharidosis clinical trials, highlighting significant clinical activity and potential market growth. Phases I and III each hold 27%, reflecting balanced early and late-stage development. These developments are expected to positively impact innovation and treatment availability in the mucopolysaccharidosis market.
Mucopolysaccharidosis Pipeline Assessment Segmentation, By Drug Classes
The drug molecule categories covered under the mucopolysaccharidosis pipeline analysis include small-molecules, gene therapies, cell-based therapies, and biologics. The mucopolysaccharidosis report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for mucopolysaccharidosis. Enzyme replacement therapies are emerging as promising treatment options in the Mucopolysaccharidosis market. For instance, Tividenofusp alfa, developed by Denali Therapeutics Inc., is a central nervous system-penetrant therapy under investigation for Mucopolysaccharidosis Type II. It combines the iduronate-2-sulfatase enzyme with Denali’s TransportVehicle™ platform to target both cognitive and physical symptoms, aiming to improve patient outcomes.
Mucopolysaccharidosis Clinical Trials – Key Players
The EMR report for the mucopolysaccharidosis pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed mucopolysaccharidosis therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in mucopolysaccharidosis clinical trials:
- JCR Pharmaceuticals Co., Ltd.
- Generium
- Denali Therapeutics Inc.
- GC Biopharma Corp.
- Immusoft of CA, Inc.
- Orchard Therapeutics
- Regenxbio Inc.
- Ultragenyx Pharmaceutical Inc.
- Abeona Therapeutics, Inc.
- Takeda
- QED Therapeutics
Mucopolysaccharidosis – Emerging Drugs Profile
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for mucopolysaccharidosis. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of mucopolysaccharidosis drug candidates.
Drug: JR-141
JR-141 is being evaluated in this Phase III STARLIGHT study sponsored by JCR Pharmaceuticals. This trial is examining the safety and efficacy of pabinafusp alfa, a recombinant fusion protein combining an anti-transferrin receptor antibody with iduronate-2-sulfatase. The drug uses J-Brain Cargo® technology to continuously penetrate the blood–brain barrier and deliver the missing enzyme for MPS II. The study aims to determine whether JR-141 improves central nervous system symptoms while maintaining systemic therapeutic activity. Administered intravenously, the therapy aims to reduce CNS substrate accumulation, with final study completion expected in 2027.
Drug: GNR-055
GNR-055 is an investigational recombinant Iduronate-2-Sulfatase enzyme replacement therapy being developed by AO Generium for mucopolysaccharidosis type II. The drug is designed to penetrate the blood–brain barrier, reducing harmful glycosaminoglycan accumulation and potentially preventing neurocognitive decline. Administered intravenously, it aims to improve systemic and neurological outcomes. The ongoing Phase 2/3 study is examining its safety, pharmacokinetics, pharmacodynamics, and efficacy across multiple age groups, with final study completion expected in 2028.
Drug: DNL126
DNL126 (ETV:SGSH) is an investigational IV enzyme-replacement therapy sponsored by Denali Therapeutics Inc. and is being evaluated in an ongoing Phase 1/2 study for pediatric Mucopolysaccharidosis Type IIIA. The study is examining its safety, tolerability, PK, PD, and early clinical efficacy. DNL126 is designed to cross the blood–brain barrier using Enzyme Transport Vehicle technology, delivering SGSH directly into the brain and body tissues to reduce behavioral, cognitive, and physical symptoms of MPS IIIA. The study completion is estimated for 2028.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
Key Questions Answered in the Mucopolysaccharidosis Pipeline Insight Report
- Which companies/institutions are leading the mucopolysaccharidosis drug development?
- Which company is leading the mucopolysaccharidosis pipeline development activities?
- What is the current mucopolysaccharidosis commercial assessment?
- What are the opportunities and challenges present in the mucopolysaccharidosis pipeline landscape?
- What is the efficacy and safety profile of mucopolysaccharidosis pipeline drugs?
- Which company is conducting major trials for mucopolysaccharidosis drugs?
- Which companies/institutions are involved in mucopolysaccharidosis collaborations aimed at providing enhanced therapeutic alternatives for patients?
- What are the geographies covered for clinical trials in mucopolysaccharidosis?
Reasons To Buy This Report
The Mucopolysaccharidosis Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for mucopolysaccharidosis. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into mucopolysaccharidosis collaborations, regulatory environments, and potential growth opportunities.
Related Reports
Mucopolysaccharidosis Epidemiology Forecast
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Report Summary
Explore our key highlights of the report and gain a concise overview of key findings, trends, and actionable insights that will empower your strategic decisions.
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