Mucopolysaccharidosis III Pipeline 2025: Pioneering Clinical Progress by 8+ Global Leaders - DelveInsight | Featuring Ultragenyx Pharmaceutical Inc, JCR Pharmaceuticals Co., Ltd., GC Biopharma Corp, D

DelveInsight's, "Mucopolysaccharidosis III - Pipeline Insight, 2025" report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Mucopolysaccharidosis III pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Mucopolysaccharidosis III (MPS III) is emerging as a significant global health concern, closely linked to serious comorbidities such as cardiovascular disease, diabetes, and certain cancers-driving the urgent need for safer and more effective therapies. According to DelveInsight, the MPS III pipeline currently includes 8+ pharmaceutical and biotech companies advancing over 10 therapeutic candidates across multiple stages of clinical and preclinical development. This reflects strong innovation and commitment toward tackling one of the most challenging rare diseases worldwide.

DelveInsight's "Mucopolysaccharidosis III Pipeline Insight 2025" report offers an in-depth and strategic analysis of the current R&D landscape. It highlights clinical trial advancements, novel mechanisms of action, emerging therapies, competitive dynamics, and major company initiatives. The report acts as a vital resource for stakeholders-including researchers, investors, and policymakers-providing insights into the evolving MPS III therapeutics market and the transformative breakthroughs shaping its future direction.

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During the ERS Congress 2025 in September 2025, several biotechnology and pharmaceutical companies shared notable advancements in pulmonary and cardiopulmonary research:

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In September 2025, Diagonal Therapeutics presented new preclinical data highlighting the potential of its lead clustering antibody, DIAG723, as a disease-modifying therapy for pulmonary arterial hypertension (PAH).

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In September 2025, a study revealed that babies raised with dogs may have a reduced risk of developing childhood asthma.

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In September 2025, Savara shared data from the Phase 3 IMPALA-2 trial of Molgramostim Inhalation Solution for patients with autoimmune pulmonary alveolar proteinosis (aPAP).

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In September 2025, AllRock Bio, Inc. reported encouraging Phase 1 results for ROC-101, a first-in-class oral pan-ROCK inhibitor for cardiopulmonary and fibrotic diseases.

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In September 2025, Gossamer Bio, Inc., in collaboration with Chiesi Group, announced five scientific presentations on seralutinib for PAH and PH-ILD.

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In September 2025, Boehringer Ingelheim presented pooled analyses from the Phase III FIBRONEER Trademark program on nerandomilast, an investigational oral PDE4B inhibitor, showing a nominally significant reduction in mortality risk across IPF and PPF.

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In September 2025, Kaia Health launched a pilot program of Kaia Breathe, a digital pulmonary rehabilitation solution for COPD patients, in partnership with MedImprove.

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In September 2025, aTyr Pharma, Inc. shared additional findings from the Phase 3 EFZO-FIT Trademark study of efzofitimod in 268 patients with pulmonary sarcoidosis, a key form of interstitial lung disease.

Key Takeaways from the Mucopolysaccharidosis III Pipeline Report

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DelveInsight's Mucopolysaccharidosis III pipeline report highlights a dynamic landscape with 8+ active companies developing over 10 therapeutic candidates for the treatment of MPS III.

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Ultragenyx Pharmaceutical Inc. has submitted a Biologics License Application (BLA) to the FDA for UX111 (ABO-102), an adeno-associated virus (AAV) vector-based gene therapy for MPS IIIA. The FDA has accepted the application under Priority Review, setting a PDUFA action date of August 18, 2025. This submission is supported by results from the Transpher A clinical trial, which showed a meaningful reduction in cerebrospinal fluid heparan sulfate levels-a critical biomarker of disease activity.

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Leading companies such as GlaxoSmithKline, Medytox, Theravidam, Candesant Biomedical, and others are also advancing novel therapies to enhance the treatment landscape for MPS III.

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Promising pipeline candidates currently under investigation include Sofpironium bromide, ET-01, DMT410, and several others, spanning various stages of development.

Mucopolysaccharidosis III Overview:

Mucopolysaccharidosis III (MPS III), also known as Sanfilippo syndrome, is a rare inherited lysosomal storage disorder transmitted in an autosomal recessive manner. It results from enzyme deficiencies that hinder the degradation of heparan sulfate, leading to its accumulation within lysosomes and subsequent multi-organ damage. The condition is classified into four subtypes-A, B, C, and D-each linked to a deficiency of a specific enzyme.

The disease primarily manifests as progressive neurodegeneration beginning in early childhood, with hallmark symptoms such as cognitive decline, motor function loss, and behavioral disturbances. Life expectancy is generally limited to the second or third decade, though milder and less common variants may allow survival into later adulthood. Subtype A is most frequently seen in Northern Europe, whereas subtype B is more prevalent in Southern Europe.

Clinical features of MPS III include coarse facial characteristics (thick eyebrows, prominent eyelashes, coarse/dry hair), skeletal abnormalities causing growth delays and joint issues, hepatosplenomegaly, macrocephaly, and hearing impairment. However, the most defining aspect is the gradual decline of the central nervous system (CNS), resulting in intellectual disability and hyperactivity. Children often appear normal at birth but begin showing signs-such as speech delay, learning difficulties, and behavioral issues-between ages 1 and 3. By ages 3 to 5, more severe behavioral problems, including hyperactivity, sleep disorders, and aggression, typically arise. Over the next 5-10 years, affected individuals experience worsening neurological and physical decline, eventually losing the ability to speak, walk, and swallow. In advanced stages, many enter a vegetative state, with death occurring anywhere from adolescence to, in rare cases, the sixth decade of life.

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Mucopolysaccharidosis III Pipeline Analysis

The Mucopolysaccharidosis III pipeline insights report 2025, provides insights into:

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Provides comprehensive insights into key companies developing therapies in the Mucopolysaccharidosis III Market.

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Categorizes Mucopolysaccharidosis III therapeutic companies by development stage: early, mid, and late-stage.

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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

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Reviews emerging Mucopolysaccharidosis III drugs under development based on:

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Stage of development

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Mucopolysaccharidosis III Route of administration

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Target receptor

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Monotherapy vs. combination therapy

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Mucopolysaccharidosis III Mechanism of action

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Molecular type

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Offers detailed analysis of:

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Company-to-company and company-academia collaborations

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Mucopolysaccharidosis III Licensing agreements

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Funding and investment activities supporting future Mucopolysaccharidosis III market advancement.

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Mucopolysaccharidosis III Emerging Drugs

UX111: Ultragenyx Pharmaceutical Inc

UX111 (rebisufligene etisparvovec) is an investigational in vivo gene therapy under development for Mucopolysaccharidosis III type A (MPS IIIA). Delivered as a single intravenous infusion, it employs a self-complementary AAV9 vector to introduce a functional copy of the SGSH gene into patient cells. By addressing the underlying cause-sulfamidase enzyme deficiency-the therapy aims to prevent the pathological buildup of heparan sulfate in the brain, which drives progressive neurodegeneration and cellular damage.

UX111 has received multiple regulatory designations in the U.S., including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Rare Pediatric Disease, and Orphan Drug status, along with PRIME and Orphan Drug designations in the EU. It is currently in the preregistration stage for MPS III.

Trehalose: Seelos Therapeutics

Trehalose (SLS-005), being developed by Seelos Therapeutics, is a potential therapy for Mucopolysaccharidosis III (MPS III). This small disaccharide has the unique ability to cross the blood-brain barrier and stimulate autophagy, a key cellular mechanism responsible for clearing misfolded proteins and accumulated waste. By boosting autophagy, Trehalose is designed to limit neurodegeneration and support improved neurological outcomes in patients with MPS III. The therapy has been granted Orphan Drug Designation by the FDA and is currently in Phase II clinical trials for this indication.

DNL126: Denali Therapeutics Inc.

DNL126 is an experimental enzyme replacement therapy designed to treat Mucopolysaccharidosis III type A (MPS IIIA). Utilizing Denali's proprietary Enzyme Transport Vehicle (ETV) technology, it crosses the blood-brain barrier through receptor-mediated transcytosis, delivering the SGSH enzyme directly to the central nervous system. By replenishing the deficient enzyme, DNL126 aims to reduce harmful heparan sulfate accumulation in the brain, potentially alleviating neurological symptoms and enhancing patient outcomes. This recombinant SGSH enzyme is engineered to target both neurological and systemic effects of MPS IIIA. The therapy is currently undergoing Phase I/II clinical trials.

Mucopolysaccharidosis III Pipeline Therapeutic Assessment

Mucopolysaccharidosis III Assessment by Product Type

- Mono

- Combination

- Mono/Combination

Mucopolysaccharidosis III By Stage

- Late-stage products (Phase III)

- Mid-stage products (Phase II)

- Early-stage product (Phase I) along with the details of

- Pre-clinical and Discovery stage candidates

- Discontinued & Inactive candidates

Mucopolysaccharidosis III Assessment by Route of Administration

- Oral

- Parenteral

- Intravenous

- Subcutaneous

- Topical

Mucopolysaccharidosis III Assessment by Molecule Type

- Recombinant fusion proteins

- Small molecule

- Monoclonal antibody

- Peptide

- Polymer

- Gene therapy

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Table of Contents

1. Report Introduction

2. Executive Summary

3. Mucopolysaccharidosis III Current Treatment Patterns

4. Mucopolysaccharidosis III - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

6. Mucopolysaccharidosis III Late-Stage Products (Phase-III)

7. Mucopolysaccharidosis III Mid-Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Mucopolysaccharidosis III Discontinued Products

13. Mucopolysaccharidosis III Product Profiles

14. Mucopolysaccharidosis III Key Companies

15. Mucopolysaccharidosis III Key Products

16. Dormant and Discontinued Products

17. Mucopolysaccharidosis III Unmet Needs

18. Mucopolysaccharidosis III Future Perspectives

19. Mucopolysaccharidosis III Analyst Review

20. Appendix

21. Report Methodology

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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