Sanfilippo Syndrome Pipeline 2025: Pioneering Clinical Developments by 8+ Global Leaders - DelveInsight | Featuring Ultragenyx Pharmaceutical Inc, JCR Pharmaceuticals Co., Ltd., GC Biopharma Corp, Den

DelveInsight's, "Sanfilippo Syndrome - Pipeline Insight, 2025" report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Sanfilippo Syndrome 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.
With Sanfilippo syndrome becoming increasingly prevalent worldwide and contributing to comorbidities such as diabetes, cardiovascular disease, and certain cancers, there is a growing need for safer and more effective treatment options. DelveInsight reports that the Sanfilippo syndrome pipeline includes 8+ pharmaceutical and biotech companies actively developing over 10 therapeutic candidates. These treatments are at various stages of clinical and preclinical development, reflecting substantial innovation and commitment to addressing this significant public health challenge.

DelveInsight's "Sanfilippo Syndrome Pipeline Insight 2025" offers a comprehensive and strategic analysis of the R&D landscape. The report examines clinical trial progress, emerging therapies, mechanisms of action, competitive dynamics, and key company initiatives. It serves as a vital resource for stakeholders-researchers, healthcare investors, and decision-makers-seeking insights into the evolving Sanfilippo syndrome therapeutics market and the advancements shaping its future.

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Key Takeaways from the Sanfilippo syndrome Pipeline Report

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DelveInsight's Sanfilippo syndrome pipeline report highlights a dynamic landscape with over 8 active companies developing more than 10 therapeutic candidates for Sanfilippo syndrome.

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In February 2025, the U.S. Food and Drug Administration (FDA) accepted Ultragenyx Pharmaceutical's Biologics License Application (BLA) for UX111 (ABO-102), an AAV-based gene therapy for Sanfilippo syndrome Type A. The FDA granted Priority Review for this application, shortening the review timeline to six months, with a Prescription Drug User Fee Act (PDUFA) target date of August 18, 2025.

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Denali Therapeutics' DNL126, an investigational enzyme replacement therapy engineered to cross the blood-brain barrier, was selected for the FDA's Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program, designed to accelerate the development of novel rare disease treatments.

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Key companies such as Ultragenyx Pharmaceutical Inc., JCR Pharmaceuticals Co., Ltd., GC Biopharma Corp, Denali Therapeutics Inc., Orchard Therapeutics plc, Seelos Therapeutics, and others are actively advancing new therapies to enhance the treatment landscape for Sanfilippo syndrome.

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Promising pipeline therapies currently in development include UX111, OTL-201, DNL126, and additional candidates.

Sanfilippo syndrome Overview:

Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is a rare genetic disorder caused by deficiencies in enzymes responsible for breaking down heparan sulfate, resulting in its accumulation within cells. The condition is divided into four subtypes-A, B, C, and D-each associated with a deficiency in a specific enzyme. The disease primarily leads to severe neurodegeneration beginning in early childhood, manifesting as cognitive decline, motor impairments, and behavioral challenges. Life expectancy generally ranges from the teenage years to the 30s, although milder cases may survive longer. Subtype A is more common in Northern Europe, whereas subtype B is more prevalent in Southern Europe.

Physical manifestations can include coarse facial features, skeletal abnormalities, enlarged liver and spleen, macrocephaly, and hearing loss. Early development often appears normal, but between ages 1 and 3, cognitive delays, speech difficulties, and behavioral issues such as aggression emerge. Behavioral problems typically intensify between ages 3 and 5, often accompanied by hyperactivity and sleep disturbances. As the disease progresses, patients gradually lose the ability to speak, move, and swallow, ultimately entering a vegetative state and succumbing to the condition during adolescence or later, depending on disease severity.

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Sanfilippo syndrome Pipeline Analysis

The Sanfilippo syndrome pipeline insights report 2025, provides insights into:

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Provides comprehensive insights into key companies developing therapies in the Sanfilippo syndrome Market.

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Categorizes Sanfilippo syndrome 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 Sanfilippo syndrome drugs under development based on:

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Stage of development

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Sanfilippo syndrome Route of administration

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Target receptor

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Monotherapy vs. combination therapy

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Sanfilippo syndrome 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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Sanfilippo syndrome Licensing agreements

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Funding and investment activities supporting future Sanfilippo syndrome market advancement.

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Sanfilippo syndrome Emerging Drugs

OTL-201: Orchard Therapeutics

OTL-201 is an ex vivo autologous gene therapy under development for treating Sanfilippo syndrome. The therapy employs a modified viral vector to deliver a functional copy of the SGSH gene into a patient's cells. It has been granted Rare Pediatric Disease Designation by the FDA and is currently being assessed in an ongoing proof-of-concept clinical trial. OTL-201 remains investigational and has not yet received approval from any regulatory or health authority.

DNL126: Denali Therapeutics Inc.

DNL126 is an investigational enzyme replacement therapy being developed for Sanfilippo Syndrome type A (MPS IIIA). Utilizing Denali's proprietary Enzyme Transport Vehicle (ETV) technology, it crosses the blood-brain barrier through receptor-mediated transcytosis to deliver the SGSH enzyme directly to the central nervous system. By replenishing the deficient enzyme, DNL126 aims to reduce the buildup of heparan sulfate in the brain, potentially alleviating neurological symptoms and improving patient outcomes. This recombinant SGSH enzyme is designed to target both neurological and systemic manifestations of Sanfilippo Syndrome A. DNL126 is currently in Phase I/II clinical trials.

Sanfilippo syndrome Pipeline Therapeutic Assessment

Sanfilippo syndrome Assessment by Product Type

- Mono

- Combination

- Mono/Combination

Sanfilippo syndrome 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

Sanfilippo syndrome Assessment by Route of Administration

- Oral

- Parenteral

- Intravenous

- Subcutaneous

- Topical

Sanfilippo syndrome 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. Sanfilippo syndrome Current Treatment Patterns

8. Sanfilippo syndrome - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

10. Sanfilippo syndrome Late-Stage Products (Phase-III)

7. Sanfilippo syndrome 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. Sanfilippo syndrome Discontinued Products

13. Sanfilippo syndrome Product Profiles

18. Sanfilippo syndrome Key Companies

15. Sanfilippo syndrome Key Products

110. Dormant and Discontinued Products

17. Sanfilippo syndrome Unmet Needs

18. Sanfilippo syndrome Future Perspectives

19. Sanfilippo syndrome 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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