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 Sanfilippo Syndrome pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Sanfilippo Syndrome Pipeline Report

• In May 2025, JCR Pharmaceuticals Co., Ltd. announced a Phase I/ II study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.
• In May 2025, Denali Therapeutics Inc. announced a phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). The core study period is 25 weeks (approximately 6 months) and is followed by a 72-week (approximately 18 month) open-label extension (OLE). Participants with MPS IIIA will be enrolled in two planned cohorts, and additional participants with MPS IIIA may be enrolled in three optional cohorts.
• DelveInsight’s Sanfilippo Syndrome Pipeline report depicts a robust space with 8+ active players working to develop 10+ pipeline therapies for Sanfilippo Syndrome treatment.
• The leading Sanfilippo Syndrome Companies such as Ultragenyx Pharmaceutical Inc, JCR Pharmaceuticals Co., Ltd., GC Biopharma Corp, Denali Therapeutics Inc., Orchard Therapeutics plc, Seelos Therapeutics and others.
• Promising Sanfilippo Syndrome Pipeline Therapies such as JR-446, anakinra, SAF-301, UX111, SOBI003, DNL126, SBC-103, AX 250 and others.

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Sanfilippo Syndrome Emerging Drugs Profile

• UX111: Ultragenyx Pharmaceutical Inc.

UX111 (rebisufligene etisparvovec) is an investigational novel in vivo gene therapy under evaluation for Sanfilippo syndrome type A (MPS IIIA). UX111 is designed to be dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells. The therapy is designed to address the underlying sulfamidase enzyme deficiency responsible for abnormal accumulation of heparan sulfate, a glycosaminoglycan, in the brain that results in progressive cell damage and neurodegeneration. The UX111 program has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., and PRIME and Orphan Drug designations in the EU. In December 2024, Ultragenyx Pharmaceutical announced the submission of a Biologics License Application to the US Food and Drug Administration seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A.

• OTL-201: Orchard Therapeutics

OTL-201 is an ex vivo autologous gene therapy being developed for the treatment of Sanfilippo syndrome. It uses a modified virus to insert a functional copy of the SGSH gene into a patient’s cells. OTL-201 has received rare pediatric disease designation from the FDA and is currently being evaluated in an ongoing proof-of-concept clinical trial. OTL-201 is an investigational therapy and has not been approved by any regulatory agency or health authority.

• DNL126: Denali Therapeutics Inc.

DNL126 is an investigational enzyme replacement therapy developed for the treatment of Sanfilippo Syndrome type A (MPS IIIA). Utilizing Denali’s proprietary Enzyme Transport Vehicle (ETV) technology, DNL126 is designed to effectively cross the blood-brain barrier through receptor-mediated transcytosis, allowing for targeted delivery of SGSH to the central nervous system. By replenishing the deficient enzyme, DNL126 aims to reduce the toxic buildup of heparan sulfate in brain tissues, potentially alleviating neurological symptoms and improving overall outcomes for patients. DNL126 is a recombinant SGSH enzyme engineered to cross the blood-brain barrier, replace the SGSH enzyme and treat neuropathic and systemic forms of the Sanfilippo syndrome A. Currently, the drug is in Phase I/II stage of its clinical trial for the treatment of Sanfilippo Syndrome.

The Sanfilippo Syndrome Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Sanfilippo Syndrome with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Sanfilippo Syndrome Treatment.
• Sanfilippo Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Sanfilippo Syndrome Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Sanfilippo Syndrome market

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Sanfilippo Syndrome Companies

Ultragenyx Pharmaceutical Inc, JCR Pharmaceuticals Co., Ltd., GC Biopharma Corp, Denali Therapeutics Inc., Orchard Therapeutics plc, Seelos Therapeutics and others.

Sanfilippo Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Sanfilippo Syndrome Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Unveil the future of Sanfilippo Syndrome Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight’s expert analysis @ Sanfilippo Syndrome Market Drivers and Barriers

Scope of the Sanfilippo Syndrome Pipeline Report

• Coverage- Global
• Sanfilippo Syndrome Companies- Ultragenyx Pharmaceutical Inc., JCR Pharmaceuticals Co., Ltd., GC Biopharma Corp, Denali Therapeutics Inc., Orchard Therapeutics plc, Seelos Therapeutics and others.
• Sanfilippo Syndrome Pipeline Therapies- JR-446, anakinra, SAF-301, UX111, SOBI003, DNL126, SBC-103, AX 250 and others.
• Sanfilippo Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Sanfilippo Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Get the latest on Sanfilippo Syndrome Therapies and clinical trials. Download DelveInsight’s in-depth pipeline report today! @ Sanfilippo Syndrome Companies, Key Products and Unmet Needs

Table of Contents

1. Introduction
2. Executive Summary
3. Sanfilippo Syndrome: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Sanfilippo Syndrome– DelveInsight’s Analytical Perspective
7. Late Stage Products (Preregistration)
8. UX111: Ultragenyx Pharmaceutical Inc
9. Drug profiles in the detailed report…..
10. Mid-Stage Products (Phase II)
11. Drug Name: Company Name
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. DNL126: Denali Therapeutics Inc.
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug Name: Company Name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Sanfilippo Syndrome Key Companies
21. Sanfilippo Syndrome Key Products
22. Sanfilippo Syndrome- Unmet Needs
23. Sanfilippo Syndrome- Market Drivers and Barriers
24. Sanfilippo Syndrome- Future Perspectives and Conclusion
25. Sanfilippo Syndrome Analyst Views
26. Sanfilippo Syndrome Key Companies
27. Appendix

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