Sanfilippo Syndrome Pipeline 2025: Key Companies, MOA, ROA, and Clinical Trials Evaluation by DelveInsight

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Sanfilippo Syndrome pipeline constitutes 8+ key companies continuously working towards developing 10+ Sanfilippo Syndrome treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Sanfilippo Syndrome Pipeline Insight, 2025" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Sanfilippo Syndrome Market.

The Sanfilippo Syndrome Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

Some of the key takeaways from the Sanfilippo Syndrome Pipeline Report: https://www.delveinsight.com/sample-request/sanfilippo-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Sanfilippo Syndrome treatment therapies with a considerable amount of success over the years.
• Sanfilippo Syndrome companies working in the treatment market are Abeona Therapeutics/Ultragenyx, Allievex, JCR Pharmaceuticals, Ultragenyx Pharmaceutical Inc, Orchard Therapeutics, Denali Therapeutics Inc., and others, are developing therapies for the Sanfilippo Syndrome treatment
• Emerging Sanfilippo Syndrome therapies in the different phases of clinical trials are- ABO-102 (UX111), AX 250, JR-441, UX111, OTL-201, DNL126, and others are expected to have a significant impact on the Sanfilippo Syndrome market in the coming years.
• In April 2025, Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical firm dedicated to advancing and commercializing innovative therapies for neurological disorders with high unmet needs, announced a new corporate strategy alongside the acquisition of tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B (MPS IIIB).
• In July 2025, The U.S. Food and Drug Administration (FDA) has issued a complete response letter to Ultragenyx Pharmaceutical, denying approval of its investigational gene therapy UX111. Designed as an adeno-associated virus (AAV) therapy for mucopolysaccharidosis (MPS) type IIIA, also known as Sanfilippo syndrome, the rare metabolic condition impacts up to 1.89 per 100,000 live births. The FDA's decision was based on manufacturing-related issues, particularly concerns with the company's production processes and facilities in California.
• In February 2025, According to new trial results, Ultragenyx Pharmaceutical's investigational gene therapy UX111 has been shown to slow or stop brain damage that impairs communication, cognition, and motor abilities in children with Sanfilippo syndrome type A. Younger children who received the one-time therapy demonstrated improvements in these functional areas compared to untreated peers of the same age, while older patients or those with more advanced disease were able to preserve important skills.
• In September 2023, Medipal Holdings and JCR Pharmaceuticals entered into an agreement for JR-441 on MPS III treatment.
• In July 2023, the PEI (Paul-Ehrlich Institute) accepted the clinical trial application (CTA) for JR-441 to start global Phase I/II clinical trial for the treatment of mucopolysaccharidosis Type III A (Sanfilippo Syndrome).

Sanfilippo Syndrome Overview
Sanfilippo Syndrome, also known as Mucopolysaccharidosis type III (MPS III), is a rare inherited metabolic disorder that primarily affects the brain and nervous system. It occurs due to a deficiency of certain enzymes needed to break down heparan sulfate, a complex sugar molecule. The buildup of this substance leads to progressive brain damage and other health issues. Symptoms often appear in early childhood and include developmental delays, speech difficulties, behavioral problems, sleep disturbances, and gradual loss of motor skills. Over time, the disease worsens, and currently, there is no cure, though supportive care and research into gene therapies are ongoing.

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Emerging Sanfilippo Syndrome Drugs Under Different Phases of Clinical Development Include:
• ABO-102 (UX111): Abeona Therapeutics/Ultragenyx
• AX 250: Allievex
• JR-441: JCR Pharmaceuticals
• UX111: Ultragenyx Pharmaceutical Inc
• OTL-201: Orchard Therapeutics
• DNL126: Denali Therapeutics Inc.

Sanfilippo Syndrome Route of Administration
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
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Sanfilippo Syndrome Molecule Type
Sanfilippo Syndrome Products have been categorized under various Molecule types, such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Sanfilippo Syndrome Pipeline Therapeutics Assessment
• Sanfilippo Syndrome Assessment by Product Type
• Sanfilippo Syndrome By Stage and Product Type
• Sanfilippo Syndrome Assessment by Route of Administration
• Sanfilippo Syndrome By Stage and Route of Administration
• Sanfilippo Syndrome Assessment by Molecule Type
• Sanfilippo Syndrome by Stage and Molecule Type

DelveInsight's Sanfilippo Syndrome Report covers around 10+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Further Sanfilippo Syndrome product details are provided in the report. Download the Sanfilippo Syndrome pipeline report to learn more about the emerging Sanfilippo Syndrome therapies at:
https://www.delveinsight.com/sample-request/sanfilippo-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Some of the key companies in the Sanfilippo Syndrome Therapeutics Market include:
Key companies developing therapies for Sanfilippo Syndrome are - JCR Pharmaceuticals, BioMarin Pharmaceutical, Ultragenyx Pharmaceutical, Denali Therapeutics, Orchard Therapeutics, Seelos Therapeutics, and others.

Sanfilippo Syndrome Pipeline Analysis:
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 key 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.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Sanfilippo Syndrome drugs and therapies-
https://www.delveinsight.com/sample-request/sanfilippo-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Sanfilippo Syndrome Pipeline Market Drivers
• Rising R&D investment in rare genetic disorders, especially in gene therapy and enzyme replacement approaches.
• Growing awareness and advocacy from patient foundations and organizations driving research and funding.
• Advancements in genetic diagnostics enabling earlier identification and intervention.
• Regulatory incentives such as Orphan Drug Designation, Fast Track, and Priority Review encouraging innovation.
• Collaborations and partnerships between biotech firms, academic institutions, and research centers accelerating pipeline progress.

Sanfilippo Syndrome Pipeline Market Barriers
• High cost of therapy development due to the complexity of gene and enzyme replacement therapies.
• Limited patient population making recruitment for clinical trials challenging.
• Scientific and technical hurdles, including delivery of therapies across the blood-brain barrier.
• Regulatory uncertainties in approving novel genetic treatments for ultra-rare conditions.
• Long development timelines before therapies reach commercialization.

Scope of Sanfilippo Syndrome Pipeline Drug Insight
• Coverage: Global
• Key Sanfilippo Syndrome Companies: Abeona Therapeutics/Ultragenyx, Allievex, JCR Pharmaceuticals, Ultragenyx Pharmaceutical Inc, Orchard Therapeutics, Denali Therapeutics Inc., and others
• Key Sanfilippo Syndrome Therapies: ABO-102 (UX111), AX 250, JR-441, UX111, OTL-201, DNL126, and others
• Sanfilippo Syndrome Therapeutic Assessment: Sanfilippo Syndrome current marketed and Sanfilippo Syndrome emerging therapies
• Sanfilippo Syndrome Market Dynamics: Sanfilippo Syndrome market drivers and Sanfilippo Syndrome market barriers

Contact Us:
Gaurav Bora
gbora@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

This release was published on openPR.