Gene therapy to deliver a replacement enzyme for Sanfilippo A syndrome
Official title: Gene Therapy that Systemically Produces Brain-penetrating Replacement Enzyme for MPS IIIA (Sanfilippo A Syndrome)
Funded at Biostrategies, Lc · NIH-10930137 · Data updated 2026-11-12
This study is testing a new gene therapy for people with Sanfilippo A syndrome that aims to deliver a helpful enzyme to the brain and other parts of the body without needing any invasive procedures, with the hope of easing symptoms and improving daily life.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Biostrategies, Lc NIH-funded |
| Lab location | 1 site (State University, United States) |
| Project start | September 15, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $1,498,067 |
| NIH project number | 5R44NS134453-02 |
| Project ID | NIH-10930137 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with mucopolysaccharidosis type IIIA, particularly children who are experiencing early symptoms of the disease.
✕Less likely to help
Patients with other types of mucopolysaccharidosis or those who do not have a confirmed diagnosis of MPS IIIA may not benefit from this research.
What this research studies
This research focuses on developing a novel gene therapy that aims to treat mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) by delivering a functional replacement enzyme across the blood-brain barrier. The approach utilizes an adeno-associated virus (AAV) to achieve systemic therapeutic effects without the need for invasive procedures like intrathecal administration. Patients may benefit from a potential treatment that could alleviate the debilitating symptoms and improve quality of life. The therapy is designed to be effective not only in the central nervous system but also in other body tissues.
Why it matters
Potential benefit: If successful, this research could provide the first clinically effective treatment for Sanfilippo A syndrome, potentially improving patient outcomes and quality of life.
How similar studies have gone: While gene therapy for other conditions has shown promise, this specific approach for MPS IIIA is novel and has not been extensively tested in clinical settings.
Where this research is happening
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Biostrategies, Lc
State University, United States Active
Researchers
- Principal investigator: Pattada, Shivakumar Devaiah — Biostrategies, Lc
- Study coordinator: Pattada, Shivakumar Devaiah
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.