Gene therapy to treat Morquio A syndrome
Cell-based Gene Therapy for MPS IVA
This study is testing a new gene therapy for people with Morquio A, a rare condition that makes it hard for the body to break down certain sugars, and it aims to help improve movement and overall health by fixing the genetic issue causing the problem.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mandalmed, INC. NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-10922052 on NIH RePORTER |
What this research studies
This research focuses on developing a cell-based gene therapy for Mucopolysaccharidosis type IVA (Morquio A), a rare genetic disorder that affects the body's ability to break down certain sugars, leading to severe health issues. The approach aims to correct the genetic defect responsible for the disease by delivering a functional copy of the gene that encodes for the enzyme needed to process these sugars. By using advanced techniques to modify cells, the therapy seeks to improve the function of affected tissues and reduce the need for invasive surgeries and other treatments currently available. Patients may be monitored for improvements in mobility, organ function, and overall quality of life as part of this innovative treatment strategy.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Mucopolysaccharidosis type IVA, particularly those experiencing severe symptoms and complications.
Not a fit: Patients with other types of Mucopolysaccharidosis or those who do not have a confirmed diagnosis of Morquio A may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve the quality of life for patients with Morquio A by reducing the need for surgeries and enhancing physical function.
How similar studies have performed: Previous research in gene therapy for other lysosomal storage diseases has shown promising results, indicating potential for success in this novel approach for Morquio A.
Where this research is happening
San Francisco, United States
- Mandalmed, INC. — San Francisco, United States (Active)
Researchers
- Principal investigator: Mounzih, Khalid — Mandalmed, INC.
- Study coordinator: Mounzih, Khalid
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 below.