Bioengineered extracellular vesicles to help people with inherited metabolic diseases like Gaucher
Official title: Bioengineered extracellular vesicles as a therapeutic platform for inborn errors of metabolism
Funded at Brigham and Women's Hospital · NIH-11452248 · Data updated 2026-11-12
A researcher at Brigham and Women’s Hospital is developing a new cell-sized delivery system to bring enzyme therapy directly to where it’s needed in patients with Gaucher disease and related conditions.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $171,720 |
| NIH project number | 1K08HD120691-01A1 |
| Project ID | NIH-11452248 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include individuals with Gaucher disease or related inborn errors of metabolism who may benefit from improved enzyme delivery to the central nervous system.
✕Less likely to help
Patients with conditions unrelated to Gaucher disease or those without potential CNS involvement may not receive direct benefit from this specific research.
What this research studies
This project aims to create engineered extracellular vesicles that carry the enzyme glucocerebrosidase to improve treatment of Gaucher disease and other inherited metabolic disorders. It combines active loading of the enzyme into vesicles, targeted delivery to the brain, and highly sensitive methods to measure success. The work focuses on making a safer, more effective enzyme replacement approach that could reduce the need for frequent infusions. The study is designed to be explored in animal models first, with the goal of moving toward human studies if results are favorable.
Why it matters
Potential benefit: If successful, this approach could provide more effective and less burdensome treatment for patients with Gaucher disease and similar conditions, potentially improving CNS outcomes and reducing infusion frequency.
How similar studies have gone: Previous work in EV-based enzyme delivery shows promise, but applying active loading and CNS targeting is an innovative step with mixed early results.
Where this research is happening
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Melkonian, Arek — Brigham and Women's Hospital
- Study coordinator: Melkonian, Arek
Related research
Other NIH-funded projects on the same topics.
-
Targeted therapies for glioblastoma in adults and kids
A large NIH program is exploring new brain cancer therapies, including drugs that reach the brain and work against glioblastoma in both children and adults.
-
Statins to Prevent Cancer-Associated Blood Clots in Cancer Patients
A large study will test whether taking rosuvastatin every day for a year can lower the chance of dangerous blood clots in people with cancer, without raising the risk of bleeding.
-
Preterm infant body composition and early-life health after NICU stay
This grant studies how body composition in very preterm babies relates to later brain development and risk of obesity or diabetes, with findings meant to improve care from NICU through early childhood.
-
Rapid Emergency Skills for Prehospital Optimization and Delivery (RESPOND) for Guatemala
A lightweight, AI-enabled prehospital training and triage program helps volunteer firefighters in Guatemala deliver faster, better care to trauma patients.
-
Rapid Emergency Skills for Prehospital Optimization and Notification in Guatemala
A mobile, AI-powered training and triage program helps volunteer emergency responders in Guatemala provide faster, safer trauma care and notify hospitals more quickly.
-
Understanding antibiotic allergy to improve care for patients
Scientists are testing new ideas to understand why some people develop severe allergic reactions to antibiotics and how to prevent or treat them.
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.