Designing new methods to deliver mRNA therapies for cystic fibrosis
Official title: Computationally-Inspired Design of Non-Viral Gene Delivery Vehicles for mRNA-Based Cystic Fibrosis Therapies
Funded at Nanite INC. · NIH-10760605 · Data updated 2026-11-12
This study is working on new ways to help deliver mRNA treatments for cystic fibrosis more effectively, making it easier for these therapies to reach the lungs and improve care for people living with this condition.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Nanite INC. NIH-funded |
| Lab location | 1 site (Watertown, United States) |
| Project start | August 10, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $299,995 |
| NIH project number | 1R43HL170835-01 |
| Project ID | NIH-10760605 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those who have not responded well to existing therapies.
✕Less likely to help
Patients with cystic fibrosis who are already effectively managed with current therapies may not see additional benefits from this research.
What this research studies
This research focuses on developing innovative non-viral gene delivery vehicles specifically for mRNA-based therapies aimed at treating cystic fibrosis (CF). By addressing the challenges of delivering complex molecules like mRNA through the thick mucus that characterizes CF, the project seeks to enhance the effectiveness of gene therapies. The approach involves computational design to create delivery systems that can effectively penetrate the mucus layer and reach the target cells in the lungs. This could potentially lead to improved treatment options for patients suffering from CF.
Why it matters
Potential benefit: If successful, this research could provide more effective gene therapy options for cystic fibrosis patients, potentially improving their lung function and overall health.
How similar studies have gone: While gene therapy for cystic fibrosis is a growing field, this specific approach using non-viral delivery systems is relatively novel and has not been extensively tested.
Where this research is happening
-
Nanite INC.
Watertown, United States Active
Researchers
- Principal investigator: Murthy, Shashi — Nanite INC.
- Study coordinator: Murthy, Shashi
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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.