How ASO drugs behave and may cause side effects in people using a human liver-kidney model
Official title: Investigating the Impact of Metabolic Disease States on the Pharmacodynamics and Toxicity of FDA-Approved Antisense Oligonucleotides Using an Integrated Liver-Kidney Model
Funded at Javelin Biotech · NIH-11316401 · Data updated 2026-11-12
This grant tests how FDA-approved antisense oligonucleotides travel through and affect human liver and kidney tissues using a combined microphysiological system.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Javelin Biotech NIH-funded |
| Lab location | 1 site (Billerica, UNITED STATES) |
| Project start | July 1, 2026 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $349,677 |
| NIH project number | 1R43TR005374-01A1 |
| Project ID | NIH-11316401 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are patients who may receive ASO therapies or have conditions that could benefit from ASO treatments in the future.
✕Less likely to help
Patients not receiving ASO therapies or not involved in translational studies may not directly benefit.
What this research studies
If you have a disease that could be treated with antisense oligonucleotides, this project uses a human liver-kidney chip to study how these drugs are absorbed, processed, and cleared, and to identify potential liver or kidney toxicity early. The study combines advanced tissue models to better mimic human responses and looks at four approved ASOs to understand safety and performance. The goal is to reduce unexpected ADRs and improve therapeutic outcomes by understanding ADME and toxicity in a human-relevant system. Results could guide safer dosing and design of future ASO medicines.
Why it matters
Potential benefit: It could lead to safer ASO therapies with fewer adverse reactions and more predictable dosing in patients.
How similar studies have gone: Previous non-human models showed limitations; this study aims to provide a more human-relevant understanding of ASO ADME and toxicity.
Where this research is happening
-
Javelin Biotech
Billerica, United States Active
Researchers
- Principal investigator: Cirit, Murat — Javelin Biotech
- Study coordinator: Cirit, Murat
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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.