RNA-based therapy for a rare brain disorder
Official title: Developing RNA therapeutics for rare neurodevelopmental disorders
Funded at Dayi Therapeutics, INC. · NIH-11251787 · Data updated 2026-11-12
A patient-facing plan to develop targeted RNA medicines that could help people with a rare brain disorder caused by a single gene mutation.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dayi Therapeutics, INC. NIH-funded |
| Lab location | 1 site (New York, UNITED STATES) |
| Project start | May 1, 2023 |
| Project end | December 31, 2026 |
| Funding year | 2026 |
| Award (this year) | $845,187 |
| NIH project number | 5R42HD111138-03 |
| Project ID | NIH-11251787 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with a severe neurodevelopmental disorder caused by a specific single-gene mutation, and who may be eligible for early-phase RNA therapy trials.
✕Less likely to help
Patients with disorders not caused by the targeted single-gene mutation or those who are not candidates for RNA-based therapy may not benefit.
What this research studies
This project aims to create a precision RNA therapy using antisense oligonucleotides to restore the function of a faulty gene in a severe neurodevelopmental condition. The work will be driven by a platform that identifies natural gene-regulating regions to guide treatment. If successful, this approach could modify the disease by correcting gene expression and protein function in the brain. The research is conducted by DAYI Therapeutics, a spin-out from Columbia University, in collaboration with clinicians and researchers. The ultimate goal is to move a first therapy toward testing in people affected by this condition.
Why it matters
Potential benefit: If successful, the therapy could improve brain function and daily living for patients with this devastating developmental disorder.
How similar studies have gone: Previous similar RNA-based approaches have shown promise in early-stage development for other genetic diseases, but this exact therapy is novel for this syndrome.
Where this research is happening
-
Dayi Therapeutics, INC.
New York, United States Active
Researchers
- Principal investigator: Lotti, Francesco — Dayi Therapeutics, INC.
- Study coordinator: Lotti, Francesco
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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.