Investigating the safety of oligonucleotide treatments for neurological disorders
Safe-OPTION: Optical Physiology To Interrogate Oligonucleotide Neurotoxicity
This study is working on a new way to check if certain treatments for severe brain disorders, like Angelman syndrome, are safe before they are tested on animals, using smart computer techniques to help find any possible harmful effects.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Quiver Bioscience INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-11068749 on NIH RePORTER |
What this research studies
This research focuses on developing a new platform to assess the safety of antisense oligonucleotides (ASOs), which are treatments for severe neurological disorders like Angelman syndrome. By using advanced machine learning techniques, the project aims to predict potential neurotoxic effects of ASOs before they are tested in animal models. This approach seeks to reduce the need for costly and time-consuming in vivo studies, ultimately leading to safer and more effective treatments for patients. The research will explore how ASOs interact with the central nervous system and identify those that may cause harmful side effects.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with neurological disorders such as Angelman syndrome, Dravet syndrome, ALS, or Huntington’s Disease.
Not a fit: Patients with conditions unrelated to neurological disorders or those not eligible for antisense oligonucleotide therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer oligonucleotide therapies for patients with severe neurological disorders.
How similar studies have performed: Previous research has shown promise in using machine learning to predict drug toxicity, indicating potential success for this novel approach.
Where this research is happening
Cambridge, United States
- Quiver Bioscience INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Simao, Fabricio — Quiver Bioscience INC.
- Study coordinator: Simao, Fabricio
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.