Safer antisense (ASO) treatments for neurological conditions
Safe-OPTION: Optical Physiology To Interrogate Oligonucleotide Neurotoxicity
This project builds lab and computer tests to find antisense medicines that are less likely to harm the brain for people with disorders like Angelman syndrome, ALS, Huntington’s disease, and Dravet.
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-11332274 on NIH RePORTER |
What this research studies
This project will create lab and computer tests to spot ASOs that can harm the brain before they advance to costly animal or human studies. The team combines machine learning–based ASO design with optical physiology experiments in preclinical models to detect acute and delayed neurobehavioral effects. By identifying patterns linked to neurotoxicity, they plan to filter out risky ASOs early in development. The goal is to speed creation of safer ASO treatments for conditions such as Angelman syndrome, ALS, Huntington’s disease, and Dravet.
Who could benefit from this research
Good fit: People with genetic neurological conditions targeted by ASO therapies (for example Angelman syndrome, ALS, Huntington’s disease, or Dravet syndrome) are the populations who could benefit and might be eligible for related future trials.
Not a fit: People without a relevant neurological genetic disorder or those seeking an immediate treatment option would not directly benefit from this preclinical development project.
Why it matters
Potential benefit: If successful, this work could reduce harmful side effects and speed delivery of safer ASO treatments to patients with severe neurological genetic disorders.
How similar studies have performed: Antisense therapies have produced major successes such as treatments for spinal muscular atrophy, but using optical physiology combined with machine learning specifically to predict CNS toxicity is a newer approach with limited prior human testing.
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.