Biomarkers to measure how well QTE therapy works for Friedreich's ataxia
Official title: Development and Validation of Efficacy Biomarkers for QTE Therapy in Friedreich's Ataxia
Funded at Anekabio LLC · NIH-11420527 · Data updated 2026-11-12
Aiming to find blood and muscle signals that show whether a new FRDA treatment (QTE therapy) is working.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Anekabio LLC NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | September 23, 2025 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $245,560 |
| NIH project number | 5R41NS143585-02 |
| Project ID | NIH-11420527 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Friedreich's ataxia or researchers seeking biomarkers that could apply to FRDA patient care and trial participation.
✕Less likely to help
Healthy individuals or those without Friedreich's ataxia are unlikely to benefit from FRDA biomarker development.
What this research studies
This project will look for measurable biological indicators in blood and muscle that reflect QTE therapy's effect in Friedreich's ataxia. Researchers will use mouse models and advanced proteomic and transcriptomic methods to identify candidates, then test their reliability as biomarkers. The goal is to enable future human trials by providing clear ways to monitor treatment response and safety. If successful, these biomarkers could help guide dosing and decision-making in patients with FRDA.
Why it matters
Potential benefit: If successful, patients with Friedreich's ataxia could see more precise monitoring of treatment response and faster advancement to human trials for effective therapies.
How similar studies have gone: Previous biomarker efforts in FRDA have shown promise but require validation in human trials; this project focuses on identifying and validating biomarkers in preclinical models for future clinical use.
Where this research is happening
-
Anekabio LLC
Gainesville, United States Active
Researchers
- Principal investigator: Chandran, Vijayendran — Anekabio LLC
- Study coordinator: Chandran, Vijayendran
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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.