Oral, once-daily MC16 medicine for retinitis pigmentosa
Official title: Novel gene mutation agnostic small molecule to treat retinitis pigmentosa
Funded at Mitochem Therapeutics INC. · NIH-11401308 · Data updated 2026-11-12
A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mitochem Therapeutics INC. NIH-funded |
| Lab location | 1 site (Charleston, United States) |
| Project start | September 30, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $320,790 |
| NIH project number | 1R43EY037591-01A1 |
| Project ID | NIH-11401308 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with retinitis pigmentosa or related retinal diseases who could potentially benefit from an oral, once-daily therapy.
✕Less likely to help
Patients without RP or those unable to take oral medications may not benefit.
What this research studies
Retinitis pigmentosa is a group of inherited eye diseases that cause progressive vision loss. This project aims to move MC16 from topical use to an easy-to-take oral tablet, so patients can take it once daily. The team will test different oral doses in mice that model RP to find a minimally effective amount and evaluate safety and how the drug behaves in the body. The approach focuses on correcting a cellular dysfunction involving FUS protein that contributes to retinal damage. If successful, this could offer a non-gene-therapy option for many RP patients.
Why it matters
Potential benefit: If successful, patients could have an easier daily treatment that helps slow vision loss.
How similar studies have gone: Preclinical animal studies have shown promising results with MC16; human data are not yet available.
Where this research is happening
-
Mitochem Therapeutics INC.
Charleston, United States Active
Researchers
- Principal investigator: Voevodsky, Michael — Mitochem Therapeutics INC.
- Study coordinator: Voevodsky, Michael
Related research
Other NIH-funded projects on the same topics.
-
Inflammation in MERTK-related retinitis pigmentosa
This project aims to understand whether losing the MERTK gene causes retinal inflammation that leads to vision loss in people with MERTK-related retinitis pigmentosa.
-
Enhancing cone survival in RP through targeted cell-specific CRISPR therapy
A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.
-
Role of IPM Proteoglycans in Retinal Health and Inherited Blindness
This grant studies how specific retinal extracellular matrix proteins (IMPG1 and IMPG2) affect eye health and contribute to retinitis pigmentosa, with potential implications for people who have or are at risk for inherited blindness.
-
NAC Attack: a late-stage trial for preserving vision in RP patients
A large, multi-site study is testing whether taking a daily NAC pill can help people with retinitis pigmentosa keep more of their vision.
-
Non-invasive biomarker for mitochondria health in retinitis pigmentosa
Aims to test a new, non-invasive OCT-based biomarker to explain and predict how mitochondria function affects cone cells in people with retinitis pigmentosa.
-
Understanding and Treating Inherited Retinal Degeneration to Preserve Vision
A clinician-scientist at Stanford is pursuing gene-agnostic therapies to slow or prevent vision loss from inherited retinal diseases like Retinitis Pigmentosa.
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.