During our ADOA day on Saturday, November 1, 2025, Aishwarya Kundu, Senior Director of Translational Research, shared the latest PYC results. You can read them in this edition of "The Road to Treatment."
A short guide for patients and families
ADOA, or autosomal dominant optic atrophy, is caused by a defect in the OPA1 gene. This causes the body to produce too little of an important protein called OPA1.
OPA1 ensures that mitochondria—the cell's energy factories—continue to function properly. When there are too few mitochondria, the cells of the optic nerve begin to deteriorate, leading to gradual vision loss.
Currently, there is no treatment that can slow or stop this process. But PYC Therapeutics is developing a new approach: PYC-001, an RNA-based therapy designed to increase OPA1 protein levels precisely where they are needed most.
How does PYC-001 work?
PYC-001 doesn't permanently alter the DNA. Instead, it helps the body produce more OPA1 protein, regardless of which OPA1 mutation you have. This is important because ADOA can be caused by many different genetic changes.
Unlike some gene therapies, PYC-001 is administered via a simple injection into the eye. This is a procedure already widely used for other eye conditions.
PYC-001 uses a special carrier protein that delivers the RNA drug directly to the cells of the optic nerve, which are most affected in ADOA.
What do we know so far?
Early laboratory tests using cells from ADOA patients showed that treatment with PYC-001 can repair the mitochondrial problem. Mitochondrial function improved due to an increase in the amount of OPA1 protein.
Follow-up research was conducted in healthy animals to confirm that PYC-001 can indeed increase OPA1 protein levels—a key step in demonstrating the drug's effectiveness in a living organism. Based on these results, PYC initiated the first human study in Australia, called SUNDEW. This study tested the safety of a single injection of PYC-001 at three doses: low, medium, and high.
The results showed that PYC-001 was safe and well-tolerated in all nine participating patients and at all doses tested. In addition, there were promising early indications that PYC-001 improved visual acuity and the ability to recognize dim or low-contrast images—exactly the kind of vision people need in everyday life, not just what's measured on a brightly lit chart at the ophthalmologist's office.
Next steps
Based on the safety and initial positive effects from the SUNDEW study, PYC has initiated a multicenter global study, called MYRTLE. This study will investigate the safety and efficacy of multiple injections of PYC-001, administered at 2- or 3-month intervals.
The goal is to show that repeated treatments over a year can produce the same, or even better, visual improvements in all participating patients.
Why is this important?
PYC-001 is still in early stages of research, but it represents a significant step forward. For the first time, scientists are developing treatments that address the underlying cause of ADOA.
RNA therapies like these offer a way to restore normal cell function without permanently altering the genetic material. As research progresses, we're learning more about how ADOA develops and how we can combat it.
A reason for cautious hope
There's still much work to be done, and clinical studies must demonstrate both safety and actual benefits. But every step forward brings us closer to understanding—and hopefully one day treating—ADOA.
