The search for an effective treatment for ADOA has reached a major milestone. During the annual meeting of the Neuro-Ophthalmology Society of Australia (NOSA) in September 2026, Dr. Clare Fraser presented new data on PYC-001: a candidate treatment that does not focus on symptom management, but addresses the underlying genetic cause of ADOA.
This is a promising step towards a potential treatment, but it is emphatically not yet a proven or approved therapy.
What is PYC-001 and how does it work?
In ADOA, there is a mutation in the OPA1 gene. In ADOA, one of the two copies of the OPA1 gene is damaged, causing the body to produce too little of the essential OPA1 protein from the remaining healthy copy (about half). This deficiency — or haploinsufficiency — places a heavy strain on the energy factories of the optic nerve cells and causes vision loss.
PYC-001 is a modern drug composed of genetic material (an RNA-like therapy) that is administered directly into the gel-like fluid of the eye. The clever thing about this drug is that it gives the little factories in the cell a boost. It binds to the piece of hereditary code (the mRNA) from which cells get their instruction manual. Normally, there is a kind of brake on this, causing too little protein to be produced. PYC-001 removes that brake, as it were, so that the cell can once again produce more of the much-needed OPA1 protein.
Experiments in the laboratory and on test animals showed that this indeed ensures a significant increase in the amount of this protein and that the cells restore their energy production and structure.
First results in humans: safety and early signs
The drug is currently being tested for safety and efficacy in humans in an international study (divided in practice under the names SUNDEW and MYRTLE).
The initial safety results are positive. At the investigated dosages — ranging from very
low to higher, and also in patients who received multiple injections — no serious or concerning side effects caused by the drug have been reported to date.
In addition, doctors look for early signs of whether vision remains more stable or improves. In treated eyes, researchers observed tentative, positive signals: for instance, treated eyes performed better on average in terms of contrast vision (the ability to distinguish shades and contours) than the untreated eyes of the same patients, and there was less stress on the retina.
For whom is this being investigated?
To keep expectations clear, it is good to know for whom these current studies are intended. The study focuses on adults (aged 18 and older) with ADOA due to an OPA1 mutation who have moderate vision impairment.
In this phase, a deliberate choice is also made to select patients who do not use specific dietary supplements or other substances such as idebenone or Q10, in order to accurately measure the effect of this new medication alone.
Important nuance
Although these results are very encouraging, it is important to remember that this concerns data from a manufacturer's presentation at a medical trade fair, and not yet a definitive, independently reviewed scientific article.
The research is in a phase where it is being examined whether the concept works in practice. Before a product can ultimately come to market and be approved, larger and longer follow-up studies are required.
For the longer term, this offers a tangible perspective: for the first time, there is a treatment in clinical trials that attempts to address the underlying deficit in ADOA.
