What is the STK-002 Clinical Trial for ADOA?

STK-002 is an experimental treatment being investigated for the classic form of Autosomal Dominant Optic Atrophy (ADOA). It is designed to address the root cause of the condition by increasing the amount of essential proteins in the eye. While you may have read about this emerging therapy, it is important to understand how it works, its current development phase, and what researchers typically look for in trial participants.

How Does STK-002 Work?

STK-002 is a type of targeted therapy known as an antisense oligonucleotide (ASO) ^[1]. ASOs are small, synthetic pieces of genetic material that can change how cells read genetic instructions ^[2].

Most people with classic ADOA have one healthy copy of the OPA1 gene and one mutated copy. This results in haploinsufficiency — meaning the body only produces about half the normal amount of the OPA1 protein, which is necessary for the survival of the optic nerve cells ^[1].

Instead of trying to fix the mutated gene, STK-002 targets the healthy OPA1 copy. Normally, some of the genetic instructions from this healthy copy contain a “stop” signal (a nonproductive exon) that limits protein production ^[1]. STK-002 blocks this signal, forcing the cell to produce significantly more functional OPA1 protein ^[1]. Preclinical studies in lab-grown human cells and animal models have demonstrated that STK-002 successfully boosts OPA1 levels and improves the energy function of these cells ^[1].

Trial Phase and General Eligibility

As of mid-2026, STK-002 has primarily been focused on preclinical development (testing in labs and animals) to support its progression toward human clinical trials ^[1]. Because the therapy is in these early stages, specific and final human enrollment criteria are not fully detailed in the current medical literature ^[1][3].

However, based on standard practices for clinical trials involving retinal gene and ASO therapies, researchers typically require participants to meet specific criteria ^[4][5]. If you are looking to join a trial for STK-002 or a similar treatment, general eligibility requirements will likely include:

• Confirmed Genetic Diagnosis: You will need a genetic test confirming that your ADOA is caused by a specific type of OPA1 mutation that leads to low protein levels (haploinsufficiency) ^[1]. If you have not had a comprehensive genetic panel done yet, talking to your doctor about getting one now is a great proactive step.
• Age Requirements: Early-phase trials usually begin with adults to ensure safety before expanding to include children ^[6].
• Specific Visual Function: Trials usually require a specific window of best-corrected visual acuity (BCVA) (how well you see with your best glasses or contacts) ^[7]. Your vision cannot be too good (making it hard to measure improvement) or too poor (meaning there may not be enough surviving nerve cells to save) ^[8].
• Retinal Health: Researchers will use imaging tests to ensure you have enough preserved retinal structure for the treatment to be effective ^[8].
• Willingness for Eye Injections: Like many ASO therapies for the eye, STK-002 is delivered via intravitreal injection (an injection directly into the eye) ^[1]. While this sounds intimidating, the eye is thoroughly numbed with an anesthetic beforehand, making it a very common and manageable procedure in eye clinics. However, it does carry standard risks, such as temporary eye inflammation or increased eye pressure, which trials will monitor closely.

How to Track Enrollment Status

Because clinical trial statuses can change rapidly, the best way to monitor for open enrollment is by using official clinical databases and patient organizations:

• ClinicalTrials.gov: This database lists federally and privately supported clinical trials. You can search the site periodically using keywords like “ADOA,” “OPA1,” and “STK-002.” You can also set up an email alert or RSS feed for these keywords to automate your tracking.
• Patient Registries: Joining a registry is one of the most effective ways to be notified about upcoming trials, as researchers frequently use these to find matching patients ^[9]. Consider looking into the My Retina Tracker registry (managed by the Foundation Fighting Blindness) or registries associated with the Cure ADOA Foundation.
• Your Medical Team: Keep in close contact with a neuro-ophthalmologist or a specialist in inherited retinal diseases. They often receive early notifications about trial site openings and can help determine if you are a good candidate.