NurExone Demonstrates Reproducible, Dose-Dependent Vision Recovery in Preclinical Glaucoma Model
NurExone Biologic announced new preclinical results showing that its lead candidate ExoPTEN produces a reproducible, dose-dependent therapeutic effect in an eye model of glaucoma.
The study, conducted in collaboration with Prof. Ygal Rotenstreich and his team at the Goldschleger Eye Institute, Sheba Medical Center—one of the world’s leading ophthalmic research hospitals—demonstrated that ExoPTEN’s biological activity increases with higher dosing levels in animals with optic nerve injury. The results showed consistent and measurable recovery of visual function, confirming that ExoPTEN’s regenerative effect is both reproducible and quantifiable.
“Reproducibility is the key challenge in science, and now we have a first validation of the results,” said Prof. Michael Belkin, Professor Emeritus of Ophthalmology at Tel Aviv University and Scientific Advisor to NurExone. “These results confirm that ExoPTEN has the potential impact of a true therapeutic, producing reproducible repair of damaged optic nerves in small animals, advancing our ability to address vision loss in patients with optic nerve damage, such as glaucoma and related conditions.”
“We are seeing a clear, dose-dependent effect of ExoPTEN in the eye, with stronger functional recovery at higher doses,” added Dr. Tali Kizhner, Director of Research and Development at NurExone. “It highlights the one-two punch of our platform: exosomes that reach and protect neural tissue, and the siRNA cargo that switches on the regenerative response. This dose-response data marks an essential step toward future clinical trials and brings us closer to translating this therapy to patients.”
Study Highlights: Dose-Dependent Functional Recovery
The latest study marks the third independent preclinical investigation of ExoPTEN’s regenerative activity in the optic nerve crush (ONC) model, a well-established method that mimics the nerve damage seen in glaucoma.
Researchers tested low and high doses of ExoPTEN delivered via extrachoroidal injection directly to the eye. Functional measurements of retinal activity using scotopic threshold response electroretinography (STR-ERG) showed that both doses improved visual signal strength in animals with optic nerve injury, with the high-dose group achieving response amplitudes comparable to those of uninjured eyes.
These findings demonstrate substantial functional recovery and provide clear evidence of a dose-dependent therapeutic effect, consistent with ExoPTEN’s proposed mechanism of action—namely, promoting neural regeneration through targeted gene silencing and exosome delivery.
Figure 1: Dose-Dependent Restoration of Retinal Response Following ExoPTEN Treatment
In the ONC model, visual responses are considered detectable when retinal signal amplitudes exceed 5 µV; signals below this threshold indicate minimal or no retinal activity. Both low- and high-dose ExoPTEN-treated eyes exhibited recovery of retinal electrical response relative to saline and naïve exosome controls. The high-dose group achieved STR amplitudes comparable to normal eyes, confirming a dose-dependent restoration of visual function.
Supporting Previous Findings
The results build upon NurExone’s previously announced preclinical data from June 2024 and December 2024, which demonstrated structural preservation and survival of retinal ganglion cells following ExoPTEN administration. Together, these studies provide mounting evidence that ExoPTEN can promote functional and structural recovery in optic nerve injury.