Researchers develop formula to deliver drugs to the back of the eye

The researchers believe the technology, which uses nanoparticles to deliver an RUNX1 inhibitor, could have applications in treating diseases relating to sight loss


A team of researchers have developed an eye drop to effectively deliver drugs to the retina and other tissues located in the back of the eye.

Potential applications for the technology could include the treatment of several diseases relating to sight loss, researchers believe.

Outlined in Nature Scientific Reports, the new experimental treatment is composed of nanoparticles called eNano-Ro5 and delivers a small molecule inhibitor of the transcription factor RUNX1 to the back of the eye.

The scientists involved in developing the new formulation previously linked excessive function of RUNX1 to the abnormal growth of blood vessels observed in patients with advanced diabetic eye disease (proliferative diabetic retinopathy). The research, reported in Diabetes in 2017, showed that an injection of a small molecule inhibitor of RUNX1 into the eye was effective at curbing growth in preclinical models.

In the new study, the treatment was administered as daily eye drops to preclinical models of proliferative vitreoretinopathy or PVR. The report found that eNano-Ro5 delivered effective amounts of the RUNX1 inhibitor to the back of the eye, resulting in reduced severity of PVR in preclinical models.

Researchers anticipate the technology could support the development of new methods of treatment to address conditions that lack a medical treatment or require the administration of approved drugs using regular eye injections.

Leo A. Kim, co-corresponding author, retina surgeon at Mass Eye and Ear and assistant professor of ophthalmology at Harvard Medical School, commented: “It is clear the RUNX1 plays a role in multiple pathological conditions within the eye and beyond.

“Retinal detachment and PVR has been a vexing problem for retinal surgeons. Developing a topical agent that effectively treats PVR using a relevant in vivo model is a significant step forward in our treatment of this sight threatening condition.”

Co-corresponding author, Joseph F. Arboleda-Velasquez, assistant scientist at Schepens Eye Research Institute of Mass Eye and Ear and assistant professor of Ophthalmology at Harvard Medical School, said that when work began on the eye drop formulation, he saw it as a “matter of convenience” that patients would have an alternative to eye injections.

“But now I see it as a matter of necessity because there is an urgent clinical need for drugs that patients can self-administer under social distancing precautions,” he said.