Retinal disease treatment strategies: could a new ophthalmic drop-delivered drug be the way forward?

A new investigational drug could be administered by ophthalmic drops for the treatment of age-related macular degeneration and diabetic retinopathy.

Dec 15, 2017

Recent data presented at the XIII Annual Meeting of the Oligonucleotide Therapeutics Society, which took place in Bordeaux, France, suggest that a new compound developed by the biotechnology company Sylentis (Madrid, Spain) could be able to penetrate the retina for the treatment of age-related macular degeneration and diabetic retinopathy.

The current treatment of choice for such conditions are administered via intraocular injections; however, this new compound could be delivered to the target area of the eye through the administration of ophthalmic drops.

Research and Development Manager at Sylentis, and main author of this recently presented study, Covadonga Pañeda (Madrid, Spain), highlighted the need to develop new treatment methods: “patients suffering from age-related macular degeneration or diabetic retinopathy have to go to the hospital where the treatments are performed with ocular injections, which is annoying and painful, and also involves a significant financial investment for the health system”.

Pañeda further explained that this new drug, known as SYL136001v10, “is a small interfering RNA (siRNA) designed to silence the expression of NRARP, a protein that controls the formation of new blood vessels in the retina”.

“The drug exerts its action by entering the cells of the retina, where it prevents the synthesis of this protein and blocks the formation of new vessels, which is one of the fundamental characteristics of degenerative diseases of the retina”.

Current treatments (which are based on large antibodies) are unable to penetrate the retina due to their large size, and so can only be administered by injection.

"siRNA, like SYL136001v10, are up to 10 times smaller than these treatments,” stated Pañeda, “Their size allows them to penetrate the retina and inhibit the formation of new blood vessels after their application in ophthalmic drops, as we have shown in animal models."

"Efficacy studies have shown that the reduction of NRARP in the retina by means of siRNA leads to the regression of angiogenic retinal lesions and that the observed reductions are equivalent to those of anti-VEGF, which is the current treatment standard for these diseases with ocular injections,"

Whilst this drug is still at the preclinical stage, it has demonstrated effectiveness in animal models and trials with humans are scheduled to start towards the end of 2018.


Covadonga Pañeda et al. “Targeting NRARP with siRNA based compounds for the treatment of retinal neovascularization.”  Annual Meeting of the Oligonucleotide Therapeutics Society (2017); http://

Hannah Makin

Commissioning Editor at Future Science Group, Future Science Group

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