13 December 2017

Drops from blindness

The drug in the form of eye drops in animal studies has shown its effectiveness for the treatment of retinal diseases such as age-related macular degeneration and diabetic retinopathy. Previously, their treatment involved only intraocular administration of medication. The new drug is able to reach retinal cells and prevent the growth of pathological newly formed vessels.

The drug SYL136001v10, presented by PharmaMar at the Annual Congress The Oligonucleotide Therapeutics Society is currently in the preclinical phase of trials. Human studies are scheduled to begin at the end of 2018.

Newly formed pathological vessels are the main link in the pathogenesis of age–related macular degeneration and diabetic retinopathy, which, without treatment, lead to complete loss of central vision.

The presented preparation SYL136001v10 contains a small interfering RNA (siRNA), which suppresses the expression of genes encoding the synthesis of the NRARP protein. NRARP has been proven to control the growth of new vessels in the retina.

The drugs currently used to treat the retina contain large molecules of anti-VEGF (vascular endothelial growth factor antagonists), therefore, they have a therapeutic effect only when directly injected into the eye cavity near the retina (intravitreal administration). This procedure is performed in a hospital setting, is expensive and involves risks of infection and damage to the retina during injection.

The siRNA component in the SYL136001v10 has a small size, so if you drip the drug into the conjunctival cavity, the active substance easily reaches the retina. As studies on animal models have shown, the drug inhibits the growth of newly formed vessels and improves the condition of the retina.

According to the effect of siRNA in animal studies, it did not differ from the currently approved anti-VEGF, the effectiveness of which in suppressing the growth of new vessels has been proven.

The manufacturer of the drug SYL136001v10 intends to conduct efficacy studies in humans and license it with a positive result.

Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru based on EurekAlert: A drug to treat retinal diseases with drops instead of injections.


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