A new compound, produced by the biotech company Sylentis, can permeate the retina to take care of age-related macular degeneration and diabetic retinopathy, simply with the administration of this new Ophthalmic drops. The drug SYL136001v10, shown by the PharmaMar group company, continues to be in the preclinical stage. “Its efficiency has shown in pet models and tests with humans will commence by the end of 2018”, said Covadonga Pañeda, R&D Manager at Sylentis.
Pañeda is the key author of a report on SYL136001v10, that was presented by the end of Sept at the XIII Total annual Conference of the Oligonucleotides Therapeutics Society, held in Bordeaux (France).
The objective of their research was to silence the expression of the NRARP protein, this protein is responsible for controlling the formation of new arteries in the retina. the small interfering RNA (siRNA) mediates this silencing, Pañeda told.
In this manner, “the medicine exerts its action by getting into the cells of the retina, where it helps prevent the formation of this proteins and blocks the forming of new vessels, which is one of the essential characteristics of degenerative diseases of the retina“, she added. Presently used treatment to take care of these diseases derive from large antibodies that cannot permeate from the ocular surface to the retina, so they need to be implemented by intravitreal injections.
Therefore, “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”, says Covadonga Pañeda.
However, she notes, “siRNA, like SYL136001v10, are up to 10 times smaller than these treatments. Their size allows these to permeate the retina and inhibit the forming of new arteries after their application in new Ophthalmic drops, as we’ve shown in animal models”.”.
In particular, “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”, stresses the R&D manager. The firm, located in Tres Cantos (Madrid), plans to take charge of the first clinical efficacy studies. Once the proof of concept in humans has been demonstrated, it will try to license the drug to a multinational.
Source- News Medical