Madrid, Spain-based firm Sylentis has developed a drug for treating age-related macular degeneration and diabetic retinopathy. The new compound is a small molecule that features a relatively simple administration through ophthalmic drops.
Compounds currently used to treat these disease are based on large antibodies that cannot penetrate the ocular surface of the retina and thus require delivery by intravitreal injections. "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," said Covadonga Pañeda, R&D Manager at Sylentis.
The new compound SYL136001v10 is a small interfering RNA (siRNA) is designed to enter retinal cells and prevent the protein synthesis of NRARP. This blocks the formation of new vessels, which is one of the fundamental characteristics of degenerative diseases of the retina.
"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," adds Pañeda.
SYL136001v10 has been shown to be effective in animal models and human trials will begin at the end of 2018.
Reference:
See full press release here.
Covadonga Pañeda et al. "Targeting NRARP with siRNA based compounds for the treatment of retinal neovascularization". Annual Meeting of the Oligonucleotide Therapeutics Society (September 2017)
Source: Spanish Foundation for Science and Technology