RNAi gene silencing technology in form of ophthalmic drops completes Phase 1a clinical trial

In Spain, the first product based on interference RNA (RNAi) technology is an ophthalmic treatment in the form of drops to treat elevated IOP and glaucoma. SYL040012 has just completed its first clinical trial, having been administered to 30 healthy volunteers.

The endpoint of the Phase 1a trial was to determine the tolerance and safety of the ophthalmic drops. Initial results, say Sylentis, the drug's developer, have shown good local and systemic tolerance.

In its press release the company said that in preclinical trials with SYL040012, the siRNAs administered topically to treat ocular hypertension associated with open angle glaucoma have proven effective both in vitro and in vivo. In those trials, SYL040012 effectively prevented the increase in intraocular pressure induced in the ocular hypertension model that was used. Ocular tolerance studies have shown excellent local and systemic tolerance to the administration of SYL040012 in much larger doses than required for therapeutic effectiveness.