Sylentis presents new data on a new eye drop siRNA for the treatment of retinal diseases avoiding intraocular injections

  • This new investigational treatment, based on RNA interference, reaches the retina when administered in eye drops. This feature is expected to positively affect patients’ quality of life by eluding the use of current standard treatments administered by intraocular injections.


Madrid, 25 September 2017. Sylentis presents, for the first time, positive data on the efficacy of SYL136001v10, a siRNA (small interference RNA) for the treatment of neovascular diseases of the retina such as Age-Related Macular Degeneration (AMD) and Diabetic Retinopathy (DR). SYL136001v10 reaches the retina when administered topically, demonstrating that the siRNA may be applied in eye drops changing the treatment paradigm for patients suffering these diseases. “Currently, patients are treated in a hospital setting where they receive anti-VEGFs by intraocular injection. This procedure is not only painful and uncomfortable for the patient but also entails increasing financial commitments for our national health system”, notes Covadonga Pañeda, R&D Manager at Sylentis, and principal author of the studies.

The data will be presented at the XIII Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) taking place in Bourdeaux, France between the 24th and the 27th of September and where the latest advances in oligonucleotide therapeutics will be presented.

Sylentis has identified NRARP (NOTCH-regulated Ankyrin Repeat Protein) as a potential target for the treatment of angiogenic diseases of the retina. NRARP is a key regulator of the molecular pathways leading to vessel formation; as such, the consequences of modulating the expression of this gene with RNAi has been studied in animal models of retinal neovascularization.

The results of the efficacy studies have shown that reduction of NRARP in the retina using RNAi leads to regression of neovascular lesions and that the lesion reductions observed are equivalent to those observed in response to anti-VEGF treatment, current standard treatment administered by intraocular injection.

Sylentis studies at the XIII Annual Meeting of the Oligonucleotide Therapeutics Society:

  • Targeting NRARP with siRNA based compounds for the treatment of retinal neovascularization
    Session: September 24th 18:30 to 20:30 horas. Presenting author: Covadonga Pañeda et al. R&D Manager, Sylentis, España.
  • Clinical and preclinical study correlation for SYL1001, a new treatment for dry eye disease (Posterboard number:
    Session: September 25th 17:00 to 19:30 horas. Presenting author: Covadonga Pañeda et al. R&D Manager, Sylentis, España.


About RNA interference (RNAi)
RNA interference (RNAi) is a natural cellular process that regulates the expression of certain genes, providing a role in innate defense and development in animal and plants. This process is used to specifically silence genetic transcripts that encode protein-causing diseases. The therapeutic application of targeted siRNAs is booming given the specificity of gene silencing for a particular protein in a given tissue and the lack of side effects. This new approach to drug discovery is a promising technology that is rapidly moving in the translational research space[i],[ii].

About Sylentis
Sylentis, a company of PharmaMar (MSE:PHM), is a biotechnology company fully owned that develops innovative therapies harnessing the technology of post-transcriptional gene silencing or RNA interference (RNAi). Sylentis has developed an approach to efficiently design RNAi-based therapeutics that can be used to silence numerous disease-causing genes. We currently have a robust therapeutic program in ophthalmology with two candidates under development in Phase II studies for glaucoma (bamosiran)[iii] and ocular pain (SYL1001)11. Sylentis is also developing new products for the treatment of several eye diseases such as ocular allergies and retina diseases. To know more about us, please visit us at

This document is a press release, not a prospectus. This document does not constitute or form part of an offering or invitation to sell or a solicitation to purchase, offer or subscribe shares of the company. Moreover, no reliance should be placed upon this document for any investment decision or contract and it does not constitute a recommendation of any type with regard to the shares of the company.


[i] Elbashir SM1, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 2001 May 24;411(6836):494-8

[ii] Soutschek J1, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Röhl J, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature. 2004 Nov 11;432(7014):173-8

[iii] Moreno-Montañes J, Sádaba B, Ruz V, Gomez-Guiu A, Zarranz J, Gonzalez MV, Pañeda C, Jimenez AI. Phase I Clinical Trial of SYL040012, A Small Interfering RNA Targeting β-Adrenergic Receptor 2, for Lowering Intraocular Pressure. Mol Ther. 2014, 22(1):226-32

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