Drug intelligence / Profile preview

SYL1801

Development stage
Phase 2
Lead developer
Sylentis
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Ophthalmic, Topical
01

Overview

SYL1801 is a chemically modified small interfering RNA (siRNA) developed by Sylentis, a PharmaMar Group company, for the treatment of retinal diseases such as neovascular (wet) age-related macular degeneration (AMD). It is administered as an eye drop solution and works by selectively inhibiting the synthesis of NRARP (Notch-Regulated Ankyrin Repeat Protein), a protein involved in choroidal neovascularization. By downregulating NRARP expression through RNA interference, SYL1801 targets abnormal blood vessel formation in the retina—a key pathological process in wet AMD. Unlike current antiangiogenic therapies that require intravitreal injections, SYL1801 offers a non-invasive topical alternative. The drug has completed Phase I clinical trials and is currently in Phase II development for wet AMD[2][4][7].

Other names
syl1801syl-1801syl 1801
02

Targets

NRARP (Notch-regulated ankyrin repeat protein)

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