Drug intelligence / Profile preview

ALY-103

Development stage
Preclinical
Lead developer
Alys Pharmaceuticals
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Topical
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Overview

ALY-103 is a small interfering RNA (siRNA) therapeutic conjugated to a lipid moiety, designed for the topical treatment of vitiligo. Developed by Alys Pharmaceuticals using technology originally from Aldena Therapeutics, the drug specifically targets the mRNA of Janus kinase 1 (JAK1). By silencing JAK1 expression locally in the skin, ALY-103 aims to inhibit the JAK/STAT signaling pathway, which is a key driver of the autoimmune destruction of melanocytes in vitiligo. The proprietary siRNA-lipid conjugate platform is engineered to overcome the skin barrier, providing targeted delivery to the dermis while minimizing systemic absorption and the side effects typically associated with oral JAK inhibitors.

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Targets

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