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SIS-201-CD
- Development stage
- Preclinical
- Lead developer
- SiSaf
- 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, Gene Therapies
- Administration
- Topical, Ophthalmic
01
Overview
SIS-201-CD is a topical, non-invasive small interfering RNA (siRNA) gene therapy developed to inhibit the mutated Transforming Growth Factor Beta I (TGFBI) gene. Its primary mechanism is to prevent the overproduction and aggregation of TGFBI protein in patients with hereditary Type II Corneal Dystrophy, a genetic eye disorder characterized by abnormal material accumulation in the cornea leading to visual impairment. The drug uses SiSaf's Bio-Courier delivery platform for targeted ocular administration as an eye drop, representing a first-in-class approach for this indication. Development is led by SiSaf and Avellino Labs[1][2][3].
- Other names
- TGFBI gene inhibitor (SiSaf/Avellino)TGFBI siRNA therapy (SiSaf/Avellino)
02
Targets
TGFBI (Transforming growth factor-beta-induced protein ig-h3)
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