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SIS-202-CDC
- Development stage
- Preclinical
- Lead developer
- SiSaf
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Topical, Ophthalmic
01
Overview
SIS-202-CDC is a topical CRISPR-Cas9 gene-editing therapy developed using SiSaf's Bio-Courier technology. It is designed to permanently edit the mutated Transforming Growth Factor Beta I (TGFBI) gene in patients with type II Corneal Dystrophy. By employing a double-cut CRISPR-Cas9 approach, the drug aims to stop the overproduction and aggregation of TGFBI protein, which underlies disease pathology. The therapy represents an innovative approach for treating corneal dystrophies, which are genetic eye disorders characterized by abnormal material accumulation in the cornea leading to visual impairment. As of March 2025, SIS-202-CDC remains in preclinical development[1][2][4].
02
Targets
TGFBI (Transforming growth factor-beta-induced protein ig-h3)TGFB1 (Transforming growth factor Beta-1 proprotein)
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