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SIS-202-CDC

Development stage: Preclinical
Lead developer: SiSaf
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration: Topical, Ophthalmic

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].

Targets

TGFBI (Transforming growth factor-beta-induced protein ig-h3)

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