SIS-102-ACH

SIS-102-ACH is a systemic Bio-Courier small interfering RNA (siRNA) therapeutic developed to inhibit the mutated Fibroblast Growth Factor Receptor 3 gene (FGFR3), which acts as a negative regulator of bone growth. By specifically targeting and silencing the mutant FGFR3 allele, SIS-102-ACH aims to address the root cause of achondroplasia, a genetic disorder characterized by impaired bone growth and dwarfism. Unlike other therapies that may only affect certain bones, this siRNA therapy is designed to promote growth in all bones throughout the body. The drug is administered via injection at weekly or longer intervals and is initially focused on pediatric patients with potential future use in adults[1][2][3]. **Developer:** SiSaf SIS-102-ACH represents a first-in-class approach for achondroplasia by directly inhibiting the disease-causing mutation without affecting normal FGFR3 function. As of March 2025, it remains in preclinical development[1][2].