SanegeneBio's CFB-Targeting siRNA Drug SGB-3383 Granted IND Approval in China for Complement-Mediated Kidney Diseases

On May 20, SANEGENEBIO announced that the clinical trial application for its self-developed CFB-targeted small nucleic acid (siRNA) drug SGB-3383 injection has recently received tacit approval from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for clinical trials, intended for the treatment ofComplement-mediated kidney diseases (including IgA nephropathy, C3 glomerulopathy, immune complex-mediated membranoproliferative glomerulonephritis, atypical hemolytic uremic syndrome, etc.)。

Screenshot source: CDE official website

The complement system is an important component of the body's innate immunity, playing a crucial role in immune and physiological functions. The activation of the complement system occurs through three pathways: the classical pathway, the alternative pathway, and the lectin pathway.Complement Factor B (CFB), as a crucial component of the alternative complement pathway, is a key element involved in complement activation and plays an important role in both cell injury and inflammatory processes.CFB is involved in the onset and progression of various diseases. Current research has found that CFB is associated with atypical hemolytic uremic syndrome, age-related macular degeneration, cardiovascular diseases, tumors, and other conditions. Targeting CFB therapy, by modulating complement activation, may treat various diseases caused by alternative pathway dysfunction while not affecting other complement pathway-mediated immune responses to microbial invasion, showing potential therapeutic effects in reducing patients' infection risks.

According to the SANEGENEBIO press release, SGB-3383 is an RNAi therapy targeting CFB.Treating Complement-Mediated Kidney Diseases by Suppressing CFB Expression in the Liver Through RNAiPreclinical trial data show that SGB-3383 can significantly and durably reduce the levels of CFB in the liver and blood, with good safety and tolerability.

For complement-related diseases, in addition to SGB-3383, SANEGENEBIO's SGB-9768, an siRNA drug targeting complement C3, has also achieved positive results in preclinical and phase 1 clinical trials, demonstrating stronger protein knockdown activity and longer-lasting effects. It has now advanced to phase 2 clinical trials.