Tides Express | SanegeneBio's SGB-9768 (C3 siRNA) Receives NMPA Approval for Clinical Trials in PNH and aHUS

SGB-9768 has previously received clinical trial approval for complement-mediated kidney diseases, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).

SGB-9768 is an siRNA candidate drug targeting C3, currently in Phase II clinical trials for complement-mediated kidney diseases. In the completed Phase I clinical trial, a single subcutaneous dose of SGB-9768 achieved dose-dependent, significant, and sustained reduction in C3 levels and inhibition of the complement pathway activity, demonstrating good safety and tolerability, and showing best-in-class potential.

The advancement of SGB-9768 into hematological indications further highlights its broad therapeutic potential and brings new opportunities to address significant unmet clinical needs in complement-mediated diseases.

PNH and aHUS are rare and severe hematological disorders driven by the abnormal overactivation of the alternative complement pathway (AP). This abnormality can lead to immune-mediated damage to the body's own blood cells. PNH is characterized by chronic hemolysis and an increased risk of thrombosis, while aHUS manifests as microvascular thrombosis, which can cause multi-organ damage, particularly renal impairment. Although currently approved complement inhibitors have significantly improved clinical outcomes for patients with PNH and aHUS, there remains a significant unmet clinical need. Existing therapies primarily target the terminal pathway of the complement system, and some patients may not achieve complete disease control. Additionally, long-term intravenous administration also imposes a high treatment burden on patients.

AboutSANEGENEBIO

SANEGENEBIO is a company focused on innovation.RNA InterferenceSANEGENEBIO is a clinical-stage biotechnology company dedicated to the development of RNA interference (RNAi) therapeutics. Founded in 2021 by a pioneering team in the field of RNAi, the company is headquartered in Suzhou and Shanghai, China, and Boston, USA, with research and development centers supported by several globally renowned venture capital firms. We firmly believe that RNAi technology has the potential to generate blockbuster drugs across multiple therapeutic areas, thereby improving the quality of life and extending the lifespan of patients worldwide. Leveraging its industry-leading and differentiated RNAi drug development platform (including the tissue-selective LEAD™ delivery technology), SANEGENEBIO is committed to developing innovative therapies with "Best-In-Class" and "First-In-Class" potential to address unmet clinical needs in immune-mediated diseases, obesity, and cardiometabolic disorders.

Source:

1.SANEGENEBIO