BOSTON, SHANGHAI, and SUZHOU – May 19, 2026, SanegeneBio, a clinical-stage biotechnology company developing innovative RNAi therapeutics, announced that China’s National Medical Products Administration (NMPA) has approved a new clinical trial application (CTA) for SGB-9768 in complement-mediated hematologic disorders, including Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS). SGB-9768 had previously received CTA approvals in complement-mediated kidney diseases, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
SGB-9768 is an investigational RNAi therapeutics designed to reduce hepatocyte production of complement component 3 (C3) and is currently being evaluated in a Phase 2 clinical study for the treatment of complement-mediated kidney diseases, including IgAN, C3G, and IC-MPGN. In two Phase 1 clinical trials in healthy volunteers, a single subcutaneous administration of SGB-9768 demonstrated dose-dependent, significant, and sustained reductions in serum C3 levels and complement pathway activity, along with a favorable safety and tolerability profile, supporting its potential as a best-in-class therapy.
The advancement of SGB-9768 into hematological indications further underscores its broad therapeutic potential and highlights the opportunity to address significant unmet medical needs across multiple complement-mediated diseases.
Dr. Yuyan Jin, Senior Vice President of Clinical and Nonclinical Development at SanegeneBio, stated:
“The potent and durable activity, together with the favorable safety profile demonstrated by SGB-9768 in clinical studies, provides a strong foundation for expansion into complement-mediated blood disorders. This approval further validates the multi-indication potential of our RNAi platform and marks an important step in extending our pipeline beyond kidney diseases into broader therapeutic areas. As we continue to expand the clinical development of SGB-9768, we believe it has the potential to bring meaningful new treatment options to patients across multiple complement-mediated diseases.”
About Complement-Mediated Hematologic Disorders (PNH and aHUS)
Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) are rare, severe hematologic disorders driven by the pathological overactivation of the alternative complement pathway (AP). This dysregulation results in the immune-mediated destruction of host blood cells. PNH is characterized by chronic hemolysis and a high thrombotic risk, while aHUS involves microvascular clot formation leading to multi-organ damage, particularly renal failure. Although currently approved complement inhibitors have significantly improved clinical outcomes for patients with PNH and aHUS, important unmet medical needs remain. Existing therapies primarily target the terminal complement pathway and may provide incomplete disease control in some patients. Moreover, the requirement for long-term intravenous injections imposes a high treatment burden on patients.
About SanegeneBio
SanegeneBio is a global, venture-backed, fully-integrated biotechnology company focused on developing RNAi-based therapeutics. Founded in 2021 and led by a team of RNAi veterans, the company has R&D operations in Boston, Shanghai and Suzhou. Their clear vision is that RNAi technology will power blockbuster medicines in diverse therapeutic areas, improving the quality and longevity of life for patients worldwide. This vision is being realized by advancing a fast-growing pipeline, which includes experimental medicines for autoimmune nephropathies, obesity, and cardiometabolic indications. SanegeneBio has initiated clinical trials for four experimental medicines to-date, and is committed to developing potential best-in-class and first-in-class therapeutics which leverage our industry-leading and differentiated LEAD™ tissue-selective RNAi delivery technology. For more information, please visit: www.sanegenebio.com and engage on LinkedIn.
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