SanegeneBio Receives NMPA Approval to Initiate Clinical Trial of SGB-3383 for Complement-Mediated Blood Disorders
Sep 19, 2025
— A new approval to study evaluates efficacy and safety in complement-mediated blood disorders: Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) —
BOSTON, SHANGHAI, and SUZHOU – September 19, 2025, SanegeneBio, a clinical-stage RNAi therapeutics company, today announced that China's National Medical Products Administration (NMPA) has approved SGB-3383 to initiate a clinical trial to treat complement-mediated blood disorders: Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS).
SGB-3383 is an experimental RNAi medicine targeting complement factor B (CFB). It previously had already received approval in China to initiate a Phase 1 clinical trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SGB-3383 in healthy volunteers.
Dr. Yuyan Jin, Senior Vice President of Clinical and Non-Clinical Development at SanegeneBio, stated:
About Complement-Mediated Blood 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 manifests as chronic hemolysis and a high thrombotic risk, while aHUS involves microvascular clot formation leading to vital organ damage, particularly renal failure.
Current complement inhibitors for PNH and aHUS have significant limitations: They only target the terminal complement pathway, leading to suboptimal efficacy. Moreover, the requirement for long-term intravenous injections imposes a high treatment burden on patients.
About SGB-3383
SGB-3383 is an RNAi-based experimental medicine targeting the complement Factor B (CFB) mRNA. It is being developed for the treatment of complement-mediated nephropathies, including IgA nephropathy (IgAN) and C3 glomerulopathy (C3G), as well as complement-mediated blood disorders such as Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS).
For PNH and aHUS, SGB-3383 can suppress the overactivation of the complement alternative pathway (AP), providing potent and sustained upstream inhibition. Compared to current therapies, SGB-3383 has the potential to overcome suboptimal efficacy and offer less frequent subcutaneous therapy to patients.
SGB-3383 leverages SanegeneBio proprietary RNAi technology to achieve superior potency, duration and tolerability. Preclinical data demonstrated that SGB-3383 can effectively and continuously inhibit the production of CFB in the liver and bloodstream, while exhibiting an unparalleled safety profile. Currently, no RNAi-based medicines targeting the complement system have been approved globally.
About SanegeneBio
SanegeneBio is a global, venture-backed, fully-integrated biotechnology company focused on developing RNAi-based therapeutics. Founded in 2021, SanegeneBio is led by a team of RNAi pioneers with unrivaled experience in this Nobel Prize-winning technology. With R&D operations in Boston, Shanghai and Suzhou, our vision clear – RNAi technology will power blockbuster medicines in diverse therapeutic areas, improving the quality and longevity of life for countless patients in the coming years. Our fast-growing pipeline includes experimental medicines for autoimmune nephropathies, obesity, and cardiometabolic indications. SanegeneBio has initiated clinical trials for several experimental medicines, 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 with us on LinkedIn.
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