--SGB-3383 is an siRNA drug targeting CFB for complement-mediated kidney diseases and related systemic conditions, showing its potential to be "best-in-class".
--Preclinical studies demonstrated that SGB-3383 can effectively and continuously inhibit the production of CFB, while exhibiting favorable safety and tolerability profiles.
On May 20, 2025, SanegeneBio, a clinical-stage biotech company focused on developing innovative RNAi therapeutics, announced that its self-developed siRNA drug candidate SGB-3383 targeting complement factor B (CFB), has recently received clinical trial approval from the Center for Drug Evaluation (CDE) under China's National Medical Products Administration (NMPA), for the treatment of complement-mediated kidney diseases, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), Immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), atypical Haemolytic Uraemic Syndrome (aHUS), etc.
The complement system, a key component of innate immunity, serves critical immunological and physiological roles, protecting the body from infections and clearing dead cells and apoptotic material. There are three pathways of complement activation: the classical pathway, the alternative pathway, and the lectin pathway. CFB plays a crucial role in the complement system, particularly in the activation of the alternative pathway. Inhibition of CFB is sufficient to reduce cell damage and inflammation [1]. CFB is involved in the occurrence and development of various diseases, including atypical hemolytic uremic syndrome, age-related macular degeneration, cardiovascular disease, tumors, and other diseases [2]. CFB-targeted therapies, by regulating complement activation, may treat diseases caused by alternative pathway dysfunction without affecting the immune responses to microbial invasion mediated by other complement pathways, showing potential therapeutic effects in reducing the risk of infection in patients.
Developed based on SanegeneBio's proprietary GalNAc platform, SGB-3383 is delivered to liver cells to inhibit CFB expression, thereby treating complement-mediated kidney diseases. Preclinical data demonstrated that SGB-3383 can effectively and continuously inhibit the production of CFB in the liver and bloodstream, while exhibiting favorable safety and tolerability profiles. Currently, no siRNA drugs targeting the complement system have been approved globally. SGB-3383 has the potential to become the China's first and world-leading siRNA drug targeting CFB.
In the field of complement-mediated diseases, SanegeneBio is also developing another siRNA drug candidate SGB-9768, which has achieved positive results from preclinical and Phase I clinical trial, demonstrating superior potency and durability. SGB-9768 has now advanced to phase II clinical trials.
Dr. Weimin Wang, Founder and Chief Executive Officer of SanegeneBio, stated: " SGB-3383 consolidates SanegeneBio's leading position in the field of siRNA therapeutics for complement-related diseases, providing greater flexibility and strategic options for late-stage clinical development and product planning. Up to now, four drug candidates developed on SanegeneBio's proprietary GalNAc platform have entered the clinical stages, with robust clinical data consistently validating the platform's high efficiency, safety, and stability. Moving forward this year, the company will continue to accelerate clinical research of these drug candidates and advance more high-value extrahepatic pipelines into clinical trials, striving to bring more treatment options to patients as soon as possible."
Reference
[1] Kavanagh D, Barratt J, Schubart A, Webb NJA, Meier M, Fakhouri F. Factor B as a therapeutic target for the treatment of complement-mediated diseases. Front Immunol. 2025 Feb 14; 16:1537974. doi: 10.3389/fimmu.2025.1537974.
[2] 郭楠楠, 王兴智. 补体B因子的研究进展[J]. 国际免疫学杂志,2022,45(01): 102-107.
About SanegeneBio
SanegeneBio is an emerging RNAi-based company developing innovative RNA interference (RNAi) therapeutics driven by the cutting-edge delivery technology. Founded in 2021, SanegeneBio was led by a team of industry-leading experts and has operations in both the US and China. Since its incorporation, SanegeneBio has successfully established proprietary chemical modification platform, hepatic and extrahepatic delivery platforms, enabling tissue-specific delivery of a wide range of RNA therapeutics to efficiently knock down disease-causing genes. Our fast-growing RNAi portfolio covers a broad range of therapeutic areas including cardiovascular and metabolic diseases, immunology-related diseases, and nervous system diseases. Among them, our four RNAi drugs have entered into the clinical stage, and several pipelines are in progress simultaneously. SanegeneBio is committed to developing transformational RNAi medicines through striving for scientific innovation to address unmet medical needs worldwide and change the lives of patients and families. For further information, please visit: www.sanegenebio.com and engage with us on LinkedIn.
