SanegeneBio's SGB-3383 (CFB siRNA) Receives NMPA Approval for Phase II Clinical Trial in Complement-Mediated Hematologic Diseases

SANEGENEBIO Was Incubated from BioTOP to BioBAY in 2022Continue to DevelopSANEGENEBIO is a clinical-stage global biotechnology company focused on the development of RNAi therapies. It has established a multi-pipeline portfolio of innovative drugs targeting obesity, cardiometabolic diseases, and immune-mediated conditions. Leveraging its proprietary LEAD™ (Ligand-Enhanced And Delivery) technology platform, SANEGENEBIO overcomes the challenges of multi-tissue delivery for RNAi drugs, achieving liver/adipose/muscle/immune cell targeting, long-term gene silencing with a single dose, and high safety and efficacy validated by preclinical and clinical studies.

SGB-3383 Adds New Phase II Clinical Indication for Hematological Diseases, Expanding Its Scope from Renal Diseases to Hematological Diseases

As a novel RNAi therapy, SGB-3383 has the potential to bring transformative treatment options for patients with PNH and aHUS.

▲SANEGENEBIO's CFB-Targeted siRNA Candidate Drug SGB-3383

The Complement System: A Key Component of Innate Immunity and Its Role in DiseaseThe complement system is a crucial component of innate immunity, playing a key role in protecting the body from infections, clearing dead cells, and removing apoptotic materials. However, once dysregulated or excessively activated, the complement system can induce inflammation and damage self-tissues, causing immune injury. This is closely associated with the development and progression of certain hematological, ophthalmic, and renal diseases. Complement-mediated hematological disorders are caused by the overactivation of the alternative complement pathway, which leads to the destruction of the body's own blood cells. Common diseases include PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome). Both PNH and aHUS are rare diseases mediated by the complement system, affecting very small patient populations but imposing a heavy disease burden. If the condition is not effectively controlled, it progressively causes multi-organ damage involving the kidneys, nerves, and heart, posing severe health threats to patients. These patients typically face long and challenging diagnostic processes, along with recurring serious symptoms such as anemia, thrombosis, and worsening renal function, presenting significant challenges to their quality of life.

Currently, complement inhibitors are the main treatment in clinical practice. These drugs have improved the survival and quality of life for patients with PNH and aHUS, but they still have many limitations: they only block the terminal stage of the complement system activation and cannot inhibit the activation of upstream pathways; some patients show poor efficacy with these drugs; long-term intravenous administration is required, leading to a heavy treatment burden and higher risk of infection. Therefore, innovative drugs that can precisely regulate the complement system from its origin and provide long-acting and convenient treatment have become an urgent clinical need.

RNAi Therapy Offers Transformative New Option for Treating Complement Diseases by Silencing Pathogenic Gene Expression. SGB-3383, an RNAi candidate drug targeting CFB, has the potential to treat various diseases caused by overactivation of the alternative pathway and may reduce patients' risk of infection. Based on SANEGENEBIO’s proprietary RNAi technology platform, this drug demonstrates excellent therapeutic efficacy, prolonged effectiveness, and safety. Preclinical trial data show that SGB-3383 can efficiently and durably inhibit the expression of CFB in the liver and blood while exhibiting favorable safety. Currently, no RNAi drugs targeting the complement system have been approved globally.SGB-3383 is expected to be the first breakthrough, leveraging its outstanding potential to become a "Best-In-Class" RNAi therapy targeting CFB.。

Dr. Yuyan Jin, Senior Vice President of Clinical and Non-clinical at SANEGENEBIO, stated:“We are committed to leveraging cutting-edge RNAi technology, focusing on major diseases with large patient populations while consistently addressing the needs of patients with rare diseases that carry a heavy disease burden. This strategic approach is reflected in the positioning of this product—SGB-3383 not only offers a new treatment option for chronic kidney disease, which affects a vast number of patients, but also provides new therapeutic possibilities for rare complement-mediated diseases such as PNH and aHUS. Moving forward, SANEGENEBIO will continue to enhance its platform innovation capabilities, accelerate pipeline development, and strive to establish RNAi therapy as a standard clinical treatment across a wide range of disease areas.”