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On February 26, SANEGENEBIO announced its independently developedsiRNA Drug SGB-9768Recently received approval from New Zealand's Medicines and Medical Devices Safety Authority (Medsafe) and the Health and Disability Ethics Committee (HDEC) to conduct a Phase 1 clinical trial in New Zealand, aiming to developFor the treatment of complement-related diseasesSGB-9768 isAn RNAi Therapy Targeting Complement C3 Protein, is SANEGENEBIO's second siRNA drug to enter the clinical trial stage.
The complement system is an important component of innate immunity, playing a regulatory role in adaptive immune responses and having functions in immune surveillance and maintaining tissue homeostasis. The complement system performs crucial immune and physiological roles in the human body; however, abnormal regulation or excessive activation of the complement system can induce inflammation and damage self-tissues, causing immune injury. It is closely related to the occurrence and development of diseases in fields such as hematology, ophthalmology, and nephrology, including age-related macular degeneration (AMD), paroxysmal nocturnal hemoglobinuria (PNH), generalized myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), C3 glomerulopathy (C3G), IgA nephropathy (IgAN), and thrombotic microangiopathy (TMAs), among other immune-related diseases.The complement system comprises more than 30 soluble proteins, membrane-bound proteins, and complement receptor components, among which complement C3 is the most abundant and serves as a common pathway member in all complement activation pathways, making it a powerful therapeutic target for diseases.。SGB-9768 isA siRNA-GalNAc Conjugate Targeting Complement C3, utilizing SANEGENEBIO's uniquely innovative next-generation LEAD™ GalNAc technology for delivery to liver cells, inhibits the synthesis of C3 in the liver through RNAi. The safety, efficacy, and stability of GalNAc-delivered siRNA drugs have been extensively validated by a large amount of data. SGB-9768 can achieve a dosing frequency of once every 3 or 6 months, offering the advantages of low treatment frequency, good patient compliance, and long-lasting efficacy. Preclinical study data shows that SGB-9768 can effectively and continuously reduce C3 synthesis.The phase 1 study approved this time in New Zealand is a randomized, double-blind, placebo-controlled, single-ascending-dose study. Its primary objective is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SGB-9768 in healthy volunteers.Founder and Chief Executive Officer (CEO) of SANEGENEBIODr. Weimin WangSaid: "SGB-9768 is the first candidate drug from SANEGENEBIO to be approved for clinical trials in the field of immune-related diseases., which utilizes the company's proprietary LEAD™ GalNAc delivery platform, has demonstrated significant advantages in preclinical studies, including excellent activity, durability, and safety. We will accelerate the Phase 1 clinical trial of SGB-9768, anticipating that its outstanding research results will soon be validated and showcased. Meanwhile, the company will continue to explore the application potential of the C3 target comprehensively, advancing its development in various complement-related diseases to provide more and better treatment options for patients with immune-related disorders."[1] SANEGENEBIO's siRNA Drug Targeting Complement C3 Protein Approved for Phase I Clinical Trial in New Zealand. Retrieved Feb 26, 2024, from https://mp.weixin.qq.com/s/hgHioHWyz_VtPV882tjo6A
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