On Feb. 26, 2024, Sanegene Bio Inc. (SanegeneBio) announced that its siRNA drug SGB-9768 for the treatment of complement mediated diseases has recently been approved by the New Zealand Medicines and Medical Devices Safety Authority (Medsafe) and the Health and Disability Ethics Committee (HDEC) to conduct Phase I clinical trial in New Zealand. SGB-9768 is a RNAi drug targeting the complement C3 (C3) protein, and is SanegeneBio's second siRNA drug to enter into clinical development.

The complement system is an important component of innate immunity. It regulates the adaptive immune response, and plays a role in immune surveillance and maintaining homeostasis. Complement plays an important role in the immunological and physiological functions in the human body. However, dysregulation or overactivation of the complement system can induce inflammation and destroy self-tissues, causing immune damage, which is closely related to the occurrence and development of some diseases in hematology, ophthalmology and nephrology, such as age-related macular degeneration (AMD), paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), C3 Glomerulopathy (C3G), IgA Nephropathy (IgAN), thrombotic microvascular disease (TMAs), and other immune related diseases. The complement system consists of over 30 soluble proteins, membrane-bound proteins, and complement receptor components, of which C3 is the most abundant component. It is the convergence of all complement activation pathways and therefore a potential potent therapeutic target in the relevant diseases. Currently, there is only one approved drug targeting C3 globally, indicating a huge unmet clinical need in this area.

SGB-9768, an siRNA-N-acetylgalactosamine (GalNAc) conjugate targeting C3, is delivered to liver using SanegeneBio's novel LEAD™ GalNAc platform to reduce production of complement component 3 through RNA interference. The advantages of GalNAc platform have been also fully verified, including safety, effectiveness, and stability. SGB-9768 can be administered every 3 or 6 months, with the advantages of low dosing frequency, good patient compliance, and long-term efficacy. SGB-9768 can continuously reduce C3 synthesis and has been shown in preclinical studies with superior efficacy than benchmark compounds, showing its potential to be the best-in-class siRNA drug targeting C3.

The Phase I, randomized, double-blind, placebo-controlled, and single-dose escalation study is designed to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic characteristics of SGB-9768 in adult healthy volunteers.

"SGB-9768 is SanegeneBio's first siRNA drug in the field of immune related diseases to enter into the clinical development, using our proprietary LEAD™ GalNAc delivery platform, and has demonstrated excellent potency, duration, and safety in the preclinical studies. We will accelerate the phase I clinical trial of SGB-9768 and look forward to the verification and demonstration of the excellent compound in clinical as soon as possible. At the same time, SanegeneBio will continue to deeply explore the full potential of C3 program, push forward its development in various complement mediated diseases, and provide more and better treatment options for patients with immune related diseases." said Dr. Weimin Wang, Founder and Chief Executive Officer of SanegeneBio.

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 two 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.