-FDA Grants Orphan Drug Designation to SGB-9768 for C3 Glomerulopathy, a rare and devastating kidney disease-
-SGB-9768 has the potential to transform the treatment landscape of C3G and offer a new option to patients-
Boston, Suzhou & Shanghai – October 21, 2025 – SanegeneBio, a clinical-stage RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SGB-9768 for the treatment of C3 Glomerulopathy (C3G). As a novel RNAi therapy, SGB-9768 may significantly improve the lives of C3G patients with a subcutaneously-administered safe and effective medicine that can be dosed as infrequently as twice yearly.
SGB-9768 is an experimental RNAi therapeutic targeting complement C3 for the treatment of complement-mediated kidney diseases.
FDA ODD Accelerates Efforts to Address Unmet Clinical Needs
ODD is an FDA policy established under the Orphan Drug Act to encourage the development of drugs for the treatment of rare diseases or conditions. Under this policy, companies receiving ODD are eligible for various incentives, including tax credits, FDA assistance in clinical trial design, waiver of certain application fees, and up to seven years of market exclusivity upon drug approval. This milestone marks a significant step forward in the development of SGB-9768 for the treatment of a rare disease and is expected to accelerate its clinical development and regulatory progress, ultimately benefiting C3G patients in urgent need of effective treatments.
C3G is a rare kidney disease with a global incidence of 1-2 per million people per year, primarily affecting young adults. Approximately 50% of patients progress to end-stage renal disease within 10 years, requiring dialysis or kidney transplantation; post-transplantation, the disease recurs in 50%-70% of patients. Although rare, C3G is a severe progressive disease that significantly threatens both patient survival and quality of life. The primary cause of the disease is overactivation of the complement system. Previous supportive care and immunosuppressive therapies have shown limited efficacy. As an emerging technology capable of silencing disease-causing genes, RNAi therapeutics offer a mechanism that intervenes at the root cause, potentially providing a new option for C3G.
Potential for a New Therapeutic Approach; Phase II Clinical Studies Progressing
Developed using SanegeneBio’s proprietary GalNAc platform, SGB-9768 delivers RNAi specifically to hepatocytes to suppress C3 expression, thereby downregulating complement activation. Preclinical and Phase I data presented at the 2024 Complement-Based Drug Development Summit demonstrated:
- Robust, dose-dependent, and sustained reduction in C3 levels following a single subcutaneous dose
- Superior C3 gene silencing potency and durability compared to benchmark siRNAs
- Favorable safety profile and greater durability
Beyond its development for C3G, SGB-9768 has potential in other complement-mediated diseases with larger patient populations, such as IgA nephropathy (IgAN) and Immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). The drug is currently in multi-center Phase II clinical trials.
Dr. Weimin Wang, Founder and CEO of SanegeneBio, stated: “The FDA’s Orphan Drug Designation for SGB-9768 is a significant recognition of its innovative mechanism and therapeutic potential, and we believe it can address the urgent medical needs of C3G patients. Given the long-standing limited treatment options, we are committed to leveraging our cutting-edge RNAi technology to develop potentially transformative medicines. Moving forward, we will advance the clinical development of SGB-9768, aiming to bring new hope to patients as soon as possible.”
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.