Rona Therapeutics' siRNA Candidate Drug RN0361 Targeting APOC3 Receives FDA IND Approval, Phase 2 Clinical Trial to Commence.
LongRiver Jiangyuan Investment led the A+ round and consecutively invested in Rona Therapeutics for two rounds, supporting its advancement in innovative nucleic acid drug development.
Rona Therapeutics, a global leader in nucleic acid technology in the RNA-targeted therapeutics field, announced that its small interfering RNA (siRNA) candidate drug RN0361 targeting APOC3 has received Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) and will initiate Phase 2 clinical trials.RN0361 is a potential best-in-class small interfering nucleic acid drug (siRNA) targeting APOC3. It utilizes Rona Therapeutics' proprietary GalNAc-siRNA conjugation and oligonucleotide chemistry platform GAIA to achieve potent and long-lasting liver cell-specific delivery and silencing via subcutaneous injection. This therapy achieves durable gene silencing through optimized chemical modifications, significantly inhibiting APOC3 mRNA expression and reducing APOC3 protein levels.The drug is intended for the treatment of severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS).Based on the favorable safety profile and durable triglyceride (TG)-lowering effects observed in a 6-month single-dose Phase 1 clinical trial, Rona Therapeutics is poised to initiate a randomized, double-blind, placebo-controlled global multicenter Phase 2 trial. Over a 9-month follow-up period, the company will evaluate the efficacy, safety, and durability of RN0361 in patients with hypertriglyceridemia.Rona Therapeutics founder and CEO Yibin Shi said,The approval of this IND reflects the company's commitment to developing transformative RNAi therapies for patients with severe dyslipidemia. The Phase 1 data of RN0361 validates its potential as the best-in-class small interfering nucleic acid drug (siRNA), combining long-lasting efficacy with convenient dosing advantages. Rona Therapeutics looks forward to collaborating with global researchers and suppliers to accelerate the development of this drug.Alex M. DePaoli, MD, Chief Medical Officer and Executive Vice President of Translational Medicine at Rona Therapeutics, stated,The Phase 2 study of RN0361 will further validate its efficacy in patients with metabolic abnormalities associated with hypertriglyceridemia, offering a potential therapeutic tool for this group with significant unmet needs. The potent and durable lipid-lowering effects observed in the single-dose first-in-human study have laid a solid foundation for subsequent development.Rona Therapeutics is a global leading nucleic acid innovative drug platform company, focusing on the treatment of metabolic diseases and nervous system diseases.Rona Therapeutics is dedicated to developing best-in-class and first-in-industry siRNA drugs with differentiation and innovation to address unmet clinical needs and improve treatment outcomes for cardiovascular diseases, obesity, and metabolic disorder-associated nonalcoholic steatohepatitis (NASH).In addition, Rona Therapeutics is fully committed to unlocking the enormous potential of extrahepatic delivery technology for neurological diseases and adipose-muscular delivery targeting various metabolic syndrome treatments.
AboutLongRiverJiangyuan Investment
LongRiver Jiangyuan Investments (LongRiver Investments) isA long-term investment institution focused on technological innovation. The term of its first U.S. dollar fund is 10 years, with an AUM of approximately $400 million. The investors are all international large financial groups and professional asset management institutions. The team mainly focuses on two major directions: healthcare and technology, primarily investing in industries such as biopharmaceuticals, medical devices, new energy, new materials, information technology, and advanced manufacturing, covering stages from early to later growth phases.
Founder and CEO Zhang Jiang is a senior technology investor.With 20 years of experience in R&D, strategic development, operations management, and investment in the global healthcare and information technology sectors. HePreviously worked at a global leading integrated financial group, strategic consulting firm, European technology giant, and state-owned energy and power industry group. Over the past decade, Zhang Jiang has led his team in raising multiple US dollar and RMB funds, investing in more than 60 healthcare and technology companies worldwide, nearly 20 of which have been listed on stock exchanges within and outside of China.
The investment and management experience of large insurance groups, the R&D and operational accumulation in the technology industry, and a global perspective have endowed LongRiver Jiangyuan Investment with comprehensive capabilities of "finding, understanding, assisting, and exiting." In the future, LongRiver Jiangyuan Investment will leverage the innovative advantages of emerging platforms, connect with new drivers of global industrial development, and support and cultivate the next generation of industry leaders.
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