Disclaimer: Due to limited proficiency, errors are inevitable, and some information may not be the most up-to-date. Comments are welcome to point out any issues. This article is only an introduction to drugs related to healthcare and is not a recommendation of treatment plans (if involved). This article does not constitute any investment advice.
At the forefront of biomedicine, nucleic acid drugs have become a powerful weapon to tackle difficult diseases due to their unique mechanisms of action and therapeutic advantages, drawing significant attention from the global scientific community and pharmaceutical industry. In January and February of 2025, the nucleic acid drug sector continued to gain momentum, with multiple new drug clinical trial applications (INDs) approved, injecting new vitality into the field’s development and bringing hope of a cure to countless patients.On January 10, CirCode's self-developed circular RNA drug - hm2002 injection received clinical trial approval (IND) from the National Medical Products Administration (NMPA) for the treatment of ischemic heart disease.HM2002 Injection, developed based on CirCode's self-innovative technology platform, promotes angiogenesis and myocardial perfusion by stably expressing vascular endothelial growth factor in the myocardium, thereby accelerating cardiac function recovery.Compared with traditional linear RNA, circular RNA has higher stability and lower immunogenicity. This approval marks a key progress for CirCode in the development of circular RNA drugs, and also enhances China's leading position in this field.
Visirna
In January, the marketing authorization application for Visirna's liver-targeted apoc3 siRNA candidate drug — VSA001 Injection (Pulesiran Sodium) — was officially accepted by the NMPA for the treatment of familial chylomicronemia syndrome. In December 2024, the drug is proposed to be included in the NMPA’s priority review and is also in the application stage for market approval in the United States. If approved, it will become the world’s first marketed drug targeting apoc3. Additionally, Visirna has three other siRNA candidate drugs that have entered clinical stages, with VSA012 completing the first dosing in healthy subjects in January.
Ribo Life Science and Boehringer Ingelheim Achieve Milestone in CollaborationIn January, Ribo Life Science announced its collaboration withBoehringerIngehanCollaborative R&DThe first milestone has been reached. The two parties collaborated a year ago to jointly develop innovative small nucleic acid therapies for the treatment of non-alcoholic or metabolic dysfunction-associated steatohepatitis, with a total transaction value exceeding 2 billion US dollars. Ribo Life Science's core technologies include liver-targeted delivery technology, tumor-targeted delivery technology, and second-generation modification technology. It currently has 8 clinical-stage products, 5 of which are based on liver-targeted delivery technology. The achievement of this collaborative milestone has injected strong momentum into its R&D efforts in this field.
Rikang Life
On February 5, Lekang Life Technology's self-developed tumor neoantigen mRNA vaccine product —— lk101 injection successfully obtained IND approval from the U.S. FDA.This is China's first tumor neoantigen mRNA vaccine product approved by the FDA. It has previously received IND approval from the China National Medical Products Administration and has entered Phase I clinical trials, with preliminary data showing excellent safety and efficacy.LK101 Injection is an individualized neoantigen-targeted cancer vaccine that combines the advantages of mRNA and dendritic cells. It precisely identifies tumor mutations through high-throughput sequencing and AI analysis, activating the immune system to attack tumor cells.
In February, Haobo Medicine's HBV ASO candidate drug —— AHB-137 completed dosing in the Phase 2b clinical trial and officially entered the unblinding stage. In the preliminary results of the previous Phase 2a study, serum HBsAg levels rapidly decreased after treatment in all subjects following at least 12 weeks of treatment. The core technology of Haobo Medicine is MED-oligo™Multi-segment enhanced and dual-action small nucleic acid platform, achieving higher efficacy and safety through innovative chemical modifications, while also modulating immune function. The completion of this Phase 2b clinical dosing brings new hope for hepatitis B treatment.February,Ribo Life SciencePreclinical data of RBD4059, a self-developed GalNAc-conjugated siRNA candidate drug for anticoagulation/antithrombotic therapy, published in an authoritative cardiovascular journal. Existing Phase 1 clinical results show that RBD4059 demonstrates sustained FXI activity and protein reduction effects with good safety. This candidate drug is currently undergoing Phase 2 clinical trials in Europe, offering the potential for a new treatment option in anticoagulation/antithrombotic therapy.The relevant progress of nucleic acid drug IND in January and February fully demonstrates the vigorous development and unlimited innovation potential in this field. From heart disease to cancer, from rare diseases to infectious diseases, every approval and significant progress signifies a solid step forward in the journey to conquer major diseases.Reference: Public Information
