Nucleic Acid Therapeutics 'Circle of Friends' Expands: Innovent, BeyondSpring, CSPC, and Ractigen Report Breakthroughs in July | Latest Updates to Be Showcased at August Nucleic Acid Drug Development Forum in Suzhou

The development of nucleic acid drugs has a history of several decades, with a tumultuous journey. In recent years, they have gained widespread recognition due to the "explosive popularity" of mRNA vaccines for COVID-19. However, even before that, nucleic acid drugs had already been applied in the fields of rare diseases and complex conditions. The 2023 Nobel Prize in Physiology or Medicine once again affirmed the potential of nucleic acid drugs, marking a "highlight moment" for this class of drugs. With their significant efficacy and shorter development cycles, nucleic acid drugs are becoming a focal point in clinical medication and new drug research. As of July 2024, multiple companies have made further progress!

July 25,Ractigen TherapeuticsRactigen Therapeutics announced that its self-developed small activating RNA (saRNA) drug RAG-18 has received the Pediatric Rare Disease Designation (RPDD) from the U.S. FDA for the treatment of Duchenne Muscular Dystrophy. RAG-18 is a first-in-class double-stranded saRNA drug that specifically targets and activates the expression of the UTRN gene in muscle cells through the RNA activation (RNAa) mechanism.

On July 18, according to the announcement on the CDE official website,Afana Bio and Anke BioThe IND application for AFN0328 Injection, a Class 1 new drug jointly developed, has been approved. It becomes the second HPV-related tumor mRNA therapeutic vaccine in China to be approved for clinical trials, and also the second mRNA product of Afana Bio to receive clinical trial approval this year.

July 11,Jingyin PharmaAnnounces the successful completion of dosing for all subjects in the Phase I clinical trial of its self-developed small nucleic acid drug (SRSD107 injection). SRSD107 injection is a double-stranded small interfering nucleic acid (siRNA) drug with independent intellectual property rights developed by Jingyin Pharmaceuticals. It specifically targets liver-directed coagulation factor XI (FXI) mRNA, inhibits FXI protein expression, and blocks the activation of the intrinsic coagulation pathway, thereby achieving anticoagulant/antithrombotic effects.

On July 10, according to the announcement on the CDE official website,Giantstone Biologics (Shiyao Group)The 1.2 class new drug RSV mRNA vaccine has obtained clinical tacit permission for the prevention of lower respiratory tract diseases caused by respiratory syncytial virus (RSV) infection.

On July 8, according to the announcement on the CDE official website,Innovent Biologics and SinoGenThe small nucleic acid (siRNA) drug SGB-3908 (IBI3016) injection, co-developed for clinical use, has been approved for clinical trials for the treatment of primary hypertension. SGB-3908 is an siRNA drug targeting AGT, developed based on Saint Biotech's proprietary small nucleic acid drug development platform LEAD™ (Ligand and Enhancer Assisted Delivery).

July 1,ArgoAnnounced that the first dosing of BW-03, a self-developed siRNA new drug for chronic hepatitis B, has been completed in China's Phase 1 clinical trial on June 27, 2024.(The above report is a comprehensive compilation from: Pharma Intelligence New Drug Morning Report, may not be entirely exhaustive, feel free to add more in the comment section!)

For current trends and challenges in the nucleic acid drug industry, as well as more cutting-edge advancements and technological progress, we welcome you to register for the 2024 Biomedical Innovation Expo.Frontiers in Nucleic Acid Drug Development Forum！

*Schedule updating, subject to the conference site.

09:00-09:20 mRNA Vaccines and mRNA Drugs

Hui Aimin Hui Zhengqi Medicine, Founder

09:20-09:40 Regulatory Challenges in the Development of Oligonucleotide Drugs

09:40-10:00 Preclinical Pharmacokinetic Studies of Small Nucleic Acid Drugs

Liu WenYue Huaxi Haiqi, Chief Technology Officer of Pharmacokinetics

10:00-10:20 Delivery and Clinical Progress of Small Interfering RNA Drugs

Yang XianbinChief Sales Officer (CSO) of Greater China, Sirnaomics; General Manager of Suzhou, China Headquarters, Sirnaomics

10:20-10:40 Sources of Impurities in Oligonucleotide Drugs

Feng Bing  Zhong Peptide Biochemical,Senior Director of Oligonucleotide Research and Development

10:40-11:00 Innovation Practice of CSPC Group in Small Nucleic Acid Drugs

Xiaoye Su  Head of Nucleic Acid Drug Research Institute, CSPC Group

11:00-11:20 Nucleosidic phosphoramidites: an optimized approach in their synthesis, quality evaluation and impurities characterization

Beatrice Trucchi Flamma S.p.A. - Italy R&D Project Manager

11:20-12:00 BH-RACETM New Nucleic Acid Drug Platform

Hu Han  BinHui Bio, Vice President

12:00-13:30 Lunch

13:30-13:50 Delivery and Clinical Progress of Small Interfering Nucleic Acid Drugs

Yuan Xudong Aikang Pharmaceuticals, CEO

13:50-14:10 Novel siRNA Molecular Design for Treating Liver and Lung Diseases

Huang Yi  Guowei Pharmaceuticals, Vice President of the Research Institute

14:10-14:30 Quanhecheng Nucleic Acid Drug Delivery Platform

Song Yanmin Quan Hecheng, Chairman

14:30-14:50 Development of Long-Acting Small Nucleic Acids for Chronic Disease Cases(Tentative, subject to change based on the actual event)

Wanjin Bridge Xianyan Bio, General Manager

14:50-15:10 AI-Driven Efficient mRNA Vaccine Development

Caiyi FeiChengshi Biotech, Head of Nucleic Acid Drug Research and Development

15:10-15:30 Dual-Target Nucleic Acid Aptamer Technology

Wang Xueqiang Hangzhou Institute of Medicine, Chinese Academy of Sciences, Researcher

15:30-15:50 Challenges and Case Studies in DMPK Research of Oligonucleotide Drugs

Guiying Chen  Director of Bioanalysis Department, Chemistry Drugs, Wuhan Hongren Biology

*The ranking of guests is不分先后, according to the initial letter of their names from A to Z.