Antaisens Therapeutics Completes RMB 20 Million Pre-A Funding Round to Advance Global Leadership in Oligonucleotide Drug Development

Recently, ASOcura (hereinafter referred to as "ASOcura" or "the Company") has completed a pre-A round of financing of 20 million yuan. This round of financing was exclusively invested by HeRui Venture Capital. The funds will be used for new product development, clinical trials, and registration applications.

ASOcura, established on April 1, 2017, is located in Suzhou Industrial Park. The company is the first in China to target RNA splicing and RNA editing for the development of antisense oligonucleotide (ASO) drugs and owns several of the latest international ASO technologies. The company has multiple projects underway, with significant progress already made in drug development for diseases such as DMD (Duchenne Muscular Dystrophy) and SCA3 (Spinocerebellar Ataxia Type 3). The founder of the company, Dr. Yimin Hua, is a world-leading scientist in the field of ASO drugs and one of the principal inventors of Nusinersen (marketed as Spinraza), the most globally sold small nucleic acid drug. Building on existing technologies, the company has optimized and upgraded its platform, pioneering new-generation ASO-based drug development technologies including 5D-ASO, which intervenes in RNA splicing, and ANISPR, a novel ASO that induces RNA editing.

In 2020, impacted by the COVID-19 pandemic, nucleic acid-based biopharmaceutical technologies, represented by mRNA vaccines, developed rapidly. Small nucleic acid drugs also gradually expanded in scale globally. According to relevant materials, the global ASO drug market is expected to reach 64.64 billion US dollars by 2030. ASO, as the most mature technological route among small nucleic acid drugs, represents one of the development directions of RNA-targeted drugs.

Effective delivery of oligonucleotide therapies to various tissues remains a major challenge. Oligonucleotides are generally large, hydrophilic poly-anions (single-stranded ASOs are approximately 4–10 kDa, double-stranded siRNAs are about 14 kDa), which means they do not easily pass through the plasma membrane. So far, almost all approved nucleic acid drugs on the market have focused on local administration (e.g., eyes) or delivery to the liver, while the first blockbuster nucleic acid drug, nusinersen, is delivered to the spinal cord via lumbar puncture. Professor Yimin Hua, founder of ASOcura, is the inventor of the splicing-switching ASO technology that promotes the inclusion of SMN2 exon 7, and was a key scientist in the preclinical development of nusinersen. In SMA patients, mutations in the SMN1 gene prevent it from expressing SMN protein. Fortunately, humans also have the SMN2 gene, which can produce a small amount of SMN protein. However, under normal circumstances, during the splicing of SMN2 pre-mRNA, exon 7 is excluded (normal SMN1 retains exon 7), resulting in a truncated, non-functional, and unstable protein. Nusinersen ensures that SMN2 mRNA retains exon 7, allowing for the translation of normal SMN protein and achieving therapeutic effects. The company's next-generation technology can enhance ASO efficiency by more than five times, bringing hope to the development of ASO drugs for extrahepatic tissue diseases, especially rare diseases.

Dr. Huayimin Hua, the founder of the company, obtained his Ph.D. from Sun Yat-sen University in 1998. He went to the United States in 2000 to conduct research on Spinal Muscular Atrophy (SMA). In 2015, he returned to China full-time as a Distinguished Professor and doctoral supervisor at Soochow University. In 2017, he founded ASOcura. Dr. Hua has published over 40 high-standard papers in top international journals such as Nature and Science, with more than 6,000 citations. While working at Cold Spring Harbor Laboratory in the U.S., he invented Spinraza, an antisense nucleic acid drug for treating SMA. The drug was approved by the U.S. FDA on December 23, 2016, launched in China in early 2019, and currently generates annual sales of approximately 2 billion US dollars. Over the years, Nusinersen has saved the lives of thousands of children with SMA worldwide.