十年长跑，小核酸药物迎来“中国时刻”

Core Viewpoint - The article emphasizes the importance of transforming scientific research into practical applications, highlighting the journey of scientists who become entrepreneurs in the field of technology innovation, particularly in Jiangsu province [1]. Group 1: Scientific Breakthroughs - Zhang Chenyu's discovery of "extracellular small RNA" has opened a new research field in life sciences, recognized as a paradigm shift in biological research by Nature magazine [2]. - The ER2001 drug represents a significant advancement in gene therapy, being the first small nucleic acid drug to deliver to the central nervous system via intravenous injection [4][5]. Group 2: Clinical Trials and Innovations - The ER2001 clinical trial for Huntington's disease is notable for being the first of its kind conducted outside Europe and the U.S., utilizing innovative methods to overcome the blood-brain barrier [3]. - Initial results from the ER2001 trial show significant improvements in patients' motor functions and cognitive abilities, indicating a potential breakthrough in treatment [9][10]. Group 3: Commercialization and Investment - The establishment of Aima Biotechnology (Nanjing) Co., Ltd. was a pivotal moment, with the company securing 162.6 million yuan in contracts for patent transfers from Nanjing University [6]. - The collaboration with Dinghui Investment was crucial for funding, driven by the publication of key research papers that attracted investor interest [11]. Group 4: Strategic Partnerships - The partnership with top medical institutions facilitated the rapid progression of ER2001 into clinical trials, showcasing the importance of building a clinical resource network [12]. - The combination of scientific expertise and management experience within the team has been essential for navigating the complexities of drug development and commercialization [8].