晶泰科技赋能国内首个"AI + RNA"小分子创新药获批临床，ReviR溪砾科技实现罕见病首创新药突破

Core Viewpoint - ReviR, a company incubated by JingTai Technology, has received approval from the National Medical Products Administration (NMPA) for its small molecule pipeline RTX-117, aimed at treating Charcot-Marie-Tooth disease (CMT), with Phase I clinical trials set to begin in Q1 2026 [1] Group 1: Company Developments - RTX-117 is the first Class 1 innovative drug in China targeting CMT and is the first project to enter clinical trials from a series of rare disease drug pipelines developed through AI and robotic-assisted research [1][3] - JingTai Technology will receive milestone payments and has the right to participate in revenue sharing from sales and subsequent licensing of the RTX-117 pipeline [1] - The drug has already received IND approval and orphan drug designation from the FDA, allowing for priority review and extended market exclusivity [1] Group 2: Market Potential - CMT is listed as the 17th rare disease in the first batch of rare diseases published by the National Health Commission in 2018, with a global prevalence of 17.7 to 40 per 100,000, affecting over 2.6 million patients [2] - The global CMT treatment market is projected to grow from $1.013 billion in 2024 to $11.394 billion by 2035, reflecting a compound annual growth rate (CAGR) of 24.62% [2] - The median age of onset for CMT patients is 7.3 years, with a long diagnostic cycle and a lack of effective treatment options, indicating a significant unmet clinical need [2] Group 3: Innovation and Technology - RTX-117 targets the key physiological mechanisms causing CMT by inhibiting the Integrated Stress Response (ISR) pathway, showing significant preclinical efficacy in restoring motor function in model animals [4] - The development of RTX-117 exemplifies the potential of AI and RNA modulation in accelerating breakthroughs in rare disease therapies [3][4] - JingTai Technology's platform integrates quantum physics, AI, and large-scale robotic experimentation, establishing a leading automated drug development infrastructure [6] Group 4: Industry Challenges and Opportunities - The global rare disease market is characterized by a significant commercial opportunity, with only 5% of the over 7,000 identified rare diseases having clear treatment options [5] - Domestic rare disease patients face challenges such as high costs and low accessibility of imported drugs, highlighting the need for innovative solutions [5] - The AI-driven approach to drug development is expected to accelerate the creation of clinically valuable and marketable therapies, providing new opportunities in the rare disease treatment landscape [5]