Domestic AI Pharma Scores Breakthrough as First-in-Class "AI+RNA" Small Molecule Drug RTX-117 Doses First Patient

　　【Pharmaceutical Network Industry DynamicsIn recent years, AI pharmaceuticals have been on the rise. Data shows that as of December 2025, there are over 350 AI pharmaceutical companies globally, including no fewer than 100 in China, with more than 10 having entered the growth and maturity stages. Recently, the AI pharmaceutical sector has received more good news.
 

On March 2, ReviR Therapeutics (hereinafter referred to as "ReviR"), an incubated company of XtalPi, announced that the first subject has been dosed in its small molecule drug pipeline RTX-117. This marks the official entry into the clinical development stage for RTX-117, a small molecule drug discovered through the collaboration between XtalPi and ReviR using “AI+RNA” technology, which is expected to provide therapeutic options for patients with neurological disorders such as Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM).
 

It is reported that RTX-117, combined with XtalPi's AI+RobotThe drug discovery platform, combined with ReviR's deep understanding of RNA biology, has led to the collaborative discovery, design, and optimization of a small-molecule candidate drug that intervenes in disease pathogenic pathways at the molecular level. This showcases the efficiency of XtalPi's AI drug discovery platform and ReviR's strong pipeline advancement capabilities.
 

RTX-117 restores normal mRNA translation by inhibiting the abnormally activated ISR pathway, achieving therapeutic effects by targeting the molecular root cause of diseases. This pathway is not only crucial for treating neurological disorders such as Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM), but also considered closely related to various other neurological conditions including Amyotrophic Lateral Sclerosis (ALS), Parkinson’s disease (PD), Alzheimer’s disease (AD), and brain injuries. With the steady progress of its Phase I clinical trials, RTX-117 is expected to expand its application to more indications in the future.
 

XtalPi Strengthens Its Position as an AI-Driven Drug Discovery Leader through High-Impact Collaborations and Clinical TranslationXtalPi is reinforcing its advantages and commercial value as an AI-powered pharmaceutical company by actively participating in and effectively advancing innovation breakthroughs and clinical translation in high-value therapeutic pipelines. Recently, XtalPi announced partnerships with Yaotang Biotech, JW Pharmaceutical, and VISEN Pharmaceuticals to explore the application of AI across various stages of drug discovery. Earlier, Dongyang Guangya Pharmaceutical (DGY) established a joint venture with XtalPi, planning to invest hundreds of millions of yuan in building an “AI + Robotics” joint laboratory. A representative from DGY stated that the strategic partnership between DGY and XtalPi, formed through a joint venture rather than a simple project licensing agreement, aims to establish a long-term, deeply integrated technology symbiosis. "Traditional project collaborations are often limited to single-point technology applications, whereas a joint venture enables full-chain synergy across 'data-model-pipeline-commercialization,' creating a closed-loop iterative AI-driven drug discovery engine."
 

Some institutions have indicated that under the traditional model, developing a new drug from discovery to market typically takes over a decade and requires at least $1 billion in investment. However, with the deep involvement of AI technology, this landscape is quietly changing: "AI models can reduce compound design time by 70% and increase success rates tenfold." Another institution also stated that AI can cut the R&D costs from target to lead compound stage from $94 million to $200,000, saving over 90%; the R&D cycle can be shortened from 1 year to 2 months, improving efficiency by 83%.
 

At the same time, improving operational efficiency while ensuring scientific rigor and deepening innovation capabilities is also a common challenge faced by pharmaceutical companies today. The application of AI in this field is evolving from being an "efficiency tool" to a "scientific tool." Industry experts indicate that the innovative value of AI lies in its ability to "Think out of the box." In traditional research and development pathways, compound design is limited by existing rules and building block libraries, whereas AI, through models such as generative adversarial networks and reinforcement learning, can achieve data-driven autonomous molecular design and generation.
 

The preclinical data and clinical approval speed of the aforementioned RTX-117 have both exceeded the expectations of both parties, once again demonstrating the efficiency of XtalPi's AI drug discovery platform and ReviR's strong pipeline advancement capabilities. In the successful development of RTX-117, XtalPi's AI drug discovery platform showcased its drug design capabilities. Combined with ReviR’s VoyageR AI platform and its deep insights into underlying disease mechanisms, this collaboration holds promise for achieving more efficient RNA translation modulation and innovative target discovery.
 

Disclaimer: Under no circumstances shall the information or opinions expressed in this article constitute investment advice to any person.