ReviR Therapeutics and CureCMT Forge Strategic Alliance to Build World’s First iPSC + AI Virtual Cell Precision Medicine Platform for Charcot-Marie-Tooth Disease

On April 21, 2026, ReviR Therapeutics (hereinafter referred to as "ReviR Therapeutics") announcedStrategic Collaboration with CureCMT Foundation to Jointly Develop the World’s First AI Precision Medicine Platform for Charcot-Marie-Tooth Disease (CMT)The platform will integrate isogenic induced pluripotent stem cell (isogenic iPSC) disease models with AI Virtual Cell (AIVC) technology, enhancing the precision of patient stratification while accelerating the development of the core pipeline RTX-117, laying the foundation for personalized treatment in this disease area.

CMT is a group of peripheral nervous system disorders with high genetic heterogeneity, and its complex biological diversity has long been a major obstacle in drug development. The lack of standardized resources capable of systematically evaluating treatment responses across different subtypes further complicates the challenge.Patient screening and efficacy evaluation in clinical development face significant challenges.。To address this pain point, ReviR Therapeutics and CureCMT will combine their resources to jointly buildThe World's First AI Precision Medicine Platform for CMTThe platform will integrate isogenic induced pluripotent stem cell (iPSC) models of various CMT subtypes obtained through CRISPR/Cas9 gene editing. AI virtual cell (AIVC) models based on gene perturbation data will be developed to predict the responses of patients with different genotypes to RTX-117, providing scientific guidance for precise clinical medication.

This collaboration aims toTo build a set of underlying infrastructure for the entire industry, and will be open to the global CMT research community,As a shared resource to empower academic institutions, biotechnology companies, and clinicians to explore disease mechanisms, validate drug responses and targets in a computer-simulated environment, and provide customized strategies for the CMT patient population.。As the platform covers an increasingly wide range of subtypes, the predictive performance of AI models will also continue to iterate, driving a leap in R&D efficiency across the entire CMT field.

The platform's modeling and patient stratification capabilities will be first applied to ReviR Therapeutics' core pipeline, RTX-117.RTX-117 is a potential "First-in-class" oral small molecule therapy that modulates the integrated stress response (ISR) by activating the key protein translation initiation factor eIF2B, restoring normal mRNA translation and reestablishing protein expression homeostasis to achieve therapeutic effects. Given the widespread dysregulation of ISR in various subtypes of Charcot-Marie-Tooth disease (CMT), such as those related to aminoacyl-tRNA synthetase mutations and mitochondrial dysfunction, RTX-117 has the potential to become a core therapy addressing multiple CMT subtypes. Currently, this pipeline has received clinical trial approval (IND) from the U.S. FDA and obtained orphan drug designation (ODD). It has also been granted clinical approval by China's NMPA for Charcot-Marie-Tooth disease (CMT)/Vanishing White Matter disease (VWM) and is currently in Phase I clinical trials.