An isogenic iPSC and AI-enabled disease modeling platform spanning multiple genetically defined CMT subtypes will be established to support patient stratification, accelerate RTX-117 development, and create a shared foundation for next-generation therapies
BRISBANE, Calif., April 21, 2026 /PRNewswire/ — ReviR Therapeutics today announced a strategic investment from CureCMT to support the launch of a precision medicine platform for Charcot-Marie-Tooth disease (CMT). The platform will combine isogenic induced pluripotent stem cell (iPSC) models spanning multiple genetically defined CMT subtypes with AI-driven drug perturbation modeling to advance patient stratification, accelerate development of RTX-117, and support future targeted therapies.
CMT represents a highly heterogeneous group of inherited neuropathies, and therapeutic development has historically been complicated by substantial genetic and biological diversity. There remains no centralized, scalable, and subtype-resolved cellular resource capable of systematically evaluating therapeutic responses across the breadth of CMT biology, making clinical development and patient selection particularly challenging.
To address this gap, ReviR and CureCMT are collaborating to build a comprehensive platform centered on a first-of-its-kind panel of isogenic iPSC models across genetically defined CMT subtypes, while generating perturbation data to train computational models. The platform will also incorporate disease-specific virtual cell models to predict responses to RTX-117 across genotypes and ultimately guide patient selection in the clinic.
Unlike prior efforts, the ReviR-CureCMT collaboration is designed to serve as a foundational resource for the field and will be made broadly available to the global CMT community. The isogenic iPSC panel will enable side-by-side comparison of disease phenotypes and drug responses across mutations, providing a valuable tool for rational trial design, biomarker discovery, computational modeling of therapeutic response, and development of next-generation therapies. The platform will create a shared infrastructure for academic investigators, biotechnology companies, and clinical researchers to interrogate disease mechanisms, predict drug response in silico, validate targets, and develop tailored therapeutic strategies for specific CMT patient populations. As the platform expands across a broader range of CMT subtypes, the accompanying AI models are expected to grow in predictive power and utility, helping to accelerate the broader therapeutic development process.
As the lead therapeutic application of the platform, RTX-117 is expected to directly benefit from its disease modeling and patient stratification capabilities. RTX-117 is ReviR’s investigational therapy designed to modulate the integrated stress response (ISR) through activation of eIF2B, restoring translational homeostasis in cells experiencing chronic stress. Dysregulation of the ISR has been implicated in multiple CMT subtypes, including those associated with tRNA synthetase mutations and mitochondrial dysfunction. By normalizing this pathway, RTX-117 has the potential to address a fundamental disease mechanism across genetically distinct forms of CMT.
“This collaboration represents a decisive step toward precision medicine approaches in CMT,” said Paul August, PhD, Chief Scientific Officer of ReviR Therapeutics. “RTX-117 targets a core cellular stress pathway that appears to be convergent across multiple CMT subtypes. The deployment of isogenic iPSC models, together with virtual cell models, not only strengthens our clinical strategy but also establishes a durable framework for future therapeutic development.”
“CureCMT is committed to transforming how therapies are developed for patients with CMT,” said Patrick Livney, Chief Executive Officer of CureCMT. “Our goal is to build that resource in partnership with ReviR and make it accessible to the entire CMT community. We believe this will catalyze a new wave of innovation, enabling not only transformative therapies like RTX-117 but also future targeted therapies to reach patients more efficiently. CureCMT is distinguished by its comprehensive and patient-centered approach, integrating therapeutic development with surgical innovation, bracing solutions, and functional outcomes research. This investment reflects the foundation’s commitment to advancing a full spectrum of interventions that can meaningfully improve the lives of patients.”
“We are honored to partner with CureCMT, an organization that brings both strategic vision and urgency to the field,” said Peng Yue, PhD, Chief Executive Officer of ReviR Therapeutics. “What excites us most is the convergence of isogenic disease modeling and AI. By generating perturbation data across genetically defined CMT subtypes, we are building the foundation for an AI platform that can predict drug response before we enter the clinic. This not only accelerates RTX-117 development but also creates a shared, openly accessible resource that can benefit the broader CMT ecosystem for years to come.”
By integrating precision disease models with clinical development, the collaboration aims to redefine the therapeutic paradigm for CMT, moving from a one-size-fits-all approach to one grounded in genetic and mechanistic specificity, while establishing a scalable model for future therapeutic development in other genetically heterogeneous diseases.
About CureCMT
CureCMT is a leading patient-driven foundation dedicated to advancing comprehensive solutions for Charcot-Marie-Tooth disease. Through strategic investments, collaborative research initiatives, and a commitment to therapeutic, surgical, and supportive care innovation, CureCMT is working to deliver meaningful improvements in outcomes and quality of life for patients.
About ReviR Therapeutics
ReviR Therapeutics is a global biotechnology company committed to discovering and developing innovative therapies for patients with serious genetic diseases. ReviR was founded in 2021 by leaders in computational biology, RNA biology, genomics, and drug discovery, with the aim to create disease-modifying therapies that are highly specific, efficacious, and safe. ReviR’s lead candidate, RTX-117, is an investigational small molecule therapy for Charcot-Marie-Tooth disease (CMT)/Vanishing White Matter disease (VWM). In addition, ReviR’s pipeline includes other orally administered small molecules designed to modulate RNA function by splicing. For more information about ReviR Therapeutics, visit www.revirtx.com.
About RTX-117
RTX-117 is a potential first-in-class, orally administered small molecule therapy developed by ReviR Therapeutics, with discovery and molecular design powered by XtalPi’s AI and robotics drug discovery platform. The therapy targets the Integrated Stress Response (ISR) pathway by activating eIF2B, a key protein translation initiation factor. By restoring protein synthesis homeostasis and improving neuromuscular function, RTX-117 aims to address the underlying molecular pathology of related genetic disorders. The candidate is currently undergoing a Phase 1 clinical trial, with Investigational New Drug (IND) clearance already secured for both Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM).
