New York, New York, September 23, 2026
Apertura Gene Therapy announced that it will provide Lexeo Therapeutics with access to its TfR1 CapX™ AAV capsid for a program being developed to address Friedreich ataxia (FA), a rare genetic and progressive multisystem disorder. The collaboration gives Lexeo access to a next-generation gene-delivery technology designed for intravenous administration and blood-brain barrier (BBB) crossing, potentially expanding approaches for delivering genetic medicines to the central nervous system (CNS). The companies are evaluating the technology in the context of Lexeo’s broader Friedreich ataxia development strategy, including its existing LX2006 gene-therapy program.
TfR1 CapX Designed for CNS Gene Delivery
TfR1 CapX is a proprietary, second-generation adeno-associated virus (AAV) capsid engineered to target human transferrin receptor 1 (TfR1). According to Apertura, the capsid is designed to use TfR1-mediated transport to cross the blood-brain barrier following intravenous administration and deliver therapeutic genetic cargo throughout the brain and spinal cord. Apertura describes the technology as an evolution of its first-generation BI-hTFR1 capsid, with preclinical research supporting improved CNS delivery. The company’s platform is being developed specifically around human-relevant receptor biology with the goal of enabling systemic delivery of genetic medicines to neurological tissues. The ability to deliver gene therapies to the CNS following intravenous administration is an important area of research because many neurological disorders affect tissues that are difficult to reach with conventional systemic delivery. Apertura said TfR1 CapX has been supported by preclinical development programs, regulatory engagement, and manufacturing activities with contract development and manufacturing organizations (CDMOs). The company has also reported that multiple organizations have validated and licensed the technology.
Lexeo Expands Friedreich Ataxia Gene-Therapy Strategy
For Lexeo Therapeutics, access to TfR1 CapX adds another potential CNS-delivery approach to its growing Friedreich ataxia research program. Lexeo is developing LX2006, a gene-therapy candidate intended to address the underlying genetic basis of Friedreich ataxia by delivering a functional copy of the FXN gene. The company has been exploring strategies that could complement systemic administration of LX2006 by providing additional approaches for delivering therapeutic material to the CNS. Lexeo has described the Apertura collaboration as an opportunity to evaluate a potentially less invasive route of CNS administration following initial systemic treatment. The companies will need to establish the scientific and clinical feasibility of the approach through further research and development. The current agreement provides access to the technology for evaluation and does not represent regulatory approval of TfR1 CapX or establish clinical efficacy in Friedreich ataxia.
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Gene-Delivery Innovation Targets Rare Neurological Disease
Friedreich ataxia is caused by mutations affecting the FXN gene, resulting in reduced production of frataxin, a protein important for mitochondrial function. The disease can progressively affect the nervous system, coordination, movement, and cardiac function. Apertura and Lexeo are therefore investigating technologies that could improve delivery of genetic medicines to tissues involved in the disease. The collaboration is part of a broader expansion of Lexeo’s Friedreich ataxia platform. In September 2026, Lexeo announced several strategic collaborations covering CNS-targeted gene-therapy delivery, sequential dosing, and immune-management approaches designed to complement its systemic LX2006 program. The company said these approaches will be evaluated against predefined scientific, clinical, and financial criteria as it determines which programs advance toward clinical development.
Apertura said its TfR1 CapX program is advancing toward clinical development, with the company targeting clinical entry during the second half of 2026. The technology is intended to provide a scalable platform for CNS delivery across neurological and genetic diseases, although its therapeutic effectiveness and safety in specific clinical indications will require clinical evaluation. The collaboration highlights the growing importance of AAV capsid engineering and blood-brain barrier-crossing technologies in genetic medicine. By combining Apertura’s CNS-targeted delivery platform with Lexeo’s Friedreich ataxia therapeutic programs, the companies are exploring another potential pathway for reaching disease-relevant tissues. Future research and clinical studies will determine whether TfR1 CapX can translate its preclinical delivery characteristics into a clinically useful approach for patients with Friedreich ataxia.
Source: Apertura Gene Therapy, Lexeo Therapeutics press release
