Apertura Gene Therapy to Provide Lexeo Therapeutics Access to TfR1 CapX™ for Program Designed to Treat Friedreich Ataxia

NEW YORK, Sept. 23, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, will provide Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage company focused on reshaping the path of genetic diseases with high unmet need, access to TfR1 CapX™ for a program designed to treat Friedreich ataxia. Apertura's TfR1 CapX is an intravenously delivered AAV capsid designed to target human transferrin receptor 1 (hTfR1) and cross the blood-brain barrier.

"As an intravenously delivered capsid designed to cross the blood-brain barrier, TfR1 CapX holds the potential to help unlock new approaches to treat Friedreich ataxia," said Diego Garzón, Ph.D., Chief Business Officer at Apertura Gene Therapy. "We're proud to support Lexeo's research and development efforts and believe that this agreement can make a meaningful difference for the Friedreich ataxia patient community."

Friedreich ataxia (FA) is a genetic, progressive, degenerative multi-system disorder with a prevalence of approximately 1:50,000 in the United States. It is caused by a mutation in the FXN gene that disrupts the normal production of the protein frataxin, which is critical to the function of mitochondria in a cell and to the maintenance of cardiac function. Absence of fully functional frataxin leads to damage to peripheral nerves and the parts of the brain that control movement and balance, leading to neurological symptoms that include impaired muscle coordination, or ataxia, that worsen over time.

"We are excited to partner with Apertura Gene Therapy and gain access to this promising technology," said Louis Tamayo, Chief Financial Officer of Lexeo Therapeutics. "This agreement expands the tools available to us as we evaluate innovative approaches to address the significant unmet needs of patients living with FA, including the potential for a less invasive route of administration to the CNS following initial systemic administration of LX2006."

TfR1 CapX™ is a leading IV-administered, BBB-crossing capsid. Multiple for-profit and non-profit organizations have validated and licensed TfR1 CapX, and other groups are in discussions to license the technology. Clinical readiness has been supported by several preclinical development programs, including regulatory engagement and manufacturing by contract development and manufacturing organizations (CDMOs). TfR1 CapX is a proprietary, second-generation capsid that demonstrates superior CNS delivery compared to Apertura's first-generation capsid, BI-hTFR1. Research on the first-generation BI-hTFR1 capsid was published in Science.

Apertura Gene Therapy develops genetic medicines and next-generation AAV capsids that engage human-relevant receptors, aiming to enable more effective and selective gene delivery. The company's lead capsid, TfR1 CapX™, leverages human transferrin receptor 1 to enable intravenous delivery to the brain and spinal cord. This established transport mechanism has a strong clinical track record in pediatric and geriatric populations, expanding its potential to treat serious neurological and genetic diseases. Apertura has licensed its next-generation capsids to multiple partners, with several programs expected to enter clinical trials over the next 12 months. Founded in 2021 on technology from the Broad Institute and supported by Deerfield Management, Apertura Gene Therapy is headquartered in New York City. Learn more at aperturagtx.com and follow us on LinkedIn.

Lexeo Therapeutics is a New York City-based, clinical stage genetic medicine company dedicated to reshaping heart health by applying pioneering science to fundamentally change how cardiovascular diseases are treated. The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including LX2006 in Friedreich ataxia (FA), LX2020 in plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and others in devastating diseases with high unmet need.

Investor Response: Ashley Kaplowitz [email protected]