Apertura Grants Lexeo Access to TfR1 CapX AAV Capsid for Friedreich Ataxia Gene Therapy Program
核心洞察
Apertura Gene Therapy (搜索) will give Lexeo Therapeutics access to its TfR1 CapX (搜索) capsid for a gene therapy program designed to treat Friedreich ataxia (搜索).
TfR1 CapX (搜索) is an intravenously delivered AAV capsid that targets human transferrin receptor 1 (搜索) and is designed to cross the blood-brain barrier.
Lexeo said the agreement expands its tools for addressing unmet needs in Friedreich ataxia (搜索), including a potentially less invasive CNS route after systemic LX2006 dosing.
Apertura Gene Therapy (搜索) will provide Lexeo Therapeutics access to its TfR1 CapX (搜索) capsid for a program designed to treat Friedreich ataxia (搜索), the two companies announced on Sept. 23, 2026. TfR1 CapX is an intravenously delivered AAV capsid engineered to target human transferrin receptor 1 (搜索) (hTfR1) and cross the blood-brain barrier.
"As an intravenously delivered capsid designed to cross the blood-brain barrier, TfR1 CapX (搜索) holds the potential to help unlock new approaches to treat Friedreich ataxia (搜索)," said Diego Garzón, Ph.D., Chief Business Officer at Apertura Gene Therapy (搜索). "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."
Lexeo is a clinical stage genetic medicine company whose portfolio includes LX2006 in Friedreich ataxia (搜索) and LX2020 in plakophilin-2 (搜索) (PKP2) arrhythmogenic cardiomyopathy. Louis Tamayo, Chief Financial Officer of Lexeo Therapeutics, said the agreement expands the tools available to the company as it evaluates approaches to the 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."
The Capsid Platform
TfR1 CapX (搜索) is described by Apertura as a leading IV-administered, blood-brain barrier-crossing capsid. It is a proprietary, second-generation capsid that the company says demonstrates superior CNS delivery compared with its first-generation capsid, BI-hTFR1, research on which was published in Science.
According to Apertura, multiple for-profit and non-profit organizations have validated and licensed TfR1 CapX (搜索), 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.
Apertura develops genetic medicines and next-generation AAV capsids that engage human-relevant receptors, aiming to enable more effective and selective gene delivery. Its lead capsid, TfR1 CapX (搜索), leverages human transferrin receptor 1 (搜索) to enable intravenous delivery to the brain and spinal cord. The company states that this transport mechanism has a strong clinical track record in pediatric and geriatric populations, which expands 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 (搜索) is headquartered in New York City.
Friedreich Ataxia Disease Burden
Friedreich ataxia (搜索) 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 (搜索) that disrupts normal production of the protein frataxin (搜索), which is critical to mitochondrial function in a cell and to the maintenance of cardiac function.
The absence of fully functional frataxin (搜索) leads to damage to peripheral nerves and to the parts of the brain that control movement and balance. This produces neurological symptoms that include impaired muscle coordination, or ataxia, which worsen over time.
Lexeo Therapeutics is a New York City-based, clinical stage genetic medicine company focused on cardiovascular disease, advancing therapeutic candidates directed at the underlying genetic causes of conditions with high unmet need.