Lexeo Therapeutics (NASDAQ: LXEO) has gained access to Apertura Gene Therapy's TfR1 CapX platform, an intravenously delivered adeno-associated virus (AAV) capsid engineered to target the human transferrin receptor 1 and cross the blood-brain barrier.
The agreement centers on Lexeo's LX2006 therapeutic candidate, which targets Friedreich ataxia (FA), a rare genetic disease affecting approximately one in 50,000 people in the United States. FA is caused by a mutation in the FXN gene that disrupts production of frataxin, a protein critical to mitochondrial function and cardiac health. Lack of functional frataxin damages peripheral nerves and brain regions controlling movement and balance, leading to progressive impairment of muscle coordination.
TfR1 CapX has been licensed by multiple for-profit and non-profit organizations. Several preclinical development programs have supported its clinical readiness, including regulatory engagement and manufacturing through contract development organizations, according to the source information.
Apertura Gene Therapy was founded in 2021 on technology from the Broad Institute, is backed by Deerfield Management, and is headquartered in New York City. Lexeo is a clinical-stage company focused on genetic and cardiovascular diseases, also based in New York.
Louis Tamayo, Lexeo's chief financial officer, said the agreement expands the tools available to the company as it evaluates 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 central nervous system following initial systemic administration of LX2006.
