news

한어Русский языкEnglishFrançaisIndonesianSanskrit日本語DeutschPortuguêsΕλληνικάespañolItalianoSuomalainenLatina

Zhitong Finance APP learned that on July 16, Lexeo Therapeutics (LXEO.US) announced positive interim data for LX2006, a gene therapy for the treatment of Friedreich's ataxia (FA) cardiomyopathy. In the SUNRISE-FA Phase 1/2 clinical trial and another Phase 1A trial, patients' cardiac biomarkers improved by 53.3%, which is clinically significant. Based on this positive result, Lexeo plans to accelerate the development of the therapy, including discussing the design of key trials with regulatory authorities and applying for potential accelerated approval.

It is understood that LX2006 is an adeno-associated virus (AAV)-based gene therapy that aims to promote the expression of FXN protein and restore mitochondrial function in cardiomyocytes by delivering a functional FXN gene through intravenous injection. In preclinical studies, LX2006 reversed cardiac abnormalities in FA disease models, showed improvements in cardiac function and survival, and exhibited good safety. The FDA has granted LX2006 rare pediatric disease qualifications, fast track qualifications, and orphan drug qualifications for the treatment of FA cardiomyopathy.