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LX2006

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Frataxin gene-replacement therapy in recruiting pivotal Phase 2 development for Friedreich-ataxia cardiomyopathy.

Modality
systemic AAVrh10 gene therapy delivering functional frataxin
Availability (US)
Pivotal Phase 2 SUNRISE-FA 2 study Recruiting; Phase 1/2 long-term follow-up ongoing
Biomarkers
left-ventricular mass index; cardiac troponin; frataxin expression; modified Friedreich Ataxia Rating Scale; treatment-emergent adverse events
Hallmark targeted
mitochondrial dysfunction, iron-sulfur-cluster biology, cardiomyopathy
Kind
investigational gene therapy
Clinical trials
NCT07721025 — Phase 2, Recruiting on September 2, 2026 update; NCT05445323 — Phase 1/Phase 2, Active, not recruiting on December 23, 2025 update