Lexeo Therapeutics’ gene therapy provides a healthy version of the frataxin gene, which is critical for energy production in ...

Nomlabofusp is a protein replacement therapy aimed at addressing the root cause of FA by delivering frataxin to mitochondria. The FDA’s decision to include it in the START program was based on ...

The company revealed encouraging news from the U.S. Food and Drug Administration (FDA), which has expressed openness to consider skin frataxin (FXN) concentrations as a reasonably likely surrogate ...

11 of 12 participants saw an over 25% drop in troponin I and a 115% average frataxin boost in the high-dose group at 3 months. Feel unsure about the market’s next move? Copy trade alerts from ...

The fresh data come from a pooled analysis of two studies of LX2006, a gene therapy designed to treat cardiac dysfunction in FA by increasing expression of the heart muscle protein frataxin.

frataxin expression to be evaluated for any increase from baseline rather than numerical threshold Interim update from cohort 1 of LX2020 HEROIC-PKP2 Phase 1/2 trial: observed 71% and 115% ...

The firm plans to use improvement in left ventricular mass index and increased frataxin protein expression after LX2006 treatment as co-primary endpoints.

Participants have experienced clinically meaningful improvements across multiple measures, as well as increased frataxin expression in the heart, all of which underscore the potential of LX2006 to ...

Regulatory Update: Further alignment on elements of the accelerated development pathway following a Type B RMAT meeting with the U.S. Food and Drug Administration (FDA): Frataxin expression to be ...