Daily Dosing of CTI-1601 Appears to Rescue Frataxin Levels

Daily Dosing of CTI-1601 Appears to Rescue Frataxin Levels

294160

Daily Dosing of CTI-1601 Appears to Rescue Frataxin Levels

Daily dosing of CTI-1601, a lab-made, modified version of frataxin — the protein lacking in people with Friedreich’s ataxia (FA) — for 13 days led to a dose-dependent increase in frataxin levels compared to placebo, top-line data from a Phase 1 study show. Patients treated with the two highest doses of CTI-1601 tested, 50 mg and 100 mg, saw their frataxin levels reach similar or even surpassing the levels of carriers — those with approximately…

You must be logged in to read/download the full post.