After 2 years in HGB-205, patients with TDT (n=4) and SCD (n=3) had a median follow-up of 49.6 (40.5-60.6) and 28.5 (25.5-52.5) months, respectively.
The second study, HGB-205, treated 3 patients with sickle cell disease and 4 with β-thalassemia, with follow-up ranging from 20 months to more than 3 years.
Patients were then observed in long-term follow-up studies LTF-303 (NCT02633943) and LTF-307 (NCT04628585) for TDT and SCD, respectively.
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HGB-205 is a phase 1/2, open-label, single-arm, non-randomized interventional study of 2-year duration at a single center, followed by observation in long-term follow-up studies LTF-303 ( NCT02633943 ) and LTF-307 ( NCT04628585 ) for TDT and SCD, respectively.
Long-term outcomes of lentiviral gene therapy for the β ... - Nature
A total of 23 patients were enrolled and received treatment, with a median follow - up of 29.5 months (range, 13.0 to 48.2). Transfusion independence occurred in 20 of 22 patients who…
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