Key clinical point: With a median follow-up of 6.7 years in 10 patients aged 18-64 years who were treated with scAAV2/8-LP1-hFIX–comediated gene therapy, factor IX expression has been persistent and stable.
Major finding: Factor IX expression averaged 1.9%-2.3% at the lower doses, and 5.1% at the high dose at up to 8.6 years.
Study details: An interim follow-up data for 10 patients in a phase 1/2 study.
Disclosures: Dr. Reiss reported having no disclosures.
Reiss UM et al. ASH 2018, Abstract 491.