Blood: BAX 335 gene therapy is expected to overcome type B haemophilia.

By transferring the functional human F9 gene (coded coagulation factor IX) into liver cells, gene therapy has the potential to maintain therapeutic coagulation factor IX (FIX) levels at normal levels in patients with type B haemophiliac patients.
study is an open-label, incremental phase 1/2 study to study the efficacy of BAX 335 (AskBio009, AAV8.sc-TTR-FIXR338Lopt), a Gland-related virus (AAV) serotony type 8-based FIX Padua gene therapy for haemophilia B.
diagram of BAX 335 This report is primarily an interim analysis of safety, pharmacodynamics, effects on FIX activity, and immune response at 12 months.
8 adult male patients (20-69 years old, FIX activity range: 0.5%-2.0%) received the first dose of BAX 335 (IV, 3 times): 2.0×1011, 1.0×1012 or 3.0×1012 vector/kg genome.
three subjects had four severe adverse reactions (SAEs), all of which were considered unrelated to BAX 335.
no SAE resulted in death.
no clinical embolism, inhibitors, or other FIX Padua targeted immunity was observed.
OFX activity in different patients was detected in 7 patients, and the peak OFIC activity showed dose dependence (in Group 3: 32.0%-58.5%).
one subject received continuous therapeutic FIX activity (about 20% of active levels) with no bleeding or alternative therapy for up to 4 years;
contrary to previous studies, corticosteroid hormone therapy does not stabilize FIX activity.
speculated that the loss of GM may have been caused by congenital immune response stimulation.
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