Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.

Background: Wiskott-Aldrich syndrome is a rare, life-threatening, X-linked primary immunodeficiency characterised by microthrombocytopenia, infections, eczema, autoimmunity, and malignant disease. Lentiviral vector-mediated haemopoietic stem/progenitor cell (HSPC) gene therapy is a potentially curative treatment that represents an alternative to allogeneic HSPC transplantation. Here, we report safety and efficacy data from an interim analysis of patients with severe Wiskott-Aldrich syndrome who received lentiviral vector-derived gene therapy.

Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome.

Background: Thrombocytopenia is a serious issue for all patients with classical Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) because it causes severe and life-threatening bleeding. Lentiviral gene therapy (GT) for WAS has shown promising results in terms of immune reconstitution. However, despite the reduced severity and frequency of bleeding events, platelet counts remain low in GT-treated patients.

A randomized, double-blind, multiple-dose, placebo-controlled, dose escalation study with a 3-cohort parallel group design to investigate the tolerability and pharmacokinetics of albaconazole in healthy subjects.

Albaconazole is a triazole antifungal agent discovered at Palau Pharma SA (Barcelona, Spain) and currently being developed for the treatment of fungal infections of the nails. This randomized, double-blind, placebo-controlled, phase 1 study evaluated the safety, tolerability, pharmacokinetics, and effects on electrocardiogram parameters of albaconazole administered orally at escalating supratherapeutic doses. Healthy subjects received 400 mg albaconazole every 24, 12, or 8 hours for 5 days.