Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.

ABSTRACT: Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34⁺ cell gene therapy (GT) following busulfan reduced-intensity conditioning is a promising therapeutic approach for ADA-SCID, but long-term data are warranted. Here we report an analysis on long-term safety and efficacy data of 43 patients with ADA-SCID who received retroviral ex vivo bone marrow-derived hematopoietic stem cell GT. Twenty-two individuals (median follow-up 15.4 years) were treated in the context of clinical development or named patient program. Nineteen patients were treated post-marketing authorization (median follow-up 3.2 years), and two additional patients received mobilized peripheral blood CD34⁺ cell GT. At dat

SUBMITTER: Migliavacca M 

PROVIDER: S-EPMC7615698 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature