Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

ABSTRACT:

Background

Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency.

Methods

We treated 50 patients with ADA-SCID (30 in the United States and 20 in the United Kingdom) with an investigational gene therapy composed of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a self-inactivating lentiviral vector encoding human ADA. Data from the two U.S. studies (in which fresh and cryopreserved formulations were used) at 24 months of follow-up were analyzed alongside data from the U.K. study (in which a fresh formulation was used) at 36 months of follow-up.

Results

Overall survival was 100% in all studies up to 24 and 36 months. Event-free surv

SUBMITTER: Kohn DB 

PROVIDER: S-EPMC8240285 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature