simoladagene autotemcel

Simoladagene autotemcel (OTL-101) is an autologous ex vivo hematopoietic stem cell (HSC) gene therapy developed for the treatment of adenosine deaminase deficiency-severe combined immunodeficiency (ADA-SCID), a rare and life-threatening genetic disorder. The therapy involves the extraction of a patient's own CD34+ hematopoietic stem and progenitor cells, which are then genetically modified ex vivo using a self-inactivating lentiviral vector to insert a functional copy of the human adenosine deaminase (ADA) gene. Following non-myeloablative busulfan conditioning, the modified cells are re-infused into the patient. Once engrafted in the bone marrow, these cells proliferate and differentiate into various immune cell lineages, providing a continuous source of the ADA enzyme. This restoration of enzyme activity prevents the accumulation of toxic metabolites such as deoxyadenosine triphosphate (dATP), thereby allowing for the development and maintenance of a functional immune system and protecting against life-threatening infections.

Brand names

OTL-101OTL101OTL 101

Other names

autologous CD34+ cells transduced with lentiviral vector encoding human ADA genesimoladagene autotemcel