NEW YORK – The first patient with sickle cell disease has begun the process of receiving Bluebird Bio's gene therapy Lyfgenia (lovotibeglogene autotemcel) outside of clinical trials, the company said Monday.
Sickle cell disease is a rare and inherited blood disorder caused by mutations in the gene that encodes hemoglobin, resulting in misshapen red blood cells that can't carry oxygen throughout the body. Lyfgenia is a one-time treatment that involves collecting a patient's hematopoietic stem and progenitor cells and genetically engineering them ex vivo with a functional copy of a modified beta-globin gene, so that it can produce anti-sickling hemoglobin that works like normal, adult hemoglobin.
The first commercial cell collection for delivering the gene therapy to a patient took place at Children's National Hospital in Washington, D.C.
"We are thrilled to be the first center in the country to commercially collect cells from a person living with sickle cell disease and are proud to be the trailblazers in using this new approach," David Jacobsohn, chief of the division of blood and marrow transplantation at Children's National, said in a statement.
Somerville, Massachusetts-based Bluebird received the US Food and Drug Administration's approval to market Lyfgenia in December, at the same time that the agency also approved Vertex Pharmaceuticals and CRISPR Therapeutics' gene-editing sickle cell treatment Casgevy (exagamglogene autotemcel). Lyfgenia and Casgevy represent the first two gene therapies to reach the market for this disease.