First Patient Receives Lyfgenia Gene Therapy for SCD

A 12-year-old patient with sickle cell disease (SCD) became the first person to receive the one-time gene therapy Lyfgenia (lovotibeglogene autotemcel), The New York Times reported.¹

Lyfgenia was approved by the US Food and Drug Administration in December 2023 for patients 12 years of age and older with a history of vaso-occlusive events, according to a press release from Bluebird Bio, the manufacturer of the therapy.²

Clinicians at Children’s National Hospital in Washington, DC, collected millions of isolated bone marrow stem cells, which Bluebird Bio will genetically modify at their lab in Allendale, New Jersey. Technicians will add a healthy hemoglobin gene to the bone marrow cells to correct the mutated genes that cause SCD. Three months later, the modified cells will be returned for transplantation.¹

“This is a big effort,” David Jacobsohn, MD, SCM, MBA, Division Chief of Blood and Marrow Transplantation at Children’s National Hospital, told The New York Times.¹

The “time-consuming” process, which costs $3.1 million, is estimated to treat the cells of 85 to 105 patients per year, including those with beta thalassemia.¹

At least 20 patients at Children’s National Hospital are eligible for Lyfgenia, and deciding which patient received the first treatment was based on two factors: insurance approval for the treatment and who was the sickest. Gene therapy recipient Kendric Cromer has experienced symptoms such as pain crisis and avascular necrosis since SCD onset at age three.¹

Lyfgenia was evaluated in the nonrandomized, open-label, multisite, single-dose phase I/II HGB-206 study, in which severe vaso-occlusive events were resolved in 94% of patients six to eight months after infusion. The most common grade 3 and higher adverse events were stomatitis, thrombocytopenia, neutropenia, febrile neutropenia, anemia, and leukopenia.²

References

1. Kolata G. First patient begins newly approved sickle cell gene therapy. The New York Times. May 6, 2024. Updated May 7, 2024. Accessed May 7, 2024. https://www.nytimes.com/2024/05/06/health/sickle-cell-cure-first.html
2. Bluebird Bio press release, December 2023.