First Patient Set to Receive bluebird’s Approved Sickle Cell Gene Therapy, Lyfgenia


Lovo-cel was approved as Lyfgenia in December 2023.

David Jacobsohn, MD, MBA, chief, division of Blood and Marrow Transplantation, Children’s National,

David Jacobsohn, MD, MBA

Credit: Children's National Hospital

The first patient with sickle cell disease (SCD) is set to receive bluebird bio’s lovotibeglogene autotemcel (lovo-cel) after completing a cell collection at Children’s National Hospital in Washington.1,2 Lovo-cel was approved as Lyfgenia by the FDA in December 2023.3

"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. The recent approval of gene therapies to treat patients with sickle cell is an enormous breakthrough in patient care and a silver lining to families witnessing their children’s struggles with this condition. In the face of immense burden, gene therapy is a groundbreaking treatment alternative and a beacon of hope for a better future," David Jacobsohn, MD, MBA, chief, division of Blood and Marrow Transplantation, Children’s National, said in a statement.1

The patient, a 12-year-old boy named Kendric Cromer, according to reporting from the New York Times, is receiving the gene therapy covered under insurance. Lyfgenia has a listed price of $3.1 million. The therapy consists of autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and apheresis, transduced with BB305 lentiviral vector (LVV) encoding the human beta-A-T87Q globin gene.

"Seeing people living with sickle cell disease receive gene therapy in the real world is a vision that has fueled bluebird for more than 10 years," Andrew Obenshain, president and CEO, bluebird bio, added.1 "This historic moment comes nearly a century after sickle cell disease was the first genetic disorder to be characterized at the molecular level, and almost a decade after bluebird initiated clinical development for LYFGENIA. We are grateful to the patients, caregivers, researchers, and clinicians whose work made this milestone possible, and look forward to continued partnership with the sickle cell disease community."

WATCH NOW: Mark Walters, MD, on Lessons Learned With Investigating Lovo-Cel for Sickle Cell

Lyfgenia was approved based off of efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 clinical trial (NCT02140554) and 2 patients in the phase 3 HGB-210 clinical trial (NCT04293185), as well as safety data from 50 patients treated across multiple clinical trials, 6 patients of which had at least 6 years of follow-up. Its efficacy was assessed based on the complete resolution of vaso-occlusive events (VOEs) between 6 and 18 months after infusion, at which time 88% (n = 28) of the 32 included patients achieved this milestone.

“It's going to be very complicated, because we need to support sickle cell disease centers in delivering this product, as well as the product itself, which are sort of 2 different price streams,” Julie Kanter, MD, director, Adult Sickle Cell Clinic and associate professor of hematology and oncology, University of Alabama Birmingham, previously told CGTLive®. “I think it'll be a little bit complicated as we wander into this new territory of high-priced therapies. But I think the patients that I have taken care of would tell you it is absolutely worth it. It is truly a transformative therapy." Kanter has served as an investigator on multiple trials assessing lovo-cel.

1. bluebird bio Announces Completion of First Cell Collection for LYFGENIA™ Gene Therapy. May 6, 2024.
2. First Patient Begins Newly Approved Sickle Cell Gene Therapy. Article. New York Times. May 6, 2024.
3. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 8, 2023. Accessed December 8, 2023.
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