Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral

gene therapy Gene therapy is Health technology, medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. The first attempt at modifying human DNA ...

used for the treatment of

sickle cell disease Sickle cell disease (SCD), also simply called sickle cell, is a group of inherited Hemoglobinopathy, haemoglobin-related blood disorders. The most common type is known as sickle cell anemia. Sickle cell anemia results in an abnormality in the ...

. The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with chemotherapy and underlying disease. The US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) approved lovotibeglogene autotemcel in December 2023.

Medical uses

Lovotibeglogene autotemcel is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for the treatment of people aged twelve years of age and older with sickle cell disease and a history of vaso-occlusive events. The recipient's blood stem cells are genetically modified to produce HbA (T87Q), a gene-therapy derived

hemoglobin A Hemoglobin A (HbA), also known as adult hemoglobin, hemoglobin A1 or α2β2, is the most common human hemoglobin tetramer, accounting for over 97% of the total red blood cell hemoglobin. Hemoglobin is an oxygen-binding protein, found in Red blood ...

, which is similar to the normal adult

hemoglobin Hemoglobin (haemoglobin, Hb or Hgb) is a protein containing iron that facilitates the transportation of oxygen in red blood cells. Almost all vertebrates contain hemoglobin, with the sole exception of the fish family Channichthyidae. Hemoglobin ...

produced in persons not affected by sickle cell disease. Red blood cells containing HbA (T87Q) have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the recipient. The gene therapy is made from the recipient's own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, the recipient's own stem cells are collected, and then the recipient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in lovotibeglogene autotemcel.

Side effects

The US FDA label contains a black box warning about hematologic malignancy (blood cancer).

History

The safety and effectiveness of lovotibeglogene autotemcel is based on the analysis of data from a single-arm, 24-month multicenter study in participants with sickle cell disease and history of Vaso-occlusive episodes (VOEs) between the ages of twelve and fifty years. Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between six and eighteen months after infusion with lovotibeglogene autotemcel. Twenty-eight (88%) of 32 participants achieved VOE-CR during this time period. The FDA granted the application for lovotibeglogene autotemcel

priority review Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

, fast track, and regenerative medicine advanced therapy designations. The FDA granted approval of Lyfgenia to Bluebird Bio Inc. In March 2025, Cohen Children's Medical Center announced that through the use of this treatment the first person in New York State was cured of sickle cell anemia.

Society and culture

Economics

The cost effectiveness threshold of the therapy is estimated to be between to $2.05 million.

Brand names

Lovotibeglogene autotemcel is the international nonproprietary name. Lovotibeglogene autotemcel is sold under the brand name Lyfgenia.

References

External links

* Approved gene therapies Drugs acting on the blood and blood forming organs Lentiviruses Orphan drugs Sickle-cell disease {{Pharma-stub