Lovotibeglogene autotemcel

Lovotibeglogene autotemcel (also known as Lenti-D) is a gene therapy product developed by Bluebird Bio for the treatment of cerebral adrenoleukodystrophy (CALD), a rare, genetic neurological disorder. It is an ex vivo gene therapy, which means that it involves modifying the patient's cells outside the body before reinfusing them.

Mechanism of Action

Lovotibeglogene autotemcel works by introducing a functional copy of the ABCD1 gene into hematopoietic stem cells (HSCs) collected from the patient. The ABCD1 gene is responsible for producing a protein that breaks down certain types of fatty acids. In people with CALD, mutations in the ABCD1 gene lead to the accumulation of very long-chain fatty acids (VLCFAs) in the brain, causing progressive damage to the myelin sheath that protects nerve cells.

The patient's HSCs are collected and then treated with a lentiviral vector carrying a functional copy of the ABCD1 gene. The modified cells are then reinfused into the patient, where they can produce new blood cells that express the functional ABCD1 protein, helping to prevent further accumulation of VLCFAs.

Clinical Trials and Approval

Lovotibeglogene autotemcel has undergone several clinical trials to evaluate its safety and efficacy. The results of these trials have shown that the therapy can halt the progression of CALD in the majority of patients, with some experiencing improvements in neurological function.

In 2021, the European Medicines Agency (EMA) granted conditional marketing authorization for lovotibeglogene autotemcel, making it the first gene therapy approved for the treatment of CALD.

Side Effects

As with any medical treatment, lovotibeglogene autotemcel can cause side effects. The most common side effects reported in clinical trials include nausea, vomiting, fever, and headache. Serious side effects can include infection, low blood cell counts, and graft-versus-host disease (GVHD).

See Also

• Gene therapy
• Cerebral adrenoleukodystrophy
• Bluebird Bio