Gene Therapy Offers New Treatment Option for Sickle Cell Disease

 

There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone with sickle cell disease qualifies for it. Bluebird Bio, a biotechnology company, has been working on a new option for treatment: gene therapy. They work with a number of different diseases, including cerebral adrenoleukodystrophy, sickle cell disease, β- thalassaemia, and multiple myeloma. Through a variety of gene therapies, they aim to improve treatment options for those who have these diseases.

About Sickle Cell Disease

Sickle cell disease is a group of disorders that is characterized by malformed red blood cells, which resemble sickles. The most well known version of this disease is sickle cell anemia, which is also the most common and severe type. In sickle cell disease, the deformed cells get caught along the walls of blood vessels, causing blockages and restricting blood flow.

The malformation of the cells is caused by a mutation in the gene that is responsible for making hemoglobin, which carries oxygen throughout the body. The gene for this disease is recessive, meaning both parents must pass down a copy to their child. Sickle cell disease is most common in people of African American descent. It also disproportionately affects those of Hispanic descent.

Symptoms of sickle cell disease include pain crisis, swelling of the hands and feet, and symptoms associated with anemia like fatigue, jaundice, and delayed growth. While adults constantly feel the effects of these symptoms, children usually only experience them during pain crisis. Regardless of age, damage can occur to the organs that face blocked blood flow. The organs that are most commonly damaged are the brain, eyes, spleen, liver, kidneys, lungs, heart, skin, joints, and bones.

There is no cure for sickle cell disease. While some can get bone marrow and blood transplants, not everyone is eligible for this procedure. Other forms of treatment are symptomatic and meant to prolong life.

About Bluebird Bio’s Gene Therapy

Bluebird Bio works with three different forms of gene therapy, including gene editing, cell therapy, and gene addition. They recently released the results of Phase 1/2 HGB-206 Group C clinical trial of LentiGlobin gene therapy. The study included 26 participants and followed results for four years. The gene therapy aims to produce hemoglobin that is not deformed into the sickle shape. Results found that there was a substantial reduction of deformed cells, which paired with no episodes of acute chest syndrome (ACS) or intense vaso-occlusive crisis. Results of this study were reinforced by exploratory assays.

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