Sickle Cell Disease Treatment Receives Rare Pediatric Disease Designation

Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is meant to treat a serious or life-threatening rare disease that primarily affects those under the age of 18. The designation also comes with incentives like a priority review voucher.

About Sickle Cell Disease (SCD)

Sickle cell disease (SCD) includes a group of disorders that are characterized by malformed red blood cells that look like sickles. The most common and severe form of this disease is sickle cell anemia. In sickle cell disease, deformed cells cause blockages and restricting blood flow as they get caught along the walls of blood vessels. This disease is caused by a mutation in the gene responsible for producing hemoglobin, which carries oxygen throughout the body. This gene is inherited in an autosomal recessive pattern, meaning both parents must pass down a copy to their child. Sickle cell disease is most common in people of African and 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. Adults tend to constantly feel the effects of this disease, but children usually only experience them during pain crisis. Regardless of age, damage usually occurs to the organs that are affected by the blocked blood flow. The most commonly damaged organs are the brain, eyes, spleen, liver, kidneys, lungs, heart, skin, joints, and bones. There is no cure for sickle cell disease. While some people qualify for bone marrow and blood transplants, not everyone is eligible for this procedure. Other forms of treatment are symptomatic and meant to prolong life.

About EDIT-301

EDIT-301 uses CRISPR technology and is an autologous cell therapy. It is intended to edit the HBG1/2 promoter region in the beta-globin locus, with the goal of increasing levels of fetal hemoglobin. If successful, it would provide a significant benefit to those with sickle cell disease.

The next step for this cell therapy is to file an investigational new drug application, and Editas is planning to submit one by the end of this year. Hopefully this treatment continues to show positive results, as it has the potential to better the lives of those with SCD.

Find the source article here.

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