FDA Approves New Treatment for Sickle Cell Disease

For Immediate Release:

December 08, 2023

Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy. 

Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. It is most common in African Americans and, while less prevalent, also affects Hispanic Americans. The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues. This mutation causes red blood cells to develop a crescent or “sickle” shape. These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events or crises can lead to life-threatening disabilities and/or early death. 

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.” 

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Minority Blood Donations Needed!!!!

African-American blood donors hold the power to save other African-Americans in need of a blood transfusion. That’s because donors with genetically-similar blood are more likely to be a match for patients from the same ethnic background.

When it comes to blood donations, researchers have found that African-Americans are under-represented among donors and that minority donors in general are less likely to become regular donors.

Why should African-Americans donate blood?
Increasing African-American donations is vital because blood types O and B, the blood types of about 70 percent of African-Americans, are also the blood types most in demand.

Genetically-similar blood is preferred for those who need repeated blood transfusions, and for conditions like sickle cell disease (SCD), which primarily affects African-Americans. Blood that closely matches that of a patient is less likely to be rejected by the patient and can mean fewer complications after a transfusion.

African-American blood donors are more likely to be a match for the majority of individuals with SCD in the United States, making them ideal donors to help SCD patients receive regular blood transfusions.

For more information on Blood Donations please visit the following links

Organizations
American Red Cross (ARC)

America’s Blood Centers (ABC)

Blood Centers of America (BCA)

Community Blood Center (CBC)

Hoxworth Blood Center

Versiti

There’s no Tymia without the A #Missing Type

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