NFL Alumni, Genetix team up to raise awareness of sickle cell treatments
Campaign will highlight one-time genetic therapies now available in the U.S.
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Genetix Biotherapeutics is teaming up with the National Football League Alumni Association for a campaign to increase awareness of one-time genetic treatments for sickle cell disease (SCD).
“Despite hundreds of patients now treated, the vast majority of eligible patients remain untreated, primarily due to lack of awareness that these treatment options are available today,” David Meek, CEO of Genetix, said in a company press release. “We are excited that together with the National Football League Alumni Association, we will expand education and foster meaningful community conversations to improve the quality of life of those living with sickle cell disease.”
Sickle cell disease disproportionately affects Black communities
According to Genetix, roughly 100,000 people in the U.S. have SCD, a disease that disproportionately affects Black communities.
“Many former NFL players and their families understand firsthand the devastating impact of this disease and the importance of ensuring patients and families have knowledge and access to available treatment options,” said Brad Edwards, CEO of the National Football League Alumni Association. “By elevating personal stories with local community engagement from current and former players, we aim to raise awareness and connect patients and families with educational resources that support actionable next steps.”
SCD is a genetic disorder that affects hemoglobin, the protein red blood cells use to carry oxygen through the bloodstream. The abnormal hemoglobin can cause red blood cells to take on a sickle shape, making them prone to breaking down and becoming stuck in blood vessels. These blockages can trigger painful episodes called vaso-occlusive crises (VOCs).
In late 2023, the U.S. Food and Drug Administration (FDA) approved two one-time genetic therapies for SCD. Both work by modifying a patient’s own blood-forming stem cells so the body can produce forms of hemoglobin that help prevent red blood cells from sickling. The stem cells are collected from the patient, modified in a lab, and then infused back into the patient as part of a stem cell transplant.
Genetix, formerly known as Bluebird Bio, markets one of these therapies, Lyfgenia (lovotibeglogene autotemcel), which is approved by the FDA for SCD patients ages 12 and older who have a history of VOCs. Lyfgenia adds a gene to the stem cells that allows them to produce a form of hemoglobin with anti-sickling properties. The goal is to reduce VOCs and other disease complications.
“For decades, Genetix has been committed to the sickle cell disease community, working alongside patients, families, advocates, and healthcare providers to advance care,” Meek said. “Partnering with this community led to the development of an FDA-approved, one-time transformative treatment, making genetic medicine a reality for many people living with this debilitating disease.”
Casgevy offers another one-time genetic treatment option
The other one-time therapy, Casgevy (exagamglogene autotemcel), was developed by CRISPR Therapeutics and Vertex Pharmaceuticals. It uses gene editing to increase production of fetal hemoglobin, a form of the protein normally produced during fetal development that can help prevent red blood cells from sickling. Casgevy is FDA-approved to treat SCD patients ages 2 and older who experience recurrent VOCs.
Data from clinical trials have shown that one-time treatment with either Lyfgenia or Casgevy can substantially reduce VOCs, with many patients remaining free of these crises for years after treatment.
These therapies carry potentially serious safety risks that need to be considered on a case-by-case basis. Still, they can be transformative, with the potential to provide a functional cure.
According to Genetix, many families affected by SCD are unaware that FDA-approved one-time genetic therapies are already available in the U.S. The new campaign aims to increase awareness of these treatment options among families affected by SCD.
“The National Football League Alumni Association is proud to partner with Genetix to widen awareness and promote education on treatment options for people with sickle cell disease,” Edwards said.
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