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Genetix Biotherapeutics is teaming up with the National Football League Alumni Association for a U.S. awareness and education drive centered on sickle cell disease (SCD). The collab is designed to “raise greater awareness of the difficulties of living with sickle cell disease, while helping individuals, families and healthcare providers better understand available treatment options and support services,” Genetix said in a statement . It added that “many families are unaware that FDA-approved genetic medicines” for the condition, including, “one-time transformative gene therapies, are available today and have proven to dramatically transform the lives of many people impacted by the disease.” The biopharma sells one-time gene therapy Lyfgenia for sickle cell disease, as well as Zynteglo for beta thalassemia and Skysona for early cerebral adrenoleukodystrophy. SCD encompasses a group of inherited blood disorders in which abnormal hemoglobin causes red blood cells to become hard, sticky, and crescent-shaped. These cells can break down fast and block small blood vessels, leading to chronic anemia, painful episodes, and serious organ damage if left untreated. The disease heavily impacts the Black community. “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,” NFL Alumni Association CEO Brad Edwards said in the release. “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,” Edwards said. “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.” Genetix is the rebranded name of bluebird bio. The company went back to its roots last September by renaming itself under its original moniker. That changeup came after the drugmaker sold itself to a pair of private equity firms to go private and then install new leadership.

Vertex has secured a label expansion through the FDA's Commissioner’s National Priority Voucher (CNPV) program, which allows children as young as 2 years of age to receive the gene therapy Casgevy for the one-time treatment of sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). The groundbreaking therapy, which was co-developed by CRISPR Therapeutics, had already been approved for patients ages 12 and older. The expansion opens the use of Casgevy to an additional 5,500 children in the United States, according to the Boston-based company, giving those patients and their families an opportunity to reduce the risk of organ damage that can result from either disorder. “Earlier access to the transformative potential of this therapy will allow clinicians and families to consider treatment before years of cumulative damage from these life-shortening diseases take hold,” Haydar Frangoul, M.D., the Medical Director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, said in a release. The expansion is supported in part by results from two ongoing phase 3 trials. In one, which included 11 children aged 5 to 12 with SCD, of the eight patients who were evaluable for efficacy, all achieved the primary endpoint with no severe VOCs for at least 12 consecutive months, according to a separate approval release from the FDA. In the other trial of 15 TDT patients in the same age group, eight of the nine who could be evaluated for efficacy achieved the same standard, with a median duration of transfusion independence of 20 months. Expanding the indication down to 2 years of age was also based on the known characteristics of Casgevy and clinical study data in both indications, Vertex said. “The remarkable consistency of results across age groups reinforces the potential of Casgevy to deliver durable, transformative benefits to those who have historically had limited options,” Vertex CEO Reshma Kewalramani, M.D., said in Vertex's announcement. The approval falls under the CNPV program established by former FDA commissioner Marty Makary, M.D., who announced his resignation in May amid a turbulent tenure at the regulator. The CNPV program was designed to drastically shorten review timelines for certain products and companies aligned with U.S. national interests. When Casgevy was originally approved in 2023 for SCD, it was hailed as the first medicine developed using the revolutionary CRISPR gene-editing system, which earned its inventors a Nobel Prize in 2020 and holds tantalizing potential to cure other diseases for which there are no current treatments. On the same day that the FDA originally approved Casgevy, the U.S. regulator also signed off on bluebird bio’s SCD gene therapy Lyfgenia. While uptake has been slow for both treatments—owing in part to their respective prices of $2.2 million and $3.1 million—they are building momentum. Sales for Casgevy came in at $116 million in 2025 with 64 patients receiving infusions, including 30 in the fourth quarter. In the first three months of this year, Vertex listed Casgevy sales at $43 million. The company added in its report that more than 500 patients have initiated treatment with Casgevy since it reached the market. Meanwhile, in 2025, more than 150 patients completed initial cell collection for Lyfgenia, with more than 100 receiving infusions, according to private Genetix Biotherapeutics, which does not report sales of the treatment. After bluebird was sold last year, private equity companies Carlyle and SK Capital established Genetix. Lyfgenia remains approved for SCD and TDT patients ages 12 and older.