# Gene therapies offer sickle cell cure, but few can access them > Casgevy and Lyfgenia can eliminate severe pain crises, yet high costs, demanding treatment and specialist shortages leave thousands unable to reach the breakthrough. - Title: Gene therapies offer sickle cell cure, but few can access them - Summary: Casgevy and Lyfgenia can eliminate severe pain crises, yet high costs, demanding treatment and specialist shortages leave thousands unable to reach the… - Keywords: sickle cell disease, casgevy, lyfgenia, gene therapy, healthcare access, medicaid, vertex pharmaceuticals, genetix biotherapeutics, fda approval, rare disease, business, finance - Source: CNBC — https://cnbc.com/2026/10/16/sickle-cell-gene-therapies-offer-new-hope-accessing-them-is-hard.html - Author: Steven Ross Johnson - Published: 2026-10-16T13:19:09+00:00 - Read time: 3 min - Topics: sickle cell disease, casgevy, lyfgenia, gene therapy, healthcare access, medicaid, vertex pharmaceuticals, genetix biotherapeutics, fda approval, rare disease, business, finance ## A century of pain, then hope Sickle cell disease affects around one hundred thousand people in the U.S., more than ninety percent of them non-Hispanic Black or African American. ## Decades of slow progress From nineteen ten until about twenty fifteen, there was very little progress in disease-modifying treatments for sickle cell disease. > from 1910 until about 2015, there was very little progress in disease-modifying treatments for sickle cell disease. ## Hydroxyurea changes the game In nineteen ninety eight, the FDA approved hydroxyurea, the first medication shown to modify the health effects of sickle cell disease. ## Gene therapy arrives In twenty twenty three, the U.S.Food and Drug Administration approved Casgevy and Lyfgenia, the first two gene therapies for patients with severe forms of sickle cell disease. ## A potential cure Dr.Edward Donnell Ivy, chief medical officer for the Sickle Cell Disease Association of America, said the breakthrough opens up a world of individuals potentially being cured of sickle cell disease. > That opens up a whole world of individuals potentially being cured of sickle cell disease. ## Slow uptake despite potential By the end of twenty twenty five, a total of sixty four patients worldwide had received infusions of Casgevy, according to Vertex Pharmaceuticals.Genetix Biotherapeutics reported that one hundred fifteen U.S. ## A demanding treatment process Gene therapy requires collecting the patient's blood-forming stem cells and modifying them to produce healthy hemoglobin, then returning those cells to the patient's body. ## Permanent side effects The conditioning process often causes permanent infertility or sterility.Casgevy is available for patients as young as two years of age, while Lyfgenia is available for patients twelve years and older. ## Who qualifies for treatment Likely candidates include patients who have experienced at least four pain crises or acute chest syndrome episodes within twenty four months prior to seeking treatment. ## A patient's difficult choice Dr.Edward Donnell Ivy, who experiences four to five pain crises a year before taking hydroxyurea, said he opted to forego gene therapy because of the demands on his body and time. > If I was younger, I might strongly consider it for sure. But there are a lot of things to consider. ## Prohibitive cost The list price for a single-course treatment ranges from two point two million to three point one million dollars.This cost does not include healthcare expenses such as hospital stays, chemotherapy, or the specialized care needed. ## Medicaid coverage efforts Efforts have been made to address potential cost disparities between patients with private health insurance and the majority of sickle cell disease patients who receive coverage through Medicaid or the Children's Health Insurance Program. ## The CMS access model In twenty twenty four, the Centers for Medicare and Medicaid Services launched the Cell and Gene Therapy Access Model, a program where the agency enters into agreements with states and manufacturers to set pricing for… ## Coverage varies by state Dr.Alexias Thompson, an attending physician at Children's Hospital of Philadelphia and spokesperson for the American Society of Hematology, acknowledged that the type of services covered through the program can vary. > Depending on the state that someone is in, some of the coverage may be more comprehensive than others. ## Specialist shortage is the real barrier While cost is a concern, finding a qualified healthcare provider able to provide treatment may be a larger challenge. ## Rural patients face long drives The number of specialized care centers leaves care gaps for people living in more rural areas, requiring many to frequently travel for hours across their state or to another state to access treatment. > there is a mismatch between the number of patients and the number of providers, that means some patients are driving from up to three hours away to gain access to sickle cell expertise. ## Building support systems To bridge the gap, the Medical University of South Carolina's Shawn Jenkins Children's Hospital in Charleston employs social workers, patient navigators and virtual health visits. ## National network for standardization The National Alliance of Sickle Cell Centers, with one hundred fifteen member centers across the country, works to share information among specialists and raise awareness among the broader medical community. > One thing sickle cell disease has lacked for a very long time is standardization of care and really bringing light to disease. ## Key takeaway Two new gene therapies approved in 2023 offer potential cures for severe sickle cell disease, but access remains limited by cost, treatment complexity and lack of specialist centres.