New therapies for genetic differences
cystic fibrosis, sickle cell disease
Promising advances have been made in treatment of cystic fibrosis and sickle cell disease thanks to new precision therapies.
These approaches are due in part to the integration of precision medicine that considers genome factors, disease severity and genetic variability, researchers say.
CF
Cystic fibrosis is a genetic disorder characterized by mutations in the CFTR gene. Gene therapy and personalized medicine have helped make advances, but they still face obstacles, according to a March 2025 article in the National Library of Medicine.
Mutations are numerous and vary from patient to patient.
“While personalized and gene therapies represent promising frontiers in the treatment of cystic fibrosis (CF), their widespread application and efficacy are still developing,” the article states. “The personalized approach, primarily through CFTR modulators, has made significant strides, especially for patients with specific mutations.
“However, its reach is limited by the diversity of mutations and variability in patient responses. Many patients either do not qualify for these therapies or do not respond optimally due to the specific mutations they carry.”
CF can cause persistent lung infections and affect the pancreas, liver, and intestines.
SCD
Sickle cell disease is now being targeted by two new gene therapies approved by the U.S. Food and Drug Administration.
One is known as Casgevy, the other Lyfgenia.
They were approved for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises. Approximately 16,000 people may be eligible for such treatment, according to insideprecisionmedicine.com
Casgevy is a genome-edited cellular therapy administered via a hematopoietic stem cell transplant procedure.
Lyfgenia, also known as lovo-cel, is gene therapy custom-designed to treat the underlying cause of sickle cell disease.
Sickle cell disease, also called sickle cell anemia, is a group of inherited disorders that affect hemoglobin, which carries oxygen in red blood cells. Sickle cell disease can trigger sudden, strong episodes of pain because of blocked blood flow. They often require medical attention.
For more CF details, see pmc.ncbi.nlm.nih.gov/articles/PMC12026723/
For more SCD details, see insideprecisionmedicine.com/topics/precision-medicine/two-gene-therapies-for-sickle-cell-disease-get-fda-nod-including-first-ever-crispr-based-therapy.
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