New gene therapies for sickle cell disease are approved but access is low due to high costs, complex treatment, and uneven healthcare.
01Summary
- 01In 2023, the US FDA approved Casgevy and Lyfgenia, the first two gene therapies for severe sickle cell disease.
- 02A 2020 analysis found NIH research funding for sickle cell disease averaged $812 per patient from 2008 to 2018, against $2,807 for cystic fibrosis.
- 03By the end of 2025, 64 patients worldwide had received Casgevy infusions and 115 US patients had been treated with Lyfgenia.
- 04Gene therapy can take up to a year to complete and involves chemotherapy, blood transfusions, and risk of permanent infertility.
- 05Between 25% and 35% of US sickle cell patients are estimated to meet the clinical criteria for gene therapy.
02Why it matters
After decades of slow progress, these therapies offer a potential cure for a painful genetic disorder affecting around 100,000 Americans, but most eligible patients still cannot access them due to high costs and a complex treatment journey.
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