Beyond the Cure: What is a Drug That Opens the Doors to a New Drug?
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5 min read
In 2024, the FDA approved Casgevy, the first CRISPR-based gene therapy for sickle cell disease, demonstrating how a foundational scientific discovery can become a drug that opens the doors to a new drug, validating a new modality of medicine entirely. This landmark approval was the culmination of decades of research and has fundamentally shifted the paradigm for treating genetic disorders.
