Researchers identified a peptide that selectively blocks pain signaling in sensory neurons and packaged it into a viral-based gene therapy in a step towards treating chronic pain without the risk for addiction.
For decades, treatment options for sickle cell disease have been extremely limited. Soon, gene therapies and a host of pharmaceuticals could alleviate the immense burden of this disease.
Currently, the only cure for alpha thalassemia is a bone marrow transplant, but the success of gene therapies for related conditions points to a potential new cure.
Forty years after the Orphan Drug Act passed, researchers advance drug development for neglected rare conditions everywhere from the lab bench to backstage.
Most vaccines and biologics must stay cold until ready for use, but new thin film technology stabilizes these therapeutics for months at room temperature.
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