Selective Antisense Oligonucleotide-based JAK2 Inhibitor Receives FDA Orphan Drug ...
FDA granted orphan drug designation to VGT-1849A, a selective ASO-based JAK2 inhibitor for treating polycythemia vera (PV). PV, a rare hematologic malignancy, affects 1 in 2000 individuals in the US and is caused by a JAK2 V617F mutation. VGT-1849A aims to reduce JAK2 activity, potentially offering targeted efficacy and improved safety over current JAK2 inhibitors.
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Selective Antisense Oligonucleotide-based JAK2 Inhibitor Receives FDA Orphan Drug ...
FDA granted orphan drug designation to VGT-1849A, a selective ASO-based JAK2 inhibitor for treating polycythemia vera (PV). PV, a rare hematologic malignancy, affects 1 in 2000 individuals in the US and is caused by a JAK2 V617F mutation. VGT-1849A aims to reduce JAK2 activity, potentially offering targeted efficacy and improved safety over current JAK2 inhibitors.