VGT-1849A / Vanda - LARVOL DELTA

Selective ASO-based JAK2 inhibitor for the Treatment of Polycythemia Vera: Preclinical Efficacy and Safety (IMMUNOLOGY 2025) - "This study showcases an antisense oligonucleotide (ASO) therapeutic agent VGT1849A that reduces JAK2 transcriptional expression by ASO-mediated exon masking...This may serve as a more efficacious treatment option with a more favorable side effect profile, resulting in a higher quality of life for PV patients. Bottom of FormKeywords: Animals Human; Molecules Protein Kinases/Phosphatases; Processes Gene Regulation Hematopoiesis; Techniques/Approaches Gene Therapy"

Preclinical • Essential Thrombocythemia • Gene Therapies • Hematological Disorders • Infectious Disease • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytosis • JAK1 • JAK3 • TYK2

Vanda Pharmaceuticals Announces Orphan Drug Designation Granted for VGT-1849A, a Novel and Selective Antisense Oligonucleotide Candidate for the Treatment of Polycythemia Vera (PRNewswire) - "Vanda Pharmaceuticals Inc...today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849A...for the treatment of polycythemia vera (PV), a form of a rare hematologic malignancy that is estimated to affect 1 in 2000 Americans."

Orphan drug • Polycythemia Vera