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Mast Cell Derived Cancers
HT- KIT is a New Molecular Entity – classified as Antisense Oligonucleotide (ASO). It directly targets and inhibits the proto-oncogene c-KIT (including mutations in cKIT) providing a broad range of potential therapeutic indications: Aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL); Acute myeloid leukemia (AML); Gastrointestinal stromal tumors (GIST); Small lung cell carcinoma. It also has a secondary indication for the prevention of anaphylaxis. HT-KIT received Orphan Designation in 2022 for treatment of mastocytosis. API manufacturing and formulation development has been initiated.