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THOR-427

Development stage: Preclinical
Lead developer: THOR Therapeutics
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Parenteral

Overview

THOR-427 is an antisense oligonucleotide (ASO) designed to target and knock down glial cell line-derived neurotrophic factor family receptor alpha-like (GFRAL) expression. Developed by Thor Therapeutics, THOR-427 is being investigated for the treatment of refractory cancer cachexia and as an immuno-oncology (IO) booster. By silencing GFRAL expression, the drug blocks the GDF15-GFRAL signaling axis, which is activated by tumors to drive appetite loss, muscle and fat wasting, and immune suppression. THOR-427 is currently in IND-enabling preclinical development.

Targets

GDF15 (Growth differentiation factor 15)RET (Rearranged during transfection receptor tyrosine kinase)GFRAL (Glial-derived neurotrophic factor family receptor alpha-like/ret receptor complex)

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