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HTX-001
- Development stage
- Preclinical
- Lead developer
- HAYA 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
01
Overview
HTX-001 is a modified antisense oligonucleotide (ASO) developed by HAYA Therapeutics, designed to target the long noncoding RNA (lncRNA) WISPER. WISPER is highly expressed in heart cells, particularly in patients with hypertrophic cardiomyopathy (HCM), and drives excessive interstitial fibrosis—a key contributor to the pathophysiology of HCM and heart failure. By silencing WISPER, HTX-001 aims to reduce heart tissue scarring and potentially reverse existing fibrosis, offering a targeted treatment for non-obstructive hypertrophic cardiomyopathy and related cardiac conditions[2][3][5]. The drug is currently in preclinical development for these indications.
- Other names
- Wisper
02
Targets
WISPER (Wisp2 super-enhancer–associated RNA)
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