Drug Profile

HTX 001

Alternative Names: HTX-001

Latest Information Update: 03 Feb 2026

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At a glance

Originator Centre Hospitalier Universitaire Vaudois
Developer Centre Hospitalier Universitaire Vaudois; HAYA Therapeutics
Class Antifibrotics; Antisense oligonucleotides; Cardiovascular therapies
Mechanism of Action Gene silencing; Long noncoding RNA expression inhibitors; Long noncoding RNA inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

03 Feb 2026 HTX 001 is still in preclinical development in Hypertrophic-cardiomyopathy in Switzerland (Parenteral) (HAYA Therapeutics pipeline, February 2026)
28 Jun 2025 No recent reports of development identified for preclinical development in Fibrosis in Switzerland (Parenteral)
28 Jun 2025 No recent reports of development identified for preclinical development in Hypertrophic-cardiomyopathy in Switzerland (Parenteral)

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