Restoring vascular stability through pericyte-directed small molecule therapies

At RougeTx, we are developing small molecule first in class therapies designed to restore vascular integrity by targeting pericyte biology. Pericytes play a central role in supporting and stabilising blood vessels, and their dysfunction is increasingly recognised as a key driver of vessel fragility in HHT.

Lead programme

Our lead programme, RTX-001, is designed to restore pericyte–endothelial interactions, improve vessel stability and support a disease-modifying unique approach for HHT.

Key HHT statistics

Underserved patient population with no approved therapy
(palliative care)

  • 1:3800

    prevalence worldwide

  • 1.4mn people

    affected worldwide

  • ~500k patients

    in 7 major markets

  • 2nd most common

    vascular bleeding disorder

  • > 96% loss of function

    mutations in ENG (HHT1) or ACVRL1 (HHT2) genes, essential for endothelial BMP signalling and normal vascular development

Disease overview

Hereditary Haemorrhagic Telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome, is a rare inherited vascular disease in which blood vessels develop abnormally.

HHT is the second most common genetic bleeding disorder.
The disease is characterised by blood vessel structural instability and pathological angiogenesis leading to fragile blood vessels known as telangiectasias and arteriovenous malformations (AVMs), where arteries and veins connect without a normal capillary bed.

These abnormal blood vessels are prone to bleeding and can occur in the nose, skin, gastrointestinal tract, lungs, liver and brain.

HHT symptoms typically start to present in late teenage years or older and can have a significant impact on a person’s quality of life and in some cases cause life-threatening bleeding events.

People living with HHT often experience recurrent debilitating nosebleeds (epistaxis), and gastrointestinal bleeding leading to iron-deficiency anaemia.

In some cases, arteriovenous malformations (AVMs) in vital organs can cause serious complication:

  • Brain AVMs can lead to migraines, seizures or stroke
  • Lung AVMs can result in right to left blood shunting, increasing stroke risk, infection, pulmonary bleeding and chronic respiratory disorders
  • Liver AVMs can contribute to heart failure, pulmonary arterial hypertension and progressive liver dysfunction.

Currently, treatment is largely focused on managing symptoms and preventing complications. There remains a significant need for novel therapies that address the underlying vascular instability driving the disease.

HHT patient resources