
Rett Syndrome Drug Development Program
Our secondary drug development program.
Rett syndrome (RTT) is a rare progressive neurodevelopmental disorder affecting approximately 1 in 10,000 female births worldwide. Around 200 girls in Australia have been diagnosed with the disease and around 350,000 girls around the world are living with Rett syndrome.

A lack of effective therapies for Rett syndrome
Conventional treatment approaches for Rett syndrome, including physiotherapy and speech therapy aim to improve movement and communication, while medication can be taken to manage seizures. Trofi netide – a drug that reduces infl ammation in the brain has been approved to treat RTT patients over 2 years of age. However, the drug dose is significantly high and severe side effects such as diarrhoea and weight loss can occur.
Our Approach to Reduce Rett Syndrome Symptoms
ProGenis Pharmaceuticals aims to reduce Rett syndrome symptoms by developing an RNA medicine that targets the gene responsible for RTT complications. Our lead drug molecule has been shown to be effective in reducing the expression of the disease-causing gene in cellular models of RTT.
