Pifithrin analogues and methods of treating rett syndrome

EP4766356A1Pending Publication Date: 2026-07-01RGT UNIV OF CALIFORNIA
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Patent Information

Authority / Receiving Office
EP · EP
Patent Type
Applications
Current Assignee / Owner
RGT UNIV OF CALIFORNIA
Filing Date
2024-08-12
Publication Date
2026-07-01

AI Technical Summary

Technical Problem

Rett Syndrome, an X-linked neurodevelopmental disorder caused by mutations in the MECP2 gene, lacks a known cure and current treatments only manage symptoms.

Method used

Development of Pifithrin analogues, such as 2-(2-Imino-5,6-dihydro-2H-cyclopenta[d]thiazol-3(4H)-yl)-1-(p-tolyl)ethan-1-one hydrogen bromide (MXL026), which are administered to treat Rett Syndrome by inhibiting neuronal senescence and restoring brain function.

Benefits of technology

The Pifithrin analogues effectively reduce symptoms of Rett Syndrome, including reduced mobility, dystonia, and learning delays, by crossing the blood-brain barrier and inhibiting p53 target genes associated with senescence.

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Abstract

Disclosed herein are Pifithrin analogues and methods of treating Rett Syndrome with the Pifithrin analogues.
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