Pifithrin analogues and methods of treating rett syndrome
EP4766356A1Pending Publication Date: 2026-07-01RGT UNIV OF CALIFORNIA
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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