Overview
Angelman syndrome is a genetic disorder caused by UBE3A gene dysfunction, leading to cognitive impairment, motor coordination issues, and developmental delays. Research suggests that oligodendroglial dysfunction, which affects myelination in the brain, contributes to these conditions. Based on the research, this invention discloses a novel method for treating Angelman syndrome (AS) and autism spectrum disorders (ASD) using repurposed non-steroidal selective estrogen receptor beta (ERβ) agonists. The method includes ERβ agonists as a targeted treatment, leveraging their ability to restore oligodendrocyte progenitor cell (OPC) function, improve myelination, and mitigate cognitive and motor impairments. The therapy involves compounds such as AC-186, AC-131, and LY500307, which selectively activate ERβ, promoting neural repair and neurodevelopmental improvements.
Market Opportunity
AS is a rare neurodevelopmental disorder, but its overlap with autism spectrum disorders presents a broader clinical market. AS affects 1 in 15,000 births, with no FDA-approved treatments targeting the underlying pathology. ASDs, impacting 1 in 36 children in the U.S., have similar neurodevelopmental and myelination deficits, making this approach relevant for a much larger population. The global ASD therapeutics market is projected to grow substantially, driven by increasing diagnosis rates and demand for targeted treatments. Current interventions rely mainly on behavioral therapy and symptomatic management, highlighting the urgent need for disease-modifying therapies. ERβ agonists represent a first-in-class therapeutic solution with the potential to transform the treatment landscape for AS and ASD by directly targeting neuronal and glial dysfunction.
Innovation and Meaningful Advantages
This patent introduces a highly selective, non-steroidal ERβ agonist therapy designed to correct oligodendroglial dysfunction, a key contributor to cognitive and motor symptoms in AS and ASD. Unlike traditional treatments, which focus on symptom relief, this approach restores neural function at a molecular level by enhancing OPC proliferation, differentiation, and myelination. There are many key advantages to use this therapy, which includes that this is the first disease-modifying therapy, it is a precision-targeted approach, it has a potential for early intervention to delay or prevent sever neurodevelopment impairments, and has been identified to have multiple administration routes.
Collaboration Opportunity: We are interested in exploring research collaborations and licensing opportunities.
References:
Principal Investigator
Yu-Wen Alvin Huang, MD, PhD
GLF Translational Assistant Professor of Molecular Biology,
Cell Biology & Biochemistry
Brown University
yu-wen_huang@brown.edu
https://vivo.brown.edu/display/yhuan149
Contact
Neil Veloso
Executive Director
Brown Technology Innovations
neil_veloso@brown.edu
Brown Tech ID 3343