Imagine the brain's wiring is like a complex electrical grid; in some people, a genetic glitch makes the signals too strong, causing seizures and learning difficulties. This project creates a 'blueprint' of these glitches using stem cells and animal models to find the right chemical switches to turn the signals back to normal. It also works on better ways to get these medicines past the brain's natural security fence.
Patients with RASopathies suffer from severe intellectual disabilities and seizures that currently lack approved therapeutic options. There is a critical need for accurate biomarkers and effective delivery methods to get treatments into the brain.
The project developed a patient-based registry, iPSC-derived neuronal disease models, automated drug screening pipelines, and new brain-targeted drug delivery technologies.
If you are a drug discovery firm dealing with a lack of targets for rare genetic diseases — this project developed high-content screening pipelines using patient-derived neurons that identify effective drug candidates. This allows for faster identification of repurposed drugs for neurocognitive symptoms.
If you are a biotech company dealing with the difficulty of crossing the blood-brain barrier — this project developed and validated new technologies for efficient drug delivery to the brain. This enables the delivery of antisense oligonucleotides and other therapies to the target site.
If you are a diagnostic provider dealing with underdiagnosed neurodevelopmental disorders — this project developed cellular and molecular disease signatures as biomarkers. These tools allow for earlier and more accurate diagnosis of RASopathies.
Based on available project data, there is no information regarding the cost or pricing of the therapies.
The project uses automated high-resolution, high-content pipelines for phenotypical screenings, which suggests a design intended for high-throughput scalability.
Based on available project data, specific IP and licensing agreements are not detailed in the summary.
The project runs from 2023-06-01 to 2027-05-31, focusing on preclinical proof-of-concept trials during this period.
The consortium includes a patient organization as a formal partner to implement a patient report-based registry for cohort stratification and treatment evaluation.
The consortium is heavily research-oriented with 10 universities and 4 research institutes, but it maintains a 12% industry ratio with 2 SMEs. This balance suggests a strong focus on fundamental discovery and early-stage development, while the inclusion of a patient organization ensures the research is aligned with actual clinical needs across 8 countries.
Contact the Universitätsklinikum Erlangen in Germany
Contact SciTransfer to explore licensing opportunities for the drug delivery technologies developed by EURAS.
