Encapsulated cell-based delivery of biologics to the brain for targeted therapies of neurodegenerative disorders

Purpose and goal

The purpose of our project is to develop new therapies for Parkinson´s and other neurodegenerative diseases, for which there is currently a lack of disease modifying treatments. Our solution is a promising drug delivery technology, encapsulated cell biodelivery (ECB), which we use for "combinatorial gene therapy", a novel therapeutic concept for optimal effect. With the project, we have shown that the ECB is a working drug delivery technology for treating the brain with various biologics

Expected results and effects

We have developed ECB therapies with different classes of biologics and also combinations of several biologics in the same therapy. The therapies are tailored for hereditary forms of Parkinson´s (PD) and frontotemporal dementia (FTD) and are designed to correct the effects of disease - causing mutations and to restore the function of diseased neurons. We have demonstrated therapeutic effect with 4 different therapies in patient-derived cells and animal models for different subtypes of FTD and PD. The next step is safety studies before we start testing the therapies in patients.

Planned approach and implementation

Sinfonia Biotherapeutics develops precision therapies for genetically defined neurodegenerative diseases. The company has key competencies in cell therapy, pharma, med tech and neurosurgery. In product development, we have teamed up with academic expertise in patient and animal models, protein production, clinical development including biomarkers and access to patient organizations. The collaborative efforts have resulted in a careful characterization, validation and testing of novel therapies with good results in animal models and patient-derived cellular models.