Imagine the brain has a cleaning crew called microglia that fails to take out the trash in some dementia patients. This project modifies a patient's own stem cells to create a high-performance cleaning crew that can clear out toxic plaques. These upgraded cells are delivered directly into the brain to fix the root cause of the disease rather than just masking the symptoms.
Current Alzheimer's and Nasu-Hakola Disease treatments only manage symptoms and fail to stop the progression of the disease. There is a critical need for curative therapies that can restore the brain's ability to clear toxic protein accumulations.
A clinical-grade manufacturing protocol for TREM2-engineered stem cells and an IP-protected delivery method for the brain lateral ventricles.
If you are a gene therapy developer dealing with the low efficacy of symptomatic dementia drugs — this project developed a lentiviral vector method to express TREM2 in the CNS that reduces Aβ accumulation. This allows for a curative rather than symptomatic treatment path.
If you are a neurology drug manufacturer dealing with the lack of disease-modifying therapies for Nasu-Hakola Disease — this project developed a method to transplant engineered HSPCs that restore physiological scavenging functions. This targets the specific genetic mutations causing the disease.
If you are an ATMP clinic dealing with the difficulty of selective brain engraftment — this project developed an IP-protected delivery route in the brain lateral ventricles. This ensures engineered cells reach the CNS exclusively and safely.
Based on available project data, there is no information regarding the cost or pricing of the treatment.
The project is developing innovative and advanced protocols for lentiviral vector and cell manufacturing in clinical grade conditions via INSERM to enable translation.
The project utilizes a delivery route in the brain lateral ventricles that is explicitly described as IP-protected.
The project runs from 2024-04-01 to 2027-03-31, aiming to launch a first-in-human Phase I clinical trial upon completion.
It involves a reduced intensity conditioning regime and the transplantation of engineered hematopoietic stem/progenitor cells via the brain lateral ventricles.
The consortium consists of 4 partners across 3 countries (IT, FR, CZ). It is heavily academic and research-oriented, with 1 university and 2 research organizations, and 0% industry participation. This indicates the project is in a high-risk, high-reward translational phase where the primary goal is generating clinical data rather than immediate commercial production.
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