AADAD - Project
The Grand Challenge
Some forms of dementia are rare but the disease is always devastating. Current treatments only address symptoms rather than the root cause. The AADAD project aims to develop a highly precise genetic therapy that targets the underlying mutations driving these rare forms of Alzheimer’s disease, offering hope for disease-modifying treatments.
The consortium
AADAD brings together leading researchers, clinicians, and patient advocates across Belgium, the UK, and the USA. By integrating expertise from basic science, translational research, and clinical care, the project ensures a patient-focused, innovative approach to tackling genetic forms of dementia.
Objectives
- Develop antisense oligonucleotides (ASOs), a targeted genetic therapy with the potential to reduce harmful proteins that cause neurotoxicity and disease progression.
- Conduct proof-of-concept studies in non-human primates to validate safety and effectiveness.
- Generate the preclinical data package necessary for regulatory discussions and the transition to clinical trials.
This precision therapy is designed to halt or potentially reverse the progression of genetically driven Alzheimer’s disease by addressing the disease at its root, rather than just alleviating symptoms.
Engaging key stakeholders to boost impact
The project actively involves clinicians, patients, and key stakeholders to ensure that the therapy is developed ethically, safely, and with accessibility in mind. Early collaboration with a clinical research organization (CRO) ensures compliance with FDA, EMA, and national safety standards.
Looking ahead
AADAD aims to deliver a robust therapeutic platform ready for clinical trials, with pathways for commercial partners to ensure global access. By combining cutting-edge science, patient engagement, and ethical development, the project has the potential to transform treatment options for genetically driven neurological diseases.