Our Strategy In an urgent effort, our current focus is on funding innovative research strategies that look at A-T in new ways.
Our greatest interest is in funding translational and clinical research projects focused on the neurological problems faced by all patients with A-T. We are eager to deliver new ideas from A-T research with practical relevance for patients into the hands of experts involved in drug discovery and clinical development. We have interest in disease-modifying strategies that would prevent, slow, or stop the progression of the disease as well as symptom-improving strategies that address neural circuit function, immune system health and lung function – but especially the neurological symptoms.
Accelerating A-T Research:
In recent years, researchers have achieved substantial progress towards finding treatments for A-T, making notable advancements on several fronts. They have been exploring innovative methods to discover new drugs and refining gene therapy and editing techniques to better address A-T. Additionally, they have made efforts to establish robust measurement approaches for A-T, enabling more precise and objective assessments of clinical trial outcomes.
Our ongoing work involves fostering collaborative scientific brainstorming sessions and forming close partnerships with pharmaceutical and biotech companies interested in pioneering A-T treatments. We are also committed to expanding our global reach to identify more children affected by A-T and conducting experiments with novel A-T cell-based strategies. Furthermore, we are actively engaged in the development of "proof-of-concept" gene-editing and gene-delivery treatments designed to target all known A-T mutations.
Because of this work, we hope to continue to attract academic researchers and cutting-edge companies that are advancing new DNA-focused technologies to pursue A-T.