CTAD taskforce: genetic therapies in Alzheimer’s disease

There are an increasing number of genetic approaches to treating Alzheimer’s disease and other dementias, with some promising results from early-phase trials. This prompted the convention of the first EU-US CTAD Task Force on genetic therapies in Alzheimer’s disease in October 2024. Preclinical studies and clinical trials of genetic therapies in Alzheimer’s disease and other dementias are presented here with key lessons for the field. Importantly, there are several challenges and opportunities unique to neurogenetic therapies which were reviewed and discussed, including means of genetic manipulation, adverse events, monitoring, timing of therapy, and the importance of patient involvement in trial design. Continued collaboration across disciplines will accelerate development of neurogenetic therapeutics.