AAV belongs to the Parvoviridae family and is highly attractive for the treatment of neurodegenerative diseases due to their neuronal tropism and their good safety profile demonstrated in dozens of clinical studies. In therapeutic gene delivery, the viral genes are replaced by the desired transgene expression cassette and referred as recombinant AAV (rAAV). These transgenes encoded within rAAV can form circular concatemers that persist as episomes in the nucleus of transduced cells without integrating into the host genome. The final designed rAAV is a non-replicating recombinant that has a low immunogenicity and is not known to cause disease.  These properties make it ideal for the CNS-selective expression of SIRT6. 

The SIRT6 AAV drug is designed to deliver functional SIRT6 genes directly into the affected brain cells. This innovative approach aims to augment the activity of SIRT6, leading to potential neuroprotection, reduced inflammation, and enhanced cellular repair mechanisms. By targeting the underlying causes of neurodegeneration, the SIRT6 AAV drug offers a novel and focused therapeutic strategy. Its ability to address the root mechanisms of neurodegenerative diseases holds promise for advancing treatment options and improving patient outcomes with the specific goal of treating neurodegenerative diseases such as Alzheimer’s disease (AD), Parkinson’s disease (PD), and amyotrophic lateral sclerosis (ALS)