BGT-DTDS

BGT-DTDS is an adeno-associated virus (AAV) serotype 2-based gene therapy designed for the treatment of Dopamine Transporter Deficiency Syndrome (DTDS). Developed by Bloomsbury Genetic Therapies, the therapy utilizes an AAV2 vector to deliver a functional copy of the *SLC6A3* gene, which encodes the dopamine transporter (DAT) protein. DTDS is a rare, progressive, and life-limiting neurometabolic disorder caused by biallelic mutations in *SLC6A3*, leading to impaired dopamine reuptake and severe motor dysfunction. By restoring DAT expression in the brain, BGT-DTDS aims to normalize dopaminergic signaling and alleviate the clinical symptoms of the disease. The program is currently in the IND-enabling stage of development.