PDR-001

PDR-001 (AAV9-tat-βsyn-deg) is a novel peptide-based targeted protein degrader (TPD) delivered via an adeno-associated virus type 9 (AAV9) vector for the treatment of early-to-moderate stage Parkinson's disease. Developed by Prodegre (Shanghai) Pharmaceutical in collaboration with Ruijin Hospital, it represents the world's first peptide-based protein degrader to enter human clinical trials. The therapy is administered as a one-time bilateral stereotactic injection into the subthalamic nucleus, where the AAV9 vector turns infected cells into 'cell factories' that continuously express a three-segment therapeutic peptide (tat-βsyn-deg). This peptide selectively targets and clears pathological alpha-synuclein aggregates using a non-CRLIM-mediated degron mechanism, aiming to halt or delay disease progression by addressing the root cause of dopaminergic neuron death.

Other names

tat-βsyn-deg