DRD1A receptor therapy

DRD1A receptor therapy is an adeno-associated virus (AAV)-based gene therapy being developed by Pathways Neuro Pharma for the treatment of Juvenile Parkinson's Disease (JPD). The therapy utilizes an engineered AAV6.2FF vector to deliver optimized dopamine receptor D1 (DRD1A) gene constructs to affected neuronal populations. Unlike traditional dopaminergic treatments like L-dopa or dopamine agonists that provide transient relief, this approach is designed as a non-agonist therapy that aims to restore dopaminergic signaling and normalize dopaminergic tone through durable, steady-state receptor expression. Preclinical studies have demonstrated that the therapy can provide sustained therapeutic benefits for up to 15 months, improving motor function and providing neuroprotective effects. The therapy's mechanism also involves modulation of GPR139 and serotonin 5-HT4 receptors to further stabilize dopaminergic signaling.

Other names

dopamine D1A receptor therapyDRD1 gene therapyDRD-1 gene therapyDRD 1 gene therapyAAV-DRD1A therapyAAV-DRD-1A therapyAAV-DRD 1A therapy