Gene therapy
Targeted Receptor Therapy (TRT) for Juvenile Parkinson’s Disease
Our Targeted Receptor Therapy (TRT) platform leverages AAV6.2FF-mediated gene therapy to restore dopaminergic receptor function in Juvenile Parkinson’s Disease (JPD) without relying on traditional dopamine agonists. This approach aims to increase receptor density and responsiveness, offering a more sustained and natural restoration of dopaminergic signaling with an improved safety profile compared to existing therapies.
Core Approach
- DRD1A Gene Therapy: Instead of using an agonist to force dopamine receptor activation, we introduce an optimized DRD1A gene sequence, increasing dopamine receptor availability and enhancing the body’s ability to respond to endogenous dopamine.
- GPR139 Modulation: Given its role in the habenula and its connection to dopaminergic pathways, we are also investigating GPR139 as a potential neuroregulatory target in JPD.
Technology & Intellectual Property
We have filed for intellectual property protection on our AAV6.2FF-based DRD1A gene therapy and potential combination strategies integrating receptor-based gene therapy with pharmacological modulation.
Key Research Objectives
- Develop and optimize DRD1A and GPR139 gene constructs for targeted expression in the brain.
- Engineer AAV6.2FF vectors for efficient and precise gene delivery.
- Validate protein expression and receptor functionality in preclinical models.
- Assess biodistribution and therapeutic impact following both intravenous and intracranial administration.
Future Applications & Pipeline Potential
While our primary focus is on Juvenile Parkinson’s Disease, our TRT gene therapy platform may have broader applications in rare pediatric neurodegenerative diseases beyond JPD.
- GPR139 and Neuroprotection: Given its role in dopaminergic and habenular signaling, we are exploring GPR139’s potential therapeutic applications in other neurodevelopmental disorders.
- Serotonergic Modulation (5HTR4): The 5HTR4 receptor, a key player in serotonin signaling, represents another target of interest for expanding our TRT-based gene therapy approach in future rare pediatric disease indications where serotonin receptor dysfunction contributes to pathology.
- Pipeline Expansion Potential: Our approach may be pplicable to other rare pediatric movement disorders, including conditions such as Huntington’s Disease, where targeted receptor modulation could provide a novel therapeutic avenue.
