Pharmaceutical Discovery

Our pharmaceutical discovery program is dedicated to pioneering Targeted Receptor Therapy (TRT) for Juvenile Parkinson’s Disease (JPD). Central to this initiative is the development of gene therapies that enhance dopaminergic signaling by targeting the Dopamine Receptor D1 (DRD1).

Current Research Focus:

We are actively conducting preclinical studies utilizing the engineered AAV6.2FF vector to deliver optimized DRD1 gene constructs. This approach aims to restore dopaminergic function in relevant neuronal populations. The AAV6.2FF vector has been selected for its enhanced transduction efficiency and reduced immunogenicity, which are critical for effective central nervous system delivery.  

Preclinical Development Phases:

1. In Vivo Efficacy Studies:

• Objective: Assess the therapeutic potential of AAV6.2FF-mediated DRD1 gene delivery in animal models that replicate the pathophysiology of JPD.
• Approach: Evaluate motor function improvements and neuroprotective effects following targeted delivery of the DRD1 gene.

2. Pharmacokinetic and Biodistribution Analysis:

• Objective: Determine the distribution, persistence, and expression levels of the therapeutic gene within the central nervous system and peripheral tissues.
•  Approach: Utilize quantitative assays to map vector dissemination and transgene expression profiles.

3. Safety and Toxicology Assessments:

• Objective: Evaluate the safety profile of the AAV6.2FF-DRD1 therapy, focusing on potential off-target effects and immunogenic responses.
• Approach: Conduct comprehensive histopathological examinations and monitor biomarkers indicative of immune activation.

Future Directions:

Upon successful completion of these preclinical studies, we plan to advance our lead candidate into clinical trials. This progression underscores our commitment to translating innovative gene therapy solutions from bench to bedside, offering hope for patients affected by Juvenile Parkinson’s Disease.