Enhancing Translational Validity Through Animal Models in Ophthalmic Gene Therapy Research
Time: 1:00 pm
day: Pre-Conference Workshop Day Preclinical Track PM
Details:
Robust preclinical research is essential for ensuring the safety and efficacy of gene therapy interventions in treating
ocular diseases. This workshop delves into the challenges of current animal models, the need for innovative approaches, and the foundational role of preclinical research in advancing ophthalmic gene therapy.
Key Discussion Points:
- Leveraging Animal Models for Ophthalmic Gene Therapy Development: Assessment of disease-specific animal models like retinitis pigmentosa pigs and canine models for translational research. Exploration of challenges and opportunities associated with leveraging animal models to understand disease mechanisms and evaluate gene therapy efficacy.
- Overcoming Challenges in Preclinical Model Development: Identification of suitable animal models for inherited retinal diseases and strategies for developing more predictive models. Exploration of innovative approaches such as iPSCderived cell models and larger animal models for better mimicking human ocular diseases.
- Conducting Comprehensive Safety Studies: Importance of safety studies to evaluate potential toxicity, immunogenicity, and off-target effects of gene therapy in animal models. Assessment of long-term effects of gene expression on ocular structures and function in preclinical research.
By the end of this workshop, participants will gain understanding of the challenges and opportunities in preclinical research for ophthalmic gene therapy and insights into leveraging animal models, innovative approaches, and conducting comprehensive safety studies, contributing to safe and effective gene therapy interventions for ocular diseases.