Pre-Conference Workshop Day
PRECLINICAL TRACK
9:00 am Advancing Ocular Gene Therapy Delivery: Suprachoroidal Innovations
Synopsis
Optimizing ocular gene therapy delivery methods is crucial for enhancing treatment efficacy and minimizing invasiveness. This workshop focuses on the emerging technique of suprachoroidal delivery, offering insights into its benefits, challenges, and potential to revolutionize ocular gene therapy.
Key Discussion Points:
- Overview of Suprachoroidal Delivery: Exploring the concept and potential benefits of suprachoroidal delivery in ocular gene therapy. Understanding the challenges and limitations, including precision targeting within the small space of the eye.
- Advancements in Ocular Delivery Technologies: Investigating emerging approaches like RNA editing and splicing for enhancing therapeutic agent delivery to the retina. Exploring lipid nanoparticle-based delivery systems and their applications in ocular gene therapy.
- Suprachoroidal Delivery and Bioavailability: Assessing the role of suprachoroidal delivery in enhancing bioavailability of therapeutic agents in ocular gene therapy. Overview of Clearside Biomedical’s expertise and experience in developing suprachoroidal delivery technologies.
- Financial Investment and Regulatory Pathways: Discussing the financial investment required for developing suprachoroidal delivery devices. Exploring regulatory pathways and considerations for the approval of suprachoroidal delivery systems in ocular gene therapy.
By the end of this workshop, participants will gain a comprehensive understanding of suprachoroidal delivery and acquire insights into its potential to enhance bioavailability, challenges
CLINICAL TRACK
9:00 am Optimizing Endpoint Selection in Ophthalmic Gene Therapy Trials
Synopsis
Choosing appropriate endpoints is pivotal for accurately evaluating the efficacy of ophthalmic gene therapy. This
workshop provides a cohesive exploration of innovative strategies to enhance endpoint selection, ensuring validity,
reliability, and patient-centricity in ocular gene therapy trials.
Key Discussion Points:
- Advancing Visual Function Assessment: Exploring advancements in visual function assessment methodologies tailored for gene therapy trials, including the integration of active machine learning algorithms to optimize data collection and analysis.
- Enhancing Endpoint Validity and Reliability: Addressing challenges in determining appropriate endpoints, evaluating novel assessment methods, and discussing collaborative efforts to establish standardized endpoint criteria for improved trial design and interpretation.
- Defining Meaningful Endpoints: Examining the discrepancy between regulatory expectations and patient perspectives on meaningful outcomes, and discussing regulatory considerations for redefining clinical endpoints.
- Navigating Regulatory Expectations: Discussing adaptive trial designs, the potential of biomarkers as surrogate endpoints, and emphasizing patient-centered outcomes in compliance with regulatory standards, ensuring flexibility and validity in endpoint selection.
By the end of this workshop, participants will gain insights into the complexities of endpoint selection and acquire practical strategies to optimize endpoint selection.
12:00-1:00 Lunch Break & Networking
1:00 pm Enhancing Translational Validity Through Animal Models in Ophthalmic Gene Therapy Research
Synopsis
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.
1:00 pm Navigating Regulatory Pathways for Ophthalmic Gene Therapies
Synopsis
With the evolving landscape of gene therapy and regulatory frameworks, understanding and effectively navigating
regulatory pathways are crucial for the development and approval of ophthalmic gene therapies. This workshop
provides insights into regulatory challenges, collaboration strategies, and innovative approaches to accelerate the
approval process for breakthrough ocular gene therapies.
Key Discussion Points:
- Regulatory Landscape Overview: Understanding diverse regulatory frameworks and challenges across regions. Discussing harmonization efforts and strategies for compliance with regulatory guidelines.
- Addressing Safety and Efficacy Concerns: Highlighting complexities in long-term safety assessment and strategies for risk mitigation. Exploring translational challenges and robust risk-benefit analyses in regulatory decision-making.
- Regulatory Considerations for Emerging Technologies: Examining pathways for novel delivery platforms like suprachoroidal and subretinal systems. Discussing standardization challenges and the role of real-world evidence in supporting submissions.
- Accelerating Regulatory Approval for Breakthrough Therapies: Overview of expedited pathways and emphasizing proactive engagement with regulatory agencies.
By the end of this workshop, participants will gain an understanding of the regulatory landscape and acquire practical strategies to navigate challenges, collaborate effectively with regulatory agencies, and expedite the approval process for breakthrough ocular gene therapies.