8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
TRANSFORMATIVE ADVANCES IN OPHTHALMIC TREATMENTS: RNA INNOVATIONS, GENE EDITING & OPTOGENETICS BREAKTHROUGHS
9:00 am Advancing ASO Therapies for Ophthalmic Disorders: Addressing Challenges & Expanding Applications in Ophthalmology
Synopsis
- Overview of antisense oligonucleotides (ASOs) and their role in treating ophthalmic disorders
- Exploring the potential of ASOs in broader applications beyond splicing modulation
- Overview of recent advancements in ASO design and delivery methods
- Highlighting efforts in developing functional assays and organoid models for assessing ASO efficacy
9:30 am Innovative Non-Viral Gene Editing: Harnessing Synthetic AAV Constructs
Synopsis
- Explore how synthetic AAV genomes offer a novel approach to gene editing, providing a versatile platform for precise genetic modifications
- Discuss the potential of synthetic AAV constructs as efficient non-viral gene delivery vehicles, circumventing limitations associated with traditional viral vectors
- Uncover preclinical data for a non-CRISPR alternative to gene editing, paving the way for innovative treatments with enhanced safety and efficacy profiles
10:00 am Innovative Approaches to Patient Testing and Clinical Readiness in Optogenetics for Ocular Disorders
Synopsis
- Discuss the complexities and challenges associated with testing patients with severe visual impairments, including mobility mazes, peripheral vision tests, contrast sensitivity assessments, and quality of life measurements
- Share insights into the importance of reliable baseline measurements and the ability to capture changes from baseline to assess treatment efficacy
- Discuss the significance of reliable data and preparation for clinical trials in ensuring successful translation from preclinical to clinical stages
10:30 am Morning Break & Speed Networking
Synopsis
Our speed networking is the ideal opportunity to get face-to-face time with the brightest minds working in ocular gene therapy and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against industry leaders and establish meaningful business relationships to pursue for the conference and beyond.
REFINING OCULAR GENE THERAPY DELIVERY: INNOVATIONS & CHALLENGES IN SUBRETINAL AND INTRAVITREAL TECHNIQUES
11:30 am Suprachoroidal Gene Delivery for Chorioretinal Diseases: Advancements, Potential and Challenges
Synopsis
- Microinjector-based suprachoroidal (SC) gene delivery for minimally invasive office-based gene therapy
- SCS Microinjector®-based AAV gene therapy: Preclinical and clinical safety, efficacy, and durability
- Non-viral particle-based SC gene delivery for repeatable gene therapy
- Challenges and future direction
12:00 pm Panel Discussion: Precision Delivery to the Retina: Optimizing Gene Therapy for Enhanced Efficacy and Safety
Synopsis
- Analyzing the importance of uniform drug distribution within the retina to maximize therapeutic efficacy and minimize off-target effects in gene therapy interventions
- Evaluating strategies and technologies aimed at achieving uniform retinal delivery, including advances in drug formulation, administration techniques, and pharmacokinetic modeling
- Exploring future priorities in precision targeting and uniform distribution, with a focus on personalized approaches tailored to individual patient needs and disease characteristics
12:30 pm Lunch & Networking
OPTIMIZING CAPSID DESIGN & VECTOR ENGINEERING FOR OCULAR GENE THERAPY
1:30 pm Machine Learning for Enhanced Vector Design in Ocular Delivery
Synopsis
- Gaining insights into the latest breakthroughs in capsid engineering with Dyno eCap1, a pioneering product designed for intravitreal delivery.
- Exploring how advanced algorithms are revolutionizing the design and optimization of capsids and learn how high-throughput screening and generative AI are shaping the future of drug delivery systems
- Discover how these innovations are driving the development of targeted therapies with efficacy and safety profiles
2:00 pm Advancing Gene Therapy Vectors: The Role of scAAVengr Platform in Proprietary AAV Vector Generation
Synopsis
- Discussing the utilization of library approaches to generate diverse vector variants aimed at enhancing delivery efficiency and specificity within the eye
- Exploring how the computationally guided scAAVengr platform facilitates the identification of optimal vector candidates with enhanced infectivity and celltype specificity crucial for effective treatment outcomes
- Discovering how machine learning algorithms facilitate the interpretation of complex data sets, enabling the identification of key vector characteristics correlated with superior performance in ophthalmic gene therapy
2:30 pm Unraveling the Complexity: Capsid Tropism, & Transduction in Ophthalmic Gene Delivery
Synopsis
- Examining the interplay between capsid design, vector tropism, and desired transduction levels
- Evaluating the impact of promoter selection and cassette design on vector performance and safety
- Exploring the potential of directed evolution techniques to optimize vector design, enhance tropism, and mitigate safety concerns
3:00 pm Afternoon Break & Poster Session
Synopsis
Celebrate innovation and research breakthroughs in ophthalmic gene therapy. Engage with leading experts and explore cutting-edge advancements. Don’t miss this opportunity to network and discover the future of ocular gene therapies.
3:45 pm Unconventional Capsids for Suprachoroidal Injection
Synopsis
- Utilizing cutting-edge techniques to engineer AAV capsids, optimizing tissue specificity and transduction efficiency for targeted delivery in ophthalmic disorders
- Selecting and designing capsids that enhance suprachoroidal injection, ensuring effective gene delivery while prioritizing safety and scalability
- Pioneering methods in customizing capsids to precisely target specific cell types or organelles, revolutionizing gene therapy for ophthalmic disorders
CHARTING THE COURSE: FINANCING OPHTHALMIC GENE THERAPY ADVANCEMENTS
4:15 pm Roundtable Discussion: Securing Funding for Ophthalmic Gene Therapies: Understanding Investor Priorities & Navigating the Investment Landscape in Ophthalmic Innovation
Synopsis
- Exploring the criteria that investors consider when evaluating gene therapy projects, including scientific innovation, market potential, and feasibility
- Discussing the importance of demonstrating a clear path to clinical translation and commercialization to attract investment
- Discussing the impact of regulatory approvals, clinical trial outcomes, and market dynamics on investor sentiment and funding decisions
- Identifying emerging opportunities and challenges in securing investment for ophthalmic gene therapy projects and strategies to address them