8:55 am Chair’s Opening Remarks
Establishing Recent Clinical Data to Strengthen Chances of Rare Ophthalmic Regulatory Submissions
9:00 am Updating on Lighthouse Clinical Trial of ATSN-201 to Bring XLRS Patient Closer to Life-Changing Treatment
Synopsis
- Presenting the progress of Part B of the Lighthouse study to evaluate the subretinal injection of ATSN-201
- Illustrate the nature of AAV.SPR capsids to ensure safe delivery of AAV gene therapy
- Compare the success and challenges of Part B to Part A of phase II/III to optimize phase II/III trials and beyond
9:30 am Showcasing Phase 2 Clinical Data for Beacon Therapeutics Laru-Zova to Apply Learnings to Phase 3 Trials
Synopsis
- Understanding the success of the subretinal administration of lara-zova to maximize delivery
- Analyzing phase 2 safety and efficacy results of DAWN trial to ensure quality takeaways for future clinical trials
- Discussing the pitfalls and side effects during the phase 2 trials to prevent similar problems during the next clinical trial
10:00 am Accelerating Progression of TTX-381 through the Value Chain to Address Blindness in CLN2 Batten Disease
Synopsis
- Discussing the need for rapid development of Tern Therapeutics TTX-381 to preserve vision in patients with CLN2 Disease
- Highlighting the steps taken to maximize speed of development, including novel preclinical, clinical, regulatory, and CMC approaches
- Evaluating implications for rapid development of gene therapies for other rare ophthalmic diseases
10:30 am Advancing mRNA Therapies in Rare Ophthalmic Disorders: Lessons from Clinical Development
Synopsis
- Differentiate mRNA therapies from DNA-based gene therapies, highlighting unique opportunities and challenges in ocular indications
- Share lessons learned from rare disease trial execution, including patient population considerations and regulatory navigation
- Explore the role of novel and alternative endpoints in inherited retinal disorder studies to better capture efficacy
11:00 am Morning Refreshment Break & Speed Networking
Diversifying Funding Models & Partnership Strategies to Overcome Investment Barriers for Rare Ophthalmic Gene Therapies
12:00 pm Interview the Investor: Engaging Investor Perspectives on Ophthalmic Gene Therapy Investment Criteria to Unlock Funding Opportunities for Novel Ocular Gene Therapies
Synopsis
- Understanding investor requirements for clinical efficacy data and risk mitigation strategies to meet evolving investment standards
- Exploring alternative value propositions for investment to address rare market size limitations
- Identifying strategic partnership opportunities and funding diversification approaches to make gene therapy development financially sustainable
1:00 pm Lunch Break & Networking
Spearheading Bi-Functional Gene Therapy Technology to Induce Optimal Efficacy Against Prevalent Ocular Diseases
2:30 pm Developing Dual-Acting Gene Therapy to Combat Geographic Atrophy & Prevent AMD Progression
Synopsis
- Engineering bi-functional IKAR-001 gene therapy to provide synergistic complement targeting, inflammation control, and neuroprotection for GA treatment
- Utilizing advanced preclinical models to validate therapeutic efficacy and establish proof-of-concept for dual-protein approach
- Outline the role of PEDF and sCD46 in wet-AMD to emphasis the effectiveness of IKAR-001 gene therapy technology
Strengthening Patient Partnership Models & Retention Strategies to Enhance Clinical Trial Outcomes & Regulatory Engagement
3:00 pm Roundtable Discussion: Harnessing Patient-Led Regulatory Engagement to Establish Precedents & Streamline Approval Pathways for Ophthalmic Gene Therapies
Synopsis
- Positioning patients as regulatory advocates to push for approval pathway concessions to establish precedents that benefit the entire industry
- Demonstrating how patient voices carry unique regulatory influence to achieve more efficient development requirements and reduce trial burdens
- Creating a “trojan horse” approach where patient-driven regulatory wins to unlock streamlined pathways for larger companies to leverage
4:00 pm Addressing Patient Compliance Challenges to Ensure Long-Term Follow-Up Success in Ophthalmic Gene Therapy Trials
Synopsis
- Understanding patient motivations to develop strategies that maintain engagement beyond initial treatment administration
- Implementing support systems for aging patient populations and changing life circumstances to overcome barriers like transportation limitations and caregiver responsibilities
- Creating flexible follow-up protocols that accommodate patient needs over multiyear studies to reduce dropout rates while maintaining regulatory-compliant data collection