8:00 am Check-In & Coffee

8:50 am Chair’s Opening Remarks


9:00 am Extracting Insights from Novartis for Future Gene Therapy Development

  • Stephen Poor Executive Director & Head of Global Program Clinical, Novartis AG


  • Analyzing factors contributing to failures in gene therapy development for ophthalmic disorders
  • Highlighting pitfalls and challenges encountered in clinical trials and translational research
  • Presenting case studies of failed gene therapy programs and the key lessons learned from these setbacks

9:30 am Panel Discussion: Maximizing Ophthalmic Gene Therapy Trial Success Strategies

  • Stephen Poor Executive Director & Head of Global Program Clinical, Novartis AG
  • Claire Gelfman Senior Scientific Advisor, Foundation Fighting Blindness
  • Abraham Scaria Chief Scientific Officer, Beacon Therapeutics
  • Kenji Fujita Chief Medical Executive, Atsena Therapeutics


  • Discussing strategies for improving trial design to mitigate risks and enhance success
  • Exploring the regulatory landscape and its impact on gene therapy trials, including approval processes and compliance requirements
  • Detailing actionable steps derived from past failures to inform future gene therapy endeavours and maximize trial outcomes

10:00 am Morning Break & Networking

11:00 am Roundtable: Navigating Phase 2 Trials: Ensuring Safety & Efficacy in Gene Therapy for Ophthalmic Disorders

  • Kali Stasi Senior Vice President - Clinical Development, Adverum


  • Exploring the intricate balance between safety and efficacy assessments in phase 2 trials for gene therapy interventions targeting ophthalmic disorders
  • Addressing the unique hurdles faced in validating safety parameters and defining efficacy endpoints, particularly in slowly progressive diseases characteristic of ophthalmic disorders
  • Strategies for seamless transition from phase 2 to phase 3 trials, emphasizing the critical importance of robust data interpretation, regulatory compliance, and maintaining momentum towards commercialization

11:30 am Advancements in Gene Therapy for Ophthalmic Disorders: Ocugen’s Clinical Program Updates

  • Arun Upadhyay Chief Scientific Officer, Head of Research and Development, Ocugen


  • Advancing the transition from preclinical to clinical stages and a gene-agnostic trial approach for diverse patient recruitment
  • Optimizing the enrolment progress and Phase 3 developments for Leber Congenital Amaurosis (LCA)
  • Uncovering the safety profile and efficacy data for Dry Age-Related Macular Degeneration (AMD) program

12:00 pm Exploring XLRS Cohort Update: Dosing, Safety, and Efficacy Insights


  • Explore dosing strategies employed within the XLRS cohort, analyzing the rationale behind dosage adjustments and their impact on treatment outcomes
  • Examine the safety profile observed during the trial, highlighting any notable findings and addressing concerns regarding adverse events
  • Dissect the efficacy data gleaned from the XLRS cohort, presenting key findings and potential implications for patient outcomes
  • Conduct a comparative analysis to elucidate any discernible differences between the XLRS cohort

12:30 pm Lunch & Networking


1:30 pm Preclinical Insights Into Intravitreal Gene Therapy for Rare Retinal Diseases


  • Explore the pioneering use of AAV based intravitreal gene therapies for treating Blue Cone Monochromacy and Achromatopsia
  • Examine preclinical data and draw insights from preclinical models to assess the efficacy and safety profiles of intravitreal gene therapy for rare inherited retinal diseases
  • Explore strategies for optimizing the delivery of gene therapies via intravitreal administration such as dosage and administration techniques that influence the therapeutic outcome and durability of treatment effects

2:00 pm Overcoming Gene Editor Delivery Challenges to Maximize Translational Success

  • Adam Silverman Vice President, Protein Sciences, Spotlight Therapeutics


  • Explore methodologies to assess safety and efficacy in preclinical models and highlight the importance of early-stage validation to mitigate risks and optimize translational success
  • Share progress in developing a non-viral, engineered ribonucleoprotein (eRNP) CRISPR gene editor platform enabling safe and effective gene modification via subretinal administration
  • Examine strategies to navigate translational challenges in ophthalmic disorders and discuss how we are working toward overcoming model limitations

2:30 pm Insights from Preclinical Disease Modeling in Ophthalmic Gene Therapy


  • Delving into the intricacies of preclinical disease modeling, exploring how advanced techniques and diverse disease models inform the development of effective ophthalmic gene therapies
  • Gaining valuable insights into prioritizing preclinical research efforts, focusing on key aspects such as vector engineering, delivery routes, and expression data to optimize therapeutic strategies for optic disorders
  • Exploring the journey from preclinical to clinical research, uncovering the pivotal role of disease modeling in designing regulatory pathways and translating promising therapies from bench to bedside

3:00 pm Afternoon Break


4:00 pm Navigating Challenges in Site Selection & CRO Management for Ophthalmic Clinical Trials

  • Jean Chang Vice President - Clinical Operations, Nanoscope Therapeutics Inc


  • Exploring the critical process of identifying capable and experienced principal investigators for ophthalmic clinical trials, emphasizing the unique requirements of gene therapy studies in ophthalmology
  • Delving into the pivotal role of contract research organizations (CROs) in site monitoring and data management within ophthalmic clinical operations
  • Gaining insights from recent experiences with databases and how to implement strategies for continuous improvement and success in clinical operations

4:30 pm Navigating Clinical Trial Readiness: Strategies for Resourcing & Supply Chain Management


  • Learn how to anticipate and mitigate challenges related to resourcing to maintain trial momentum and efficiency
  • Examine the critical role of supply chain management in clinical trial readiness, particularly in the context of ocular therapies
  • Discover strategies for enhancing supply chain resilience and flexibility to adapt to evolving trial requirements and unforeseen circumstances
  • Learn proactive approaches to address risks and maintain regulatory adherence throughout the trial lifecycle, ultimately safeguarding patient safety and data integrity

5:00 pm Chair’s Closing Remarks