8:00 am Morning Refreshments & Check In

8:25 am Chair’s Opening Remarks

ACCELERATING LONGER LASTING, LESS INVASIVE & PRECISE RETINAL DRUG DELIVERY FOR IMPROVED PATIENT COMPLIANCE IN AGING POPULATION

8:30 am Delving Into the Next Generation of Ophthalmic Drug Delivery

Synopsis

  • Understanding the latest advancements in surgical tools, catheters and active implant
  • Obtaining insights into the design, development and manufacturing scale-up processes that transform these concepts into market-ready solutions
  • Unlocking how AI/ML driven analytics are revolutionizing ophthalmic delivery

9:00 am Panel Discussion & Live Audience Q&A: Optimizing Drug Delivery to the Macula

Synopsis

  • Exploring the implications of using topical solutions for dry AMD and GA considering the spread of disease across the retina
  • Overcoming the hurdles of delivery of cell and gene therapies to understand how they can be improved to make delivery of protein therapeutics easier
  • Exploring how oral therapeutic might be the answer to achieving non-invasive retinal delivery and equal exposure to both eyes at the same time

EXPLORING NOVEL TREATMENT MODALITIES TO REDUCE INJECTION FREQUENCY & RETINAL INFLAMMATION

9:30 am Novel Orally Administered Small Molecule (Xiflam) Targeting Auto- Inflammation in Patients with Intermediate AMD

Synopsis

  • Understanding the pathological Cx43 hemichannel and its role in inflammasome mediated disease progression in intermediate AMD
  • Clinically developing a novel oral therapy to inhibit the pathologically open Cx43 hemichannel to modulate ATP activation and perpetuation of the inflammasome.
  • Sharing preclinical data showing the effect of Xiflam vs Placebo in maintaining retinal integrity and visual function, in a model of the Dry form of Macular Degeneration

10:00 am Morning Break & Networking

10:30 am Examining a Novel Fas Receptor Inhibitor for Dry AMD

Synopsis

  • Discussing the pre-clinical data regarding the role of Fas in retinal disease and AMD
  • Discussing the development of ONL1204, a novel inhibitor of the Fas receptor
  • Discussing the results of the Phase 1 study of ONL1204 in patients with GA secondary to AMD and the upcoming Phase 2 trial

11:00 am Developing AI-based Strategies to Leverage Imaging & Functional Biomarkers in Patient Selection for Geography Atrophy Trials

  • Deepayan Kar Ophthalmology Biomarker, Translational Development Scientist, Apellis Pharmaceuticals

Synopsis

  • Discussing the pioneering work in developing OCT-based imaging and other diagnostic tools for more objective, faster biomarkers and functional endpoints
  • Establishing the current endpoints for geographic atrophy, measuring the death of RPE and photoreceptor cells, predict lesion growths and functional outcomes for regulatory approvals
  • Uncovering the recent advancements in imaging GA by using AI algorithms to measure the extent of dead photoreceptor and RPE cells and identifying the right patients for treatment in clinical trials

11:30 am Advancements in Adult RPE stem cells for Dry AMD

  • Rajesh Rao Ophthalmology & Visual Sciences Professor, University of Michigan

Synopsis

  • Exploring the latest advancements in using iPSCs to generate RPE cells for cell replacement therapy in late-stage AMD
  • Discussing the process of reprogramming iPSCs into functional RPE cells and the challenges associated with ensuring their proper integration and functionality within the retina
  • Presenting preclinical and clinical evidence supporting the safety and efficacy of iPSC-derived cell therapy in restoring retinal function and preserving vision in patients with advanced AMD

12:00 pm Lunch & Networking Break

1:00 pm Fire-Side Chat: Discussing the Journey of Gaining Approval for GA Drugs & Current Challenges In the Market

Synopsis

  • Discussing the path to gain approval for complement inhibitors for GA by the FDA and the rejections from the EMA
  • Minimizing toxic side effects and reducing retinal inflammation when delivering via intravitreal injections
  • Debating the optimal less invasive, longer lasting administration route to reduce frequence of injections for elderly patient population
  • Understanding how the GA drug can be improved to stop disease progression and be used to treat intermediate AMD before disease reaches atrophic stage

UTILIZING GENE AND CELL THERAPY TO MAINTAIN RPE HEALTH IN AGING PATIENT POPULATIONS

1:30 pm Discussing Innovative Approaches in Gene Therapy for AMD by Targeting Inflammation, Establishing Retinal Bio-Factories as Long- Lasting Solutions

Synopsis

  • Exploring cutting-edge research in gene therapy targeting inflammatory pathways associated with the complement system dysregulation in AMD
  • Discussing gene delivery vectors and viral vectors engineered to express therapeutic proteins locally in the retina, effectively turning it into a bio-factory
  • Overcoming the challenge of frequent, invasive injections by developing long-lasting gene therapies as a once-and-done treatment for late-stage AMD

2:30 pm Delivering Cell Replacement Therapy for Geographic Atrophy by Designing Precise & Specialized Formulations for Subretinal Injection

  • Max Cotler Translational Medicine Consultant, GelMEDIX

Synopsis

  • Using subretinal delivery to locally deliver cell replacement therapy to disease area with atrophy around the macula
  • Overcoming patient concerns related to subretinal delivery and addressing alternative less invasive delivery mechanisms
  • Ensuring that the cell therapy is not lost due to high pressure when being delivered via subretinal injections

3:00 pm Delving into Long-term Follow-Up of Subjects in a Phase 1/2a Clinical Trial of an Allogeneic, Bioengineered hESC-Derived RPE Implant for Advanced Dry Age-Related Macular Degeneration: Next Steps in Development

Synopsis

  • Design of CPCB-RPE1
  • Results of Phase 1/2a Clinical Trial Through Long-term Follow-up
  • Next Steps in the Development Path for the Product

3:30 pm Chair’s Closing Remarks & End of Conference