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
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
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 Multi-Characteristic Opsin for Treating Geographic Atrophy
Synopsis
- Development of GA model in multiple species
- Assessing the latest preclinical data on intravitreal Multi-Characteristic Opsin therapy of GA in NHP
- Use of a laser delivery of MCO for treatment of GA
2:00 pm Investigating the Opportunity for a New Oral Chemical Entity, Gildeuretinol, for the treatment of Geographic Atrophy Secondary to AMD
Synopsis
- Summarizing the gildeuretinol program and Alkeus’ efforts to understand mechanisms of disease
- Discussing results of SAGA, a 2-year, randomized, double-masked, placebo-controlled study
- Sharing next steps in the development pathway for gildeuretinol
2:30 pm Delivering Cell Replacement Therapy for Geographic Atrophy by Designing Precise & Specialized Formulations for Subretinal Injection
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