8:30 am Morning Refreshments & Check In
8:55 am Chair’s Opening Remarks
SPEARHEADING DEVELOPMENT TO ACCELERATE THE NEXT GENERATION OF DRY AMD & GA THERAPEUTICS
9:00 am Stage-specific Cell Therapy Strategies to Treat Retinal Diseases
Synopsis
- Cell therapy platform offering a versatile approach to treat ophthalmic indications affecting retinal health and function
- Neuroprotective cell therapies provide support to delay the loss of a patient’s own retinal tissues
- Cell replacement therapies aim to reconstruct degenerated tissues to restore function lost in disease
9:30 am Delving Into Boehringer Ingelheim’s Pipeline: Strategy & Stages of Development
Synopsis
- Integrating multi-disciplinary research and technology to preserve vision and enhance life quality
- Targeting vascular, neuronal, and inflammatory mechanisms in retinal diseases
- Exploring various routes of administration for AMD
10:00 am Speed Networking Break
Synopsis
An optimal chance to network one-to-one with leading retinal and ophthalmology experts working across dry AMD and geographic atrophy. Learn how your peers are discovering new target pathways, novel modalities and preclinical models to fast-track earlier interventions for dry AMD into the clinic.
10:30 am Morning Break
SHOWCASING NEXT GENERATION IN VITRO & IN VIVO PRECLINICAL MODELS TO SUPERCHARGE DRY AMD DISEASE RECAPITULATION
11:00 am Unlocking Insights Into AMD Pathogenesis with Advancements in In Vitro Modelling Including Cell Cultures & Retinal Organoids
Synopsis
- Reviewing the differential susceptibility of macular RPE cells and the role of RPE dysregulation in AMD pathogenesis
- Recapitulating the morphological and functional abnormalities of dry AMD in in vitro models including spontaneously formed cell lines, immortalized cells, primary human and animal RPE cells, and embryonic and induced pluripotent stem cell (iPSC) derived RPE
- Generating retinal organoids mimicking in vivo retinal development and its molecular and cellular profiles
UTILISING GENOMICS & PROTEOMICS STUDIES TO ACCELERATE TARGET IDENTIFICATION & VALIDATION FOR EARLY THERAPEUTIC INTERVENTION
11:30 am Functional Genomics in AMD: From Genetic Associations to Understanding Disease Mechanisms
Synopsis
- Genome-wide association studies (GWAS) have identified >60 loci that are associated with AMD; main disease pathways include the complement system, lipid metabolism, and extracellular matrix
- The vast majority of AMD-associated loci have not yet been assessed in functional studies. Functional genomics approaches can help unravel causal variants, genes and disease mechanisms. Quantitative trait loci (QTLs), chromatin accessibility and chromatin capture can help prioritize causal genes in GWAS loci; recent studies have pinpointed to genes in other pathways
- A shift to functional genomics is needed to capitalize on the success of GWAS studies in AMD, including the need for additional and larger datasets, and validation studies in disease-relevant cell types
12:00 pm Delving Into the Genetics of Dry AMD: Disease Risk & Disease Progression
Synopsis
- Identification of therapeutic targets for age-related macular degenerations using genome-wide association studies (GWAS)
- Leveraging deep clinical data in large-scale biobanks for GWAS of AMD, disease subtypes, and progression
- Inferring pathogenic genes, pathways, and cell types by incorporating retina multi-omics data
12:30 pm Lunch & Networking Break
1:30 pm PolySia Nanoparticle Modulates Macrophages Response Through Siglec Agonism as Mechanism of Action for Macular Degeneration Treatment
Synopsis
- Revolutionizing the latest advancement in genomics and proteomics to improve understanding of disease pathogenesis, beyond simple genetic mutations
- Uncovering the relationships between genomic alterations and disease phenotypes, and gaining insights into how inflammation can lead to different disease manifestations
- Discussing emerging methodologies and technologies advancing the understanding of complex molecular interactions underlying disease progression and paving the way for more effective interventions and personalized medicine approaches
2:00 pm Roundtable Discussion: Overcoming Discovery, Preclinical & Clinical Challenges at Each Stage of Drug Development for Dry AMD & Geographic Atrophy
Synopsis
- Discovery: Which of the latest innovations or targets hold the greatest promise to improve disease understanding?
- Translational: What are the current safety and efficacy of preclinical models and how can they be improved for smoother translation to the clinic?
- Clinical: How to optimize endpoints being achieved in clinical trials for dry AMD and GA?
Moderator Feedback & Audience Debate:
Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open up a wider audience debate
2:30 pm Afternoon Break & Scientific Poster Session
3:00 pm Delving Into C1q Inhibition: A Unique Neuroprotective Approach to Preserve Vision in Dry AMD
Synopsis
- C1q drives synaptic pruning and elimination of neurons in many neurodegenerative diseases, including dry AMD
- ANX007 inhibition of C1q resulted in consistent, significant protection from visual function loss in dry AMD
- Preservation of function with ANX007 is supported by protection of retinal structures associated with visual function, especially structures near the foveal center
3:30 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