8:50 am Chair’s Opening Remarks
Receiving the Latest Clinical and Post-Approval Updates for Dry AMD Therapeutics
9:00 am Discovering the Latest Clinical Updates from Iveric Bio on Their Path to Approval
Synopsis
- Delving into the latest updates in clinical data
- Outlining recent developments in the approval process
- Leveraging lessons learnt to inform next steps
9:30 am Showcasing the Latest Clinical Updates from Inflammasome Therapeutics
Synopsis
- Outlining the mechanism of action
- Delving into the preclinical data
- Defining the next steps for the clinical trial
10:00 am Delving into the Long Term Follow Up of Patients in Phase 1 and Phase 2 Trials
Synopsis
- Receiving a program update from the OpRegen program
- Assessing the available clinical data to understand drug efficacy
- Understanding how to monitor and improve patient safety in dry AMD clinical trials
10:30 am Speed Networking and Refreshments
Synopsis
- Our structured networking is the ideal opportunity to get face-to-face time with many of the brightest minds working in gene therapy and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against the industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond
PRECLINICAL STREAM
Navigating how to Optimize Current Dry AMD In Vivo Models to Improve Efficacy
11:30 am Panel Discussion: Overcoming the Limitations of Dry AMD not having a Recognised Disease Model
Synopsis
- Incorporating more NHPs in preclinical testing to better inform the safety profile of dry AMD therapeutics
- Understanding why developing a relevant animal model for Dry AMD remains a pertinent challenge
12:00 pm Delving into the in vivo work to Identify a Lead Bicistronic Gene Therapy Candidate for Dry AMD
Synopsis
- Utilizing appropriate in vitro and mouse models to screen vector candidates and improve preclinical success
- Exploring different routes of delivery and vector capsids to confirm efficacy
12:30 pm Utilizing Mouse Models to Improve ComplementBased Gene Therapies for Dry AMD with C3 Driven Inhibition of Complement Activation
Synopsis
- Uncovering how to improve the accuracy of hitting the desired target
- Assessing the optimal location for delivery
- Highlighting different types of promoters inducible by complement activation
CLINICAL
Optimizing Clinical Trial Design for Dry AMD Therapeutics
11:30 am Panel Discussion: Navigating What Design Elements are Needed to Optimize Clinical Trials for Dry AMD?
Synopsis
- Understanding the optimal inclusion and exclusion criteria
- Considering how the heterogenicity of clinical symptoms, diagnosis and manifestation should impact early and latestage clinical trial planning
- Assessing the feasibility of having smaller scale clinical trials
12:00 pm Round Table Discussion: Assessing How to Improve Patient Experience in Dry AMD Clinical Trials
Synopsis
- Considering the patient burden when receiving frequent clinical treatments
- Optimizing the patient experience, particularly for elderly patients
- Exploring durable treatment options to lessen patient burden
12:30 pm Exploring the Burden of GA and AMD for Patients in Clinical Trials and Clinical Practice
Synopsis
- Understanding the impact of end points being based on anatomy but not vision
- Delving into the effects the treatment methodologies have on patients
- Considering clinical trial design elements to lessen the patient burden within dry AMD clinical trials and clinical practice
1:00 pm Lunch and Networking
2:00 pm
Leveraging the Recent Developments in In Vitro Models to Maximize Preclinical Efficacy
2:00 pm iPSC based-Dry AMD model using 3D-Bioprinted Choroid and RPE Cells
Synopsis
- Differentiation overview of human iPSC derived-endothelial cells, pericytes, fibroblasts and RPE cells.
- 3D bioprinting technology to generate highly vascularized choroid tissue and maturation into choroid/RPE combination of outer blood retinal barrier (oBRB) model.
- Investigation of dry-AMD phenotype using alternative pathway of complement system and complement factor H-knockout iPSC derivatives.
2:30 pm Utilizing Small Molecules to Regenerate RPE – Preclinical Insights
Synopsis
- Delving into the in vivo models used to represent RPE regeneration
- Expanding knowledge on toxicology and PK studies for the appraoch in different animal models
- Highlighting the importance of communication with regulatory bodies and sharing insights on the FDAs perspective
Investigating the Latest Advancements for Improving the Delivery of Dry AMD Therapeutics
2:00 pm Leveraging Clinical Data to Evaluate a Single Dose Intravitreal Administration of AVD-104
Synopsis
- Assessing the dose escalation and safety data
- Highlighting the benefits of using our chosen molecule
- Outlining the trial design for the 2 year trial with a 12 month primary endpoint
2:30 pm Leveraging Directed Evolution to Facilitate Targeted Delivery of an AAV-based Retinal Gene Therapy
Synopsis
- Overcoming barriers to efficient intravitreal delivery of AAVbased gene therapy to the retina
- Employing directed evolution in nonhuman primates to discover novel retinotropic AAV vectors for clinical gene therapy
- Exploring key translational research considerations to facilitate clinical trial development
3:00 pm Afternoon Break and Poster Session
Delving into the Crucial Impact of Advancing Imaging Techniques will have on Dry AMD Therapeutic Development
3:30 pm Delving into a Novel Imaging Technology for Dry AMD
Synopsis
- Understanding the development of the imaging method from rodents to humans
- Assessing the available anatomical and functional imaging methods of this novel approach
- Discovering how a strong RPE is needed to induce a strong signal
4:00 pm Exploring Early Imaging Biomarkers for Intermediate Dry AMD
Synopsis
- Integrating AI technology with imaging biomarkers
- Monitoring patient progress
- Preparing for the next gen of dry AMD therapeutics using early imaging biomarkers to prevent geographic atrophy
4:30 pm Mitochondrial-mediated Photoreceptor Protection and Visual Function in Geographic Atrophy
Synopsis
- Elamipretide has demonstrated a protective affect on OCT biomarkers of photoreceptor protection
- This was associated with an improvement in patients gaining 2+ lines of visual function
- This provides a novel path toward potential functional benefit for patients suffering from early GA and, potentially, intermediate dAMD.