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

  • Paul Ashton President & Chief Executive Officer, 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

  • Brian Culley Chief Executive Officer, Lineage Cell Therapeutic

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

  • Samarendra Mohanty Chief Scientific Officer & President, Nanoscope Technologies
  • Daniel Chao Senior Director and Clinical Lead, Retinal Diseases, Janssen Global Services
  • Shelley Boyd Chief Scientific Officer, Translatum Medicus

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?

  • Vicken Karageozian CEO, Allegro Ophthalmics
  • Daniel Chao Senior Director and Clinical Lead, Retinal Diseases, Janssen Global Services
  • Jason Ehrlich CEO, Ollin Biosciences, Inc
  • Stephen Poor Global Program Clinical Head, Scientific Engagement Lead, Ophthalmology Development, Novartis
  • Brian Culley Chief Executive Officer, Lineage Cell Therapeutic
  • Rachel Sartaj Associate Director - Clinical Development, Gyroscope Therapeutics

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

  • Rachel Sartaj Associate Director - Clinical Development, Gyroscope Therapeutics

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

  • Tea Soon Park Scientist Ophthalmic Genetics & Visual Function Branch (OGVFB), National Institute of Health Science

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

  • Jing Hua Director Clinical Development and Medical Affairs, Boehringer Ingelheim

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

  • Reenie McCarthy Director, President and Chief Executive Officer, Stealth BioTherapeutics

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.

5:00 pm Chair’s Closing Remarks

End of Day One