8:25 am Chair’s Opening Remarks

Receiving the Latest Clinical and Post-Approval Updates for Dry AMD Therapeutics

8:30 am Leveraging the Experiences of IVERIC Bio to Optimize the Dry AMD Therapeutic Approval Process

  • Luke Zack Senior Director & Global Head of Regulatory Strategy, IVERIC bio

Synopsis

  • Defining and building RA Strategy and Metrics of success
  • Collaborating effectively with the FDA to streamline the approval process
  • Sharing key insights to reduce future barriers to approval

9:00 am Showcasing the Latest Clinical Updates from Inflammasome Therapeutics

  • Paul Ashton President and CEO, Inflammasome Therapeutics

Synopsis

  • Outlining the mechanism of action
  • Delving into the preclinical data
  • Defining the next steps for the clinical trial 

9:30 am Delving into the Visual Function and Structural Changes of Patients in the Phase 1/2a OpRegen Study

  • Brian Culley Chief Executive Officer, Lineage Cell Therapeutic

Synopsis

  • Receiving a program update from the OpRegen program
  • Assessing the available clinical data to understand OpRegen safety and preliminary activity
  • Understanding the potential benefit of optical coherence tomography for assessing OpRegen activity

10:00 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

Navigating how to Optimize Current Dry AMD In Vivo Models to Improve Efficacy

11:00 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

11:30 am Animal Models for Dry AMD Drug Development

Synopsis

12:00 pm Delving into the In Vivo Work to Identify a Lead Bicistronic Gene Therapy Candidate for Dry AMD

Synopsis

  • Utilizing appropriate in itro 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 Complement-Based Gene Therapies for Dry AMD with C3 Driven Inhibition of Complement Activation

  • Baerbel Rohrer Endowed Chair, Gene & Pharmaceutical Treatment of Retinal Degenerative Disease, Medical University of South Carolina

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

1:00 pm Lunch and Networking

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

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 Panel Discussion Discovering the Benefits & Short Falls of Using iPSderived RPE Cells

  • Tea Soon Park Scientist Ophthalmic Genetics & Visual Function Branch (OGVFB), National Institute of Health
  • Jogin Desai Founder and CEO, Eyestem Research Pvt. Ltd.
  • Jane Lebkowski President, RPT

Synopsis

  • Strategizing how to replicate the many cell types that contribute to dry AMD in an IPS RPE model
  • Understanding how well the IPS RPE models replicate what is happening in vivo
  • Navigating the limitations of the IPS RPE models lacking a photoreceptor and exploring if co-culturing could overcome this limitation
  • Delving into the importance of growing the IPS-derived RPE cells under stressful conditions to mimic the conditions of the disease
  • Exploring the scalability capacity of iPS-derived RPE cells

3:00 pm Beyond One-Size-Fits-All; Optimizing Intraocular Drug Delivery

Synopsis

  • Navigating the challenges, insights from an ophthalmologist’s perspective
  • Assessing accuracy of syringes used for intravitreal injections
  • Understanding the implications of injection volume on intraocular pressure
  • Tailoring delivery devices for intravitreal injection

3:30 pm Afternoon Break and Poster Session

Delving into the Crucial Impact of Advancing Imaging Techniques will have on Dry AMD Therapeutic Development

4:15 pm Exploring Early Imaging Biomarkers for Intermediate Dry AMD

  • Jing Hua Director - Clinical Development & 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

4:45 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.

Optimizing Clinical Trial Design for Dry AMD Therapeutics

5:15 pm Panel Discussion: Navigating What Design Elements are Needed to Optimize Clinical Trials for Dry AMD?

  • Brian Culley Chief Executive Officer, Lineage Cell Therapeutic
  • Jason Ehrlich CEO, Kodiak Sciences
  • Daniel Chao Senior Director & Clinical Leader, Early Development & Translational Medicine, Janssen
  • Vicken Karageozian CEO, Allegro Ophthalmics
  • Stephen Poor Global Program Clinical Head, Scientific Engagement Lead, Ophthalmology Development, Novartis AG

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

6:00 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

6:30 pm Chair’s Closing Remarks

End of Day One