8:00 am Registration & Breakfast

8:50 am Chair’s Opening Remarks

Narrowing the Translatability Gap with Preclinical Models

9:00 am Ensuring Translatability Between Canine Models & Human Clinical Studies


  • Similarities and differences between canine IRDs and the human counterparts.
  • Examples of advancing therapies with dog models.
  • Advantages and disadvantages of testing potentially translatable gene therapies in dog models – do they reflect ultimate outcomes in human trials?

9:30 am Optimizing the Clinical Relevancy of Animal Model Studies


  • Exploring the need for using both mouse models of retinal degeneration and wild-type large animal models for better understanding protein expression and function levels
  • Finetuning the applicability of endpoints in animal models ahead of clinical trials
  • What does the FDA expect to see from animal model studies in your IND submission?

10:00 am Human iPSC-Derived Retina Models as a Tool for Assessing Gene Therapy Efficacy for the Treatment of Retinal Diseases

  • Valeria Chichagova Associate Director and Head of Retina and iPSC Technology, Newcells Biotech


  • Human iPSC-derived retinal models are increasingly showing utility in gene therapy applications
  • We have generated data to demonstrate their use for AAV vector efficacy screening using both retinal organoids and RPE cells derived from human iPSCs

10:10 am Leveraging the Latest Retinal Organoid Developments


  • What are the advantages of using iPSC-derived retinal organoids over traditional animal model studies?
  • Understanding the latest developments for their use in gene therapy studies
  • How close are we to seeing FDA guidance documents on retinal organoids?

10:40 am Morning Break & Networking


Exploring Different Approaches to Treating Inherited Retinal Disorders

11:20 am Assessing the Latest Developments with Nanoparticle-Based Gene Therapy

  • Viral Kansara Vice President - Preclinical Development, Clearside Biomedical


  • Outlining the status quo with the nanoparticle field in ocular gene therapy
  • Updates on progression developing a non-viral vector for large gene delivery
  • Evaluating the benefits and drawbacks of such system compared to traditional viral delivery methods

11:50 am Exploring a Gene-Agnostic Therapeutic Approach to Target Multiple IRD Etiologies


  • There are too many disease genes (>200) leading to blindness to make single gene therapy realistic for a larger number of families
  • There may be common problems across disease gene families that can be identified and approached using a more generic therapy
  • We have developed an AAV gene therapy targeting inflammation, oxidative damage, and metabolic shortcomings that benefit cone photoreceptors and vision across several mouse models


Finetuning the Design of First-in-Human Clinical Studies

11:20 am Striking a Balance with Ascending Dose Studies


  • Understanding the unique challenges behind achieving the correct dose for FIH studies
  • How many doses should drug sponsors be including in ascending dose studies?
  • Taking into consideration immunogencitiy risks when setting upper dose limits

11:50 am Designing Trials for Success & to Capture the Patient Perspective

  • Todd Durham Senior Vice President - Clinical and Outcomes Research, Foundation Fighting Blindness


  • An innovative platform approach to collecting natural history data for inherited retinal diseases
  • Incorporating learnings from natural history studies into clinical trial design
  • What are the expectations of IRD patients?

12:20 pm Lunch & Networking

Emerging Gene Therapy Glaucoma & Neuroprotection Treatment Candidates

1:20 pm Engineered Mechanosensitive Channel as an Outflow Actuator for Gene Agnostic Gene Therapy of Glaucoma

  • Adnan Dibas Principal Scientist, Nanoscope Technologies


  • Presenting an engineered mechanosensitive channel (EMC)
  • Exploring results from murine model studies, including comparisons with standard glaucoma treatment options
  • Outlining its potential future relevancy as a pathway for developing a gene therapy to target

1:50 pm XIAP Anti-Apoptotic Gene Therapy for the Treatment of Glaucoma

  • Catherine Tsilfidis Senior Scientist, Regenerative Medicine Program, Ottawa Hospital Research Institute


  • Using gene therapy to target apoptosis of the cell (the final endpoint for many ocular diseases) is a general strategy that doesn't target a specific mutation
  • The X-linked inhibitor of apoptosis (XIAP) is able to block apoptosis by preventing caspase activation, but can also block necroptosis and inflammasome formation
  • XIAP gene therapy is effective in protecting retinal ganglion cell structure and function in a mouse model of glaucoma and represents a general therapy that can be used to target various forms of retinal disease, irrespective of underlying pathology

Highlighting the Role of Gene Therapy for Age- Related Macular Degeneration

1:20 pm Driving Wet AMD Gene Therapies to Market

  • Adam Turpcu Vice President - Clinical Development & Access Strategy, Adverum Biotechnologies


  • Outlining the key clinical trial challenges in developing anti- VEGF gene therapies
  • Updates from the LUNA clinical study
  • Optimizing manufacturing platforms to ensure product quality and process control

1:50 pm Gene Therapy for Dry AMD

  • Daniel Chao Senior Director and Clinical Lead, Retinal Diseases, Janssen


  • Overview of gene therapy approaches for Geographic Atrophy in clinical trials
  • Methods and biomarkers to demonstrate biological activity and target engagement in the clinic
  • Unique considerations for dry AMD gene therapy clinical trial design

2:20 pm Afternoon Refreshments & Networking

Improving Our Understanding of Immune & Inflammatory Responses to Ocular Gene Therapies

2:50 pm Exploring the Link Between Viral Vector Design & Quality; & Ocular Inflammation


  • Evaluating which components of the vector stimulate inflammatory responses
  • Can improved promoters and enhancers be developed to deliver genes effectively without triggering the immune response?
  • Outlining the role of novel vector generation methods in reducing ocular inflammation

3:20 pm A Report on the Latest Understanding of Gene Therapy-Associated Uveitis (GTAU)

  • Christine Kay Clinical Ophthalmology Advisor, Atsena Therapeutics


  • Evaluating the key drivers of GTAU in recent gene therapy clinical trials
  • Exploring the link between GTAU and the route and method of vector administration
  • Which preclinical models are best suited to test the various variables associated with GTAU?

3:50 pm Chair’s Closing Remarks

4:00 pm End of Conference