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
- Simon Petersen-Jones Professor, Michigan State University
- 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
- Laura Moreno Leon Senior Research Scientist, Iveric Bio
- 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
- Tassos Georgiadis Vice President - Preclinical Development, MeiraGTx
- 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
- Constance Cepko Professor, Harvard University
- 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
- Arun Upadhyay Chief Scientific Officer, Ocugen
- 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
- Abraham Scaria Chief Scientific Officer, Beacon Therapeutics
- 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?