Pre-Conference Workshop Day
Tuesday September 21, 2021 | 8am - 6pm EST
Workshop A: Immune Response Mitigation Strategies for the Eye | 8am - 11am
With the eye being an immune privileged site lends ophthalmic disorders make a an optimal target for gene therapy vectors, often associated with toxic effects. However, there are still off target effects and inflammation as a result of delivery route.
Workshop will include:
- Understanding the biology of immune reactions of the eye
- Discussing inflammation and its impact on trials
- Pros & cons of subretinal delivery such as scalability
- Evaluating different strategies being used in the clinic to manage side effects
Dominik Fischer
Principal Investigator &
Consultant Ophthalmic Surgeon
Oxford Eye Hospital
Kanmin Xue
Wellcome Trust Clinician Scientist
Fellow & Honorary Consultant
Vitreoretinal Surgeon
University of Oxford
Workshop B: Translating Next Generation Vectors for the Eye | 11:30am - 2:30pm
To overcome delivery challenges, novel vectors are designed to improve specificity, mitigate off target effects and enhance efficacy.
Workshop will include:
- Discovery and preclinical data for novel vectors targeting the retina
- Exploring novel preclinical models of ophthalmic disorders and optimizing the data obtained
- Discussing current early clinical data for novel vectors
Sanford Boye
Director of Vector Core,
Powell Gene Therapy Center
University of Florida
Chief Technology Officer
Atsena Therapeutics
Ryan Boyd
Senior Research Scientist, Ophthalmology
Charles River Laboratories
Workshop C: Antisense Oligonucleotides: Evaluating Different Approaches for Ophthalmic Disorders | 11:30am - 2:30pm
Evaluating the progress with antisense oligonucleotides, cell therapy and alternative therapies for the treatment of retinal disorders to improve your understanding of the wider ophthalmology landscape. With insights from:
Robert Koenekoop, Professor, McGill University; Director,
Laboratory for Retinal Genetics & Therapeutics; Chief
Paediatric Ophthalmology, Montreal Children’s Hospital
Karmen Trzupek, Director, Clinical Trial Services & Rare
Disease Genetics Services, InformedDNA
Ben Shaberman, Senior Director, Scientific Outreach,
Foundation Fighting Blindness
Erwin van Wyk, Assistant Professor, UMC Nijmegen
Stephen Russell, Professor; Service Director, Vitreoretinal
Diseases & Surgery, University of Iowa
David Birch, Scientific Director, Retina Foundation of the
Southwest
Andy Bolan, Associate Director, Patient & Medical
Community Engagement, ProQR Therapeutics
Francesca Diodati, Information Specialist, University
Hospital of Parma
Molly Watt, Accessibility Consultant, Director, Molly Watt Talks
Aniz Girach
Chief Medical Officer
ProQR
Workshop D: Showcasing the Translatability of Suprachoroidal Delivery | 3pm - 6pm
With scalability challenges, the invasive nature and specialised skill set required for subretinal delivery, intravitreal delivery optimization is a priority for the field. Providing an alternative outpatient approach however is suprachoroidal delivery, which is showing promise in preclinical research.
Workshop will include:
• An explanation of suprachoroidal delivery and its advantages
• Recent preclinical and clinical data to support its implementation into gene therapy programs
Thomas Ciulla
Vitreoretinal Medicine & Surgery, Chief
Medical Officer & Chief Development Officer
Clearside Biomedical Inc.
Steve Pakola
Chief Medical Officer
REGENXBIO
Peter Campochiaro
Director, Retinal Cell & Molecular
Laboratory; Professor of Ophthalmology
Johns Hopkins
University School of Medicine