8:40 am Chairs Opening Remarks
Overviewing Recent Developments In The Ophthalmic Gene Therapy Space
8:50 am Panel Discussion: Gene Therapy for Ophthalmic Disorders – A Year in Review
Synopsis
- Developments and setback in the last 12 months
- Overview of instrumental work that has worked towards solving major issues
- Discussing the sustainability of ophthalmic gene therapy
- What does the coming year look like?
9:35 am Highlighting the Modifier Gene Therapy Approach for the Treatment of Retinitis Pigmentosa
Synopsis
- Discussing the preclinical data of our Retinitis Pigmentosa treatment
- Reviewing the early clinical data and outlining their indications
- Explaining the science behind the modifier gene therapy approach
10:05 am Utilizing Gene Therapy to Induce Long-Term Treatment of Wet AMD & Diabetic Retinopathy
Synopsis
- Outline the RGX-314 gene therapy program
- Discussing why suprachoroidal/subretinal delivery are optimal for this treatment
- Reviewing the current clinical data from Phase III clinical trials
10:35 am Structured Networking
Synopsis
This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the gene therapy ophthalmic field and establish meaningful business relationships to pursue for the rest of the conference.
11:31 am
PRECLINICAL STREAM
Examining Current & Next Generation Disease Models for the Eye to Provide Better Translational Data
11:35 am Highlighting the Most Efficacious Animal Models for Testing the Inflammatory Response of Gene Therapy Delivery to the Eye
Synopsis
- Considerations for model development
- Testing of anti-inflammatory therapy in a mouse mode
- Observed challenges and next steps
12:05 pm Discovering Canine Models for Inherited Retinal Diseases to Evaluate Retinal Gene Therapies
Synopsis
- Natural history of disease in dogs versus humans
- Route of delivery of AAV-mediated gene therapy
- Outcome measures of efficacy/safety studies in dogs
12:35 pm Establishing In Vitro Translational Models for Inherited Retinal Dystrophies
Synopsis
- The use of genome editing tools to generate eye disease models in vitro
- The advantages of retinal cells derived from induced pluripotent stem cells
- Understanding the mechanisms of disease
- Therapeutic approaches using cell-based models
CLINICAL STREAM
Mastering Patient Outreach & Enrolment for Better Communication with the Drug Recipients
11:35 am Understanding Gene Therapy After the Data: How Does it Reach the People in Need?
Synopsis
- Main issues in the implementation of innovative gene therapies from the country perspective (Payers, Doctors and Patients)
- Main opportunities of improvement – what have we learned so far?
12:05 pm Creating an Effective Patient Outreach Programme to Ensure Patient Comprehension & Input into Clinical Development
Synopsis
- Exploring appropriate methods of patient outreach
- Building relationships with Patient Advocacy groups
- Discovering the best methodology of ensuring patient education and expectations
- Benchmarking and recognizing the importance of the patient’s voice in clinical development
12:35 pm Looking Into Patient Perspectives on Ocular Gene Therapy
Synopsis
- Presenting on the VENTURE retinal disease natural history study
- How to ensure potential recipients of gene therapy are phenotyped and genotyped early, in preparation for upcoming trials, and what are the barriers of knowledge uptake of emerging treatments?
- Value of prospective longitudinal studies of the inherital retinal disease that treatments are being developed for
1:05 pm Lunch Break & Networking
Exploring Novel Developments in Vector Engineering & Selection to Benchmark New Therapeutic Platforms
2:05 pm Sharing Approaches for Splice Regulation & Modular Protein Function in Retinal Gene Therapy
Synopsis
- Gene therapy of the alternatively spliced gene RPGR in X-linked retinitis pigmentosa may be influenced by stringently regulator splicing of its alternative isoforms
- Modular protein function assists in designing efficacious gene therapy for large genes that are not packageable into AAV vectors
2:35 pm Addressing the Unmet Need in Retinal Gene Therapy: Focus on IVT Delivery & Cargo capacity of AAV Vectors
Synopsis
- Overview of the benefits and limitations of AAV vectors and unmet needs in retinal gene therapy
- Introduction and update on next generation vgAAV-AAV capsids
- Introduction to REVeRT dual AAV technology
3:05 pm Exploring the Laterally Spreading AAV.SPR Capsid for Treatment of Inherited Retinal Diseases
Synopsis
- Outlining the efficacy features of this novel capsid
- Detailing the safety features of AAV.SPR capsid
Approaching Commercial Challenges of Ophthalmic Gene Therapies to Help your Product Reach Market
2:05 pm Attracting Financial Investment for Biotech Startups: the Case of SpliceBio
Synopsis
- Walking through a case study of an exciting and promising new genetic platform in ophthalmology
- Sharing the processes involved in attaining investment for a novel idea in a competitive field
- Showcasing innovating science to overcome the payload capacity of the AAV vectors to demonstrate a competitive edge in ophthalmology
2:35 pm Challenges Regarding Reimbursement of Ophthalmic Gene Therapies
Synopsis
- Outlining current issues with the reimbursement of onetreatment therapies
- What the are the barriers to overcome with treatment reimbursement?
- Benchmarking a successful reimbursement platform
3:05 pm Round table: Exploring the Methods Of Overcoming Commercial Challenges
Synopsis
- Barriers in commercialization
- Luxturna case study – how is it performing?
- Reviewing reimbursement in practice
3:35 pm Afternoon Refreshments
4:05 pm Exploring the Use of Gene Therapy to Treat Dry AMD
Synopsis
- Comparing treatment for Wet AMD vs. Dry AMD
- Outlining the role of complement system in Dry AMD
- Displaying gene delivery of complement factor H
4:35 pm Session Reserved for Forge Biologics
4:45 pm Panel Discussion: Ophthalmic Gene Therapy Patient Perspective
Synopsis
Virtual Panel
- Do patients feel they know what treatments are available to them?
- Is there trial data available to patients so they can decide if they want to participate?
- How can an open line of communication be developed between patients and the industry?
5:30 pm End of Conference Day One: Chair’s Closing Remarks
5:45 pm Scientific Poster Session
Synopsis
After the formal presentations have finished for the afternoon, the learning and networking carries on. The Poster Session allows you to connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships. During this session scientific posters will be presented on the latest advancements in the gene therapy for opthalmic disorders field.