8:50 am Chairs Opening Remarks
Utilizing Gene Therapy to Restore Protein Function in Common Ophthalmic Disorders to Generate Therapies with a Large Patient Population
9:00 am Exploring Ambient Light Activatable Optogenetics for Vision Restoration in Retinal Degenerative Diseases
Synopsis
- Mutation-agnostic therapy with potential to be disease-agnostic platform
therapy preclinical data - Clinical data on Retinitis Pigmentosa
- Novel end points for low vision patients deployed in a multi-site randomized,
sham-controlled optogenetic study
9:30 am Showcasing ADVM-022 as a Treatment for Neovascular AMD
Synopsis
- Wet AMD: the rationale for using gene therapy for treatment
- Utilizing aAV.7m8 vector for intravitreal administration and preclinical and clinical
support for dosing - Clinical experience with ADVM-022 in Wet AMD
10:00 am Establishing a Sustainable Gene Therapy Pipeline for Inherited Retinal Diseases
Synopsis
- Challenges and solutions for creating a sustainable gene therapy pipeline for rare
inherited retinal diseases - Opus Genetics’ gene therapy portfolio for LCA and other classes of IRDs
- Outlining phase I clinical trial data for LCA5-IRD
10:30 am Morning Break & Networking
PRECLINICAL STREAM
Controlling Inflammation & Toxicity to Provide a Safer Treatments for the Eye
11:30 am Discussing the Involvement of Immunosuppressants in Therapies to Understand the Benefits & Drawbacks
Synopsis
- Anti-inflammatory and immunosuppressives as prophylaxisfor gene therapy associated inflammation
- Management of treatment emergent gene therapy associated inflammation
- Translational aspects of inflammation to guide immunosuppressive therapy decisions
12:00 pm Ocular Inflammation During Preclinical Studies, Towards Harmonization of Anti-Inflammatory Regimens
Synopsis
- Common ocular inflammation on IND-enabling preclinical studies for development of AAV-based gene therapy
- Outlining the contributing factors of ocular inflammation
- Discussing the lack of protocol for steroid and antinflammatory regiments in preclinical studies and how this prevents meaningful comparison
12:30 pm Panel Discussion: Discussing the Immunogenicity of the Eye
Synopsis
- Discussing the implications of treatments delivery to the
immuno-privileged eye - What are the most common causes of inflammation?
- How do we overcome ocular inflammation – is it possible?
12:50 pm Session reserved for Biomere
CLINICAL STREAM
Benchmarking Ophthalmic Clinical Trial Design to Get your Therapy to Market
11:30 am Identifying the Right Endpoints for your Therapy to Optimize your Clinical Trials
Synopsis
- Proposing a framework to select disease-specific and stage-specific endpoints including comparison measures
- Reviewing strengths and limitations of visual function tests currently used as outcome measures
- Addressing practical aspects of measuring outcomes to minimize patient burden
12:00 pm Providing an Update on Antisense Oligonucleotides for Inherited Retinal Disease
Synopsis
- Antisense oligonucleotides have so much promise in Ph1/2 clinical trials in LCA10 and Usher’s Syndrome
- Recent interim analyses in key trails have shed much light on mechanism of action of antisense oligonucleotides
- Overview of the data available from clinical trials
- Clinical trial endpoints for inherited retinal diseases
12:30 pm Clinical Trial Development for Interventional Study in Outer Retinal Degenerative Diseases
Synopsis
- Overview of the unmet need in clinical trial outcomes
- Dark adaptation as a potential outcome for intermediate AMD
- Structural and Functional parameters in X-linked retinoschisis
1:00 pm Lunch Break & Networking
Discussing the Successes & Learning Opportunities of Recent Therapies to Further Understanding of the Correct Protocols of Ophthalmic Gene Therapy Translation
2:00 pm Panel Discussion – Revolutionising Communication to Share Solutions and Techniques in Translational Gene Therapy in Ophthalmology
Synopsis
- Analysing the qualities of a successful treatment in market
- Steps from preclinical to clinical – how to increase chances of success
- Working with regulatory agencies to better formulate realistic endpoints
2:30 pm 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
3:00 pm Afternoon Break
4:00 pm Understand the Novel Protein Splicing Platform that Overcomes the Payload Capacity of AAV-Vectors
Synopsis
- Outlining the methodology of protein splicing
- Discovering split inteins: auto processing domains
- Exploring protein splicing as a novel gene therapy modality to the eye