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
  • A Phase 1 trial 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

  • Nida Sen Retina Global Clinical Development, Janssen

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 Session reserved for Biomere

12:10 pm Ocular Inflammation During Preclinical Studies, Towards Harmonization of Anti-Inflammatory Regimens

  • Josh Bartoe Vice President , Ophthalmology Services, Northern Biomedical Research

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 regimens in preclinical studies and how this prevents meaningful comparison

12:40 pm Panel Discussion: Discussing the Immunogenicity of the Eye

  • Francis Tukov Director, Preclinical Safety Assessment, Novartis

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?

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

  • Erin O’Neil Attending Physician, Children’s Hospital of Philadelphia

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

  • Catherine Cukras Director of the Medical Retina Fellowship Program, National Eye Institute (NEI)

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 Delving Into Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy

Synopsis

Virtual Presentation

  • LHON, a rare disease with unmet medical need
  • Final steps of Lumevoq clinical development
  • Importance of early access programs

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

4:30 pm End of Conference Day Two: Chair’s Closing Remarks