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 Delving Into Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy

Synopsis

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

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

  • Rachel Eclov Gene Therapy Development Project Leader, Kriya Therapeutics

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

  • William Beltran Director, Division of Experimental Retinal Therapies, University of Pennsylvania

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

  • Lauren Ayton Associate Professor, & Head - Vision Optimisation, University of Melbourne

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

  • Hemant Khanna Vice President of Preclinical Ocular Research, IVERIC Bio

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.