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

  • Arun Upadhyay Senior Vice Preisent of Research & Development, Ocugen

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

  • 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 Efficient Gene Therapy Manufacturing for Ocular Diseases

Synopsis

Gene therapy treatments targeting sensitive biological spaces, such as the eye, require highly purified and well characterized products. AAV therapeutics for ophthalmic dosing require low endotoxin, residuals, and empty particle contaminant levels. A winning commercialization strategy will include flexibility for small batch sizes during development, and a scalable process that maintains high productivity and purity of the vector. Forge has developed a platform process and proprietary technology to enable client’s ocular gene therapy programs to expedite the path from the research phase to cGMP. During this session, Forge’s Associate Director of Technical Sales, Brianna Barrett, Ph.D., will review Forge’s approach to efficient gene therapy manufacturing for ocular diseases, AAV manufacturing requirements, and considerations for partnering with a CDMO.

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.