Accelerating Gene Therapy for Ophthalmic Diseases

Life Sciences, Drug Discovery & Development, Preclinical, Cell and Gene Therapy,
  • Friday, June 09, 2023

There are a large number of patients with hereditary ophthalmic diseases worldwide, with gene therapy drugs emerging as a powerful therapeutic tool in ophthalmology due to the limited efficacy of traditional treatments. As of 2021, ophthalmology ranks second in the gene therapy pipeline according to the number of treatments in development. There are many gene therapy drugs currently on the market, but basic research and clinical translation in ophthalmology face many difficulties, such as complicated operations, a long production period for experimental models and expensive equipment.

With the continuous progress of gene-editing technology and the development of new delivery methods, the future of gene therapy for ocular diseases is very promising. Despite the complicated operations, a long production period for experimental models and expensive equipment, companies and institutions are actively investing in research and development, and increasing numbers of clinical trials are being conducted to validate the safety and efficacy of these new treatments. The goal is to bring more effective and affordable treatments to patients with hereditary ophthalmic diseases and to improve their quality of life.


In recent years, gene therapy has emerged as a powerful tool in the field of ophthalmic disease research. By correcting genetic defects, it has become a lifesaving solution for some retinal diseases previously considered to be incurable. Gene editing is a critical component of gene therapy, involving targeted editing of the genome to recreate critical phenotypes in animals. However, delivery vectors are responsible for delivering the therapy to the eye to achieve therapeutic effects, and they continue to face issues of safety, specificity and commercial viability. This is a critical factor in current research and development.

To overcome these technical challenges, it’s best to hear from the experts on how they plan to break through these bottlenecks.

Read Less...

Register for this webinar to learn about the rapid advancements in preclinical ophthalmic gene therapy research, such as how AI-guided AAV design tools can help accelerate development of effective therapeutics to patients with ophthalmic diseases.


Joseph Wekselblatt, Cyagen Biosciences

Joseph Wekselblatt, Senior Scientific Business Development Manager at Cyagen Biosciences

Joseph Wekselblatt has been in the scientific field for well over a decade, having studied the visual capabilities in species including mice and rats, barn owls, tree shrews, humans and non-human primates. Joseph Wekselblatt attended Brown University from 2004 to 2008, where he earned a bachelor’s degree in cognitive science. Joseph then attended the University of Oregon from 2011 to 2017, where he earned a Doctor of Philosophy in Neurobiology. Joseph is currently a Postdoctoral Researcher at Caltech, where he has been since 2017. In 2023, he transitioned to Cyagen as a Senior Scientific Business Development Manager.

Message Presenter

Who Should Attend?

Clinical research, drug discovery, R&D, artificial intelligence/machine learning professionals and pharmaceutical sponsors in the rare disease space, including members with the following titles:

  • VP and Head of Data Science
  • VP/Director of Biology
  • VP/Director of Research
  • Scientists
  • Biologists
  • Bioinformaticians
  • Medicinal Chemists
  • Pharmacologists
  • Data Scientists
  • Therapeutic Lead/Head
  • CIO
  • CTO

What You Will Learn

Attendees will learn about improving the quality of life and bringing more effective and affordable treatments to patients through:

  • One-stop R&D Solutions for ophthalmology gene therapy
  • Cyagen’s Eye Disease models
  • Ophthalmology CRO services
  • Artificial intelligence AAVs for gene therapy R&D

Xtalks Partners

Cyagen Biosciences

Cyagen Biosciences is a 900-employee company headquartered in Santa Clara, California, with additional locations in Germany, Japan, and China. Cyagen offers a “one-stop shop” tailored to biomedical scientists’ gene research needs. Cyagen is the world’s largest provider of custom-engineered mouse and rat models. Our custom murine model generation services range from DNA vector construction to embryonic stem cell manipulation, microinjection, breeding, and more. All projects are fully customizable and flexible. Cyagen provides comprehensive Contract Research Organization (CRO) services for cell and gene therapy (CGT) research, including viral vector development, CAR design and construction, disease models, phenotype analysis, and pharmacodynamic evaluations.

The Rare Disease Data Center (RDDC)

The Rare Disease Data Center (RDDC) is committed to providing unlimited access to comprehensive rare disease research data visualization & AI-powered pathogenic analysis tools. It integrates all reported rare disease data and information, including: epidemiological data, disease-related gene profile, variants, phenotypes, drug developmental outlines, and disease-associated mouse models. The RDDC provides a rapid analysis and visual understanding for a rare disease of interest, offering the maximum convenience for people who are dedicated to the treatments of rare diseases, such as clinical doctors, scientific researchers from hospitals and universities, and pharmaceutical companies.

You Must Login To Register for this Free Webinar

Already have an account? LOGIN HERE. If you don’t have an account you need to create a free account.

Create Account