Accelerating Gene Therapy for Ophthalmic Diseases

Overview

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. At Cyagen, we are dedicated to advancing this field with our comprehensive support for discovery and translational research, to help bring more effective and affordable treatments to patients with eye diseases.

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. As a leader in the gene therapy industry, we are dedicated to supporting and advancing this field and working towards the ultimate goal of providing hope and healing to those in need.

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.

At Cyagen, our team of experts is dedicated to supporting preclinical ophthalmic gene therapy research. Register for this webinar now to learn more about our comprehensive services for preclinical ophthalmic gene therapy research.

Key Topics

Learn how you can accelerate research and development of effective gene therapies for Ophthalmology Diseases：

One-stop R&D Solutions for Ophthalmology Gene Therapy
Cyagen’s Eye Disease Models
Ophthalmology CRO Services
AI AAVs for Gene Therapy R&D

Who Should Attend?

This session is packed with relevant insights for clinical research professionals and pharmaceutical sponsors in the rare disease space. The webinar will be of interest to members of the following fields:

Clinical Research Hospitals
Ophthalmology Diseases
Biotechnology
Pharmaceutical & Biopharma
Preclinical and Academic Research
Rare Disease Research Organizations & Non-Profits

Relevant areas:

Clinical operations
Innovation
Medical affairs
Scientific Affairs
Project Management
Research and Development

Titles may include:

Drug Discovery Professionals
VP and Head of Data Science
VP/Director of Biology
VP/Director of Research

Scientists
Biologists
Bioinformaticians
Medicinal Chemists

Pharmacologists
Data Scientists
Drug Discovery Professionals
Research & Development Professionals

Artificial Intelligence/Machine Learning Professionals
Therapeutic Lead/Head
CIO
CTO

Who Should Attend?

Presenter

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.

“The central goal of my work has been to understand the role of functionally defined brain areas along the tree shrew visual hierarchy in processing visual object representation. Importantly, tree shrews have a substantially more complex visual system than rodents and a clear experimental advantage over primate models.

I have developed expertise in the proto-primate model using recently developed techniques for whole-brain functional imaging and modulation. Existing animal models present methodological challenges: in primates, which allow the probing of complex cognition, it is difficult to record from large ensembles of neurons across multiple brain areas or manipulate genetically defined cell populations; in rodents, rudimentary cortical organization and behavioral repertoire limits the modeling of visual processing.

Over the past 5+ years I have established the brain-wide organization of the visual system using a functional ultrasound imaging – non-invasive imaging technique which allows measurements of evoked activity across the entire brain, much like fMRI, but with greatly improved spatial and temporal resolution. Next, I have targeted high density silicon probe recordings (neuropixels) to various nodes of this network (e.g. V1, V2, and IT) and measured the local activity in response to a host of visual stimuli used in classical visual physiology studies. I have also developed techniques to manipulate populations of genetically defined cells in a non-invasive and spatially targeted way in each of these regions using acoustically targeted delivery of promoter-specific virus carrying inhibitory DREADDs by transiently disrupting the blood brain barrier. This allows specific and reversible control, by decreasing the excitability of genetically defined excitatory cell populations at different nodes in this hierarchy with the systemic administration of CNO or other DREADD receptor agonists. Finally, I have developed custom behavior systems to test the behavioral effects of these targeted manipulations in the tree shrew on several different behaviors. This work provides valuable new information to researchers and clinicians about the function and computations of sensory transformations along cortical networks. “ -Joseph Wekselblatt

About Cyagen

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.

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One-Stop Preclinical Ophthalmology Research Solutions

As a comprehensive contract research organization (CRO) solution provider, Cyagen recognizes ophthalmic diseases as a breakthrough point for gene therapy and has established a platform to accelerate translational research of ophthalmic gene therapy. We have equipped the platform with state-of-the-art ophthalmic instruments for small animals and an experienced professional team. With 16 years of gene editing model construction experience, Cyagen can provide you with an array of standardized preclinical research solutions for ophthalmic gene therapy.

Our ophthalmic gene therapy platform is equipped with a full set of state-of-the-art ophthalmic detecting instruments which support a full range of verification services. Our ocular technologies include the Micron IV small animal retinal microscopic imaging system, full-field electroretinogram (ffERG), image-guided optical coherence tomography (OCT) system, and handheld ophthalmotonometer for mice. We can provide detection services for rodent models of eye-related diseases including diabetic retinopathy, retinoblastoma, macular degeneration, pediatric retinopathy of prematurity (ROP), choroidal neovascularization, and retinitis pigmentosa.

With 16 years of experience in the field of custom animal models, Cyagen has independently developed a series of gene editing models targeting ophthalmic diseases (e.g., retinitis pigmentosa (RP), retinal degeneration, Leber congenital amaurosis 2 (LCA2), macular degeneration, Leber congenital amaurosis 10 (LCA10), and endothelial corneal dystrophy). We can provide you with genetically engineered animal models, fully-humanized mouse models, and surgical models to accelerate your preclinical pharmacodynamics evaluations.