Cyagen representatives recently attended The 3rd Annual Gene Therapy for Ophthalmic Disorders meeting with 150+ stakeholders and industry leaders in ophthalmic gene therapy. The event brought together ophthalmology experts with world-class learning tracks to address the challenges faced in the preclinical stages and in clinical development, aiming to get these drugs to the ophthalmic gene therapy patients who need them as fast as possible. 

Gene Therapy for Ophthalmic Disorders Meeting

We are excited to share some of the highlights from the meeting from the perspective of our attendees. Our Chief Scientific Officer, Dr. Marvin Ouyang, provided his feedback from the event:

With so many executives and director-level leaders from biotech, big pharma, foundations from all over the world participating in this conference, I can see that there are exceptional developments coming for the application of gene therapy for ophthalmic disorders. Gene therapy has shown great promise in treating inherited ophthalmic diseases, as most of ophthalmic disorders are caused by monogenic defects, which are great candidates for gene therapy. 

Through talking with the attendees, I get the feeling that more predictive mouse models are becoming increasingly important for preclinical studies, as only data from these animal models can be translated with high accuracy. In terms of gene transfer, the AAV vector is still the preferred method. There were quite a few talks on the AAV vector improvement and optimization. I firmly believe that our capabilities with animal model generation, cell and gene therapy CRO services, and AI-powered AAV design will greatly accelerate the R&D process of ocular gene therapies.

              -  Yingbing (Marvin) Ouyang, Ph.D.

                 Executive Vice President and Chief Scientific Officer, Cyagen

We additionally have put together a video that captures the exciting progress being made in gene therapy for ophthalmic disorders:


Partnering with AmCare Genomics Lab

We attended the conference with AmCare Genomics Lab Co., Ltd. ("AmCareLab"), whom we are partnering with to accelerate development of therapeutic for ophthalmic disorders. In brief, AmCareLab will use their clinical resources to identify the most prevalent genetic mutations in ophthalmic diseases and Cyagen will generate the corresponding mouse models. 

AmCareLab is committed to using genomic testing products as a tool with continuous optimization which comprehensively and rapidly assess genetic and variant factors that cause birth defects. It creates a one-stop solution close to clinical use, and provides professional and clinically reasonable molecular detection instruments, reagents and software for medical institutions at all levels. It provides services such as clinical staff training, hospital-side consultation, and personalized genetic counseling. At the same time, it promotes the closed-loop industrial upgrading of "diagnosis, screening, prevention and treatment" of genomic diseases. In the past seven years, AmCareLab has established gene mutation databases for tens of thousands of Chinese population, and is planning for the launch of program EyeCare 100K, in which 100K patients with ophthalmic genetic diseases will be tested for the genomic alteration in five years . 

Based on this program, AmCareLab could identify the most prevalent gene mutations associated with ophthalmic disorders, for which Cyagen will generate the humanized mouse models to help accelerate clinical translation of potential therapeutics. With our united capabilities, we aim to solve the difficulties in the diagnosis of genetic rare diseases by deepening the clinical application of genomic detection technology.


1) Establish a genomic database for hereditary ocular disorders and evaluate the epidemiology and gene mutation spectrum based on these cases
2) Assist the clinicians to launch diagnosis and treatment consensus statement/guideline for hereditary ocular disorders from accumulated disease cases
3) Provide an comprehensive solutions for the integrated system of diagnosis and treatment for hereditary ocular disorders


1) Construct animal models for hereditary ocular disorders according to the mutation spectrum of patients
2) Accelerate the pre-clinical and clinical trials of ophthalmic drugs by targeting selection of ophthalmic phar-maceutical enterprises based on clinically relevant animal model

Pharmaceutical enterprises:

1) Assess the market potential for drug target
2) Develop innovate drugs based on these targets and conduct pre-clinical research
3) Enroll the suitable patients diagnosed in the cohort and conduct clinical trials in the cooperation hospital

Progress Made and Remaining Challenges for Gene Therapies

Although there are many gene therapy drugs currently on the market, basic research and clinical translation in ophthalmology faces many difficulties, such as complicated operation, long production period of experimental models, and expensive equipment. Additionally, researchers have faced limitations when it comes to obtaining animal models that are clinically relevant to the disease pathology presented in humans — thereby facilitating clinical translation. 

Humanized Mouse Models for Therapeutics Research

Humanized mouse models can provide high homology with human disease pathogenesis and improve the potential for successful clinical translation.

Diseases Genes Tech

Leber Congenital Amaurosis type 10(LCA10)



Retinitis pigmentosa (RP)



Retinitis pigmentosa (RP)


Humanization (MU: P23H)

Stargardt Disease



Usher Syndrome Type 2



Age-related Macular Degeneration




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 an ophthalmic gene therapy platform to overcome the above obstacles. 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.

About Cyagen

With 16 years of genetically modified mouse model construction experience, Cyagen has established an ophthalmic gene therapy platform to provide you with an array of preclinical research solutions. We have equipped the platform with state-of-the-art ophthalmic instruments for small animals and an experienced professional team. 

Our comprehensive contract research organization (CRO) solutions overcome the long-term difficulties faced in gene therapy research for eye diseases, CAR-T cell therapy, and more. We provide one-stop solutions for gene therapy development to meet the needs for all levels of research. Our discovery and preclinical research services include: CRISPR Cas9 Screening & Target Validation, Custom Mouse Model Generation and Cohort Breeding, Phenotype Analysis and Evaluation, and In Vitro & In Vivo Screening and Validation of Gene Therapy Drugs. 

About AmCareLab

AmCareLab was established in Guangzhou Bio Island in August 2015. It focuses on providing comprehensive solutions for the diagnosis and counseling of clinical genetic disease. It is a comprehensive biomedical enterprise with clinical inspection service, development and production of in vitro diagnostic kit . The company is committed to promoting the translational application of genomic testing technology in clinical practice.