Service Overview

Although gene therapy for eye diseases has achieved satisfactory results, in some cases, gene therapy products themselves may also produce certain toxicity in vivo, which is caused by the expressed transgenes and vectors. Meanwhile, off-target effects of gene therapy products may be another relevant toxicity factor. As a cutting-edge biotechnology research and service provider in the field of ophthalmology, Ace Therapeutics provides the following safety and toxicology analysis services for the development of ocular gene therapy products for global customers, including but not limited to.

• Carrier Toxicity Studies

Delivery systems for foreign genes targeting the eye are usually based on non-viral and viral vectors (eg, lentiviral vectors, adeno-associated viral vectors). Although some virus carriers have been attenuated through mutations or deletions, the modified virus itself may infect the normal system of the human body. Ace Therapeutics is committed to establishing the most refined service platform for vector toxicity research, including virus replication ability, immunogenicity, persistence, incubation period, tropism and route of administration, etc.

• Off-target Toxicity Studies

Off-target toxicity mainly comes from the ability of the viral vector to infect normal cells such as hematopoietic cells, neurons and reproductive system after mutation. In some cases, expressed transgenes can also be toxic in vivo. Therefore, specific assays need to be developed to analyze the off-target toxicity of transgenes and their vectors. With Ace Therapeutics' professional services and various technology platforms, we provide the best off-target toxicity analysis system for gene therapy products by utilizing appropriate animal models and designing comprehensive research practices.

• Preclinical Observations and Pathology Studies

At Ace Therapeutics' laboratory, our scientists are able to conduct many metrics to evaluate the safety and toxicity of gene therapy products for ocular diseases. Preclinical observations include, but are not limited to, cytopathic effect (CPE), animal signs, body weight, morbidity, and mortality. In addition, pathology studies refer to hematology, coagulation and urinalysis and ocular histopathological examination.

Ace Therapeutics is an excellent service provider in the field of ophthalmic gene therapy. Thanks to our advanced technology and strong R&D team, we provide world-class safety and toxicology analysis services to global customers. In addition, Ace Therapeutics scientists have developed an advanced toxicology research system to provide sufficient data for your clinical trial design and optimization. Quality control of safety and toxicology analyzes is applied to ensure reliability by applying a set of stringent standards. If you are interested in our services or need more detailed information, please feel free to contact us. Our experienced scientists are ready to help you!

• AAV Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Lentiviral Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Non-Viral Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Recombinant Adeno-Associated Virus Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Delivery System Development Services for Ocular Nucleic Acid-Based Therapy
• CRISPR/Cas9 Gene Editing Services for Ocular Nucleic Acid-Based Therapy
• Potency Testing Services for Ocular Nucleic Acid-Based Therapy

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