Gene Therapy Development Service

Gene therapy involves the delivery of genetic material into target cells to correct or compensate for defective genes responsible for disease. From vector design to preclinical studies, Protheragen offers comprehensive gene therapy development services for ophthalmic diseases.

Overview of Ocular Gene Therapy

With very little size, having easy access and being separated from severe immune reactions, the eye is a prime candidate for gene therapy. Gene therapies for eye diseases can reverse vision loss in inherited retinal disorders like Leber congenital amaurosis, and in some cases, delay retina deterioration in retinitis pigmentosa. They further enable effective therapeutics of age-related macular degeneration (AMD) by constantly producing required therapeutic proteins, offering long-lasting remission, and eliminating the need for recurrent injections.

Fig.1 From lentivirus to 3rd generation lentiviral vector system. (Arsenijevic Y., et al., 2022)

Gene Therapy Development for Ophthalmic Diseases

Recently, the gene therapy field that deals with eye diseases has improved dramatically. A lot of attention has been put to the genetic basis of different eye disorders which has resulted in refined therapies. The first-ever gene therapy approved by the FDA, Luxturna, turned out to be a major success because it was able to restore vision by treating an inherited retinal disease. Current clinical studies are still trying to test different therapeutic methodologies for other diseases such as retinitis pigmentosa, Stargardt disease, and AMD, and the results seem to be positive. These results prove how deep gene therapy can fundamentally change the therapeutic methods of diseases pertaining to the eyes.

Table 1. Summary of gene therapies for ophthalmic diseases. (Ghoraba H. H., et al., 2022)

Disclaimer: Protheragen focuses on providing preclinical research service. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.

Our Services

Protheragen knows very well that each gene therapy project has its characteristics and must be approached differently. So, we provide services that suit the specifications of our clients. From designing custom vectors to preclinical studies, our specialists work hand in hand with clients to tailor solutions that meet their needs.

Vector Design and Optimization

Protheragen specializes in the design and optimization of viral vectors, such as adeno-associated virus (AAV) vectors, which are known for their ability to efficiently deliver genetic material to target cells with minimal immune response. Our team of scientists works closely with clients to select the most appropriate vector type and optimize its design for maximum efficacy and safety.

• Viral Vector: lentiviral vectors, AAV vectors
• Non-Viral Vector: liposomal vectors, nanoparticle vectors
• Hybrid Vector: dendrimer-coated virus particles, virus particles encapsulated liposomes
• Pseudotyped Viral Vector: VSV-G pseudotyped viral vectors, LCMV-GP pseudotyped viral vectors

In Vitro and In Vivo Testing

Our state-of-the-art laboratories are equipped with advanced technologies for conducting preclinical studies in animal models. We perform extensive in vitro and in vivo testing to evaluate the safety and efficacy of gene therapies. These studies provide critical data on transduction efficiency, immune response, and long-term effects, ensuring that only the most promising candidates proceed to clinical trials.

Types of Gene Therapy Development

Protheragen is committed to advancing the field of gene therapy for ophthalmic diseases, offering a comprehensive suite of services to support the development of these innovative therapeutics. If you are interested in our services, please feel free to contact us.

References

• Arsenijevic, Yvan, et al. "Lentiviral vectors for ocular gene therapy." Pharmaceutics 14.8 (2022): 1605.
• Ghoraba, Hashem H., et al. "Ocular gene therapy: a literature review with special focus on immune and inflammatory responses." Clinical Ophthalmology (2022): 1753-1771.