Applied Genetic Technologies Corporation (AGTC)

[XenaLives]

Gene therapy is the process of modifying a patient’s cells by adding a functional copy of the mutated gene which is causing the patient disease.The new functional copy allows a patient’s own body to produce proteins to treat or prevent genetic conditions. A single treatment can provide long lasting benefits, potentially leading to a better quality of life.

Genetic therapies are complex, with interdependent components that must work in harmony.Fifteen years of gene therapy experience allows us at AGTC to design and construct all critical gene therapy elements and bring them together to develop successful treatments for patients.


AGTC is developing novel formulations for gene therapy. These formulations have shown promise in their ability to increase the concentration and stability of the gene therapy while reducing unwanted effects for the patient. Our proprietary formulation allows us to reach therapeutic doses at lower volumes which enhances the ability of the therapy to benefit patients.

Manufacturing & Characterization
AAV vectors have broad potential to treat a variety of genetic conditions. Our proprietary manufacturing system allows us to handle large volumes, reduce cost, and increase potency and purity of gene therapy treatments. We combine the consistent high yields from our manufacturing system with advanced characterization methods to accelerate the development of gene therapies into human clinical trials. This production and characterization system has been positively reviewed by the U.S. Food & Drug Administration (FDA) and European Medicines Agency (EMA) for use in human clinical trials and has been successfully transferred to multiple vendors and partners.

Vector Administration
AGTC currently employs two different methods for delivery of our ophthalmic products: intravitreal and subretinal. These delivery techniques have been carefully selected for each individual disease state in order to optimize patient outcomes, vector uptake and procedural logistics.

Intravitreal administration involves delivery of the AAV vector into the vitreous cavity, the gel-like substance within the eye. This procedure is performed in an outpatient setting and is routinely performed for delivery of other approved ophthalmic products.

Subretinal administration is a minimally invasive surgical procedure which involves delivery of the AAV vector underneath the retina. Although this delivery method is a more direct way to introduce the vector to the targeted cells, it requires general anesthesia and a refined surgical technique aimed at maximizing potential benefit while minimizing risks.

AGTC is also in the process of identifying novel capsids that would allow for treatment of additional diseases through intravitreal delivery, without the need for subretinal surgery. This will allow more clinicians to administer AGTC products with the goal of treating larger patient populations.

https://agtc.com/science/