AAV Gene Therapy
What is AAV Gene Therapy?
At Beacon, our focus revolves around utilizing Adeno-associated virus (AAV) to deliver gene constructs that encode therapeutic proteins in order to address retinal disorders.
- Gene therapy is the process of modifying a patient’s cells by adding a functional copy of the genetic factors contributing to the retinal disease. The new functional copy allows a patient’s own body to produce proteins to treat or prevent the progression of the disease. A single treatment may provide long lasting benefits to patients.
- AAVs are safe small viruses that naturally infect humans. With a very mild immune response detected, there are currently no known diseases caused by natural AAV infection.
- In the context of gene therapy, the original AAV genome is substituted with an enhanced human therapeutic gene(s), and the AAV is subsequently engineered to serve as precise vehicles for delivering therapeutic genes to the retinal cells. This modification potentially augments its safety profile and therapeutic efficacy in patients
The parts of our Gene Therapy
Cutting edge vector design
We are at the forefront of advancing next generation AAV gene therapy programs focused on transforming the landscape of retinal disorders. Our commitment to groundbreaking progress is demonstrated through the incorporation of Beacon’s novel engineered capsids and genome sequence optimization that are engineered for each disease, enhancing the precision, effectiveness, and safety of our therapeutic interventions. With our innovative approach, we aspire to transform the management of retinal conditions, providing hope for patients struggling with diseases that result in blindness.
Advanced in-house expertise in vector manufacturing
Leveraging advanced in-house expertise in vector manufacturing, our team has successfully scaled up production from research to clinical batch production. We have designed and produced investigational clinical-grade vectors with adeno-associated viral (AAV) capabilities, which were employed in clinical trials.