Our science

AAV Gene Therapy

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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

Bold science

Why Gene Therapy?

Gene therapy has growing potential in the field of retinal diseases owing to three main benefits.

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Easily accessible:

Eye is easily accessible for treatment via injections and surgical interventions. Targeted delivery of AAV vectors potentially enhances the therapy’s safety and efficacy profile. At Beacon Therapeutics we are using subretinal and intravitreal injections related to disease targets.

Sustained long-lasting Effects:

Once delivered, therapeutic genes become internalized into the patient’s own retinal cells, leading to sustained durable treatment effect.

A steady, continuous level of protein expression from one-and-done gene therapy treatment, may offer a remarkable therapeutic improvement. This marks a significant step forward in therapeutic advancements, offering the potential for more effective treatments while decreasing the need for more frequent interventions. This enhances patient convenience and minimizes healthcare burdens.

Controlled disease progression:

Gene therapy has demonstrated the ability to restore the function of mutated genes or genetic factors that are responsible for retinal disorders. Through addressing the genetic root cause, we aim to manage and control the progression of these debilitating disorders.

Key elements

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.

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Join us

Make Remarkable Happen

Beacon Therapeutics is always seeking new talent to join our teams and help deliver our mission to save and restore vision to patients with blinding retinal diseases.

If you are interested in joining our team and have the passion, skills and motivation to be a part of this transformational science, then we are keen to hear from you. Please refer to our careers information.

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