Our ApproachThe plato® Platform

ONE PLATFORM designed for state-of-the-art gene therapy

We have spent years optimizing the elements of plato®, our proprietary platform designed to bring gene therapy to patients worldwide. Plato has been developed with the goal of optimizing the safety, potency and durability of AVROBIO's investigational lentiviral gene therapies.

What is plato®?

The plato® platform includes:

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A lentiviral vector and proprietary tags designed to optimize vector copy number, transduction efficiency and resulting enzyme/protein activity in target cells.

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Advanced cryopreservation to extend shelf life and enable flexible scheduling for patients and healthcare providers.

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A personalized conditioning regimen with precision dosing of busulfan, an established conditioning agent that we believe is key to the "head-to-toe" benefits we hope to demonstrate in our therapies.

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An automated, closed manufacturing process intended to improve consistency and predictability of the drug product.

The system is self-contained and compact, which we believe will facilitate expansion of global manufacturing sites.

The technologies and processes that form plato are intended to be replicable across programs to accelerate overall pipeline development.

Why lentiviral vectors?

Lentiviral gene therapy has been used in academia and industry since the early 2000s to treat hundreds of patients in multiple clinical trials – including four clinical trials of AVROBIO’s investigational gene therapies in lysosomal disorders. We use lentiviral vectors to integrate the therapeutic gene into the patient’s own stem cells for several reasons:

  • They have sizable cargo space, which allows us to insert even large therapeutic genes into the patient’s stem cells.
  • They are capable of permanently integrating a therapeutic gene directly into the patient’s chromosomes.
    • This means that when the original batch of genetically modified stem cells divide and proliferate into trillions of daughter cells, each is expected to carry the therapeutic gene and be capable of expressing the active protein the patient needs.

The process of integrating the therapeutic gene into the cells takes place in a manufacturing facility, rather than in the patient’s body. This approach is called ex vivo gene therapy.

Our lentiviral approach is designed to enable treated stem cells to differentiate into many different cell types – including T cells, B cells and monocytes, each of which is expected to carry the therapeutic gene. Monocytes when they enter tissues become macrophages and in the central nervous system (CNS) they become microglia. Monocytes, macrophages and microglia carrying the therapeutic gene are understood to have the ability to traverse the blood-brain barrier and enter the CNS. We believe that once dispersed in the brain and CNS, these therapeutic cells may be able to slow, halt or even prevent disease progression in the CNS.

It’s important to note that our therapies are still investigational; they have not been approved by any regulatory body.

Learn more about AVROBIO’s personalized conditioning regimen.

Proprietary tagging technology facilitates targeted delivery

In some contexts, such as our programs in Pompe disease and Hunter syndrome, we use proprietary “tagging” technology to enhance the uptake of the therapeutic protein into target tissues, including CNS.

AVROBIO

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Patients and Families

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Our Approach

Learn more about our personalized approach to gene therapy.