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 gene therapy platform. It is designed as a foundation for potentially scaling gene therapies worldwide.

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 through the use of therapeutic drug monitoring.

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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.

Why lentiviral vectors?

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 when the patient’s treated cells divide, each daughter cell is expected to carry the therapeutic gene and be capable of producing the functional protein the patient needs.

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

It’s important to note that our therapies are still investigational; they have not been approved by any regulatory body. However, they are based on more than 30 years of industry research into ex vivo gene therapy, including successful treatments of dozens of  children and adults with thalessemia. Lentiviral vectors have also been studied extensively and used in the clinic with more than 350 patients undergoing ex vivo gene therapy over the last decade.

A closer look at conditioning

Conditioning is an essential step to optimize treatment. It’s used to prepare the patient’s body to receive the gene therapy. Conditioning helps the cells carrying the therapeutic gene to engraft in the patient’s bone marrow and future generations of those cells to engraft in the brain. This engraftment is crucial for the therapy to be successful.

We currently use a medication called busulfan to enable a personalized conditioning regime with precision dosing. Busulfan is an approved, extensively studied and widely used medication that has been shown over many years of clinical use to be effective in preparing the body for engraftment.

The conditioning process is associated with known side effects and does have risks. For instance, patients are more vulnerable to infection and bleeding for several weeks after conditioning. They may experience transient side effects such as fatigue, nausea, hair loss and mouth sores. There may be long-term risks to fertility; individuals undergoing conditioning may be advised to freeze their eggs or sperm in advance of the treatment.

As part of the plato® platform, we use a process called therapeutic drug monitoring for precision calibration of the conditioning dose tailored to each person receiving gene therapy. This process potentially allows us to maximize the effectiveness of conditioning — and the likelihood of successful engraftment — while minimizing the side effects.

At AVROBIO, we are committed to a process where each individual receives a personalized therapeutic approach. This may include tailoring the therapy with the conditioning agents and dosing regimen most appropriate for the individual. We believe the use of busulfan will help optimize outcomes in our clinical trials. We envision a future where other established and emerging conditioning regimens may be used, giving clinicians and patients alternatives to pair with our gene therapies.

Learn more about our commitment to individuals living with genetic disorders.


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Patients & families

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Our approach

Learn more about our personalized approach to gene therapy.