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.