AVROBIO uses a well-established conditioning medicine called busulfan, which enables engraftment and ultimately helps enable durable distribution of therapeutic protein throughout the body. Unlike other conditioning agents, busulfan crosses the blood-brain barrier and is critical to addressing diseases involving the CNS.
Similar to how keyhole surgery revolutionized surgery, we believe Target Concentration Intervention (TCI) has evolved the use of busulfan for gene therapy.
AVROBIO’s approach with TCI is designed to enable personalized conditioning deploying state-of-the-art precision dosing. Targeting is intended to reduce side effects while maximizing genetically modified stem cells engraftment in the bone marrow.
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. Some of these side effects can be managed proactively with supportive care. 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.
To learn more about Bu90-TCI, check out these resources:
- Redefining conditioning for a new era of gene therapy
Endpoints News webinar, Dec. 15. 2020. Harry Malech, M.D., Chief of the Genetic Immunotherapy Section at the NIH’s NIAID and AVROBIO Chief Scientific Officer Chris Mason, M.D., Ph.D. Sponsored by AVROBIO.
- TCI is dead. Long live TCI!
By Nick Holford, Guangda Ma, David Metz, British Journal of Clinical Pharmacology. Published online June 16, 2020
- Leading the way in bringing gene therapy to the CNS with conditioning: A renaissance for busulfan?
By Chris Mason, Cell & Gene Therapy Insights 2020; 6(9), 1299–1304. Published Oct 22, 2020
- Removing barriers to achieve widespread clinical adoption of lentiviral gene therapy
By Chris Mason, Cell & Gene 2020. Published Aug. 26, 2020