Daniel Galbraith, PhD, on Using Targeted Locus Amplification to Analyze Genetically Modified Cells

“One of the big risks [of gene therapy] is you don't want to do any damage to the patient. Obviously, these patients are ill and you want to help them as much as you can, so making sure that your treatment is not going to cause any damage is the important thing. This technology, TLA, will give us a good indication that it's not actually going to cause any cancer effects from the treatment that you're going to give.”

Although genetic modification of cells through the use of viral vectors, such as lentiviral vectors, can have a number of important therapeutic applications, such processes carry risks, as well. For example, if the vector integrates into an oncogene in a cell’s genome, it could potentially lead to malignancy. As such, tools for analyzing the genome after cells have been modified can play an important role in assessing the safety of genetically modified cell-based therapies.

One such tool is Solvias’ Targeted Locus Amplification (TLA) platform. At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2025, in New Orleans, Louisiana, Daniel Galbraith, PhD, the chief scientific officer of Solvias, presented on the use of the TLA platform for analysis of lentivirus integration and mechanisms. He noted that TLA can help determine how many copies of the vector are inserted, whether the integration disrupts important genes, and if those changes could pose risks such as activating oncogenes. He also pointed out that the technology can track how changes evolve over time as the treated cell populations grow outside the patient.

The broader goal, Galbraith emphasized, is safety: ensuring gene therapies do not cause unintended damage like cancer. He also discussed potential future avenues for the further development of the technology. Currently it is able to be used for pretreatment monitoring of patient-derived cells outside the body, but extending that monitoring to how cells behave after reinfusion could be carried out by harvesting cells for another analysis sometime after the initial treatment is given.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.