Our proprietary and optimised platform is highly innovative. Past clinical trials of investigational AAV-based gene therapies have been highly encouraging. Freeline has developed a platform approach that we believe can achieve this goal.
We have two key enabling platforms critical to realising AAV gene therapy across our indications:
AAV vector platform
In our studies to date, our proprietary AAV capsid has a high liver transduction efficiency, creating sustainable, high levels of the desired protein activity with lower AAV viral titres containing the transgene.
Our proprietary capsid, AAVS3, is an essential part of our strategy, with a promising liver transduction profile. Our proprietary AAV vector technology cassette is designed to deliver a functional copy of a therapeutic gene into the human liver to offer durable secretion of the therapeutic protein(s) into the patient’s blood. Therapeutic AAV vectors consist of two main components: the capsid and the expression cassette (that contains the gene and the promoter).
The capsid is responsible for directing the gene into the correct cells and the expression cassette guides tissue-specific synthesis of the therapeutic protein. Our research to date indicates that this technology allows high expression levels and gene product activity level per vector genome, which will be critically important to ensure lower AAV viral titres containing the transgene.