Gene Therapy Explained

Published 14 May 2020
Written by Freeline

An animation explaining what gene therapy is, how it has evolved over 50 years and the goal of providing a long-term cure for patients who receive treatment.

Freeline uses gene therapy technology to target the unmet needs of genetic diseases using a vector that targets the liver. From the liver, the newly synthesized protein will be secreted to the circulation where it can reach targeted cells throughout the body. Freeline uses an in vivo approach to deliver the gene directly into the human cells of the liver to enable a high level of secretion. Freeline is primarly focused on developing liver-based gene therapies for haemophilia B and Fabry Disease.