Gene therapy for liver disorder

Gene therapy for hereditary angioedema (HAE-CURE)

The HAE-CURE project is a public-private partnership that aims to develop innovative therapies for genetic disorders. The current project focuses on a novel treatment for hereditary angioedema a life-threatening liver disorder. The combined expertise from the academic partner Sanquin, the gene therapy company SanaGen and the expression cassette optimization tools of Annogen will be used to generate novel gene therapy vectors.

Hereditary angioedema results from severe attacks of swelling that most commonly affects the arms, legs, face, intestinal tract and the airways. Hereditary angioedema is characterized by the absence or dysfunction of C1-esterase inhibitor which results in excessive production of bradykinin which leads to the severe and unpredictable life-threatening swellings described above. The goal of the current project is to develop a gene therapy vector that allows for restoring functional C1 esterase inhibitor expression in the liver thereby alleviating the clinical symptoms in these patients. In Europe around 3500 patients suffer from this life-threatening disorder. Apart from hereditary angioedema the gene therapy platform that will be developed in this project can also be used for other congenital liver disorders that affect a large number of patients worldwide. Specifically, the current project aims to develop a novel, highly efficient liver-specific promoter that will be designed using the bioinformatic pipeline developed by Annogen. Pre-existing antibodies directed towards AAV based gene therapy vectors preclude wide applicability of the current generation of gene therapy vectors. To resolve this issue Sanquin in collaboration with SanaGen will work on next generation delivery devices that allow for efficient expression of therapeutic genes for more efficient treatment of congenital liver disorders.   

More information about Sanquin

More information about Sanagen

More information about Annogen

Summary
Hereditary angioedema (HAE) is a rare genetic disorder which leads to severe and unpredictable inflammatory events that coincide with life-threatening swelling. The current project aims to provide a permanent cure for this disorder employing a liver directed gene therapy approach.
Technology Readiness Level (TRL)
36 months
Time period
3 - 3
Partners