Haapaniemi group allocated 20m NOK to prepare a clinical trial with genome-edited T cells

The national programme for clinical treatment research – KLINBEFORSK - has allocated research funds to Haapaniemi group for developing CRISPR-Cas9 gene therapy for STAT1 Gain-of-Function disease.

Dr. Emma Haapaniemi Photo: NCMM

The project aims to develop T-cell therapy for STAT1 Gain-of-Function disease and other immunodeficiencies, where bone marrow transplant is not an immediate treatment option. The T-cells will be used in rescue therapy for life threatening or chronic infections. The project is based on preliminary data generated by PhD student Katariina Mamia, and is developed in response to the needs of the local clinicians at the Oslo University Hospital.

The proposal was developed together with Hans Christian Erichsen, a consultant pediatric immunologist, who wished more treatment options for his patients with STAT1 Gain-of-Function disease.  STAT1 gain-of-function (GOF) is a primary immunodeficiency characterized by chronic mucocutaneous candidiasis (CMC), recurrent respiratory infections, and autoimmunity. The condition is difficult to treat with standard therapies.

Gretchen Repasky, Joint Communications Director for Nordic EMBL Partnership, comments:

- This new and significant funding from KLINBEFORSK awarded to Dr. Emma Haapaniemi at NCMM enables the development of CRISPR-Cas9 gene therapy, a strong step forward in translational research. As one of the research institute nodes in Nordic EMBL Partnership for Molecular Medicine, NCMM and others in the Partnership are closing the gap from basic to clinical research and trials, ultimately raising the benefit to society, improving healthcare.

Jan Frich, deputy managing director of South-Eastern Norway Regional Health Authority and the chair of the KLINBEFORSK board, empahsised that the national clinical treatment studies receiving the grant will be of great importance to patients in all health regions, and will particularly help to strengthen the ongoing investment in advanced cell therapies in Norway.