Schlieren ZH/Amsterdam – InSphero, together with SanaGen and the Sanquin Research Institute, has been awarded a Eurostars grant for a gene therapy for the treatment of AATD. The grant amounts to 1.3 million euros.
The biotech company InSphero in Schlieren, SanaGen and the Sanquin Research Institute, both in Amsterdam, have been awarded Eurostars funding for a gene therapy that could become the first "cure" for severe alpha-1 anti-trypsin deficiency (AATD), according to a press release. The grant of 1.3 million euros is intended for the joint NOCAAVAAT project.
The NOCAAVAAT project aims to develop a breakthrough gene therapy for severe alpha-1 anti-trypsin deficiency (AATD). The Eurostars program is a joint program of EUREKA and the European Union.
AATD is a rare hereditary disease that affects one in 2000 to 5000 people in Europe and North America, the report continues. Depending on the specific genetic mutations, AATD can lead to lung or liver disease. The disease is very disabling and shortens life expectancy. An effective therapy is not yet available.
The lead applicant SanaGen is an SME based in Amsterdam that focuses on the development of gene therapies for genetic disorders. "Together, we are able to advance a breakthrough technology that will significantly improve outcomes for patients with alpha-1 antitrypsin deficiency," David Mosmuller, CEO of SanaGen, is quoted as saying.
"The use of human multicellular 3D in vitro models to test the efficacy and safety of gene therapy applications in rare genetic disorders is an invaluable accelerator to finding curable solutions for patients with these diseases," Francisco Verdeguer, Vice President of Liver Disease at InSphero, is quoted as saying. The InSphero models are one of the cornerstones for testing this therapeutic strategy.
InSphero is a spin-off of the University of Zurich and the Swiss Federal Institute of Technology Zurich and is based in the Bio-Technopark Schlieren-Zurich.ce/gba