SanaGen, InSphero and Sanquin Awarded with Eurostars Grant for a Breakthrough Gene Therapy, Poised to Become First 'Cure' for Severe Alpha-1 anti-Trypsin (AAT) Deficiency (AATD).
In a notable collaboration that underscores the power of innovation in drug discovery, SanaGen and InSphero, alongside the renowned Sanquin research institute, have been awarded a 1.3 million euro grant for the ambitious project NOCAAVAAT.
The NOCAAVAAT project, which aims to develop a breakthrough gene therapy for
severe Alpha-1 anti-Trypsin (AAT) Deficiency (AATD), was rated as one of the best project proposals of Europe. AATD is a rare, hereditary disorder which affects 1 in 2000-5000 people in Europe and North America. AATD may result in lung and/or liver disease depending on the specific genetic mutations. In their most severe form the disease manifest as emphysema, recurrent pulmonary infections, barrel-shaped chest, tachycardia, and eye-vision changes. This is intensely debilitating and reduces life expectancy. An effective therapy is not yet available.
The Swiss-Dutch consortium brings together different scientific and technological disciplines in AAV-mediated gene delivery and liver disorders.
Lead applicant SanaGen is a cutting-edge SME based in Amsterdam, Netherlands, focused on developing gene therapies for genetic disorders. The company has been dedicated to exploring innovative ways to reduce gene therapy development costs and make rare disease cures profitable, while pricing drugs to benefit the maximum number of patients possible.
David Mosmuller, MD, PhD, CEO of Sanagen: "We're thrilled to announce our successful collaboration with this esteemed consortium, securing a pivotal European grant. Together, we're poised to advance groundbreaking technology that promises to significantly improve outcomes for patients with alpha-1-antitrypsin deficiency.”
InSphero, a spin-off company of the Swiss Federal Institute of Technology (ETH) Zurich and the University Zurich, is a pioneer in 3D-cell-based assay solutions and scaffold-free 3D organ-on-a-chip technology. InSphero creates miniature organs and biological systems to better predict patient response in vitro. InSphero deploys these unique technologies for highly scalable drug discovery programs along the drug development value chain from target identification to pre-clinical testing.
Francisco Verdeguer, PhD, VP of Liver Disease at InSphero commented “The use of human 3D in-vitro multicellular models to test the efficacy and safety of gene therapy applications of rare genetic disorders is an unvaluable accelerator to find curable solutions for patients suffering these diseases. Together we SanaGen and Sanquin we tackle AATD, and our translational approach consists of modelling simultaneously the suppression of the expression of aberrant AAT while expressing a healthy AAT protein in human-derived liver spheroids. This is a novel and promising approach where InSphero models are one of the cornestones to test this therapeutic strategy.”
Sanquin Blood Supply is a not-for-profit organization with a recognized niche position within the Dutch (academic) research landscape. Sanquin operates in the fields of Blood Products, Diagnostic Services, Biotechnology and Research (basic, translational, clinical, and applied). The Department of Molecular Hematology, under the direction of Prof. Dr. Jan Voorberg will lead the research. The Voorberg-lab has developed novel and innovative capsid engineered AAV variants that efficiently target liver hepatocytes.
Prof Dr. Jan Voorberg: “We look forward to work together with InSphero and SanaGen on this project and we hope that our unique approach will allow us to develop a novel therapy for patients with this chronic disorder”.
Last week, the partners joined for a successful kick-off meeting at the InSphero offices in Zurich.
Eurostars is the largest international funding program for SMEs wishing to collaborate on R&D projects that create innovative products, processes or services for commercialization and is part of the European Partnership on Innovative SMEs. The partnership is co-funded by the European Union through Horizon Europe.