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.

Zürich — 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 in Europe. AATD is a rare, hereditary disorder that 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 manifests 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. Dr. David Mosmuller, MD, CEO of Sanagen said: “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.”

Dr. Francisco Verdeguer, Vice president 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 invaluable accelerator to find curable solutions for patients suffering these diseases. Together with SanaGen and Sanquin, we tackle AATD, and our translational approach consists of modeling 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 cornerstones 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 said: “We look forward to working 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”.

The NOCAAVAAT team at the consortium kick-off meeting in Zürich, Switzerland

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