Apertura Gene Therapy is at the forefront of developing next-generation adeno-associated virus (AAV) gene therapies, focusing on innovative capsid and payload engineering to enhance gene delivery and expression. The company is actively designing AAV capsids and regulatory elements that address existing limitations in the efficiency of gene therapy. Their research includes engineering capsids that can specifically target human receptors, which facilitates improved gene delivery to critical areas such as the central nervous system (CNS) while minimizing off-target effects, particularly in the liver.

The team at Apertura comprises experts in vector engineering, gene regulation, and machine learning, who collaborate to create a robust pipeline of advanced genetic medicines. Their work includes the development of stabilized AAV capsids and the application of machine learning techniques to optimize capsid design for better biodistribution and reduced sensitivity to neutralizing antibodies. This innovative approach aims to overcome challenges in gene therapy, particularly for rare diseases, by ensuring more effective and targeted delivery of therapeutic genes.

United States

Apertura is a biotechnology company opening opportunities in gene therapy for treating debilitating diseases with limited options. We are pioneering next-generation AAV gene therapies by simultaneously engineering capsids, regulatory elements, and payloads to overcome current limitations in genetic medicine. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, we are located at the Cure in New York City.

Apertura Gene Therapy

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