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Apertura Gene Therapy logo Cure Collaboration Residency

Apertura

Apertura is widening the aperture of gene therapy opportunities for treating debilitating diseases. By simultaneously designing AAV capsids, regulatory elements, and payloads, we are working to overcome current limitations in gene delivery and expression.

Lab research image Cure resident Apertura

Overview

Apertura is pioneering next-generation AAV gene therapies through novel capsid and payload engineering. We are a world-class team of experts in vector engineering, gene regulation, machine learning and treatment of rare diseases, collaborating to build a pipeline of next generation genetic medicines.

Apertura's Mission

Apertura is engineering next-generation gene therapies to treat unaddressed genetic diseases using its machine learning-powered delivery and expression platforms. Guided by diversity of thought, scientific technologies, and talent, we are working together to overcome genetic diseases for all patients. We are committed to growing the field of gene therapy and believe that together we maximize our impact by working with corporate and academic collaborators, patients, and disease foundations. Apertura takes a holistic, data-driven approach to creating a pipeline of next-generation gene therapies through integrated capsid and payload engineering. We have engineered a central nervous system (CNS)-targeting AAV capsid that binds to the human Transferrin Receptor 1 (TfR1). Using capsids engineered to deliver genes to the central nervous system (CNS), we are addressing devastating CNS diseases.


At-a-Glance

  • The Apertura team is designing AAV capsids, regulatory elements, and payloads to overcome limitations in gene delivery and expression.

  • We design capsids to engage human disease-relevant receptors for more effective and selective gene delivery, expanding the scope of treatable genetic diseases.

  • We are an entrepreneurial biotechnology company expanding the potential of genetic medicines by fostering a collaborative and science-driven environment.

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