Diagnosis of the Patent Landscape Related to Zolgensma®: Current Status and Opportunities

Abstract

Brazil, through the Unified Health System (SUS), has already spent approximately BRL 1 billion on onasemnogene abeparvovec (Zolgensma®), a gene therapy for spinal muscular atrophy. In this context, patents may influence access to and the price of these technologies, although their identification can be challenging because patent applications are filed before commercial and generic names are defined. This study aimed to map and investigate patents related to Zolgensma®, analyzing their validity, main technologies, and applicants. Seventeen patent families were identified, most of them filed by Nationwide Children’s Hospital. In Brazil, four families have equivalent patent applications at the Brazilian National Institute of Industrial Property (INPI), but none has been granted. The foundational patent for the technology, internationally filed in 2009, was also not filed in Brazil. These findings indicate opportunities to strengthen national research, development, and innovation in gene therapies, particularly for spinal muscular atrophy.