Prospecção de patentes para o tratamento da doença de Fabry: os avanços da última década

Abstract

Introduction: Prospecting patents for medications for Fabry Disease is the best way to assess the new advancements in treating this condition, as these intellectual properties represent the latest developments worldwide. Through these patents, we can evaluate the types of technologies being developed, their functionality, effectiveness, and cost. Fabry Disease is one of the few rare genetic disorders with approved drug treatments, with many others in development. However, due to its high development costs and low prevalence, the cost of these medications is extremely high. Methods: Patents published in the last decade, available on Espacenet, referring to the development of a new medication for the treatment of Fabry Disease were selected, and data about them were collected, including the definition of mechanisms of action, the countries of development, and the types of institutions responsible for their filing. Results: 75 patents were found, of which only 19 were selected. The majority of patents found were registered by North American institutions, followed by China, and pertained to methods of gene therapy using recombinant viral vectors. These works were published by educational and research institutions as well as pharmaceutical or biotechnology companies, with a fairly equal distribution among them. Conclusion: Gene therapy technology with viral vectors or stem cells seems to be the focus of various entities worldwide, a technology that can also aid or be researched concurrently with new treatments for more prevalent diseases such as sickle cell anemia and Parkinson's disease. Additionally, it is hoped that the presence of educational and research entities in these registrations may lead to a reduction in development costs.