Composição farmacêutica nanométrica na forma de lipossomas ou nanoemulsão que contém sequências específicas de RNA de interferência – Vitrine Tecnológica (Technology Showcase)

Technology background

The advancement of oncological therapies has driven the development of more specific and less toxic approaches, especially in the field of nanotechnology and gene therapy. In this context, the use of RNA interference (siRNA) stands out as a promising strategy for silencing genes involved in tumor progression. However, challenges such as siRNA instability in the body, its degradation by enzymes and the difficulty of reaching specific tissues — particularly the central nervous system due to the blood-brain barrier — still limit its clinical application. Therefore, there is a growing demand for efficient delivery systems that guarantee protection, therapeutic targeting and efficacy, especially in the treatment of aggressive tumors such as glioblastoma.

Technology description

A presente tecnologia refere-se a uma composição farmacêutica em escala nanométrica, based on liposomes or nanoemulsions, that incorporates specific interfering RNA sequences. These systems act as delivery vectors capable of forming stable complexes with siRNA., protecting it from degradation and allowing its targeted release into tumor tissue. The mechanism of action is based on the silencing of the NT5E gene, responsible for the expression of the CD73 protein, which is associated with the progression and aggressiveness of several tumors. The developed formulation is compatible with different routes of administration, including intravenous, intratumoral e intranasal, expanding its clinical applicability to a process.

Problem Fixed

A tecnologia proposta visa superar limitações significativas das terapias antineoplásicas convencionais, such as low selectivity, a alta toxicidade sistêmica e a complexidade de atingir o sistema nervoso central. Furthermore, faces specific gene therapy challenges, such as the instability and degradation of siRNA in the body. By employing nanometric delivery systems, the invention allows the protection of therapeutic molecules, improves its bioavailability and makes it possible to target the tumor site. Thus, contributes to a more effective and safe approach to treating tumors, especialmente aqueles de complexo acesso e significativa agressividade, of glioblastoma.

Applications

The main application of the technology is in the treatment of glioblastoma, one of the most aggressive brain tumors and difficult clinical management. However, considering the mechanism of action based on the silencing of genes associated with tumor progression, a tecnologia apresenta potencial para aplicação em outros gêneros de câncer que apresentem superexpressão da proteína CD73. Additionally, a plataforma desenvolvida contém a possibilidade de ser adaptada para o transporte de diferentes sequências de siRNA, expanding its use to other diseases that require specific gene therapies. The versatility of the administration routes also favors its integration into different therapeutic protocols..

Advantages

A tecnologia apresenta um destaque referente a sua elevada especificidade molecular, promoting targeted silencing of genes associated with tumor development. This factor contributes to the reduction of side effects, as it minimizes the action on healthy cells. A utilização de sistemas nanométricos permite maior estabilidade do siRNA, protection against enzymatic degradation and greater efficiency in delivery to the site of action, including the potential to cross the blood-brain barrier. Para além, the use of non-viral vectors increases the biological safety of the approach. A flexibilidade quanto às vias de administração e a possibilidade de adaptação da plataforma para distintos alvos terapêuticos enfatizam o potencial inovador e aplicabilidade da tecnologia.

Technology Readiness Level (TRL – Technology Readiness Level)