Descripción del proyecto
Una matriz molecular para aplicaciones de genoterapia «in vivo»
La mayoría de las aplicaciones de genoterapia «in vivo» emplean vectores víricos que pueden administrar de manera eficaz la carga (gen o genes de interés) dentro de las células diana al imitar el proceso de infección vírica. Con todo, la administración de genes mediada por virus adolece de problemas de seguridad, como la mutagénesis insercional, y requiere enfoques no víricos alternativos para administrar genes a las células. El proyecto ENDOSCAPE, financiado con fondos europeos, propone emplear moléculas que faciliten la evasión desde los endosomas, los orgánulos celulares responsables de atrapar las moléculas internalizadas y conducirlas a la degradación. La idea es crear una matriz molecular que abarque estos potenciadores, junto con el gen de interés y un ligando dirigido, lo que resolverá el problema de la administración citosólica y nucleica de las genoterapias.
Objetivo
Gene therapy is one of the most promising treatment options for future advanced therapies in a broad range of diseases. Successful gene delivery requires the recognition of target cells as well as cytosolic and nucleosolic uptake of the gene. Currently, non-viral based gene delivery such as transfection reagents are only suitable for in vitro applications, and clinical gene therapeutics delivery is accomplished via viral vectors, which still has major safety concerns as well as complex and costly manufacturing procedures, preventing future implementation for the treatment of diseases with large patients groups.
In the last 15 years, a class of secondary plant metabolites has been discovered that selectively mediates endosomal escape and cytoplasmic delivery of macromolecules specifically at low endosomal pH, thereby inducing a 40-fold enhanced gene delivery efficacy in vivo. However, endosomal escape enhancers and gene therapeutic product must be applied as two components, thus complicating therapeutic approval and clinical applicability.
The ENDOSCAPE technology platform will develop and collect proof of concept for a non-viral gene delivery technology where endosomal escape enhancers, gene therapeutic product and targeting ligand are all bound to one molecular scaffold. Proof of concept of the ENDOSCAPE technology has a major impact on the therapeutic opportunities for current and future macromolecule drugs for a broad range of diseases and large patient groups.
All this induces new biotech-based businesses, new research projects and creates new technology platforms for development of new macromolecule therapeutics for a broad range of disease indications. The non-viral based ENDOSCAPE technology will enhance therapeutic efficacy with lower therapeutic dose thereby reducing costs of healthcare, improving the health of patients worldwide, and strengthening the competitive landscape of the EU in the worldwide quest for such an advanced technology.
Palabras clave
Programa(s)
Convocatoria de propuestas
Consulte otros proyectos de esta convocatoriaConvocatoria de subcontratación
H2020-SC1-2018-Single-Stage-RTD
Régimen de financiación
RIA - Research and Innovation actionCoordinador
10117 Berlin
Alemania