Strategy and Scientific Background

“Our mission is to exploit our unique non-viral

polymer technology platform for translating the

power of gene therapy into therapeutic

medicines to the lives of patients suffering from

inherited disorders.”

We have been focussing our research and development efforts on orphan diseases that thus far have no effective treatments available. Branca Bunús Ltd utilises a distinct CRISPR nuclease: Cas9, in combination with a novel multifunctional cationic polymer delivery vector to create a medicinal therapeutic unit that can be applied directly to the body. Using these two components in unison, we can target a broad range of cell types and organs for the treatment of associated inherited disorders.

Polymer Gene Delivery:

Gene delivery is a multifaceted process whereby nucleic acids needs to be delivered through the cell membrane into target cells to enact their desired biological function. Fundamentally, clinical translation of genetic therapeutics have been hindered by a lack of viable carrier vehicles for such genetic information.

CRISPR Gene Editing:

The advent of the CRISPR based gene editing system has revolutionized the field of modern medicine with its simplicity and versatility. Fundamentally, the CRISPR system is composed of an programmable endonuclease Cas protein and an RNA guidance system. Together, these components act to locate, bind and edit the DNA of target genes. Here, the guide RNA recognizing target DNA by base pairing and the Cas endonuclease initiates DNA cleavage though a molecular scissors action, which is ultimately repaired by the cell. CRISPR gene editing has rapidly accelerated biomedical research globally and its development has been fast-tracked into clinical translation owing to its potential to treat previously incurable diseases.