Ercole and Novosom AG announce antisense delivery success

23-Mar-2004

Ercole Biotech, Inc., and novosom AG announced the successful completion of the first phase of their research collaboration. The research involves the intracellular targeted delivery of Ercole's alternative splicing oligonucleotides by using novosom's SMARTICLES® delivery system. The results confirmed the functionality of Ercole's technology in a transgenic mouse model and also confirmed that SMARTICLES can be used to target specific organs effectively and reduce the total quantity of oligonucleotide required to produce the desired effect.

Alternative splicing is a natural process in which a single gene can encode multiple related, yet distinct, protein products. Most mammalian genes are interrupted by pieces of DNA, called introns, which are selectively removed from RNA message produced from a gene through the process of RNA splicing. Cells may remove and splice together different segments of RNA from the same gene, resulting in alternatively spliced gene products. In many cases, the alternatively spliced products can have very different biological effects, with one form contributing to human disease and another form preventing human disease.

Novosom's SMARTICLES® enable the targeted delivery of oligonucleotides, plasmids and other materials directly into living cells. Smarticles represent a novel class of liposomes that are fully charge-reversible, which offers a number of advantages in delivering certain new classes of therapeutic agents. Smarticles are stable in blood and distribute in the same manner as traditional anionic liposomes. In stark contrast to anionic liposomes, Smarticles® become positively charged during endocytosis and thus provide a mechanism for endosome release and intracellular delivery.

"We are very pleased to be working with Ercole in the delivery of their proprietary oligonucleotides designed to modulate alternative splicing. This technology has the potential to be a therapeutic breakthrough for a number of diseases," commented Dr. Steffen Panzner, CEO of novosom AG. "We are also pleased to demonstrate the functionality of novosom's SMARTICLES in delivering Ercole's proprietary oligonucleotides directly into the cells of targeted tissue. These studies further confirm that Smarticles offer substantial potential for the delivery of antisense molecules. "

https://www.bionity.com/en/news/36226/ercole-and-novosom-ag-announce-antisense-delivery-success.html