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Business-Academic Team to Create RNA Delivery for Cancer

Vials and bottles

(National Cancer Institute, Unsplash)

2 Mar. 2023. A university lab and biotechnology company are developing new cancer treatments with therapies that stop RNA activity, delivered by advanced nanoscale particles. The agreement gives the company SiSaf Ltd. in Guilford, U.K., an option to license rights to research by pharmacology professor Achim Aigner at University of Leipzig in Germany, but financial terms of the deal are not disclosed.

SiSaf develops delivery mechanisms for therapies using ribonucleic acid or RNA, a basic nucleic acid with instructions from genetic codes in DNA for producing proteins in cells. Synthesized RNA is growing as a therapeutic option, but RNA on its own is unstable, thus it needs packaging for delivery. Nanoscale lipid or natural oil particles are a leading form of RNA delivery today, but SiSaf notes lipid nanoparticles themselves are fragile and unstable, requiring refrigeration or freezing from manufacture through administration in clinics. And, says the company, many lipid nanoparticles today can create toxicity when oxidized or trigger immune reactions from polyethylene glycol added to boost stability.

SiSaf says its technology called bio-courier, provides a silicon matrix for lipid nanoparticles that binds and condenses RNA payloads for greater stability. The tight binding to silicon, says the company, protects against interaction with water and disintegration, which allows for freeze-dried formulations while ensuring the RNA’s integrity. With this type of RNA delivery, says SiSaf, treatments have a longer shelf life, and can be stored and transported in ambient temperatures, rather than requiring cold-chain handling.

Achim Aigner and colleagues at University of Leipzig study therapeutic applications of RNA that interfere with normal gene expression. Much of Aigner lab’s research targets ribozymes, enzymes that induce a chemical reaction to form protein chains. As therapies, RNA interference seeks to deplete and shut off expression of disease-causing genes in cells, particularly cancer-causing genes found in tumors.

“Pancreatic cancer is an area of high unmet need”

In their agreement, researchers from SiSaf and Aigner’s lab are collaborating on delivery of micro-interfering RNAs or miRNAs, synthetic non-coding RNA molecules designed to block expression of cancer-causing genes in tumors, beginning with pancreatic cancer. Aigner and colleagues already demonstrated two types of miRNA, named miR24–3p and miRNA-506-3p, to kill tumor cells in lab animals induced with pancreatic cancer.

The deal calls for SiSaf to develop bio-courier formulations that combine miR24–3p and miRNA-506-3p molecules as a more efficient and targeted treatment for pancreatic cancer. The Aigner lab will then test the bio-courier miRNAs as treatment candidates for pancreatic cancer in preclinical models. The agreement gives SiSaf an exclusive option to later gain a worldwide license to the miRNA patent rights held by the university.

“Due to their parallel, selective effects on multiple defined targets,” says Aigner in a SiSaf statement, “miRNAs offer exceptional opportunities for the development of novel drugs that show enhanced efficacy while avoiding tumor cell resistance.” Suzanne Saffie-Siebert, CEO of SiSaf adds, “Pancreatic cancer is an area of high unmet need, and we are encouraged by Professor Aigner’s research and the potential to develop miRNA-based replacement therapies to improve outcomes for patients.”

Pancreatic adenocarcinoma is the most common type of pancreatic cancer, accounting for about 95 percent of cases. Because the pancreas is found deep in the body, symptoms do not often become apparent until more advanced stages of the disease. As a result, pancreatic cancer is not often diagnosed in its early stages, and according to American Cancer Society, the five-year survival rate drops markedly for people with pancreatic cancer that spreads beyond the localized stage.

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