A couple of University of Nebraska professors have launched a startup company with the goal of bringing to market an innovative method for delivering drugs and other therapeutics to targeted locations in the human body. The key ingredient? Milk.
Regular old milk. Turns out it’s very handy, especially in the biopharmaceutical space. New Atlas has previously reported on genetically modified cows that produce milk containing human insulin, milk produced by cows vaccinated with an HIV protein containing antibodies against the virus, and using milk nanoparticles to deliver injection-only RNA therapies orally.
Now, Janos Zempleni and Jiantao Guo, two researchers from the University of Nebraska-Lincoln, have launched a startup company, Minovacca, intending to bring to market an innovative targeted drug delivery system that uses – you guessed it – milk. The pair have committed to using their milk-based tech to treat both common and rare diseases.
“Because our technology is so versatile, we are not limited to one particular rare disease,” said Zempleni, a professor of nutrition and health sciences. “Rare disease groups are so thankful that there is maybe a light at the end of the tunnel.”
The technology relies on universal milk exosomes, nano-sized bubble-like structures released by cells that act like little messengers, carrying important materials such as proteins, fats, and genetic information. By tweaking the exosomes chemically and genetically, the researchers achieved target-specific delivery of their cargo to human cells.
Three peptides, short chains of ‘mini-proteins’ called amino acids, are attached to the membrane of each exosome. One directs the exosome to home in on a specific site in the body; another sends biochemical signals to immune cells telling them not to attack the exosome; and the third peptide improves the exosome’s survivability once it’s inside the target cell.
Ordinarily, lipids (fatty compounds) are used to attach peptides to the exosome membrane. The problem with this approach is that the lipid anchors detach when they encounter other lipophilic (lipid-loving) compounds in the body. So, the researchers tried something different. They created docking sites in a cell surface membrane protein called CD81, which is firmly anchored to the exosome. Guo used a bioorthogonal chemistry approach – bioorthogonal chemistry refers to chemical reactions that can occur inside a living system without interfering with biochemical processes – to create stable bonds between the docking sites and the peptides. In addition to improving the exosome structure’s stability, this chemistry-based adjustment also makes it more uniform, which improves the commercial viability of the innovative delivery system.
“Ensuring this homogeneous structure will allow the FDA to see that the exosomes can be produced consistently from batch to batch,” Guo said.
It also helps the exosome deliver its target to a specific location. Consider the chemotherapy used to treat cancer. It’s administered intravenously, so it travels around the entire body and damages all fast-growing cells indiscriminately, whether they’re cancerous or healthy. This widespread ‘poisoning’ contributes to side effects like hair loss, nausea, and a compromised immune system.
“Chemotherapy treatments kill not only cancer cells; they kill any cell that is proliferating fast,” said Zempleni. “And that’s something that we want to minimize with our technology.”
In addition to the targeted delivery of disease-treating drugs, the technology could deliver gene editing tools and other therapeutics. The researchers are working on licensing the tech through NUtech Ventures, the nonprofit technology commercialization affiliate of the University of Nebraska. A patent has been filed.
Forming the company has been a steep learning curve for Zempleni and Guo. They plan to keep the work ‘in-house,’ hiring University of Nebraska students whose work will ultimately result in an Investigational New Drug (IND) Application being submitted to the FDA.
Zempleni remains determined to keep his eyes on the therapeutic prize and not be distracted by the potential for money-making.
“It might sound cheesy, but if I had a choice between making $10 million in the company or saving 10 million lives, I would go for saving the lives,” he said. “I’m not into this for the money. It’s about helping people.”
Source: University of Nebraska-Lincoln
