Small Qualiber, Big Potential
Guest Post from Elizabeth Witherspoon, Ph.D., APR
Courtesy of the North Carolina Translational and Clinical Sciences Institute (NC TraCS)
In the war on cancer, it is not enough to have the most potent weapon in your arsenal. You must be able to deliver it – on target and with lethal efficiency. You need the equivalent of a guided missile system or the oncology version of SEAL Team 6.
Enter Qualiber, a spinout from the University of North Carolina at Chapel Hill. Qualiber is using nanoparticle-based technologies to develop a delivery system for small interfering RNA (siRNA) and small molecule chemotherapy drugs used in the treatment of non-small cell lung and other cancers. The unique delivery platform is called Lipid-Calcium-Phosphate (LCP) nanoparticles (NPs).
siRNAs are molecules that mediate the RNA interference (RNAi) pathway, which interferes with the expression of a specific gene. Over the last decade, researchers have been exploring the value of siRNAs’ value in killing cancer cells. Because some researchers believe siRNAs can be used to silence virtually any human gene, they hold tremendous promise as lethal agents in the war on cancer. In fact, a 2006 Nobel Prize was awarded for the discovery of RNA interference.
However, the greatest challenge has been development of an effective delivery system. Qualiber’s drug-delivering nanoparticles may offer the answer.
The small Qualiber bullet hits big cancer targets
LCP-NPs are self-assembling, biodegradable nanoparticles containing an amorphous calcium phosphate (CaP) core wrapped with a single lipid membrane to stabilize it. The acid-sensitive CaP core carries the drug payload and dissolves in the acidic endosome inside a tumor cell, destroying the endosome with it, so as to release the siRNA cargo into the cytoplasm very quickly – before the siRNA degrades in the acidic environment as well. In other words, they penetrate the tumor cell walls and explode quickly with their lethal drug payloads. Other delivery systems have been able to get siRNA to the endosome, but not released rapidly from the endosome.
All of this occurs using particles which are 25 to 30 or 75 to 100 nanometers in diameter. For a frame of reference, a single human hair is 50,000 to 100,000 nanometers thick. Another advantage of Qualiber’s unique system is that, unlike other technologies, it avoids uptake by the liver, so that the drug is more effectively taken up by the tumor, where it can do its intended damage. Dosage of the siRNA drug can be lower than with other delivery systems, thus reducing costs and potential liver toxicity.
Technology from Leaf Huang, Ph.D., suggested by his mother
The system is the brain child of Leaf Huang, Ph.D., professor and chair of the UNC Eshelman School of Pharmacy’s Division of Molecular Pharmaceutics, which is devoted to researching drug delivery. Huang has spent more than 30 years researching liposome-based drug and gene-delivery systems. No stranger to commercialization as a path to translating bench discoveries to biomedical therapies, he has co-founded four other companies as well.
In the case of Qualiber, he owes at least some of the credit to his mother, who inadvertently suggested the key ingredient of his drug delivery system. Though she was not a chemist, his mother nevertheless warned him as a boy in Taiwan not to drink too many sodas for fear that the acid in the sugary drinks would ruin his teeth. She explained to him that his teeth were calcium phosphate, which dissolves in acid.
“So when I was looking for something that would spontaneously dissolve in acid, I remembered that, and it works,” said Huang.
More family connections at kids’ science fair
Anil Goyal, Ph.D., a serial entrepreneur with more than 20 years’ experience in biotechnology companies, is Qualiber’s chief executive officer. Jackie Quay, assistant director in the UNC Office of Technology Development, played matchmaker in bringing Huang and Goyal together.
“We began talking at our kids’ science fair in March 2010,” explained Goyal, describing his initial conversation with Quay. Goyal had just finished a project and was looking for his next venture. Quay, who works with researchers at the Lineberger Comprehensive Cancer Center, asked what he thought of siRNA therapeutics, as it was of such great interest to cancer researchers. Goyal remarked that he thought the biggest problem with siRNA was finding a delivery system.
“If someone has delivery, I’d be very interested,” he said. As it turns out, Quay was working with Huang on his efforts to commercialize his delivery system for siRNA and he needed a CEO.
The company, which has completed animal testing, is pursuing two pathways in its business model. One is seeking partnerships with pharmaceutical firms for further development and future marketing of the siRNA delivery system. The other is to develop its own products, which it hopes to begin soon under federal Small Business Innovation Research (SBIR) contracts for which it has applications pending.
Biotech Center, other partners boost Qualiber’s emergence
Qualiber has reached this level of commercialization, in part, with support from Carolina KickStart (CKS). CKS is a program of the North Carolina Translational and Clinical Sciences (NC TraCS) Institute at UNC, home of its NIH Clinical and Translational Science Awards. It seeks to accelerate the pace of translation of lab discoveries to useful biomedical therapies and technologies by helping UNC scientists through the commercialization process.
CKS provided Qualiber with a grant to hire attorneys to do patent research and execute required documents and consultants to review the SBIR grant applications. CKS also has helped facilitate exposure for Qualiber to potential investors at a symposium in Boston and through the 2011 Emerging Company Showcase, held at the Friday Center in May.
“Qualiber has derived tremendous help from Carolina KickStart, OTD and the Biotech [North Carolina Biotechnology] Center,” said Goyal during his presentation at the Emerging Company Showcase. “I think without that support structure I would have gone elsewhere and started something else.”
Recently, Qualiber also won a low-interest loan from the North Carolina Biotechnology Center to assist in more of the non-scientific aspects of the commercialization process.
(Editor’s note: Earlier this year Quay secured a $50,000 Technology Enhancement Grant from the Biotechnology Center to support the advancement of Huang’s invention, because of its significant market potential.)