The patent, owned by the University of Florida Research Foundation (UFRF) and exclusively licensed to iOncologi, further consolidates the intellectual-property portfolio supporting the company’s UNIFYRs (UNiversal Immune FortifYing RNAs) platform — an innovative approach that leverages RNA-driven immune reprogramming to restore and amplify checkpoint inhibitor efficacy across multiple solid-tumor types.

iOncologi Secures Foundational U.S. Patent as Landmark Studies Link COVID19 mRNA Vaccines to Improved Survival With Cancer Immunotherapy.

In June 2000, when President Bill Clinton announced that the initial sequencing of the human genome had been completed, it was hailed as a milestone in medicine, promising cures for everything from Alzheimer’s and Parkinson’s to diabetes and cancer.

But Matt Disney had questions.

The teams sequencing the human genome had focused on DNA, counting only about 30,000 protein-coding genes, about the same number as a mustard plant or a worm. What explained human complexity and diversity if humans had similar numbers of genes to plants and invertebrates?

At the time, Disney was working on his Ph.D. at the University of Rochester, focusing primarily on ribonucleic acid, or RNA, which was thought — and has now been shown — to make up 90% of the genome.

Disney thought the answers to his questions about the human genome project, and its real potential, might lie in the RNA. But in 2000, RNA’s structure and functions were still something of a black box, and the idea of making medicines targeted to RNA was rejected as unfeasible, too risky or both.

The gaps in knowledge enticed Disney, who saw translating genetics into treatments as a worthy challenge.

“I thought, well, if we could really turn the human genome and the RNAs that they make into new targets for medicines, that would be transformative.”

Two decades later, Disney and his team at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology in Jupiter, Florida, have discovered more than 200 unique RNA-targeting compounds. His methods and discoveries have changed minds, igniting a global race to treat incurable diseases via their RNA.

The genetic scissors are a protein complex known as CRISPR-Cas12a. Until now, it had only been known to bind and cleave DNA, a molecule that holds life-giving genetic codes. Knowing that the genetic scissors can also bind RNA — the genetic cousin to DNA — gives scientists a crucial shortcut for designing future medical diagnostic tests.

By directly targeting RNA, tests for hepatitis, HIV, cancer and other disorders also might become simpler and less prone to false positives, said Piyush K. Jain, Ph.D., the study’s lead investigator, a Shah Rising Star assistant professor of chemical engineering and an affiliate assistant professor of molecular genetics in the UF College of Medicine. The group’s findings were published recently in the journal Nature Communications.

Scientists at UF Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The compound restored the health of neurons in the lab and rescued mice with the disease.

The potential medication is described this week in the scientific journal Proceedings of the National Academy of Sciences. It is designed to be taken as a pill or an injection, said the lead inventor, professor Matthew Disney, Ph.D., chair of the UF Scripps chemistry department. Importantly, experiments showed that the compound is small enough to cross the blood-brain barrier, a hurdle other approaches have failed to clear, he said.