More than 17,500 patients are living on the waiting list at any given time for a liver transplant. Unfortunately, there aren't enough of the available, donated organs to go around, leading to a critical and frequently deadly backlog. Roughly 10% of the patients on that waiting list die each year while waiting for the prospect of a new organ.
Orna is developing a new treatment that uses circular RNA and specialized lipid particles to prompt a patient's own body to produce the cell therapies needed to fight disease. The technology has the potential to "unlock an entirely new class of genetic medicines and cell therapies for patients who today have limited or no treatment options," said Francisco Ramírez-Valle, senior vice president and head of Immunology Research and Early Clinical Development at Eli Lilly, in a statement.
A fresh supply of the immune cells that keep the brain tidy might one day help to treat a host of conditions, from ultra-rare genetic disorders to more familiar scourges, such as Alzheimer's disease. In the past few months, a spate of new studies have highlighted the potential of a technique called microglia replacement and explored ways to make it safer and more effective. "This approach is very promising," says Pasqualina Colella, who studies gene and cell therapy at Stanford University School of Medicine in California. "But the caveat is the toxicity of the procedure."
