Injectable Biomaterials Enable Long-Lasting Nucleic Acid Delivery for Advanced Therapeutics
Invented by ANDRESEN; Thomas L, HENRIKSEN; Jonas R, HANSEN; Anders E, MELANDER; Carl Fredrik, BRUUN; Linda Maria, BRUS; Anja, CLERGEAUD VEIGA; Gael Francis, LØVSCHALL; Kaja Borup, JÆHGER; Ditte Elisabeth, MARCHARD; Laura Olivia Marie, BJÖRGVINSDÓTTIR; Unnur Jóna, XIN; Li
Introduction
Delivering gene therapies to the right place in the body, and keeping them there long enough to work, is a big problem in medicine. Many promising treatments with DNA or RNA, which could help fight cancer, heal injuries, or fix genetic issues, don’t work as well as hoped because the body washes them away too quickly or breaks them down. A new patent application aims to solve these problems by creating a new way to deliver nucleic acids using a special mix of hydrophobic (water-repelling) and hydrophilic (water-loving) materials. This invention could make gene therapies safer, longer-lasting, and more effective. In this article, we’ll explore the reasons behind this invention, look at what has been tried before, and explain how this new technology works in simple terms.
Background and Market Context
Gene therapies have been a hot topic in medicine for years. Scientists know that if they can control genes, they can control how cells work. This means they could turn off genes that cause disease, or turn on genes that help the body heal. DNA and RNA medicines are already being used or tested for many diseases, from cancer to genetic disorders to chronic infections. But there is a common roadblock: getting these genetic instructions to stay where they need to work, for long enough, and at the right dose, is very tough.
When doctors inject a gene therapy, the treatment often spreads throughout the body. This can cause two problems. First, the medicine might not reach high enough levels at the place it’s needed, like inside a tumor or a damaged tissue. Second, if the treatment spreads, it can cause side effects in other parts of the body. Even when the medicine gets to the right spot, DNA and RNA are fragile. The body’s watery environment and enzymes can break them down quickly. For some therapies, this means patients need frequent shots, which is uncomfortable and costly.
Drug companies and doctors want better answers. They want gene therapies that:
– Stay at the place they are needed, like inside a tumor.
– Release their medicine slowly, so patients don’t need as many injections.
– Protect the DNA or RNA so it stays active longer.
– Are safe, easy to use, and work for many types of genetic medicines.
The market for gene therapies is growing fast. Many companies are racing to find better delivery systems. The hope is that if these problems are solved, gene therapies could become routine treatments for cancer, rare diseases, immune problems, and even for delivering vaccines. The need is clear: there is a big demand for gene medicines that are safer, longer lasting, and more targeted.
Scientific Rationale and Prior Art
Let’s break down why delivering gene medicines is so hard, and what has been tried before.
DNA and RNA are big, charged molecules. They don’t slip into cells easily. The body’s cells are surrounded by oily membranes that keep out large and charged things. Plus, in the bloodstream and tissues, there are enzymes that quickly chop up DNA and RNA. So, to get gene therapies into cells, scientists have tried wrapping them in nanoparticles made from lipids (fats) or polymers (plastics). Sometimes, they use special “carriers” called liposomes or polyplexes that can stick to the DNA or RNA and help them get inside cells.
But even with these carriers, the medicines often spread too fast after injection and don’t stay put. Many carriers are made for water-based environments, which means when they enter the body, they can drift away from the injection site. This is a problem for local treatments, like injecting gene therapy into a tumor or a damaged muscle. If the medicine spreads too much, it can cause bad side effects, or simply not work where it’s needed.
Some scientists have tried making “depots” — gels or solid forms that hold onto the medicine and let it out slowly. These can be made from special sugars, fats, or polymers that are safe in the body. The idea is that the depot sits at the injection site and slowly releases the medicine over days or weeks. But this approach has its own problems. Many gene medicines are not stable in these depots, especially if water is present. The gene medicines can degrade or clump together, losing their activity.
Another trick
Click here https://ppubs.uspto.gov/pubwebapp/ and search 20250332283.
