Skip to Content

RNA Drugs Target Genes That Were Once Off-Limits

A unique class of RNA drugs could bring new treatments to cancer and other diseases.

A new class of medicines could give doctors the ability to awaken underperforming genes in patients who currently have no treatment options.

Boston-area startup RaNA Therapeutics is developing a novel kind of medicine that can boost the activity of genes that may be silenced or underactive and thus cause disease. The medicine would use a small RNA-like molecule that blocks the function of a long RNA molecule that is hampering the expression of such a gene.

By activating genes, RaNA’s medicines could do something completely new, says Jeannie Lee, a molecular biologist at Harvard Medical School and scientific founder of the company. Most drugs work by changing the function or stability of a gene product—for example, by blocking an enzyme that is overly active. “Current therapeutics go after something that is already expressed,” says Lee. “There are currently no methodologies to turn on a gene that is silent.”

RaNA’s technology, which is still at the stage of testing in lab animals, could be used to treat cancers as well as rare genetic diseases. Often, part of the changes that take place in a cancer cell includes silencing of so-called tumor-suppressor genes. And in some genetic diseases, a normal gene may be aberrantly silenced or just weakly expressed.

RaNA’s approach could also target complex metabolic diseases, says Art Krieg, the company’s CEO and cofounder. “In diabetes and other metabolic diseases, there are a lot of different targets that have been identified, and so the ability to reach in and selectively turn on just one gene, we think, is going to have very broad therapeutic applications,” Krieg says.

In lab tests, RaNA has seen a range of effects on gene expression, from a fourfold increase to a 100-fold increase. “It varies from gene to gene,” says Krieg. “It certainly is not rare to see 10-fold increases,” he says.   

The company formed less than two years ago and soon thereafter announced $20.7 million in investments from GlaxoSmithKline’s venture fund SR One, Monsanto, and others. The company hopes to start testing its products in patients in 2015, says Krieg. RaNA is keeping mum about which diseases it will target for now. Broadly, Krieg says, the company will go after diseases for which there are currently no drugs available. It’s pursuing leads in diseases involved in the nervous system, inflammation, and a muscle disease. The treatment paradigm would likely involve an infusion once every month or so, says Krieg. Depending on the disease, it could be delivered through a vein, a muscle, or a spinal tap.

Keep Reading

Most Popular

Europe's AI Act concept
Europe's AI Act concept

A quick guide to the most important AI law you’ve never heard of

The European Union is planning new legislation aimed at curbing the worst harms associated with artificial intelligence.

Uber Autonomous Vehicles parked in a lot
Uber Autonomous Vehicles parked in a lot

It will soon be easy for self-driving cars to hide in plain sight. We shouldn’t let them.

If they ever hit our roads for real, other drivers need to know exactly what they are.

supermassive black hole at center of Milky Way
supermassive black hole at center of Milky Way

This is the first image of the black hole at the center of our galaxy

The stunning image was made possible by linking eight existing radio observatories across the globe.

transplant surgery
transplant surgery

The gene-edited pig heart given to a dying patient was infected with a pig virus

The first transplant of a genetically-modified pig heart into a human may have ended prematurely because of a well-known—and avoidable—risk.

Stay connected

Illustration by Rose WongIllustration by Rose Wong

Get the latest updates from
MIT Technology Review

Discover special offers, top stories, upcoming events, and more.

Thank you for submitting your email!

Explore more newsletters

It looks like something went wrong.

We’re having trouble saving your preferences. Try refreshing this page and updating them one more time. If you continue to get this message, reach out to us at customer-service@technologyreview.com with a list of newsletters you’d like to receive.