By Jutta Schulze
Dicerna Pharmaceuticals Inc (NASDAQ:DRNA) is a biopharmaceutical company that focuses on a technology called RNA interference (RNAi). This is a technique that makes use of the human genome sequence in order to target inappropriately activated genes responsible for certain diseases in patients, and deactivate those genes.
The GalXC Platform
Dicerna Pharmaceuticals Inc (NASDAQ:DRNA) uses their proprietary GalXC™ RNAi technology platform to develop new drugs that help silence genes responsible for a range of diseases with broad therapeutic potential. In simple words, the mechanism works by taking a part of the diseased gene sequence, putting it into a particular configuration, and reintroducing it into the body through a simple injection. The body’s own natural RNAi process then takes over and turns off the corresponding gene.
Since the method is bound to one specific gene sequence, it’s highly targeted and safe with long-term therapeutic advantages, as preclinical studies have shown.
For now, Dicerna has been focusing on the liver, since this is the tissue where many metabolic diseases originate. Ailments that involve a patient’s metabolism are mostly linked to genetic deficiencies, making specific gene targeting an effective approach
As a second focus, Dicerna has been working in the area of cardiovascular diseases, because many of the actors that cause problems in the heart – like cholesterol or triglycerides- are, again, metabolized in the liver as the primary site. Other areas the company is focusing on are infectious liver diseases such as Hepatitis B, and chronic liver illnesses.
Let’s take a quick look at Dicerna’s key programs.
One involves patients suffering from Type-1 Primary Hyperoxaluria (PH1), which inhibits the production of an enzyme in the liver. The result is an overproduction of oxalate, which ultimately accumulates and overwhelms the kidneys, causing them to fail.
Until now there has been no cure or effective treatment, with kidney and liver transplants being the only options for those affected by PH1. Though the number of people affected by this rare genetic defect is comparatively small, the costs for dual organ transplants is extremely high; consequently, the market for Dicerna Pharmaceuticals Inc (NASDAQ:DRNA) is huge.
Another program involves patients with high cholesterol. Around 35 million people are being treated for hypercholesterolemia with statins in the US alone. Thus, hypercholesterolemia is considered one of the biggest healthcare challenges across the country and in the world.
Instead of controlling their cholesterol by taking one pill every day, patients could get an injection every six months. This simultaneously gives them back quality of life and helps them control their cholesterol better.
Talking With Management
In a recent interview with Small Cap Nation’s Jane King, Douglas M. Fambrough, President and CEO of Dicerna, said he’s very encouraged by the recognition the company’s platform has gotten from the pharma industry. In line with this statement, the firm is in the process of forming important collaborations with pharma partners, which will allow it to take on bigger projects in the future.
Dicerna will roll out its clinical trials to treat patients with PH1 by the end of this year. Keep tuned in for updates.
Disclosures: The author, Jutta Schulze, holds no interest in any of the securities or entities mentioned above.