Dicerna Pharmaceuticals (NASDAQ:DRNA) is a company whose primary focus is to discover and treat rare hereditary diseases. The main focus of the biopharmaceutical company is diseases involving liver and cancers, as these diseases are defined genetically. Dicerna makes use of an RNA interference technology, patented by Dicerna itself. RNAi is a process in which special double-stranded RNA molecules induce destruction of mRNAs of disease-causing genes via enzymes. The foundation of the company’s discovery approach is the double-stranded RNA molecules that can potentially optimize the RNAi potency, because they are substrates to the enzyme Dicer. The RNAi molecules are proprietary.
Dicerna Pharmaceuticals Inc. (NASDAQ:DRNA) is currently developing a drug called DCR-PH1for the treatment of Primary Hyperoxaluria 1 also called PH1. This is a rare, inherited, autosomal, recessive disorder. It affects the metabolism of the liver cells which consequently causes irreparable harm to the patient’s kidneys. Dicerna is also in the process of developing DCR-MYC for cancers related to MYC.
Dicerna Pharmaceuticals Inc. (NASDAQ:DRNA) received over 19% positive data of preclinical tests on DCR-PH1. As a result of this study, it was concluded that the gene HAO1 was potentially restricted for long-term. (HAO1 is the gene which takes part in the development of the disease PH1). The study has revealed remarkable results; about 97% of the liver disease was reduced after a single dose of the drug.
Needless to say, this news marks history; it is an important step in the development of a cure for Primary Hyperoxaluria type 1. As of today, patients who are suffering at the hands of PH1 have found no cure for their pains. The patients suffering from this disease succumb to the pain and the side-effects that come with it. The positive results shown by tests on DCR-PH1 give hope for such patients. After the successful preclinical trials, the company will now move a step ahead to perform clinical trials on the drug.
The root cause of the disease PH1 is in the liver. Dysfunction of the enzymes of the liver is caused by the defective genes in the liver cells. This leads to the liver producing excessive amount of oxalate. This high concentration of oxalate, in patients of Primary Hyperoxaluria type 1, causes the formation of calcium oxalate, which is in crystalline form that eventually develops into kidney stones. The protein, HAO1 produces oxalate. The drug DCR-PH1 targets HAO1, as mentioned above, to inhibit the production of oxalate.
Pankaj Bhargava, M.D., the chief medical officer of Dicerna Pharmaceuticals (NASDAQ:DRNA) claims that after seeing the promising results of the preclinical trials, the company is eager to perform clinical trials on human patients so that they can validate the positive effects of the drug DCR-PH1.
Because of the positive results of the preclinical trials, the stocks of Dicerna Pharmaceuticals Inc. (NASDAQ:DRNA) have soared in the market. But that is just for today. Over all on the year-to-date base, the stocks have decreased by over 50%. Let us see what progress the new findings bring for the company.