Glossary of Terms

Glossary of Terms

A clinical trial is a research study that explores whether a medical strategy, investigational treatment, or device is safe and effective for humans.

A blinded experiment is an experiment in which information about the test is hidden from the participant, to reduce or eliminate bias, until after a trial outcome is known. Double-blind means that the investigator does not know the information either.

Genes are strings of DNA that serve as the functional and physical unit of heredity passed from parent to offspring. Most genes contain the information for making a specific protein. When mutated or inappropriately activated, certain genes can drive diseases, including cancer.

Informed consent is when the patient/caregiver/legal guardian is provided clear, easy-to-understand information about a clinical trial as he/she is deciding whether to participate in a clinical trial.

Nedosiran is an investigational therapy that Dicerna is developing for the treatment of primary hyperoxaluria (PH).

Phase 1 tests safety in humans by giving the treatment or drug to healthy volunteers.

Phase 2 tests safety and effectiveness in patients living with the disease being studied.

Phase 3 tests safety and confirms effectiveness in patients with the disease and studies a larger group of people than the Phase II trial.

Phase 4 tests the drug’s effect in various populations and any side effects associated with long-term use after it is approved.

PHYOX^TM is the name of Dicerna’s clinical development program for nedosiran, which includes all phases of development.

A pivotal trial is a clinical trial intended to provide evidence for a drug marketing approval, e.g. by the United States Food and Drug Administration.

Primary hyperoxaluria (PH) is a family of severe, ultra-rare, genetic liver disorders characterized by overproduction of oxalate, a natural chemical in the body that is normally eliminated as waste through the kidneys. In patients with PH, the kidneys are unable to eliminate the large amount of oxalate that is produced, and the accumulation of oxalate can result in severe damage to the kidneys and other organs.

A randomized controlled trial is a type of study that aims to reduce bias when testing a new treatment. The people participating in the trial are randomly allocated to either the group receiving the treatment under investigation or to a group receiving standard treatment as the control.

RNA, which stands for ribonucleic acid, is a genetic material with diverse uses within the cell. Most notably, when a gene is being used, a copy of its DNA sequence is made using RNA. This RNA copy is known as messenger RNA, or mRNA. The RNAi machinery targets mRNA.

RNAi is a biologic process in which certain double-stranded RNA molecules inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of those genes.

Subcutaneous means “under the skin.” So, a therapy that is delivered subcutaneously is delivered with a needle under the skin before it enters the bloodstream.