RNAi companies represent a specialized sector within the biotechnology industry, focusing on leveraging a natural biological process called RNA interference for therapeutic purposes. These companies develop a novel class of medicines that operate by silencing specific genes that cause or contribute to various diseases. This approach targets the root cause of illnesses at the genetic level, rather than merely addressing symptoms. It allows for highly precise and effective treatments for conditions with limited or no therapeutic options.
What is RNA Interference?
RNA interference (RNAi) is a biological process that regulates gene expression. It functions as a gene-silencing mechanism. This process involves small RNA molecules that can intercept and destroy messenger RNA (mRNA) before it can deliver its instructions for protein building.
The mechanism begins when long double-stranded RNA (dsRNA) is cut into smaller fragments, about 21-23 nucleotides long, by an enzyme called Dicer. These small fragments, known as small interfering RNA (siRNA), then bind to proteins, forming a complex called the RNA-induced silencing complex (RISC). Within this complex, one strand of the siRNA is removed, and the remaining guide strand directs the RISC to a complementary mRNA sequence. The RISC then either cleaves and destroys the mRNA or prevents its translation into protein, thereby silencing the gene.
The Mission of RNAi Companies
The objective of RNAi companies is to translate the scientific understanding of RNA interference into new therapeutic products. These companies conduct drug discovery, research, and development to create novel drugs that target disease-causing genes. They develop medicines that precisely “silence” the production of specific proteins linked to disease, addressing the underlying genetic causes of various conditions.
Their work involves advancing RNAi therapeutics through research and collaborations. They focus on overcoming challenges such as efficiently delivering RNA molecules into target cells and ensuring the stability and specificity of these molecules within the body. The goal is to bring therapies to patients worldwide, improving outcomes and quality of life for individuals with serious diseases.
Diseases Targeted by RNAi Therapies
RNAi therapies target a range of diseases by addressing their genetic origins. In genetic disorders, RNAi can reduce or eliminate the production of harmful proteins. For instance, patisiran, an RNAi therapeutic, is approved for treating hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with polyneuropathy by silencing the gene that codes for the transthyretin (TTR) protein, which can misfold and cause disease.
RNAi is used for infectious diseases by targeting viral genes to prevent replication. For example, RNAi candidates reduce viral antigens in chronic hepatitis B (HBV) by targeting viral mRNA. RNAi therapies are also used for cardiovascular diseases, such as hypercholesterolemia and hypertension, by silencing genes like PCSK9 or those involved in lipoprotein(a) production to lower cholesterol levels. This approach precisely intervenes in disease pathways where specific gene expression is problematic.
Leading Companies in the RNAi Field
Several companies develop RNAi therapeutics. Alnylam Pharmaceuticals, founded in 2002, was the first company to bring RNAi-based medicines to market. Their approved therapies include Onpattro (patisiran) for hATTR amyloidosis, Givlaari (givosiran) for acute hepatic porphyria, Oxlumo (lumasiran) for primary hyperoxaluria type 1, and Amvuttra (vutrisiran) for hATTR amyloidosis with polyneuropathy. Alnylam continues to expand its pipeline, focusing on rare genetic, cardiometabolic, and central nervous system diseases.
Other companies are also advancing RNAi technology. Silence Therapeutics, for example, is developing siRNA therapies for cardiovascular diseases, hematology, and rare diseases, utilizing its mRNAi GOLD platform. Novo Nordisk acquired Dicerna Pharmaceuticals, integrating Dicerna’s RNAi platform to accelerate the development of precision medicines for chronic conditions like diabetes, obesity, and cardiovascular disease. Arrowhead Pharmaceuticals also develops candidates like ARO-AAT for alpha-1 antitrypsin deficiency and ARO-HBV for chronic hepatitis B, demonstrating the expanding reach of RNAi therapeutics.