Guest Column | July 28, 2023

5 Promising Applications Of RNA Therapeutics

By Erika L. Roberts, ELR Lab Services LLC


Advancements in scientific research have paved the way for innovative therapeutic approaches, and RNA therapeutics stand at the forefront of this revolution. Ribonucleic acid (RNA), once considered a mere messenger molecule, has now emerged as a versatile tool in the development of novel therapies. In this article, we will delve into the world of RNA therapeutics, exploring their potential and five applications that could impact the future of medicine.

1. RNA Interference (RNAi)

RNA interference, or RNAi, is a groundbreaking technology that allows scientists to silence specific genes responsible for diseases. By introducing small interfering RNA (siRNA) molecules into cells, RNAi can selectively target and degrade disease-causing mRNA. This approach has the potential to treat a broad range of diseases, including genetic disorders, viral infections, and various types of cancer.

The FDA has approved patisiran, a novel RNAi therapeutic for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This underscores the therapeutic potential of this technology. RNAi-based therapeutics continue to evolve, with ongoing efforts to enhance their delivery and target specificity. Recent advancements include the development of next-generation siRNAs with improved stability and efficacy. Researchers are also exploring novel delivery methods, such as exosome-based carriers and lipid nanoparticles, to overcome challenges associated with delivering RNAi molecules to target tissues. These advancements hold promise for treating a wide range of diseases, including neurodegenerative disorders, viral infections, and certain types of cancer.

2. Messenger RNA (mRNA) Therapeutics

mRNA therapeutics have gained significant attention in recent years, particularly due to their role in the development of COVID-19 vaccines. mRNA vaccines work by delivering a small piece of mRNA that encodes a viral protein into cells. This prompts the cells to produce viral protein, thereby triggering an immune response. Beyond vaccines, mRNA therapeutics hold immense promise in treating various diseases, including cancer, genetic disorders, and infectious diseases. Their ability to instruct cells to produce specific proteins opens a new realm of possibilities for personalized medicine. RNA-based therapeutics may have a significant impact in revolutionizing cancer treatment by enabling targeted precision medicine approaches. Researchers are using RNA technologies to develop therapies that selectively target cancer cells, leaving healthy cells unharmed. This includes the development of RNA-based nanoparticles, aptamers, and immune-stimulating molecules to deliver anti-cancer agents directly to tumor cells. Additionally, RNA-based immunotherapies are being explored to enhance the body's immune response against cancer cells. Currently, research is being conducted on a personalized mRNA vaccine against pancreatic cancer. These advancements have the potential to improve treatment outcomes and reduce side effects associated with conventional chemotherapy.

3. RNA-Based Gene Editing

Gene editing technologies, such as CRISPR-Cas9, have revolutionized the field of genetics. RNA molecules are crucial components in these systems, guiding the Cas9 enzyme to specific DNA sequences for modification. This technique allows for precise editing of the genome, offering potential treatments for genetic diseases. RNA-based gene editing holds promise in correcting disease-causing mutations and restoring normal cellular function. RNA editing technologies are gaining traction as a potential therapeutic approach for genetic disorders. Recent advancements in RNA editing tools, such as base editing and prime editing, enable precise modifications of RNA molecules. These techniques offer the potential to correct disease-causing mutations at the RNA level, providing a promising avenue for treating genetic diseases like cystic fibrosis, muscular dystrophy, and sickle cell anemia.

Ongoing research aims to refine these editing techniques and explore their therapeutic potential further. One current area of research is for human papilloma virus-driven cancers.

4. RNA Vaccines And Beyond

The success of mRNA vaccines against COVID-19 has shed light on the broader applications of RNA therapeutics. Beyond viral infections, RNA vaccines can be developed for a wide range of diseases, including cancer and autoimmune disorders. Furthermore, RNA therapeutics can be designed to deliver specific proteins, antibodies, or other therapeutic molecules to target cells, offering a more precise and tailored approach to treatment. The advent of mRNA vaccines has been a game-changer in the battle against infectious diseases. Researchers are now exploring the potential of mRNA vaccines for other infectious diseases, including influenza, Zika virus, and HIV. Currently, the FDA has just approved a vaccine for respiratory syncytial virus (RSV) in adults, children and infants. The ability to rapidly design and produce mRNA vaccines holds great promise for combating emerging infectious threats with agility and precision.

5. RNA-Based Diagnostics: Early Detection And Personalized Medicine

The use of RNA-based diagnostics is rapidly advancing, allowing for early disease detection and personalized treatment strategies. Liquid biopsies, which detect disease-specific RNA molecules in bodily fluids, hold promise for non-invasive and precise cancer detection. Additionally, RNA biomarkers are being identified for various diseases, enabling clinicians to tailor treatment plans based on individual patient profiles.

RNA sequencing (RNA-seq) allows for the detection of a wide variety of RNA species, including mRNA, non-coding RNA, pathogen RNA, chimeric gene fusions, transcript isoforms, and splice variants. It also provides the capability to quantify known, pre-defined RNA species and rare RNA transcript variants within a sample. In addition to differential expression and detection of novel transcripts, RNA-seq also supports the detection of mutations and germline variation for hundreds to thousands of expressed genetic variants, facilitating assessment of allele-specific expression of these variants. The integration of RNA-based diagnostics into clinical practice has the potential to revolutionize disease management and improve patient outcomes.

RNA therapeutics represent a new frontier in medicine, offering immense potential for treating a wide range of diseases. From gene silencing through RNAi to mRNA-based vaccines and gene editing, the power of RNA is transforming the landscape of therapeutics. As research and development in this field progress, we can anticipate more breakthroughs and a brighter future in healthcare. The potential of RNA therapeutics to revolutionize medicine is undeniable, providing hope for improved treatments, personalized medicine, and better patient outcomes.

About The Author:

Erika L. Roberts, MFS, is principal consultant and owner of ELR Lab Services LLC. Having more than 15 years of experience working in many different areas of the pharmaceutical/biotech manufacturing quality environments, she has particular expertise in sterility testing, microbial identification training, HPLC analysis, cGMP training, analytical chemistry, and pharmaceutical regulations. Roberts obtained a master’s in forensic science in 2006 with an emphasis in document examination.