RNA medicines are already changing the way diseases are prevented and treated with more than two dozen RNA-based therapies approved for patients. These include approved RNA-based medicines for patients living with rare genetic disorders, cardiovascular disease, and ALS, as well as approved vaccines to protect people from RSV and seasonal flu.
Among these advances, messenger RNA (mRNA) medicines provide incredible hope and promise for patients. Whether instructing cells to produce proteins, activating immune responses, or delivering gene-editing tools, mRNA-based medicines offer researchers a dynamic platform for developing new treatments across a wide range of diseases, including cancer, rare genetic disorders, infectious diseases, cardiovascular disease, autoimmune conditions, and more.
Although mRNA-based medicines rely on the same biological process found in every human body, different medicines are designed to accomplish different goals.
Designed specifically for an individual patient, these treatments train the immune system to recognize the unique features of a person’s tumor and help the body identify and attack cancer cells while sparing healthy cells.
Designed to be used in many patients, these treatments stimulate or direct the immune system to recognize and respond to cancer.
Stimulates an immune response within a tumor to recognize and attack cancer where it is located.
Help guide the body’s own T cells to cancer cells, improving the body’s natural ability to recognize and destroy cancer.
Instructs cells to temporarily produce a functional copy of a protein that is missing or not working properly.
Delivers mRNA directly to T cells in the body, temporarily instructing them to produce a chimeric antigen receptor (CAR) that enables T cells to attack specific cancer cells.
Delivers gene-editing tools into cells to enable targeted changes to DNA, with the goal of correcting disease-causing genetic changes. Base editing is one subtype of gene editing, enabling a change to a single DNA building block without cutting the DNA strand.
Added during the manufacturing process to improve how therapeutic cells recognize disease or enhance their ability to perform their intended function.
Prepare the immune system to protect against exposure to a disease-causing virus or other pathogen.
Administered to patients after a disease has developed.
The flexibility of mRNA technology allows researchers to tailor different therapeutic approaches to different patients with different diseases.
Cancer immunotherapy makes up the largest share of the clinical pipeline, with more than two-dozen mRNA cancer vaccine trials in development. Most are in the earlyphase of development, with cancer immunotherapy showing the highest progression to later-stage trials.
The fastest-growing share of the mRNA pipeline, with researchers developing new treatments that supply missing proteins for inherited disease.
Combines mRNA, lipid nanoparticles, and gene editing to create personalized treatments for patients with ultra-rare genetic diseases.
With its unprecedented speed and scalability, mRNA technology is enabling the development of medicines to protect against infectious diseases, including influenza, norovirus, HIV, and other threats to public health and national security.
Each marker indicates an area of research activity and does not represent the total number of therapies in development.
Before an investigational medicine can be independently reviewed by the U.S. Food and Drug Administration for approval, it typically progresses through years of laboratory research and multiple phases of clinical testing designed to meet the gold standard of evidence for safety, effectiveness, and quality.
Active clinical trials in the U.S. investigating RNA medicines
Number of states research is taking place in. 90% of these are sponsored by companies across biopharmaceutical and biotech industries
mRNA treatment programs in development across the U.S., including potential treatments for cancer, rare diseases, autoimmune conditions and more
Laboratory and animal studies to show how an investigational therapy works and whether it appears safe to study in people.
First human studies focused on safety, determining an appropriate dose, and understanding how the therapy responds in the body.
Evaluating safety while studying whether the investigational therapy shows signs of benefiting patients with a particular disease.
Larger clinical trials compare investigational therapies with existing standards of care to better understand their safety and effectiveness.
Cancer Immunotherapies
Protein Replacement
Gene & Base Editing
Cell Therapies
Intratumoral & Immune Modulation
| Clinical Stage | Respiratory | Herpesviruses | Other Viral | Bacterial |
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RNA medicine research is advancing around the world, with clinical trials underway across countries on multiple continents. Countries are competing to advance RNA research and development and establish leadership in this rapidly evolving field.
Active, non-COVID RNA trials by country of trial site — ClinicalTrials.gov, as of August 2026. Top 21 countries by trial count, United States included.
A trial with sites in several countries is counted once in EVERY country it appears in — these bars must not be summed to a global total.
* China is a FLOOR, not a count. Chinese domestic trials register in ChiCTR, not ClinicalTrials.gov, so the true figure is higher by an unknown margin; a complete picture needs WHO ICTRP or ChiCTR, which this pull does not cover. The same bias understates Japan (jRCT), South Korea and others more weakly.
RNA research is driving scientific discovery and medical innovation across the United States. From clinical trials to NIH-funded research, this innovative ecosystem is advancing the next generation of mRNA medicines while supporting patients, researchers, and communities across the country.
There are more than 2,200 active clinical trial sites advancing promising new RNA-based medicines across all 50 states and in more than 80% of U.S. congressional districts, expanding opportunities for patients to participate in cutting-edge medical research. Of all active clinical trial sites, two out of three (64%) belong to Phase 3 studies.
States with the highest concentration of RNA research include California, Texas, New York and Florida – showing that RNA crosses both state lines and party lines.
Protecting this nationwide research ecosystem will help ensure the United States remains at the forefront of medical innovation, is best prepared to address future public health and national security challenges and delivers the breakthrough RNA medicines patients need.
