Penn Medicine: The home of mRNA technology
Penn Medicine is home to the breakthrough messenger ribonucleic acid (mRNA) technology that enabled the highly successful COVID-19 vaccines from Moderna and Pfizer-BioNTech. Now the path is set for a whole new class of mRNA vaccines with the potential to eradicate countless other diseases, even cancer.
How do mRNA vaccines work?
Many vaccines use a weakened or dead version of the actual virus to stimulate an immune response against disease. Development of these vaccines can be lengthy and costly. Modifying them quickly if needed is difficult.
In contrast, mRNA vaccines use a genetic code to tell the body's cells to produce proteins that train the immune system. The result: “plug-and-play” vaccines with rapid development times and lower costs.
The future of mRNA vaccines for every imaginable infectious disease
The mRNA platform created at Penn Medicine ignited a global renaissance in RNA biology research. Scientists in industry and at universities worldwide are finding new and innovative ways to use mRNA technology to prevent and treat disease. At Penn Medicine, our researchers are using the mRNA platform to create vaccines for a wide variety of conditions.
mRNA vaccines for infectious diseases
Before COVID-19 erupted, a Penn-developed mRNA influenza (flu) vaccine was already in clinical trials. This existing work directly contributed to the speed at which drug makers could produce the mRNA COVID-19 vaccines.
With fast development and production times, mRNA vaccines are ideal for protection against new infectious diseases and variants of existing ones. Our researchers are at the forefront of mRNA vaccines for numerous infectious diseases, including the following.
Avian influenza is a disease caused by infection with avian (bird) influenza (flu) Type A viruses, which spreads among wild aquatic birds worldwide and can infect domestic poultry and other bird and animal species.
Penn Medicine researchers, led by Scott Hensley, PhD, and Drew Weissman, MD, PhD, have developed an experimental mRNA vaccine against avian influenza virus H5N1, and it is highly effective in preventing severe illness and death in preclinical models. The vaccine could potentially help manage the H5N1 virus in birds and cattle and prevent human infections.
While COVID-19 is the most recognizable disease caused by a coronavirus, it is not the only one. Penn scientists are working on an mRNA vaccine that provides the immune system with information to combat any coronavirus, including those that cause:
- Severe acute respiratory syndrome (SARS)
- Middle East respiratory syndrome (MERS)
- Future coronavirus-induced diseases
Scientists at Penn Medicine and Children’s Hospital of Philadelphia have created the first mRNA vaccine for Clostridioides difficile (C.diff), a difficult-to-treat and highly contagious infection. In early clinical trials conducted with small animals, the vaccine showed strong results—it stopped both new and repeat infections, cleared existing bacteria, and worked even when the immune system was weaker.
Herpes simplex virus 2 (HSV-2) is the most common sexually transmitted disease. Penn researchers are creating an mRNA vaccine for HSV-2 that instructs the body to:
- Block the herpes virus from entering cells
- Make sure that the virus doesn't turn off the immune system's normal protective functions
Hepatitis C is a liver infection that can become chronic and life-threatening. Penn scientists are developing an mRNA vaccine that tells the body to create antibodies that neutralize the hepatitis C virus (HCV) and prevent infection.
Human immunodeficiency virus (HIV) attacks the body's immune system and, if not treated, can lead to acquired immunodeficiency syndrome (AIDS). Penn researchers are working on an mRNA HIV vaccine that teaches the body to recognize and stop thousands of HIV variants before they cause harm.
Influenza (flu) viruses change constantly, so researchers must develop a new flu vaccine each year. Most seasonal flu vaccines are only 10 to 60 percent effective because it’s difficult to keep up with the many circulating viruses.
Penn Medicine researchers have developed a mRNA-based vaccine against all 20 known subtypes of influenza virus. If clinical trials are successful, this universal flu vaccine will give people a baseline level of immune memory against diverse flu strains and could protect against future flu pandemics.
Leptospirosis is a bacterial disease that humans can get from contact with urine or other bodily fluids (except saliva) from infected animals. Penn researchers are working on an mRNA vaccine to combat this disease, which is most common in southeast Asia.
Malaria passes to humans through bites from infected mosquitoes and causes high fevers, shaking chills, flu-like symptoms and anemia. The disease can be fatal and is a major health problem in the tropics and subtropics.
The current malaria vaccine is only 40 percent effective and must be given annually. Penn researchers are developing mRNA vaccines that are more robust and long-lasting with the goal of preventing transmission and disease.
Norovirus is a contagious viral condition that causes severe diarrhea, nausea, vomiting and stomach pain. The virus is particularly dangerous for young children, the elderly and immunocompromised people.
Scientists have had difficulty creating a norovirus vaccine using traditional methods. Penn researchers are now using mRNA technology to develop an effective, rapid-response vaccine.
Tuberculosis (TB) disease is an infectious airborne condition that usually affects the lungs but can also settle in the brain, kidneys or spine. If not treated, TB can be fatal.
Not everyone infected with TB develops TB disease right away or at all. This is called a latent TB infection. Penn researchers are working on an mRNA vaccine that prevents a latent TB infection from turning into TB disease.
mRNA vaccines for cancer
While the mRNA vaccines for COVID-19 and other infectious diseases prevent disease, mRNA technology can also help treat existing diseases like cancer. The platform's flexibility allows researchers to create mRNA cancer vaccines that activate the immune system to attack cancer cells.
mRNA treatment for food and environmental allergies and autoimmune conditions
Penn researchers are working on a vaccine to stop the body’s production of immunoglobulin E (IgE) antibodies that can cause severe attacks in people with peanut allergies. Additional efforts target dust mite allergies, as well as celiac disease and other autoimmune conditions.
mRNA and genetic diseases
Penn scientists are studying ways that mRNA could deliver replacement genes or repair defective genes to treat diseases.
An inherited (runs in families) genetic defect in the cystic fibrosis transmembrane conductance regulator (CFTR) causes cystic fibrosis, a life-threatening condition. Penn researchers are considering ways to treat cystic fibrosis by:
- Delivering mRNA into the lungs where it will instruct cells to create healthy CFTR
- Altering the DNA to fix the defected CFTR protein
Sickle cell anemia is an inherited condition in which red blood cells are deformed and stiff, resulting in numerous complications. Most advanced treatments are not possible in rural Africa and India, home to most of the 200,000 people born each year with the disease.
Penn researchers are developing a single-injection therapy that sends mRNA to bone marrow stem cells. The mRNA instructs the cells to repair the genetic defect, thereby curing the disease. If successful, this therapy could translate to other congenital (present from birth) gene defects in blood and stem cells.
mRNA for heart attack and stroke
Excess cholesterol is a leading risk factor for heart attack and stroke. Penn researchers are using mRNA technology to modify liver genes, thereby permanently reducing cholesterol levels and protecting against heart attack and stroke.
“We expect that the [vaccine’s] effect will be permanent with lifelong protection against heart disease.”
Expanding global access to mRNA vaccines
When countries can affordably and sustainably manufacture their own vaccines, global health is a greater possibility. We collaborate with world governments to establish Good Manufacturing Practices (GMP) sites and train local scientists to develop mRNA vaccines for local diseases.
Meet the Nobel Prize laureates behind mRNA science
Katalin Karikó, PhD, and Drew Weissman, MD, PhD, invented the messenger mRNA technology that serves as the foundation of the Pfizer-BioNTech and Moderna vaccines for COVID-19. The scientists received the world's most prestigious awards for their discovery science efforts, including the Nobel Prize in Medicine, Lasker-DeBakey Clinical Medical Research Award, the Breakthrough Prize and the Albany Medical Center Prize in Medicine and Biomedical Research.
Penn Institute for RNA Innovation
The Penn Institute for RNA Innovation brings together myriad scientists and vast expertise to accelerate breakthrough discoveries and develop the next generation of RNA therapies. Currently under construction, the institute is designed to harness the power of worldwide, interdisciplinary collaborations.
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