mRNA, or messenger ribonucleic acid, is a macromolecule within cells that works to synthesize protein from genes encoded in an organism’s DNA. mRNA is transcribed from DNA within a cell’s nucleus.
Our DNA, or genetic code, contains all the genes we will express in our lifetime. Within these genes are instructions that code for different proteins. Our cell’s everyday function is dependent on the availability of specific proteins.
mRNA is the “middleman” between our cell’s DNA and proteins. “Messenger RNA ” earns its name as it carries DNA’s genetic code instructions to make a specific protein. The use of mRNA in drug therapeutics have had a revolutionary effect on treating various diseases. mRNA therapies are versatile and programmable in nature and recognizable to the body through its natural synthesis of mRNA.
The Function of mRNA in Body Cells
Once DNA transcribes into mRNA, the mRNA travels to the cytoplasm and attaches to a ribosome, where protein synthesis takes place.
Proteins are responsible for nearly every task within cells.
When mRNA is transcribed from DNA, our genetic code can then be processed into proteins that can carry out the DNA’s instructions for gene expression.
These proteins can provide structural support for our cells and orchestrate their everyday functions.
How Does mRNA Therapy Work?
mRNA-based therapy involves delivering sequences of mRNA into the body. This is achieved by encapsulating the mRNA into a delivery vehicle, such as liposomes or nanoparticles. An example of mRNA therapy is the COVID-19 vaccine, which utilizes lipid nanoparticles (LNPs).
The LNPs are loaded with mRNA sequences that synthesize COVID-19 spike proteins found on the virus’s surface. When the LNPs bind to and enter cells, the mRNA is released. The body then reads these mRNA sequences and synthesizes the COVID-19 spike protein. The immune systems response to the spike protein result in antibody production that helps prevent the virus from attacking healthy cells
Why is mRNA Therapy a Good Alternative to Current Treatments?
mRNA therapy has shown to be a versatile treatment method for a range of diseases due to its programmable nature.
mRNA therapies can utilize targeted drug delivery to reach specific cells within the body while having little to no effect on surrounding areas.
Depending on the mRNA therapy’s design, various proteins can be coded for by a given sequence.
Additionally, mRNA is an incredibly stable molecule allowing easy storage and transportation. Its use also poses limited side effects since it is a naturally occurring molecule produced within the human body.
Nevertheless, mRNA therapies are breakthrough treatment methods. Much development and research are still needed to make these therapies widely available and ensure their efficacy.
mRNA Treatment Design
1. Insertion of mRNA into DNA Plasmid
The protein of interest must be identified and isolated when designing mRNA therapeutics. Once this occurs, the protein coding sequence can be inserted into an artificial DNA segment, such as a plasmid.
2. Transcription of DNA into mRNA
This DNA segment is then transcribed into mRNA. This mRNA will, which will, in turn, code for the protein of interest.
3. Loading mRNA into Delivery Vehicles and Binding and Entry into Target Cells
Once the mRNA segment is isolated, it can be loaded into a delivery vehicle such as liposomes or lipid-based nanoparticles. It is crucial to maintain the stability of the mRNA while it is enveloped within the delivery vehicle to ensure successful delivery to target cells.
4. Protein Synthesis
The mRNA is released when the delivery vehicles bind to and enter cells. Once in the body, the ribosomes of the target cells read the mRNA, and the synthesis of the protein of interest begins.
Types of mRNA Therapies
Due to mRNA’s versatile and stable nature, a range of mRNA therapies exist to treat diseases. Treatments include vaccinations and therapies that target protein availability and immune responses.
mRNA vaccines have found great success in the prevention of COVID-19. Injecting these vaccines into human muscle tissues enables nearby cells to uptake mRNA that codes for the COVID-19 spike protein. The cells then use the mRNA to synthesize antibodies that protect against COVID-19 antigens.
mRNA Protein Replacement Therapy
mRNA protein replacement therapy works to synthesize missing or defective proteins within a patient’s cells. Patients suffering from diseases that result from protein deficiencies can benefit from these treatments to restore proper cell function and alleviate the side effects of protein-deficient disorders.
mRNA Immunotherapy and Implications for Cancer Treatment
mRNA immunotherapy strengthens the body’s immune response by introducing mRNA that codes for disease-fighting proteins. This form of therapy has the largest implications for cancer treatments, as mRNA that synthesizes antibodies that recognize tumor-specific proteins can bind to and attack cancerous cells.
Despite RNA immunotherapy being in the early stages of development, it is a hopeful treatment for cancer and autoimmune diseases. It has seen greater growth since using mRNA in the novel COVID-19 vaccine.
At Phoreus Biotech, we have made great strides in developing our peptide nanocarrier technology. Our peptide technology can be engineered to effectively deliver mRNA-based cancer treatments to the body without harming healthy cells.
Due to their easily programmable nature, our peptide nanocarriers can be formulated to target tumor cells through mRNA loading that synthesizes proteins unique to cancer cells.
The Future of mRNA Therapies and FDA Approval
mRNA therapies are promising avenues for treating various diseases due to their versatile and programmable nature. Nevertheless, many mRNA technologies are still in early development, such as the use of mRNA in gene therapies.
The use of mRNA in the COVID-19 vaccine has seen success in preventing the disease and paved a successful future for mRNA therapies. Since the breakthrough of the COVID-19 vaccine, FDA has established fast tracks to making life-saving drugs readily available to the public, which only strengthens the future of mRNA therapies.
mRNA Treatment Drawbacks
mRNA therapies are breakthrough treatments still in early development. Specific limitations include:
- The cost of mRNA therapy development.
- Stability of the mRNA molecule.
- The stability of the mRNA carrier.
Cost of mRNA Therapy Development
Since mRNA therapies are in the early stages of development, high costs are associated with its technology. Nevertheless, mRNA therapies have gathered more funding for research due to the large success of their use in the novel COVID-19 vaccines. As mRNA sees more use in therapies, research, development, and production costs should also decrease.
Stability of mRNA Molecules
mRNA stability relies on a range of factors, such as temperature, cell conditions, and carrier stability. mRNA has to be kept at a low temperature to prevent it from denaturing.
Once mRNA has been loaded into its delivery carrier, it must remain stable and not lead to premature release or breakdown of the mRNA to synthesize the desired protein.
Cell conditions must also be optimal, including pH as well as avoiding interaction with enzymes that can degrade the mRNA before it can produce specific proteins.
Stability of Average mRNA Carriers
The most commonly used carriers for mRNA are lipid-based nanocarriers or LNPs. Unstable nanocarriers can have various negative consequences for mRNA therapy, including premature carrier breakdown and early release of the mRNA.
Nanocarriers made from lipid-based compounds have been shown to pose a greater risk of cell toxicity when compared to protein-based nanocarriers. Repeated doses of LNP based nanocarriers increase the possibility of triggering a negative immune response from the body.
Phoreus Biotech Uses Peptide Carriers for mRNA Therapy Delivery for Human and Veterinary Medicine
Our Branched Amphipathic Peptide Capsules (BAPC) and Corralling Amphipathic Peptide Colloids (CAPC) nanocarriers mitigate common risks posed by standard liposomal-based carriers for mRNA stability.
Our technology spans beyond human use and provides implications for the development of mRNA technology and its use in animal health.
Explore the potential of mRNA therapy with Phoreus Biotech. Experience our peptide nanocarrier manufacturing capabilities delivering and storing mRNA at room temperature while minimizing cytotoxicity. Tailor our peptide carrier methods to meet your unique pharmaceutical needs for mRNA therapy development and delivery.
Read our FAQs about Phoreus peptide technology for additional information about our revolutionary peptide carriers.