Messenger RNAs (mRNAs) work with DNA molecules to make our bodies function properly. They carry the genetic information stored in DNA, and translate them into proteins, which help control cell division and metabolism. As a result, therapies that harness mRNAs can help combat a range of diseases.
mRNA vaccines became hugely popular in the race to develop a COVID-19 vaccine. They work by using a piece of messenger RNA (mRNA), which instructs cells in the body to make a specific protein that is also present on the virus. When these proteins are made, the body’s immune system recognizes them as foreign and starts producing antibodies in response. This process effectively trains the immune system, preparing it to quickly recognize and fight the actual virus if it ever invades the body.
As mRNA technology evolves to prevent other infectious diseases, treat cancers and genetic disorders, more biotech companies have set foot into the field in recent times. Here are seven mRNA companies that have raised funds in recent years to push their candidates ahead through preclinical and clinical trials.
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Abogen Biosciences
Based in the city of Shanghai, Chinese biotech company Abogen Biosciences uses its mRNA platform to create synthetic mRNA molecules, which are delivered with the help of a lipid nanoparticle (LNP) system.
LNPs not only aid in the targeted delivery of the mRNA into specific cells but also protect the mRNA molecule from degradation. Through dynamic precision mixing technology, non-toxic, pH neutral LNPs are produced. This way, it allows for over a 90% encapsulation rate, which is the percentage of the drug that is successfully entrapped into a vector.
The focus of its pipeline is on cancer, infectious, and rare diseases. Its mRNA-based COVID-19 vaccine targeting SARS-2-COV-2 Omicron is in phase 3 clinical trials, whereas the one targeting the SARS-COV-2 wild-type strain was granted Emergency Use Authorization (EUA) in Indonesia in 2022. This clearance was based on trial results that showed that the vaccine Awcorna had higher levels of neutralization and IgG antibodies against wild-type, Delta, Omicron when compared to homologous boosters.
Its most advanced cancer therapy candidate is ABO2011, an mRNA-encoding human single-chain IL-12 protein to treat solid tumors. It is currently undergoing phase 1 trials.
Abogen’s series C financing round was deemed to be one of the largest private biotech funding rounds ever, according to a report by Fierce Biotech. The company had raised $700 million back in 2021 to fuel its COVID-19 vaccine through the clinic.
Anima Biotech
Known for its collaboration with pharma giants like Eli Lilly, Abbvie, and Takeda, Anima Biotech is a U.S.-based company that specializes in the development of small molecule mRNA drugs.
Its mRNA Lightning Platform combines artificial intelligence (AI) and mRNA biology to identify small molecules. The drug discovery approach involves screening for compounds that modulate underlying disease mechanisms. The network processes many RNA constructs in a number of cell types. From pinning down transcription sites and monitoring splicing variants within cells, all the way to tracking translation in cells, the company has created the world’s largest dataset of more than 2 billion mRNA biology images.
Anima’s most advanced drug is a preclinical, immunology candidate to treat lung fibrosis. It has demonstrated efficacy and safety in mouse models, where it reduced collagen production and fibrotic biomarkers in cells and tissue explants that were derived from patients with lung fibrosis. The clinical trial is a collaboration between Anima and Abbvie, which began last year when the latter offered $42 million to work with the biotech to discover immunology and cancer drug candidates.
CureVac
The list would be incomplete without the mention of CureVac. The German biotech company engineers mRNA to deliver messages to cells to produce disease-fighting proteins and antibodies.
CureVac has a range of candidates in its pipeline in different stages of development. The most advanced candidates are its influenza vaccines, CV0601 and CV0701, which are in phase 2. The trial is in collaboration with pharma giant GSK. Interim phase 2 results from last week revealed that the seasonal flu vaccine candidate boosted antibody titers at all dose levels and for all encoded seasonal influenza strains across younger and older adults, according to a press release. However, the candidate did struggle to meet comparator vaccine immune response levels for Influenza B.
In January, CureVac announced promising trial results where the two vaccines produced meaningful immune responses at lower doses than similar approved vaccines. However, adverse reactions were recorded in patients.
Last year, the biotech closed a $250 million public offering.
Ethris
Situated in Planegg in Germany, Ethris is devoted to using non-immunogenic messenger RNA (SNIM mRNA) to develop vaccines and therapies.
The company’s Stabilized Non-Immunogenic mRNA (SNIM® RNA) platform generates stabilized non-immunogenic mRNA. This mRNA is said to overcome the instability of mRNA because of chemical changes in their structures. This can be used to create and replace proteins that can tame diseases.
It also has an LNP platform that allows efficient transport of mRNAs via inhalation to the lungs as well as via intramuscular injection for vaccination. Ethris evades the challenges of maintaining the quality of LNPs with the help of its lipidoid formulation and optimized nebulization technology.
Ethris’ ETH42 is an inhaled drug currently being evaluated in preclinical trials to fight a rare, inherited disease called primary ciliary dyskinesia, that affects several organs in the body. Preclinical studies have been completed for ETH47, which addresses asthma exacerbations. The phase 1 study began in December 2023, and will comprise 88 participants. ETH47 can be administered by inhalation or via a nasal spray.
Gritstone bio
A self-amplifying mRNA (samRNA) molecule, like the name suggests, has the ability to self-replicate and produce more copies of itself. Gritstone leverages this mechanism to create vaccines for infectious diseases.
The American biotech’s vaccine platform helps enclose the immunogen into a vector, which is delivered to patients to induce T cell production, neutralize antibodies, and destroy target cells. Its samRNA vaccine for COVID-19 is currently in the clinic.
Interim results from a phase 1 trial explained that durable immunogenicity was achieved, and in patients who had previously been vaccinated, immunity was boosted for at least six months.
Its CORAL program SARS-CoV-2 vaccine platform delivers spike proteins – found on the surface of the virus – and other parts of the antigen in vectors that are made of the samRNA and chimpanzee adenovirus (ChAd). Its samRNA vector is based on a synthetic RNA molecule derived from a wild-type Venezuelan equine encephalitis virus (VEEV), and is delivered in an LNP formulation.
Earlier this month, the biotech raised $32.5 million as part of a public offering.
Apart from mRNA technology, this company is also invested in creating cancer and human immunodeficiency virus (HIV) vaccines.
Nutcracker Therapeutics
On a mission to crack its way into the clinic, American biotech Nutcracker Therapeutics specializes in cancer treatments. In its pipeline is lead candidate NTX-250, which is composed of three mRNA molecules. The candidate has been designed to induce a robust antitumor T-cell response as well as enhance immune cell activity and reduce immunosuppression. All three components have been shown to enhance long-term efficacy against human papillomavirus infection (HPV)-driven tumors in models.
Preclinical data showed that all the mice treated with NTX-250 saw complete regression of tumors, long-term survival, and robust immune cell infiltration, according to a press release in November 2022.
More recently, studies confirmed that two doses of NTX-250 got rid of the tumors and improved survival in mice. Its other candidate, NTX-471, which targets CD47 in tumors, showed cytotoxic activity comparable to other anti-CD47 currently in the clinic. Moreover, the candidate did not lead to anemia, which is regarded as a common side effect because red blood cells express CD47 as well.
The biotech bagged $167 million in a series C round in 2022, but, more than a year later, it laid off employees to focus on its preclinical programs.
Strand Therapeutics
American biotech company Strand Therapeutics programs mRNAs to target various kinds of proteins ranging from allosteric proteins, protein-protein complexes, and nucleic acid protein complexes to membrane proteins.
The mRNA constructs combine genes for self-replication derived from RNA viruses that are genetically programmed. Once they enter target cells, they translate the therapeutic protein they are encoding to treat the disease in the specific cells. And if they go into the wrong cells, they are degraded by those cells.
Committed to creating mRNA-based drugs, the company’s most advanced drug targets IL-12, and is used to treat melanoma, triple-negative breast cancer (TNBC), and other solid tumors. It recently obtained an Investigational New Drug (IND) clearance to embark on its clinical journey. The company was awarded $97 million in a series A round participated by pharma giant Eli Lilly back in November 2022.
Big pharma’s role in advancing mRNA technology
The global mRNA therapeutics market size was $39.9 billion in 2021 and is expected to only grow, according to a report by Grand View Research.
The pandemic led to the advancement of mRNA vaccines in the clinic and then into hospitals. Moderna’s SpikeVax and Pfizer and BioNTech’s Comirnaty were the first COVID-19 vaccines to hit the market. Besides, Sanofi has over 10 mRNA vaccines and therapies in its pipeline. And, GSK has also followed suit with its flu and COVID-19 vaccines – among others – both of which are in phase 2 in the clinic. Furthermore, AstraZeneca’s interest in the field has led to the progress of Beyfortus, a respiratory syncytial virus (RSV) vaccine, into phase 3 studies.