List of mRNA Therapies Companies in United States - 23

AGC Biologics offers deep industry expertise and uniquely customized services for the scale-up and cGMP manufacture of protein-based therapeutics (using mammalian and microbial systems), mRNA, pDNA, viral vector and cell therapy products, from pre-clinical to commercial production, including our proprietary CHEF1TM Expression System for efficient protein production with CHO cells. Our company DNA drives us to provide innovative solutions to partner with our customers in helping them reach their goals and accelerate their projects at our cGMP-compliant facilities in the US, Europe and Japan, as well as to facilitate approval and manage spending during the product lifecycle. We forge exceptionally strong partnerships with our customers and we never lose sight of our commitment to deliver reliable and compliant drug substance. Visit www.agcbio.com to learn more.

Ainos Inc. is a diversified healthcare company focused on the development of novel point-of-care testing (POCT), low-dose VELDONA interferon therapeutics, women's health, pneumonia, Ainos Pen, AI Nose, and other medical technologies. The company was founded in 1984 and is engaged in developing innovative medical technologies for point-of-care testing and safe and effective treatments for various health conditions.

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus developed Kostaive®, the first self-amplifying messenger RNA (sa-mRNA) COVID vaccine in the world to be approved. Arcturus has an ongoing global collaboration for innovative mRNA vaccines with CSL Seqirus, and a joint venture in Japan, ARCALIS, focused on the manufacture of mRNA vaccines and therapeutics. Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (over 400 patents and patent applications in the U.S., Europe, Japan, China, and other countries).

AUM LifeTech is an American Biotechnology company focused on using next-generation RNA silencing technology to develop solutions in diverse life science verticals including biomedical research, medicine, agriculture, and aquaculture. Specifically, AUM's custom products include next generation of innovative genetic tools in the area of gene silencing and manipulation for biomedical research and therapeutic development. AUM’s non-GMO products are also being developed for broad-spectrum target-specific pest management and disease control in agriculture. RNA silencing products using FANA technology provide superior alternatives to conventional RNAi and gene editing approaches like CRISPR and provide a fast track target discovery. AUM's goal is to provide innovation at the genetic level for a better life.

Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference (RNAi) with gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. Benitec is using its technology, called DNA-directed RNA interference (ddRNAi) to develop a pipeline of product candidates for the treatment of several chronic and life-threatening human diseases , including oculopharyngeal muscular dystrophy (OPMD) and hepatitis B. By combining the specificity and gene silencing effect of RNAi with gene therapy, ddRNAi has the potential to produce long-lasting silencing of disease-causing genes from a single administration, which could minimize the requirement for patients to take regular doses of medicine. Benitec’s objective is to become the leader in discovering, developing, clinically validating and commercializing ddRNAi-based therapeutics for a range of human diseases with high unmet clinical need or large patient populations and, as a result, provide a better life for patients with these diseases.

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for people with cystic fibrosis, a rare, genetic disease in which a defective gene causes a thick buildup of mucus in the lungs, pancreas, and other organs. The buildup of mucus can lead to extensive lung damage, respiratory failure, malnutrition, liver disease, and gastrointestinal issues, among many other complications. Recognized globally, the Cystic Fibrosis Foundation has led the way in the fight against cystic fibrosis, fueling extraordinary medical and scientific progress. Working alongside the CF community, the CF Foundation has fostered the development of more than a dozen CF treatments — an unprecedented number in a short span of time — and helped add decades of life for people with CF. Thanks to this work, the life expectancy of someone born with CF has doubled in the last 30 years. We are driven by a dream that one day every person with cystic fibrosis will have the chance to live a long, healthy life. Our vision is a cure for every person with cystic fibrosis and a life free from the burden of this disease. While our headquarters are in Bethesda, Maryland, we have additional offices in more than 60 locations across the country with positions in fundraising, marketing, digital, information technology, legal, finance and more – all supporting our mission to cure cystic fibrosis. At the Foundation, we are committed to creating an environment that is free from discrimination and provides a rewarding experience for all members of our team. We strive to be an organization where everyone is welcomed and where talented individuals from all backgrounds have the opportunity to thrive. The CF Foundation is a nonprofit, donor-supported organization and an accredited charity of the Better Business Bureau's Wise Giving Alliance. Please visit us at www.cff.org.

ExcepGen is a venture-backed, early stage biotechnology company located in San Francisco. The company is focused on solving fundamental underlying problems in drug discovery and applying those solutions to create a new generation of therapeutics.

GenVivo will make first- and best-in-class immune therapies that are immediately and easily deployable, highly effective, and which improve survival and quality of life beyond current therapies.

Kerna Labs is unlocking the full potential of mRNA as the universal toolkit for genetic medicine.

Kernal creates mRNA therapies to cure severe diseases including cancer. Our mRNA therapies instruct specific cells on how to make their own drugs. With roots at MIT, Harvard and Big Pharma, our team previously built a successful Biotech company and has deep expertise in mRNA space.

Landmark Bio is a collective endeavor launched by leaders from academia, the life sciences industry, and world-renowned research hospitals to accelerate development and industrialization of next-generation genomic medicine. Inspired by recent advancements in cell and gene therapy, Landmark Bio was established to remove barriers in drug development, create accessible capability, expertise, and solutions, and offer a collaboration platform to advance manufacturing technologies for the new generation of medicines to come. Founding partners include Harvard University, Massachusetts Institute of Technology (MIT), Cytiva, FUJIFILM Diosynth Biotechnologies (FDB), and Alexandria Real Estate Equities, Inc. Other collaborating institutions include Beth Israel Deaconess Medical Center, Boston Children’s Hospital, Mass General Brigham, and the Dana-Farber Cancer Institute. For more information please contact info@landmarkbio.com

Mammogen is a liquid biopsy company on a mission to unlock the quality-of-life preservation that exists at the earliest stages of diseases that affect women's health, starting with breast cancer.

At Moderna, we believe messenger RNA, or mRNA, is the “software of life.” Every cell in the body uses mRNA to provide real-time instructions to make the proteins necessary to drive all aspects of biology, including in human health and disease. Given its essential role, we believe mRNA could be used to create a new category of medicines with significant potential to improve the lives of patients. We are pioneering a new class of medicines made of messenger RNA, or mRNA. The potential implications of using mRNA as a drug are significant and far-reaching and could meaningfully improve how medicines are discovered, developed and manufactured.

Nutcracker Therapeutics is an RNA therapeutics company that has combined the power of advanced engineering with high-precision biosynthesis to deploy a complete RNA therapeutics platform. Armed with this high-tech advantage, we have developed a wholly owned pipeline of RNA therapeutic programs and established partnership initiatives with top clinical investigators at several leading institutions across the globe. With our platform’s ability to accelerate the development of life-changing RNA therapeutics, we seek to advance breakthrough RNA therapies at high velocity through all stages of development across a variety of indications. Our technology platform has the potential to significantly reduce costs and cycle times for RNA therapeutic development, with dramatic advantages in capacity scaling and point-of-care delivery over other RNA manufacturing approaches.

Omega Therapeutics, Inc. operates as a development-stage biopharmaceutical company. Its OMEGA Epigenomic Programming platform is designed to coopt nature’s operating system by harnessing the power of epigenetics, the mechanism for gene control and cell differentiation. The company is developing omega epigenomic controller (OEC) candidates to up-regulate the expression of HNF4a, a transcriptional master regulator as a potential way to restore liver-cell function in patients suffering from chronic liver diseases; to control the expression of genes that have been strongly linked to cell-growth inhibition in patients with diabetes and other conditions to restore the capacity for corneal regeneration; to down-regulate expression of the CXCL1, 2, 3, and IL-8 gene cluster; to control expression of genes implicated in patients with idiopathic pulmonary fibrosis to halt or reverse disease progression and improve disease outcomes; to down-regulate the expression of SFRP1, a protein that inhibits hair growth; and to treat non-small cell lung cancer and small cell lung cancer. It is also developing OTX-2002 to down-regulate c-Myc, an oncogene. The company was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Panorama Medicine is a VC-backed startup founded by a multidisciplinary team of world-renowned computational and experimental RNA biologists. Panorama has created PAN, a drug discovery platform that integrates genomics, big data, and computing to develop therapeutic interventions for RNA defects in disease. Research by Panorama and other companies has demonstrated that aberrant RNA splicing underlies a significant number of human diseases. Building industry leading algorithms and vast amount of public and proprietary datasets, Panorama has built the PAN platform to predict splicing events as well as design and screen modalities to address the underlying causes. We look forward to collaboration with the global drug development community to advance medicine and contribute to human knowledge of biology.

ParcelBio is developing a programmable technology to safely and specifically deliver mRNA to diverse cell types in the body. Our proprietary (nanoparticle-free) technology, which we call ‘STAmP’, stabilizes the mRNA and utilizes an interchangeable, lock-and-key-like targeting mechanism which enables fully customizable mRNA medicines.

Radar Therapeutics is a biotech company developing programmable precision therapeutics using molecular RNA sensors. The company has raised $13.4 million in seed funding to support its work in developing smart, programmable medicines. Radar Therapeutics is also the winner of the first-ever Amgen Bakar Labs Diversity, Inclusion, and Belonging Award. The company is building a business around medicines that use RNA sensors—mRNAs that gate their expression based on other RNAs in the cell.

Regulus Therapeutics Inc. (Nasdaq:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is located in San Diego, California and is led by a seasoned executive team experienced in corporate management, business, science, drug discovery and development. Regulus'​ scientific advisory board consists of world-class scientists and some of the foremost authorities in the field of microRNA research.

Repligen is a bioprocessing-focused life sciences company bringing over 40 years of expertise and innovation to our customers. We are inspiring advances in bioprocessing through the development and commercialization of high-value products and flexible solutions that address critical steps in the production of biologic drugs, principally monoclonal antibodies.

At RNAimmune, we use mRNA technology to help people live full and healthy lives. Our primary focus is development of vaccines against cancer and respiratory viruses. Our expertise in design and production of mRNA vaccines is supported by research and development efforts toward novel lipids, targeted delivery and antigen engineering. We collaborate with the world for the health of everyone.

Superfluid Dx is a biotechnology company focused on developing accurate diagnostics for neuro-degenerative diseases by harnessing the power of cell-free mRNA. They are also working on novel blood-based early detection methods.