44 mRNA Therapies Companies - Worldwide
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Company Name | Location | About |
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aceRNA Technology | Kyoto, Japan | aceRNA Technologies Co., Ltd. is a biotech startup established in 2018 based on RNA design technology developed in synthetic RNA biology research by Prof. Hirohide Saito at Kyoto University. We are developing "smart gene therapy” (mRNA-based and virus-base) that enables control of therapeutic transgene expression in cell-type and -state specific manner. By that, we are committed to develop new therapeutic approach, such as in vivo cell programming. |
AGC Biologics | 22021 20th Avenue SE, Bothell, WA 98021, US | 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 | San Diego, California | 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. |
Altamira Therapeutics | Hamilton, Bermuda | Altamira Therapeutics (former Auris Medical) is dedicated to developing therapeutics that address important unmet medical needs. The Company is currently active in three areas: • the development of RNA therapeutics for extrahepatic therapeutic targets (OligoPhore™ / SemaPhore™ platforms; preclinical), • nasal sprays for protection against airborne viruses and allergens (Bentrio™; commercial) or the treatment of vertigo (AM-125; Phase 2) • the development of therapeutics for intratympanic treatment of tinnitus or hearing loss (Keyzilen® and Sonsuvi®, Phase 3). The Company was founded in 2003, under the name Auris Medical, and is headquartered in Hamilton, Bermuda with its main operations in Basel, Switzerland. The shares of Altamira Therapeutics Ltd. trade on the NASDAQ Capital Market under the symbol “CYTO.” |
Anemocyte Srl | Milan, Italy | |
Arcturus Therapeutics | San Diego, California, United States of America | 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). |
Art Bioscience | Rehovot, Israel | ART BioScience was founded in December 2015 by Mr. Oren Ozeri and Mr. Alon Hazan, fathers of two children diagnosed with Duchenne. After co-founding the Israeli Duchenne Parent Association and increased involvement in following current research, Alon and Oren swore to do everything possible to save the lives of their children and dedicated their time and resources to trying to find a cure for the disease. The result of their intensive research and involvement in the field led them to the decision to establish ART, a company based on novel messenger RNA (mRNA) technologies for the development of new therapeutics that can potentially change the course of the disease. We are developing Dystrophin modified mRNA encapsulated in LNP (Lipid Nanoparticles) proprietary delivery system as: 1.) Protein Replacement Therapy 2.) CRISPR Cas9 mRNA as Gene Editing Therapy |
Aum LifeTech | Philadelphia, Pennsylvania, United States | 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 Biopharma | Hayward, California, United States | 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. |
BIA Separations | Villach, Austria | Separation Technology |
Biomay | Vienna, Austria | Biomay is a GMP manufacturer for plasmid DNA, mRNA and recombinant proteins. We are both technology focused and quality oriented. Our clients appreciate Biomay’s flexibility and reliability. From gene construction to market supply: Biomay delivers for the success of its clients. |
BioNTech SE | Mainz, Germany | BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules. Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here: https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html |
Bound Therapeutics | Marlton, New Jersey, United States | Bound Therapeutics LLC creates next generation healthcare solutions by developing RNA-based drugs for various diseases, immersive haptic surgical simulation, and artificial intelligence automated medical imaging analysis. |
Cansino | Tianjin, China | CanSino Biologics Inc. (CanSinoBIO, SHSE: 688185, HKEX:06185) is an innovative biopharmaceutical company dedicated to exploring best solutions to the prevention of diseases through cutting-edge research & development, advanced manufacturing and commercialization of innovative vaccine products for human use worldwide. Since its establishment in Tianjin, China in 2009, CanSinoBIO has experienced tremendous growth with 17 vaccines preventing 12 diseases, including approved vaccines for Ebola virus disease (Ad5-EBOV), COVID-19 (Ad5-nCoV, trade name: Convidecia), and meningitis (MCV2, trade name: Menphecia). CanSinoBIO has been listed on the Main Board of Hong Kong Exchange and Clearing Limited (HKEx) in March 2019. A year later, CanSinoBIO has successfully listed on the Sci-Tech Innovation Board (STAR Market) of the Shanghai Stock Exchange, making it the first "A+H" dual listing vaccine company. CanSinoBIO is focusing on continually expanding manufacturing capacity for its current vaccine candidates and further enhancing the competitiveness and the scope of its portfolio by promoting the R&D of new vaccine candidates. Leveraging broad experiences of our accomplished team of scientists and business leaders who had previously held technical and senior management positions at many of the leading pharmaceutical companies in the world, including Sanofi Pasteur, Astra Zeneca, Wyeth (now Pfizer) and CNBG (China), CanSinoBIO has developed five key platform technologies, including viral vector-based technology, synthetic vaccine technology, protein structure design and recombinant technology, mRNA technology as well as formulation and delivery technology. |
CureVac | Tübingen, Baden-Württemberg, Germany | CureVac (Nasdaq: CVAC) is a pioneering multinational biotech company founded in 2000 to advance the field of messenger RNA (mRNA) technology for application in human medicine. In more than two decades of developing, optimizing, and manufacturing this versatile biological molecule for medical purposes, CureVac has introduced and refined key underlying technologies that were essential to the production of mRNA vaccines against COVID-19, and is currently laying the groundwork for application of mRNA in new therapeutic areas of major unmet need. CureVac is leveraging mRNA technology, combined with advanced omics and computational tools, to design and develop off-the-shelf and personalized cancer vaccine product candidates. It also develops programs in prophylactic vaccines and in treatments that enable the human body to produce its own therapeutic proteins. Headquartered in Tübingen, Germany, CureVac also operates sites in the Netherlands, Belgium, Switzerland, and the U.S. Further information can be found at www.curevac.com. |
Cystic Fibrosis Foundation | Bethesda, Maryland, US | 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. |
Cytiva | 100 Results Way, Marlborough, Massachusetts, US | Cytiva, now with the life sciences business from Pall Corporation, is a global biotechnology leader dedicated to helping customers discover and commercialize the next generation of therapeutics. We bring dedicated technical expertise and a broad portfolio of tools, technologies, and services that enable the development, manufacture, and delivery of transformative medicines to patients. We are part of Danaher, a global science and technology leader. Along with other Danaher businesses, we accelerate the impact of tomorrow's science and technology innovation to improve human health. Visit cytiva.com to learn more. |
eTheRNA | Niel, Belgium | Driving discovery and solving challenges for partners across LNP formulation and RNA chemistry | www.etherna.be | www.ethernamanufacturing.com We are an RNA technology company, with over 30 years’ experience, offering professional partnership to pharma and biotech with an emerging or established interest in RNA therapeutics. Our expert team supports start-up, early stage and late stage projects by using our proprietary platforms across RNA chemistry, LNP formulation and Process Technology, to drive new pipeline development or resolve manufacturing challenges for our partners. |
Ethris | Planegg | Ethris is paving a new path from genes to therapeutic proteins using our proprietary messenger RNA technology. Developed in-house, our integrated platform enables the discovery, design and development of transcript therapies that restore missing functions in patients’ cells and tissues. We will advance transcript therapies to transform the treatment of disease independently and with our partners. |
ExcepGen | San Francisco, CA | 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. |
InnoSIGN | Eindhoven, Netherlands | InnoSIGN commercializes OncoSIGNal mRNA-based tests that measure the activity of signal transduction pathways in cancer and immune cells to predict how patients will respond to targeted drugs. |
Kernal Biologics | Cambridge, Massachusetts, United States | 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 | Watertown, Massachusetts, United States | 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 | 6 liberty, aliso viejo, california, united states, 92656 | 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. |
Moderna | Cambridge, Massachusetts, United States | 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. |
NunaBio Limited | 32 gallowgate, newcastle upon tyne, tyne and wear, united kingdom | NunaBio is a privately-owned specialist DNA synthesis and development company. We are engaged in CRO (Contract Research Organizations) and CDMO (Contract Development and Manufacturing Organisation) activities. The company has built a solid reputation for its R&D flexibility and innovative approach to complex and challenging projects. NunaBio's core expertise is research and development into cell-free DNA synthesis, process optimisation, route design, scale-up and small-scale manufacture for clinical studies. |
Nutcracker | Emeryville, California, United States | Develops the Nutcracker.® A microfluidic-based Technology Platform uses proprietary hardware and software combined with innovations in mRNA biochemistry and nanoparticle delivery to produce optimized mRNA therapies on biochips. |
Omega Therapeutics | Cambridge, Massachusetts, United States of America | 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 | 3675 Market St, 7th Floor, Philadelphia, Pennsylvania 19104, US | 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. |
Pantherna Therapeutics | Hennigsdorf, Germany | Pantherna Therapeutics is a privately held mRNA therapeutics company developing novel first-in-class therapies for indications with high unmet medical need. Pantherna’s development programs are based on two innovative and proprietary PTX platform technologies, offering robust mRNA expression constructs (PTXmRNA) and lipid nanoparticle (LNP) delivery vehicles (PTX_LNP) for localized expression of therapeutic proteins. PAN004 is Pantherna´s first development candidate derived from this platform, representing a defined mRNA-LNP formulation for selective mRNA delivery and expression of a Tie2-agonist in the vascular endothelium of the lung. Pantherna strives for expanding its pipeline with various novel mRNA-LNP candidates enabling the therapeutic use of mRNA beyond the vascular endothelium in distinct tissues such as skeletal muscle or immune cells. |
ParcelBio | 135 Mississippi Street, San Francisco, CA 94107 | 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. |
PURMX | 1-2-3 Kasumi, Minami-ku, Hiroshima, Hiroshima 734-8553, Japan | Creation of optimal nucleic acid medicine for disease treatment with proprietary technology PURMX's solution is a drug discovery seed of small RNA nucleic acid drugs that fine-tune many target genes and deliver unprecedented therapeutic effects. PURMX already possesses a large amount of functional RNA screening data, which is being further expanded. The first stage of innovative nucleic acid medicine seeds is MIRX002. MIRX002 is a nucleic acid medicine for malignant pleural mesothelioma. We have completed non-clinical trials and are conducting investigator-initiated clinical trials at Hiroshima University Hospital starting in 2021. |
Radar Therapeutics | Berkeley, California, United States | 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 | San Diego, California, United States | 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 Corporation | Waltham, Massachusetts, United States of America | 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. |
Ribopro | Kloosterstraat 9 5349 AB Oss | RIBOPRO is an innovative service provider, producing high-quality, high-performance RNA products, which are used as powerful research tools and therapeutics. RiboPro offers both off-the-shelf and custom RNA products at competitive rates and turn-around times, effectively removing all barriers to successful use of RNA in the research and development of our customers. We have more than 2 years experience satisfying the RNA-related needs of our (returning!) customers and thus we are confident that we can help you get your next project off to a good start. Let us worry about the quality and performance of your RNA, so you can focus on your next success. |
RNAimmune | Gaithersburg, Maryland, United States | 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. |
RVAC Medicines | Headquartered in Singapore w/ sites in Boston and Shangai | A mRNA platform company with a pipeline of innovative products that covers a range of disease areas. |
Sartorius BIA Separations | Ajdovščina, SI | We develop and manufactures market-leading CIM® monolithic chromatographic columns for the purification and analysis of large biomolecules, such as viruses, pDNA, and mRNA, which are applied in vaccines and gene therapies. Our Cornerstone® Biomanufacturing Development Services offer a comprehensive approach of integrated process development solutions and technology designed to improve the robustness and yield of large biomolecules production while improving the safety of therapeutic products. |
Sirnaomics | Gaithersburg, Maryland, United States | Sirnaomics is an RNA therapeutics biopharmaceutical company that focuses on the discovery and development of innovative drugs for indications with unmet medical needs and large market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics company to have a strong presence in both Asia and the United States. Based on its proprietary delivery technologies, a polypeptide nanoparticle RNAi platform and GalNAc RNAi platform, GalAhead™, Sirnaomics has established an enriched drug candidate pipeline. STP122G, which represents the first drug candidate utilizing the Group's GalAhead™ mxRNA technology, is currently in Phase I development. STP125G is the second siRNA therapeutics based on Sirnaomics proprietary GalAhead™ mxRNA technology, targeting ApoC3 mRNA for cardiovascular disease treatment. STP237G is the first dual-targeted drug based on a GalAhead™ muRNA technology and is in the late stage of preclinical evaluation. The Group has also had multiple successes with oncology applications through its clinical programs for STP705 and STP707. With the expansion of the Group's clinical pipeline and establishment of the Group's manufacturing facility, Sirnaomics focuses on a transition from a biotech company to a biopharma corporation. Learn more at: www.sirnaomics.com. |
Superfluid Dx | 259 East Grand Avenue, South San Francisco, CA 94080, United States | 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. |
Ultragenyx Pharmaceutical Inc. | Allschwil Basel-Country, Switzerland | Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community |
VaxEquity | Cambridge, United Kingdom | VaxEquity is a biotechnology company amplifying the impact of RNA vaccines and therapeutics, through using its next generation self-amplifying RNA (saRNA) platform. The Company uses saRNA to provide greatly enhanced protein expression, combining better tolerability with slower release and thermostability. VaxEquity is pioneering a next generation approach by modifying its saRNA to modulate the immune response and thereby overcoming current limitations. The flexibility of the Company’s platform ensures that its broad pipeline of assets can be designed, developed, and manufactured rapidly and at scale. VaxEquity was founded in 2020 by Professor Robin Shattock, the Head of Immunology of Infection at Imperial College London and is led by a world class team who are pioneers in the field of RNA. |
Versameb | Basel, Switzerland | Versameb AG is a privately held biotechnology company focusing on discovering and developing innovative RNA-based drugs for modulation of protein expression, including the ability to simultaneously influence several therapeutic targets, in a controlled manner, with one molecular construct, and cellular targeting. Based in Basel and fully operational from 2018, the company is led by an experienced scientific and leadership team with proven expertise in drug discovery and development from lab bench to patient. Versameb’s proprietary technology platform, VERSagile, optimizes the application of functional RNA in different disease contexts – making RNA druggable in new therapeutic areas others have been unable to solve. The pipeline includes lead candidate programs in Stress Urinary Incontinence (SUI) and solid tumors. Versameb is working towards the completion of a first in-human/proof-of-concept clinical study while advancing its platform. |