ExploRNA is open to various collaboration models including licensing its proprietary capping technology to industry partners, supporting them in mRNA manufacturing process, and jointly developing new therapeutic programs.
ExploRNA offers a range of novel cap analogs that enhance the biological properties of mRNA. These cap analogs are protected under ExploRNA-controlled intellectual property (IP) rights and have freedom to operate independently of any existing patents.
ExploRNA aims to support our partners by providing high quality cap analog material for mRNA production, facilitating technology transfer, along with high quality mRNA delivery leveraging our research laboratory and through our GMP partners.
ExploRNA is open to forming partnerships focused on developing novel mRNA-based therapeutics using ExploRNA’s proprietary technology and wide mRNA-centered know-how.
ExploRNA Therapeutics is a privately owned “spin off” company from the University of Warsaw.
ExploRNA Therapeutics is proudly backed by the Black Forest SICAV-SIF, an investment fund supporting scientific and technological advancements achieved by both Polish and foreign companies.
ExploRNA Therapeutics and Primrose bio collaborate to provide co-optimized set of cap analog and RNA polymerase. Read more
American private foundation founded by Bill Gates and Melinda French Gates. Foundation is reported as of 2020 to be the second largest charitable foundation in the world.
The Agency for Medical Research (ABM) supports ExploRNA Therapeutics in the implementation of a research project focused on the use of mRNA technology for the treatment of Acute Respiratory Distress Syndrome (ARDS).
Acute Respiratory Distress Syndrome (ARDS) is a severe and life-threatening form of lung injury that develops as a result of acute inflammatory processes, leading to non-cardiogenic pulmonary edema and impaired gas exchange. Mortality associated with ARDS remains high, reaching approximately 30–40%, while long-term complications, including pulmonary fibrosis, are frequently irreversible. The most common causes of ARDS include respiratory tract infections, generalized extrapulmonary infections, as well as mechanical and chemical lung injury.
ExploRNA Therapeutics is developing therapeutic solutions based on mRNA technology, which enables prolonged intracellular translation and sustained protein production. In preliminary preclinical studies conducted by the company, a single administration of mRNA encoding therapeutic protein resulted in production maintained for over 48 hours. In mouse models of ARDS, mRNA-based therapy demonstrated strong therapeutic effects exceeding those observed after administration of recombinant protein. The obtained results were generated using mRNA constructs requiring further structural optimization, while the lipid carriers applied in early formulations exhibited increased reactogenicity.
The Project aims to optimize the structure of mRNA encoding therapeutic protein, identify a lipid formulation characterized by reduced reactogenicity, and confirm therapeutic efficacy in preclinical models. In the course of the Project implementation, a patent application has been filed to protect the developed solution. In addition, the company holds intellectual property rights related to proprietary modifications of mRNA structure.
ExploRNA Therapeutics will achieve the aim of the Project by completing the following phases of the research and development program:
(i) design and optimization of mRNA constructs;
(ii) development and selection of lipid formulations with reduced reactogenicity;
(iii) evaluation of physicochemical and biological properties of optimized mRNA formulations;
(iv) preclinical efficacy and safety studies in relevant cellular and animal models;
(v) selection of a candidate for further clinical development.
Project co-financed by the European Union under the National Recovery and Resilience Plan (KPO), as part of Investment D3.1.1 “Comprehensive development of research in the field of medical sciences and health sciences”.
Project implemented on the basis of the Agreement on granting support No. 2024/ABM/05/KPO/KPOD.07.07-IW-07-0244/24.
Total eligible cost of the Project: PLN 4,574,894.40.
Project co-financed by the European Union from the European Regional Development Fund under the Smart Growth Operational Program.
Project implemented as part of the National Centre for Research and Development call: Fast Track 6/1.1.1/2019.
National Center for Research and Development (NCBR) funds ExploRNA Therapeutics a new research project on anticancer mRNA vaccines.
ExploRNA developed a technology to modify a cap structure at 5’ mRNA end. This modification stabilizes mRNA and significantly increases its translation efficiency. The uniqueness of mRNA for vaccine development results from the fact that in a single inoculation multiple different mRNAs encoding various tumor-associated antigens can be administered to stimulate development of adaptive immune response. This is a huge advantage over recombinant vaccines that consist of single antigens. With stable and effectively translated mRNA multiple antigens can be directly delivered to professional antigen-presenting cells that induce protective immunity. Translation of mRNA leads to production of antigens that will stimulate both T-cells and B-cells, thereby mimicking development of natural immunity. The Project undertakes the verification of the safety of this vaccination immunotherapy and its ability to induce antigen-specific T-cells in the group of patients with malignant melanoma.
ExploRNA Therapeutics will achieve the aim of the Project by completing the following five phases of the research and development program:
(i) synthesis and analyzis of physicochemical and biological properties of cap analogs and modified mRNA; (ii) synthesis optimization of the cap analogues on a larger scale and development of the mRNA synthesis methods; (iii) pre-clinical studies in advanced cellular and animal models; (iv) toxicology and pharmacokinetics in the GLP standard and selection of a candidate for clinical trials; (v) phase I clinical trial in patients with malignant melanoma.
Project co-financed by the European Union from the European Regional Development Fund under the Smart Growth Operational Program.
Project implemented as part of the National Centre for Research and Development call: Fast Track POIR.01.01.01-00-0920/19 Total value of the project: PLN 25 995 670,81 Total value of funding: PLN 19 408 810,05
Funding from the Bill & Melinda Gates Foundation will go toward understanding the benefits o novel cap analogs for therapeutic applications.
Warsaw, December 8, 2022. ExPLoRNA Therapeutics, a Polish biotech company revolutionizing mRNA vaccines and therapeutics with unprecedented translational efficiency, received funding from the Bill & Melinda Gates Foundation to further develop its unique mRNA technology. ExPLoRNA specializes in novel cap analogs, the starting part of mRNA that is crucial for enabling the successful production of the encoded protein. ExPLoRNA’s technology increases protein production beyond the scale observable so far with other standard cap analogs.
The total funding received from the Bill & Melinda Gates Foundation is $813,578 and will be used for a 14-month project. The project aims to better understand the benefits of ExPLoRNA’s cap analogs, especially in terms of reducing the mRNA dose needed for therapeutic effects in the settings of vaccination and monoclonal antibody production.
The planned experiments include the use of state-of-the-art, clinically validated lipid nanoparticle (LNP) formulations from Acuitas Therapeutics. Acuitas is known for its successful role in providing the LNP technology for one of the only two regulatory-approved mRNA-based COVID-19 vaccines.
ExPLoRNA Therapeutics is the first Polish biotech company to receive funding from Bill & Melinda Gates Foundation. Through this project, ExPLoRNA Therapeutics commits to its vision of making mRNA technology more accessible in low-income countries to help fight diseases threatening human lives and well-being.
“We are grateful for the foundation’s support to help us achieve our vision. With the help of this grant, we intend to speed up the development of future mRNA vaccines and therapies.” – comments Jacek Jemielity, CEO of ExPLoRNA Therapeutics, and professor at the University of Warsaw.
About ExPLoRNA Therapeutics:
With passion, ExPLoRNA Therapeutics (www.explorna.com) is harnessing over 20 years of cap analog research and developing ground-breaking innovations of mRNA as a therapeutic platform applies those solutions in unique therapeutic programs, and delivers technology, therapeutic platform, and therapies to pharmaceutical Partners.
About Acuitas Therapeutics:
Founded in February 2009, Vancouver-based Acuitas Therapeutics (www.acuitastx.com) is a private biotechnology company that specializes in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticles. The team works with partners to develop new therapies to address unmet clinical needs based on its internationally recognized capabilities in delivery technology.
