Uppsala Therapeutics is a biopharmaceutical company specialized in the development of nanoparticle-based therapies to deliver small molecule and oligonucleotide drugs. The innovation originates from cutting-edge biomedical research at the Department of Chemistry, Uppsala University in Sweden and the Faculty of Medicine and Health Technology, Tampere University in Finland.
We have developed two cutting-edge platform technologies that will facilitate and enable the treatment of diseases with a high unmet medical need. These are:
Blood-brain-barrier (BBB) penetrating nanoparticle: Drug delivery to the brain is a major challenge in the field as most drugs do not pass the blood-brain-barrier (BBB) because of the unique vasculature system within the central nervous system (CNS). For this reason, most of the drugs that are very effective in treating CNS diseases fail upon systemic delivery. To address this problem, we have developed a novel drug delivery system that has a unique ability to cross the BBB within few hours and deliver the cargo molecules. Such a strategy enables the evaluation of a broad spectrum of drugs (small molecules, antibodies and oligonucleotide drugs) for different CNS indications.
RNA-interference: RNA-interference (RNAi) is a Nobel Prize-winning technology that employs short (20-25 base pairs) double-stranded RNA molecules to induce gene silencing. This technology holds a large promise to find effective treatment options for many diseases. However, intracellular delivery and off-target effect remain the single greatest hurdle to the widespread adoption of in vivo RNAi methods. In contrast to the tangible and obvious effectiveness of RNAi in vitro, silencing target gene expression in vivo remains the major challenge due to multiscale barriers that require delivery reagents that are not tolerated in vivo due to toxicity. Uppsala Therapeutics provides the solution to boost efficiency while reducing undesired side effects for RNA interference (RNAi) using its proprietary technology.
Current application areas include disease targets associated with arthritis, stem cell survival, cancer, liver diseases, hyperhidrosis and keratosis.
Novel Nanoparticle Design: Our proprietary nanoparticles have demonstrated the ability to cross the blood-brain barrier when administered systemically, and to target brain tumors such as glioblastoma in pre-clinical models resulting in complete tumor elimination. Unlike other known drug delivery systems, our nanoparticle does not exhibit any cytotoxicity to healthy brain cells. Based on highly encouraging pre-clinical data, we have decided to pursue glioblastoma as the initial target indication.Furthermore, our nanoparticle technology has the potential to be used in other indications, we will further explore the development of our nanoparticles as a stand-alone treatment or as a targeted delivery platform in key therapeutic areas such as oncology and neurodegenerative diseases.
Asymmetric RNA Design: We have developed a unique RNA design that displays selective strand selection which minimizes off-target effects and enhances gene silencing efficacy. Such tailored RNAi molecules also escape degradation by endosomal nuclease while maintaining high target specificity, unlike traditional RNAi molecules. The unique RNA design is based on natural nucleotide sequences, and amenable to standard chemical modifications. The efficacy of our technology is validated for small interfering RNA (siRNA) as well as for microRNA (miRNA). This technology is currently being explored to develop a targeted delivery system to the liver as well as the central nervous system (CNS).
This technology is a spin-off of research activities from Uppsala University. Key personals in this company are as follows
Victor the Chief Executive Officer and is responsible for corporate operations and business development. He is an entrepreneur with a background in business administration and a medical degree (MD) candidate at the Faculty of Medicine, Uppsala University.
Dr. Varghese is the Chief Scientific Officer (CSO) and is the main person behind developing this pioneering technology. He also holds an Associate Professor position at the Department of Chemistry-Ångström Laboratory, Uppsala University, Sweden. His research expertise is in engineering nucleic acid-based drugs for therapeutic applications and regenerative medicine. The key highlight of his research activities includes biomaterial science and targeted drug delivery, bridging synthetic organic chemistry and biotechnology. He is specifically involved in designing novel chemical modification of nucleic acid drugs and glycosaminoglycan derivatives for cell-specific delivery of plasmid DNA, siRNA and miRNA for in vivo applications.
Dr. Hilborn is professor in Polymer Chemistry at Uppsala University in Sweden. He has management experience from life science industry, founder of several start-up companies, coordination of European projects, and advisor for EC and UK science foundations. His research interests are in the design, synthesis and preparation of biomedical polymers and specifically materials for tissue scaffolds and drug delivery. He is a frequently invited speaker at international events and has published more than 300 scientific papers (H=41) and has filed 26 patent applications.
Dr. Oommen is a partner and a co-inventor of this technology. He is currently employed as an Assistant Professor at the Faculty of Biomedical Sciences and Engineering at the Tampere University of Technology, Finland (www.tut.fi/oommen-lab). He has extensive expertise in the field of nanotechnology and targeted delivery systems for both small molecule drug to large nucleic acid drugs. He has a multidisciplinary research interest from basic organic chemistry to biomedical engineering. His ambition is to develop non-viral nanocarriers for nucleic acid drugs that could be delivered to specific tissue without eliciting the immune response which has clinical potential. His other interests are in the fields of bone and cardiac tissue engineering, cell therapy, immunotherapy and develop antifouling surfaces for biomedical applications.
Uppsala Therapeutics has teamed up with LifeScience- Belgium (LFSCNC) for successful clinical translation of our proprietary technology. Read more at, lfscnc.com!