Research and development in cell- and gene therapy
Our mission is to bridge the gap between scientific discovery and clinical practice, starting from the unmet need of disease treatment identified on the clinical side. We aim to transform patient healthcare through the power of cell-and gene therapy, development of highly efficient CRISPR/Cas9 modalities as well as oligonucleotide-based drugs.
As a joint initiative between Karolinska Institutet and Karolinska University Hospital, Karolinska ATMP Center is committed to accelerating the development and application of ATMPs - Advanced Therapy Medicinal Products.
New advanced treatments for severe diseases
Monoclonal antibodies and small molecule therapeutics have for a long time made up the cornerstones for treatment of disease. Over the past decade, new advanced treatments have emerged based on cells and genetic medicines that offer possibilities to, in a personalized fashion, treat diseases that are not amenable to treatment with current modalities.
ATMPs represent a cutting-edge category of emerging therapeutics that are based on genetic material, cells, and tissues. These therapies offer groundbreaking opportunities for treating diseases and tissue injuries, many of which currently have limited or no treatment options. Among these diseases are cancer, infectious disease, severe inflammation, and rare genetic disorders including those affecting the central nervous system.
ATMPs are divided into three main types: gene therapy, somatic cell-based medicines, and tissue-engineered products. In addition to these, nucleic acid-based therapeutics (NATs) based on chemically modified shorter RNA or DNA analogs as well as gene editors have emerged as potent regulators of gene expression and are now in clinical use to silence or correct a range of disease-causing genes.
Breakthroughs in cell and gene treatment
Research within the center is dedicated to unraveling the complexities of cell and gene therapy, investigating novel approaches to enhance the efficacy and safety of these ATMPs. The research includes development of next generation CAR-T cells as well as the use of NK cells for immunotherapy applications in cancer. Extensive effort will be made to develop CAR-T cells for treatment of solid tumors. There are also ongoing research activities into the clinical use of Mesenchymal and other stem cells for tissue regeneration and treatment of different inflammatory diseases. The Karolinska ATMP center also has the competence and capacity to develop RNA-and DNA-based vectors for cancer immunotherapy as well as for vaccination against infectious diseases. For genetic medicines to be active, they require encapsulation in nanocarriers that facilitate cellular delivery. To this end, we are committed to expand our portfolio to include next generation non-viral nanocarriers based on targeted lipid nanoparticles (LNPs) and bioengineered exosomes. These delivery systems will additionally be harnessed to deliver CRISPR/Cas9 for in vivo gene editing or for generation of in vivo CAR-T cells.
Development of nucleic acid-based therapeutics
Nucleic Acid Therapeutics (NATs) are traditionally not defined as ATMP products per se, oligonucleotide- and siRNA- based drugs can be designed to target virtually any disease relevant gene, either on RNA or DNA level. Oligonucleotide therapies have recently emerged as a promising medicine and several treatments of a wide range of genetic diseases have been FDA (U.S. Food and Drug Administration) and EMA (European Medicines Agency) approved. A core research discipline within the development of oligonucleotide therapeutics is nucleic acid chemistry and conjugation to different modalities such as various peptides, lipophilic moieties, and antibodies.
Our recently established NAT hub (NATH) at the Karolinska ATMP Center will foster a new generation of scientists within this fast-developing field.
Research facilities in Flemingsberg
Within ANA Futura in Campus Flemingsberg, the research center houses advanced laboratories equipped with cutting-edge technology for molecular biology, cell culture, and bioengineering. These labs facilitate the manipulation and analysis of genetic material and cultivation of therapeutic cells as well as production of engineered exosomes. It also houses production facility for therapeutic mRNA/DNA and LNPs as well as dedicated organic chemistry laboratories for synthesis of oligonucleotide therapeutics with state-of-the art bioconjugation capabilities.
From understanding intricate cellular mechanisms to designing targeted interventions, our research within the Karolinska ATMP Center will fuel the progress of advancing gene- and cell therapies, bringing scientific discoveries from bench to bedside. The close integration with the clinical site will also favor bedside to bench research where clinical challenges and unmet needs will feed into basic research prompting patient-close next generation innovation.