Dofinansowanie ze środków budżetu państwa
Deklaracja dostępności
In the years 2023-2025, at the Institute of Biochemistry of the Polish Academy of Sciences in the Department of Genetic Engineering a project titled: “The influence of CAG tract length polymorphism on the pathogenesis of neoplastic diseases" under the Perły Nauki program. The principal investigator is Marianna Karwacka, MSc. Neurodegenerative diseases and cancer are one of the leading diseases of the elderly age. The cause of neurodegenerative polyglutamine (polyQ) diseases, including spinal and bulbar muscular atrophy (SBMA), is the expansion of CAG trinucleotide repeats in the AR gene. At the same time, the androgen receptor, encoded by the AR gene, is a protein of great importance for the development and progression of cancer. A surprising and unexplained phenomenon is the lower incidence of cancer in patients with polyQ diseases. The aim of the project is to examine how CAG repeats of different lengths affect phenotypic features typical of cancer cells and tumor progression. The project involves the generation of cellular models of prostate and ovarian cancer containing long CAG repeats and a SBMA cellular model with short CAG repeats in the AR gene using the CRISPR-Cas9 system. Next, the cancer features of the created cell models, such as the ability to proliferate, migrate and response to cellular stress, will be examined, and their gene expression profiles will be analyzed. The obtained results will allow us to understand what mechanisms may be responsible for the protective effect of long CAG sequences in cancer diseases, which may be used in the future to develop new therapeutic approaches.
In 2021, as part of the competition for the development of innovative therapeutic solutions using RNA technology, the consortium of Warszawskie Zakłady Farmaceutyczne Polfa SA and the Institute of Bioorganic Chemistry of the Polish Academy of Sciences in Poznań received funding from the state budget (from the Medical Research Agency) in the amount of PLN 93,852,588.56. The total cost of the project is PLN 130,191,808.51. Funding was received for a commercial clinical trial project – the development of innovative therapeutic solutions using RNA technology, titled “Development of a universal fast-response platform, based on RNA technology, ensuring the national drug and epidemiological safety”. Institute of Bioorganic Chemistry of the Polish Academy of Sciences as the Consortium Member received funding in the amount not exceeding PLN 35 207 716,16. The aim of the project carried out over a period of 7 years is to develop a universal bioinformatics, chemical and biotechnological platform, unique on the Polish market. This platform is dedicated to obtaining drugs and vaccines through the use of innovative technologies and solutions based on the use of nucleic acids, in particular RNA. This will increase drug and epidemiological safety of the country while allowing for equal participation of Polish scientists and entrepreneurs in global efforts on the development of innovative therapies based on nucleic acids. We believe that in this way we will contribute to an increase in the competitiveness of the Polish economy. The project consists of eight tasks that include basic research, industrial research and development works, covering all stages of innovative drug development:
⦁ Designing therapeutic RNAs and developing bioinformatics tools and databases. ⦁ Chemical and biotechnological synthesis of short and long RNAs. ⦁ Designing and testing delivery strategies for short and long RNAs. ⦁ Evaluation of the biological activity of therapeutic RNAs in in vitro systems. ⦁ Pre-clinical evaluation of the efficacy of RNA therapeutics in animal models. ⦁ Research on formulation development of a medicinal product. ⦁ Technology transfer with scale-up. ⦁ Phase 1/2 PK/PD clinical trial evaluating the efficacy, safety and pharmacokinetics of the developed drug.
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