18.09.2023 change 18.09.2023

We are developing Polish RNA therapeutics, says expert

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‘New RNA-based therapeutics are being introduced in the medical field and we are also attempting to develop a Polish drug based on this technology,’ says Katarzyna Malik from Polpharma.

The Polish company has already introduced the first biological drugs (so-called biosimilars, equivalents of innovative, reference drugs) to the market in Poland and other countries. One of these drugs called ranibizumab was registered by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 2022. This preparation is intended for the treatment of age-related macular degeneration (AMD).

In the coming days, the FDA is expected to register another Polish biosimilar drug called natalizumab. It is a monoclonal antibody with immunosuppressive properties that can inhibit the progression of multiple sclerosis (MS).

Katarzyna Malik, R&D director at Polpharma, said at a meeting with journalists that work was also underway to develop the first Polish RNA-based therapeutic (RNA is ribonucleic acid). This method is similar to the one used to develop COVID-19 vaccines. It is also used to develop drugs for various diseases.

She told PAP: ’I cannot reveal exactly what the drug is yet, but it is to be used in the treatment of one of the neurodegenerative diseases.’ 

She added that work was also underway on a drug useful in the treatment of hypercholesterolemia. These will be second-generation drugs (commonly referred to as generics), but they require the use of modern RNA technology.

The first RNA-based therapeutic was registered in 1998, and currently 13 drugs of this type are in use, including givosiran (for the treatment of hepatic porphyria) and inclisiran (used in patients with primary hypercholesterolemia or mixed dyslipidemia).

Many more drugs are in clinical trials. Advanced work is being carried out on 80 new RNA drugs. These include both new vaccines and drugs for various types of diseases, such as cancer. Advanced research on one of the cancer drugs is conducted by BioNTech, the same company that developed the COVID-19 vaccine in mRNA technology.

RNA (RiboNucleicAcid), is a biopolymer made of four types of bases: U, C, A and G. The specific number and order of these bases creates the sequence of a given RNA. There are many types of RNA with different functions in the human cell. These are mainly mRNA, tRNA and rRNA.

In the context of drugs, the terms mRNA and RNA are most often used, which differ quite substantially in terms of structure and function. mRNA (messenger RNA) contains a unique instruction for the production of a specific protein in the cell (for example, a virus that causes an immune reaction in the case of a vaccine). These are biopolymers with long chains, containing up to several thousand bases, and their production requires special biotechnological methods.

Therapeutic RNAs are usually short-chain polymers, containing from 19 to 30 bases, obtained by chemical synthesis (not biotechnological methods). In medicinal preparations, they are used to treat diseases caused by a deficiency or dysfunction of a certain protein. They can prevent the formation of an incorrect protein by cutting the mRNA chain with its wrong instruction. Or they can change the mRNA sequence into the correct one, resulting in a protein with the correct structure being produced.

Katarzyna Malik explained that work on a Polish RNA-based therapeutic is still in the early stages. 'We expect that clinical trials of a preparation useful in the treatment of one of the degenerative diseases will begin in 2026,’ she said. Its market introduction will be possible no earlier than in 2031.

Dr. Artur Bartoszewicz from the Warsaw School of Economics, co-author of the 'Pharmaceutical Race of Nations' report published in 2022, noted that it was very important to manufacture as many drugs as possible in Poland, especially by domestic manufacturers. 'We could see this during the pandemic, when supply chains were broken,’ he said.

In his opinion, Polish manufacturers should manufacture as many drugs as possible that can be sold internationally, both in the European Union and on other continents. This applies especially to biological and RNA drugs, because they bring the greatest profits. 'The rule is that the larger the international market, the better, because it generates more revenues and more funds can be allocated to research on new drugs,’ he said. (PAP)

Zbigniew Wojtasiński

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