(Health Korea News / Lee Chung-man) Attention is focused on whether Chong Kun Dang’s RNAi-based gene therapy ‘CA102’ can become a next-generation anticancer drug that can overcome the limitations of existing anticancer drugs.
‘CA102’ is a gene therapy that inserts shRNA into a recombinant virus that recognizes molecules that are specifically expressed in various cancer cells. The shRNA was designed to simultaneously target mTOR and STAT3, genes involved in the proliferation and metastasis of cancer cells through intracellular signaling.
In April of this year, Chong Kun Dang signed a contract to introduce ‘CA102’ with Curigin, a company specializing in the development of RNAi-based gene therapy, and plans to secure global rights and proceed with exclusive research and development and commercialization. (See related article below)
‘CA102’ is emerging as a promising next-generation anticancer drug as it has the potential to be used against various types of cancer.
This drug works by simultaneously targeting mTOR and STAT3, which are expressed in various cancers. mTOR is a protein that regulates cell growth, metabolism, and protein synthesis, and STAT3 is a transcription factor that regulates cell proliferation through inflammation and immune responses.
There are countless cancer types in which these two targets are overexpressed. Representative examples include colorectal cancer, breast cancer, ovarian cancer, prostate cancer, bladder cancer, colon cancer, melanoma, and glioblastoma. Accordingly, simultaneous targeting of mTOR and STAT3 is considered a promising cancer treatment that can be used across a wide range of cancer types.
However, the development of mTOR and STAT3 target drugs has been slow due to technical difficulties. The biggest reason is that mTOR and STAT3 are involved in various cellular functions, so if their activity is inhibited indiscriminately, it can affect healthy cells and cause side effects.
On the other hand, ‘CA102’ is an RNAi-based gene therapy agent that is expected to overcome these existing limitations.
RNA is a polymer compound that acts directly when synthesizing the necessary proteins according to the genetic information contained in DNA. RNA receives instructions from DNA, produces proteins, and replicates cells.
DNA, which contains genetic information, transfers that information to RNA through a process called transcription, and RNA creates proteins through translation in the cytoplasm outside the nucleus. The process of creating proteins from this sequence information is called gene expression.
RNA therapeutics are therapeutics designed to express proteins, or to suppress or change protein expression, in this process. Among them, RNAi is an effect that suppresses RNA (a compound that acts when DNA synthesizes proteins) from being made into proteins, and is also called RNA interference.
In particular, since RNAi directly targets the RNA of a specific gene, it can easily act on proteins that are difficult or impossible to target with existing tyrosine kinase inhibitors (TKIs) or antibodies. Therefore, ‘CA102’, an RNAi-based gene therapy, is expected to theoretically have an optimized targeting ability for all proteins.
In fact, ‘CA102’ has been shown to have therapeutic potential in more than 48 types of cancer through preclinical trials.
Chong Kun Dang will first attempt to develop ‘CA102’ as a bladder cancer treatment. This is because bladder cancer has a high unmet need as the range of treatment options is limited due to the occurrence of resistance and high recurrence rate, which ultimately leads to bladder removal.
The standard first-line treatment for bladder cancer is cisplatin-based chemotherapy, but it is known that about 50% of patients are not suitable for cisplatin due to underlying diseases. These patients use a combination of gemcitabine or carboplatin, but the efficacy is lower than that of cisplatin therapy.
In relation to this, a Chong Kun Dang official said, “‘CA102’ will strengthen Chong Kun Dang’s anticancer drug pipeline and become an innovative alternative for bladder cancer treatment.”
Meanwhile, in addition to ‘CA102′, the dual mTOR and STAT3 targeting therapeutics currently under development include ▲’NM-X1′ from Novomedix, USA and ▲’Piperlongumine’ from TargTex SA, USA.
However, these two drugs are small-molecule synthetic drugs, and their target versatility is expected to be lower than that of ‘CA102’.
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