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Drug intelligence / Profile preview
RX-0201 + gemcitabine
- Development stage
- Phase 2
- Lead developer
- Opus Genetics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules
- Administration
- Intravenous
01
Overview
RX-0201 (also known as Archexin) is an antisense oligonucleotide designed to target and inhibit the expression of AKT1 by binding to its mRNA, thereby reducing downstream AKT signaling. It is being developed primarily for oncology indications, including metastatic renal cell carcinoma and pancreatic cancer. Gemcitabine is a nucleoside analog chemotherapeutic agent that acts as a prodrug; it is metabolized intracellularly into active diphosphate and triphosphate forms that inhibit ribonucleotide reductase and are incorporated into DNA, leading to inhibition of DNA synthesis and induction of apoptosis. The combination of RX-0201 with gemcitabine has been studied in clinical trials for metastatic pancreatic cancer[2][4][6].
- Brand names
- Gemzar
- Other names
- Archexingemcitabine
02
Targets
AKT1 (Proto-oncogene serine/threonine-protein kinase Akt1)DNARRM1
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