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AZD5312
- Development stage
- Phase 1
- Lead developer
- AstraZeneca
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
AZD5312 is a generation 2.5 antisense oligonucleotide (ASO) designed to specifically suppress the expression of the human androgen receptor (AR) by hybridizing with AR mRNA and blocking its translation[1][2][7]. This mechanism aims to reduce production of all known forms of androgen receptor—including splice variants such as AR-v7—thereby inhibiting AR-driven signaling pathways that are critical for the growth and progression of certain cancers[3][7]. The drug was developed for intravenous administration and has been investigated primarily in advanced solid tumors where the androgen receptor pathway is implicated, including metastatic castration-resistant prostate cancer (mCRPC), breast cancer, bladder cancer, ovarian cancer, gastric cancer, and salivary duct cancer[5][8]. The primary developer is AstraZeneca in collaboration with Ionis Pharmaceuticals[3][7].
- Other names
- androgen receptor antisense oligonucleotide AZD5312IONIS-AR-2.5RxIONIS-AR2.5RxIONIS-AR 2.5Rx
02
Targets
AR-V7 (Androgen Receptor Variant 7)
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