Drug intelligence / Profile preview

aprinocarsen

Development stage
Phase 3
Lead developer
Eli Lilly
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

Aprinocarsen is a synthetic phosphorothioate oligodeoxynucleotide that functions as an antisense molecule. It specifically inhibits the expression of protein kinase C-alpha (PKC-α) by hybridizing to its 3'-untranslated region mRNA, leading to RNase H-mediated cleavage of the hybridized mRNA and reduced PKC-α protein levels. This mechanism regulates cell differentiation and proliferation, demonstrating anti-tumor activity in various human tumor cell lines in preclinical models. It was initially developed by Isis Pharmaceuticals (now Ionis Pharmaceuticals) and later licensed to Eli Lilly & Co. for the treatment of human cancers.

Brand names
AffinitakAffinitac
Other names
Aprinocarsen sodiumDNA, d(P-thio)(GTTCTCGCTGGTGAGTTTCA) protein kinase C-alpha antisense
02

Targets

PRKCA (Protein kinase C alpha)

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