Ceptur is developing unique and differentiated genetic medicines for patients with unaddressed diseases. The Company’s proprietary U1 Adaptor technology controls gene-specific expression and affords advantageous properties for drugging difficult targets.
U1 Adaptors are bivalent oligonucleotides that engage sequence-specific mRNA and the U1 small nuclear ribonuclear protein (U1 snRNP), which is a ubiquitous intracellular machine that regulates transcription and splicing. U1 Adaptor therapeutics control gene expression at the pre-mRNA level within the nucleus.
