DNA/RNA Synthesis Modulator

NMDI14 is a nonsense mediated RNA decay (NMD) inhibitor. NMDI14 disrupts the SMG7-UPF1 interactions and inhibits NMD.

TREX1-IN-1 is a TREX1 inhibitor with an IC₅₀ value for hTREX1 less than 0.1 μM.

m7GpppAmpG ammonium (m7G(5')ppp(5')(2'OMeA)pG ammonium) is a trinucleotide 5′ end cap analog. m7GpppAmpG ammonium binds to eIF4E with a K_D value of 45.6 nM. m7GpppAmpG ammonium caps RNA with a capping efficiency of 90%. m7GpppAmpG ammonium enhances mRNA stability and translation efficiency. m7GpppAmpG ammonium is used in mRNA therapeutic research.

5-Hydroxymethyl-2’-deoxycytidine (5hmdC) is an oxidation derivative of 5-methyl-2'-deoxycytidine (5-mdC) in DNA. 5-Hydroxymethyl-2’-deoxycytidine may serve as a marker of irreversibly damaged cells.

PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model.

SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).

RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.

RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC_1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.

Myoseverin is a microtubule-binding molecule and angiogenesis inhibitor. Myoseverin can induce the reversible fission of multinucleated myotubes into mononucleated fragments. In addition, Myoseverin exerts anti-angiogenic effects by inhibiting endothelial cell function and endothelial progenitor cell differentiation.

Methylproamine is a DNA-binding radioprotector, acts by repair of transient radiation-induced oxidative species on DNA. Methylproamine also protects against ionizing radiation by preventing DNA double-strand breaks.

RNA splicing modulator 3 is an RNA splicing modulator. RNA splicing modulator 3 modulates nucleic acid splicing, including pre-mRNA splicing.

BPN-15477 is an orally active, blood-brain barrier-permeable splicing modulator compound (SMC). BPN-15477 restores the correct splicing of exon 20 of the elongator complex protein 1 (ELP1) (EC₅₀ = 1.9 μM), thereby significantly increasing the in vivo functional protein levels in all tissues including the brain. BPN-15477 can be used in the research of familial dysautonomia, such as frontotemporal dementia.

RNA splicing modulator 1 (compound 233) is a RNA splicing modulator, with an AC₅₀ value of <100 nM.

5-Hydroxymethyl-2'-deoxycytidine-d₃ (5hmdC-d₃) is the deuterium labeled 5-Hydroxymethyl-2'-deoxycytidine (HY-131394). 5-Hydroxymethyl-2’-deoxycytidine (5hmdC) is an oxidation derivative of 5-methyl-2'-deoxycytidine (5-mdC) in DNA. 5-Hydroxymethyl-2’-deoxycytidine may serve as a marker of irreversibly damaged cells.

Ultevursen (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa.

Diguanosine 5′-triphosphate (Gp3G) triammonium is a dinucleoside triphosphates. Diguanosine 5′-triphosphate triammonium also is a virus-specific oligonucleotide. Diguanosine 5′-triphosphate triammonium is needed for the synthesis of RNA during the transcription process.

ML372 inhibits survival motor neuron (SMN) protein ubiquitination, increases SMN protein stability without affecting mRNA expression. ML372 improves spinal muscular atrophy (SMA) in mice. ML372 is brain penetrant and has a reasonable exposure and half-life in vivo.

Uracil-m7GpppAmpG ammonium solution (100 mM) is a cap analog that can be used for mRNA synthesis.

Farnesylacetone acts as a transcriptional regulator, RNA synthesis modulator, and male hormone. Farnesylacetone can be extracted from the androgenic glands of the green crab (Carcinus maenas). Farnesylacetone modulates transcriptional processes, inhibits uridine incorporation into all types of RNA and leucine incorporation into ovaries, while stimulating uridine incorporation into tRNA/poly (A)⁺ RNA and leucine incorporation into testes. It suppresses electron transport in mitochondrial Complex I and Complex II. Farnesylacetone inhibits vitellogenesis in crustacean ovaries and stimulates uridine incorporation in crustacean intestines. It functions as an androgen in crustaceans.

Lorutengitide is a transcriptional regulatory peptide, and exhibits antiproliferative efficacy.