ptc

Rosolutamide (ALZ-003) is a curcumin analog, an orally active Nrf1 and Nrf2 activator.Rosolutamide modulates oxidative homeostasis, improves mitochondrial function and promotes autophagy, reduces mutant protein aggregation, and decreases intracellular/mitochondrial reactive oxygen species (ROS) levels.Rosolutamide has been used in the study of neurodegeneration such as spinal cerebellar ataxia and Huntington disease. neurodegenerative diseases such as Huntington's disease. Regulates oxidative homeostasis.

PTC-028 selectively suppresses cancer cells whereas normal cells remain unaffected. PTC-028 is an orally bioavailable inhibitor of stem cell factor BMI-1 in ovarian cancer. The depletion of BMI-1 by PTC-028 causes caspase-mediated apoptosis.

PTC-700 is an effective splicing modulator of microtubule-associated protein tau (MAPT). It reduces 4R MAPT in P301L and S305N neurons with EC50 values of 1.3 to 1.8 nM and 0.5 to 2.6 nM, respectively.

PTC-209 is a potent and selective BMI-1 inhibitor.

PTC-510 TFA salt inhibits hypoxia-induced VEGF expression.

PTC-209 hydrobromide (PTC-209 HBr) is the hydrobromide salt of PTC-209, which is a potent and selective BMI-1 inhibitor with IC50 of 0.5 μM, and results in irreversible reduction of cancer-initiating cells (CICs).

PTC299 ((4-chlorophenyl) (1S)-6-chloro-1-(4-methoxyphenyl)-1,3,4,9-tetrahydropyrido[3,4-b]indole-2-carboxylate) is an orally active inhibitor of VEGFA mRNA translation and selectively inhibits VEGF protein synthesis at the post-transcriptional level. PTC299 is also a potent inhibitor of dihydrolactate dehydrogenase (DHODH).

PTC725 is a selective HCV 1b replicons inhibitor. It has been shown to target the nonstructural protein 4B.

PTCA is a potent ligand for Ca2+/calmodulin-dependent protein kinase II α (CaMKIIα), with a pKi value of 7.2.

POPTc80-IN-1 (Compound LC-45) is a competitive inhibitor of Trypanosoma cruzi prolyl oligopeptidase (POPTc80), with an IC50 of 0.23 μM and a Ki of 0.054 μM. It inhibits the invasion of host cells by T. cruzi, with an IC50 of 46.71 μM.

PTC258 is a specific, orally active modulator of the splicing of the Elongator complex protein 1 gene (ELP1), enhancing its expression both in vitro and in vivo. Additionally, PTC258 has demonstrated good tolerability in a mouse model [1].

LAPTc-IN-1 (compound 4), with a K i of 0.27 μM, serves as a competitive inhibitor of acidic M17 leucinopeptidase (LAPTc) and stands as a potential antagonist against the parasite Trypanosoma cruzi [1].

Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk.

Votoplam HCl (PTC-518 HCl) is the hydrochloride form of Votoplam.Votoplam is a systemically acting gene splicing regulator that down-regulates the levels of huntingtin protein, which is primarily used to inhibit Huntington's disease (HD).

Unesbulin (PTC596) is an orally active, selective inhibitor of B-cell-specific Moloney murine leukemia virus integration site 1 (BMI-1).

Rhodiolin, a natural product from Rhodiola rosea, blocks glycolysis of glucose 6-phosphate isomerase (GPI) in human papillary thyroid cancer (PTC), inhibits phosphorylation of the PI3K/AKT/mTOR signaling pathway and induces apoptosis of PTC cells.

IDB-001 is an inhibitor of the ribosomal PTC that selectively represses protein synthesis by sequence. It inhibits the viability of HCC-1143 cells with an EC50 of 0.84 μM and also hinders the viability of MYC-dependent cancer cells.

RTC14, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.

GJ103 sodium salt is an active analog of the read-through compound GJ072. Some GJ072 analogs (e.g., GJ103, GJ106, GJ109, and GJ111) consistently demonstrates their activities in all three PTCs by both FCATMpSer1981 and IRIF assays. GJ103 have similar read

SRI-41315 is an eRF1 degrader that acts as a molecular glue at the center of ribosomal decoding.SRI-41315 induces an extended pause at the termination codon and inhibits cystic fibrosis-associated PTC in immortalized and primary human bronchial epithelial cells, restoring CFTR expression and function.SRI-41315 inhibits PTC by decreasing the abundance of the termination factor eRF1 NIOCH 14

SRI-37240 is a potent inhibitor of premature termination codons (PTCs) with read-across activity that induces a prolonged pause at the termination codon and inhibits PTCs associated with cystic fibrosis.SRI-37240 can be used to study PTC-related diseases.

AM-8508 is a potent and selective PI3Kδ inhibitor. AM-8508 showed good cellular potency (in vitro pAKT IC50 = 4.6 nM ). AM-8505 xhibited excellent HWB potency (HWB (pAKT) IC50 = 2.7 nM). AM-8508 inhibit KLH-specific antibodies in animal models, signifying its potential for the treatment of human inflammatory diseases. PI3Kα and PI3Kβ are ubiquitously expressed and play a role in cell growth, division, and survival.

Carotegrast methyl, also known as AJM300 and PTC-100, is an orally-active small molecule that antagonises the α4 integrin receptor. AJM300 reduces inflammation by blocking leucocyte trafficking. Oral treatment with AJM300 dose-dependently inhibited lymphocyte homing to Peyer's patches and increased the peripheral lymphocyte count in the same dose range. AJM300 dose-dependently prevented the development of experimental colitis in mice.