| Cat. No. |
Product Name |
CAS No. |
Information |
| H6872 |
SMN2 modulator 27
|
1537150-15-4 |
SMN2 modulator 27 is a novel SMN2 modulator that stabilize the SMN protein with EC50 of 0.29 uM; shows excellent in vitro properties and a promising mouse PK profile, stabilizes the SMN protein, unrelated to global proteasome or autophagy inhibition; a novel therapeutic mechanism that should complement other modalities for treatment of SMA. |
| H6871 |
SMN-C3
|
1449597-34-5 |
SMN-C3 is a selective, orally available small molecule SMN2 splicing modifier toward the production of full-length SMN2 messenger RNA with high selectivity; increases SMN protein levels, improves motor function, and protects of the neuromuscular circuit in Δ7 mice; also extends the life span of the mice. |
| H6870 |
SMN-C2
|
1446311-56-3 |
SMN-C2 is a close analogs of RG-7916 act as selective RNA-binding ligands that modulate pre-mRNA splicing; directly binds to the AGGAAG motif on exon 7 of the SMN2 pre-mRNA, and promotes a conformational change in two to three unpaired nucleotides at the junction of intron 6 and exon 7 in both in vitro and in-cell models; increases binding of the splicing modulators, far upstream element binding protein 1 (FUBP1) and its homolog, KH-type splicing regulatory protein (KHSRP), to the SMN-C2/C3-SMN2 pre-mRNA complex and enhances SMN2 splicing. |
| H6869 |
Risdiplam
|
1825352-65-5 |
Risdiplam (RG7916, RG-7916, RO7034067) is a highly potent, selective, orally active?SMN2 splicing modifier for treatment of?spinal muscular atrophy (SMA); RG7916 is undergoing clinical trials across the spectrum of spinal muscular atrophy, where it has shown promising early results.
|
| H6868 |
RG7800
|
1449598-06-4 |
RG7800 (RG-7800, RG 7800) is a potent, orally available, small molecule SMN2 splicing modifier with EC1.5X of 23 nM and 87 nM for SMN2 splicing and SMN2 protein, respectively; specifically modifies the alternative splicing of SMN2 exon 7 in SMA patient-derived cells and in two SMA mouse models; exhibits excellent pharmacokinetic and in vivo efficacy and has a favorable safety profile. |
| H6867 |
PK4C9
|
172286-77-0 |
PK4C9 (Homocarbonyltopsentin) is a small-molecule stem-loop RNA structure TSL2-binding compound (EC50=16.1 uM) that increases SMN2 exon 7 (E7) splicing and rescues downstream molecular alterations in SMA cells; modifies SMN2 E7 splicing in different systems, rescues functional SMN protein in SMA cells. |
| H6866 |
ML372
|
1331745-61-9 |
ML372 is a small molecule SMN modulator that increases SMN protein in patient fibroblasts with EC50 of 37 nM; possesses good potency, pharmacokinetics, tolerance, and CNS penetration that are able to increase levels of SMN protein in several model cell lines; increase SMN protein levels in vivo, restore motor function, and prolong survival of SMNΔ7 SMA Mice; improves the righting reflex and extended survival of a severe mouse model of SMA. |
| H6865 |
LMI-070
|
1562338-42-4 |
LMI070 (NVS-SM1, Branaplam ) is a potent SMN2 splice modulator (EC50=20 nM) that potential treatment of spinal muscular atrophy (SMA). |
| H6864 |
LDN-76070
|
838842-80-1 |
LDN-76070 is a novel potent SMN2 modulator (EC50=8.3 uM; Emax 186%), increases SMN protein in spinal cord and brain in mice (5 mg/kg). |
| H6863 |
LDN-75654
|
912798-81-3 |
LDN-75654 is a novel potent SMN2 modulator (EC50=1.8 uM; Emax 186%), increases SMN protein in spinal cord and brain in mice. |
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