Fig. 1 · Structure
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Branaplam hydrochloride
SMN2 Splicing Modulator
Branaplam hydrochloride is a potent and selective modulator of survival motor neuron 2 (SMN2) splicing, exhibiting an EC50 of 20 nM for SMN. This compound effectively enhances full-length SMN protein levels and demonstrates the ability to extend survival in a severe spinal muscular atrophy (SMA) mouse model. Additionally, Branaplam hydrochloride inhibits human ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM, making it a valuable tool for research in SMN-related therapies and spinal muscular atrophy.
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Specifications
| Manufacturer | Adooq Bioscience |
|---|---|
| Actions | Modulator |
| CAS Number | 1562338-39-9 |
| Applications | SMN2 Splicing Modulator |
| Formula | C22H28ClN5O2 |
| Molecular Weight | 429.94 |
| Synonyms | LMI070 hydrochloride; NVS-SM1 hydrochloride |
| SMILES | OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3.Cl |
Storage & Solubility
In Vitro Solubility
Reported Solubility
No reported solubility data are available for this product.
Preparing Stock Solutions
| Concentration | |
|---|---|
Values use four significant digits. Prepare to the final solution volume; this is not the volume of solvent to add.
Stock preparation is unavailable for this product molecular weight.
Calculated stock concentrations do not establish solvent solubility. Refer to the reported solubility data for experimental guidance.
Storage
Store powder at -20°C, keep desiccated.
In powder form, the chemical is stable for 36 months.
In solution, store at -20°C and use within 1 month to prevent loss of potency. Aliquot to avoid multiple freeze/thaw cycles.