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BF844
Axon 2624
CAS:
1404506-35-9
Purity:
98%
- Soluble in DMSO
- MF: C21H19ClN4O
- MW: 378.85
Description
BF844 is a first-in-class small-molecule pharmacological chaperone that stabilizes the mutant Clarin-1 (CLRN1) protein in vitro and in vivo. It is a targeted therapy designed to slow or stabilize progressive hearing and vision loss caused by the CLRN1(N48K) mutation in Usher Syndrome Type III (USH3).
CLRN1(N48K) pharmacology is relevant to disease-relevant cellular and translational research models; BF844 supports mechanistic studies of CLRN1(N48K), CLRN mutation in biochemical and cellular assays.
Key Features
- CLRN1 stabilizer
- Pharmacological tool for CLRN1(N48K) mutation mechanism studies
- Pharmacological chaperone designed to treat Usher Syndrome Type III
Applications
- CLRN1-N48K pharmacology and target-validation studies
- CLRN1(N48K) protein stabilization study
- Disease-relevant cellular and translational model systems
- Compound profiling, assay development and lead-optimization support
More Information
| Parent CAS No. | 1404506-35-9 |
|---|---|
| Chemical Name | 1-(4-chloro-3,5-diphenyl-1H-pyrazolo[3,4-c]pyridazin-1-yl)-2-methylpropan-2-ol |
| SMILES | C12N(CC(C)(C)O)N=C(C3=CC=CC=C3)C1=C(Cl)C(C1=CC=CC=C1)=NN=2 |
| MFCD | N.A. |
| InChi | InChI=1S/C21H19ClN4O/c1-21(2,27)13-26-20-16(18(25-26)14-9-5-3-6-10-14)17(22)19(23-24-20)15-11-7-4-8-12-15/h3-12,27H,13H2,1-2H3 |
| InChiKey | TVNSCKMVODVWRS-UHFFFAOYSA-N |
| CID | 66576257 |
| Short Description | CLRN1-N48K stabilizer |
