Catalog No. Size PriceQuantity
M13134-2 Contact sales@xcessbio.com for quotation $100
M13134-10 Contact sales@xcessbio.com for quotation $100


Homocarbonyltopsentin (PK4C9) is a small-molecule TSL2-binding compound, binds to pentaloop conformations of TSL2 and promotes a shift to triloop conformations that display enhanced SMN2 exon 7 (E7) splicing with EC50 value of 16 μM.

Product information

CAS Number: 172286-77-0

Molecular Weight: 370.36

Formula: C21H14N4O3



Chemical Name: 3-[5-(1H-indole-3-carbonyl)-1H-imidazole-2-carbonyl]-1H-indol-6-ol

Smiles: OC1=CC2NC=C(C=2C=C1)C(=O)C1NC(=CN=1)C(=O)C1=CNC2C=CC=CC=21


InChi: InChI=1S/C21H14N4O3/c26-11-5-6-13-15(9-23-17(13)7-11)20(28)21-24-10-18(25-21)19(27)14-8-22-16-4-2-1-3-12(14)16/h1-10,22-23,26H,(H,24,25)

Technical Data

Appearance: Solid Power

Purity: ≥98% (or refer to the Certificate of Analysis)

Solubility: DMSO : 125 mg/mL (337.51 mM; Need ultrasonic).

Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis

Storage Condition: Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.

Shelf Life: ≥12 months if stored properly.

Stock Solution Storage: 0 - 4 oC for 1 month or refer to the Certificate of Analysis.

Drug Formulation: To be determined

HS Tariff Code: 382200

How to use

In Vitro:

Homocarbonyltopsentin (PK4C9) (10-40 μM; 24 hours) shows an up to 5.2-fold decrease in the expression of E7-excluding SMN2 isoforms, and up to three-fold increase in E7-including isoforms in GM03813C cells. Homocarbonyltopsentin (PK4C9) (40 μM; 24 hours) increased 1.5-fold SMN protein expression compared to GM03813C cells treated with DMSO.


  1. Garcia-Lopez A, et al. Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes. Nat Commun. 2018 May 23;9(1):2032.

Products are for research use only. Not for human use.

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