Risdiplam

Name: Risdiplam
Brand: Tocris Bioscience
SKU: 7916

Tocris Bioscience, part of Bio-Techne | Catalog # 7916

COA / SDS

Selective SMN2 gene splicing modifier

Key Product Details

Description:	Selective SMN2 gene splicing modifier
Chemical Name:	7-(4,7-Diazaspiro[2.5]oct-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-4H-pyrido[1,2-a]pyrimidin-4-one
Purity:	≥98% (HPLC)
Molecular Weight:	401.47

View Full Technical Details for Risdiplam

Technical Data
Citations

Biological Activity for Risdiplam

Risdiplam is a selective survival of motor neuron-2 (SMN2) pre-mRNA splicing modifier. It potently enhances SMN2 splicing in vitro (EC_1.5X = 4 nM) and increases SMN protein in both brain (up to 100%) and muscle tissues (up to 49%) of transgenic mouse models of spinal muscular atrophy (SMA). Risdiplam dose-dependently increases the number of vesicular glutamate transporter 1 (vGlut1) proprioceptive inputs on motor neurons, the number of L3 to L5 motor neurons, the percentage of fully innervated neuromuscular junctions, and muscle size in the extensor digitorum longus muscles in SMN Δ7 mice.

Technical Data for Risdiplam

M. Wt	401.47
Formula	C₂₂H₂₃N₇O
Storage	Store at -20°C
Purity	≥98% (HPLC)
CAS Number	1825352-65-5
PubChem ID	118513932
InChi Key	ASKZRYGFUPSJPN-UHFFFAOYSA-N
Smiles	O=C1C=C(N=C2C=CC(=CN12)N3CCNC4(C3)CC4)C5=NN6C=C(N=C6C(=C5)C)C

The technical data provided above is for guidance only. For batch specific data refer to the Certificate of Analysis.

Tocris products are intended for laboratory research use only, unless stated otherwise.

Solubility for Risdiplam

	Solvent	Max Conc. mg/mL	Max Conc. mM
Solubility
Solubility	1eq. HCl	40.15	100 with gentle warming

Preparing Stock Solutions for Risdiplam

The following data is based on the product molecular weight 401.47.

Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Concentration / Solvent Volume / Mass	1 mg	5 mg	10 mg
1 mM	2.49 mL	12.45 mL	24.91 mL
5 mM	0.50 mL	2.49 mL	4.98 mL
10 mM	0.25 mL	1.25 mL	2.49 mL
50 mM	0.05 mL	0.25 mL	0.50 mL

Molarity Calculator

Calculate the mass, volume, or concentration required for a solution.

Mass

Concentration

Volume

M. Wt*

g/mol

*When preparing stock solutions always use the batch-specific molecular weight of the product found on the vial label and CoA (available online).

Dilution Calculator

Calculate the dilution required to prepare a stock solution.

Concentration 1 (C1)

Volume 1 (V1)

Concentration 2 (C2)

Volume 2 (V2)

Reconstitution Calculator

The reconstitution calculator allows you to quickly calculate the volume of a reagent to reconstitute your vial. Simply enter the mass of reagent and the target concentration and the calculator will determine the rest.

Volume (to add to vial)

Mass (in vial)

Desired Reconstitution Concentration

References for Risdiplam

References are publications that support the biological activity of the product.

Paik Risdiplam: A Review in Spinal Muscular Atrophy. CNS Drugs 2022 PMID: 35284988
Martin Screening strategies for identifying RNA- and ribonucleoprotein-targeted compounds. Trends Pharmacol.Sci. 2021 PMID: 34215444
Poirier Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol.Res.Perspect 2018 PMID: 30519476
Zhao SMN protein is required throughout life to prevent spinal muscular atrophy disease progression. Hum.Mol.Genet. 2021 PMID: 34368854
Markati Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA). Expert Opin.Investig.Drugs 2022 PMID: 35316106
Ratni Discovery of Risdiplam, a selective survival of motor neuron-2 (SMN2) gene splicing modifier. for the treatment of spinal muscular atrophy (SMA). J.Med.Chem. 2018 PMID: 30044619