WARNING: This product is for research use only, not for human or veterinary use.
Hodoodo CAT#: H561863
CAS#: 1825352-65-5
Description: Risdiplam, also known as RG7916 and RO7034067, is a gene splicing modulator (neuromuscular disease).
Hodoodo Cat#: H561863
Name: Risdiplam
CAS#: 1825352-65-5
Chemical Formula: C22H23N7O
Exact Mass: 401.20
Molecular Weight: 401.470
Elemental Analysis: C, 65.82; H, 5.77; N, 24.42; O, 3.99
Synonym: Risdiplam; RG7916; RG-7916; RG 7916; RO703406; RO-7034067; RO 7034067;
IUPAC/Chemical Name: 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-4H-pyrido[1,2-a]pyrimidin-4-one
InChi Key: ASKZRYGFUPSJPN-UHFFFAOYSA-N
InChi Code: InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3
SMILES Code: O=C1C=C(C2=NN3C(C(C)=C2)=NC(C)=C3)N=C4N1C=C(N(C5)CCNC65CC6)C=C4
Appearance: Solid powder
Purity: >98% (or refer to the Certificate of Analysis)
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility: Soluble in DMSO
Shelf Life: >2 years if stored properly
Drug Formulation: This drug may be formulated in DMSO
Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code: 2934.99.9001
More Info:
Biological target: | Risdiplam (RG7916) is a SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels. |
In vitro activity: | Risdiplam showed an average passive permeability in parental LLC‐PK1 cells of around 350 nm/s. Similar permeability was observed in L‐MDR1, L‐Mdra1, M‐BCRP, and M‐Bcrp cells in the presence of inhibitor. Risdiplam was found not to be a substrate of human MDR1 with an ER of 2.2, but a weak substrate of rodent Mdr1a (ER = 3.7). The predicted CSF‐to‐unbound plasma partition coefficient (pKp,uu) based on the rodent ER and model from is therefore 0.33. Risdiplam was a weak human BCRP substrate with an ER of 3.7 and a strong Bcrp substrate in rodents with an ER of 44. Reference: Pharmacol Res Perspect. 2018 Dec; 6(6): e00447. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6262736/ |
In vivo activity: | These findings were confirmed while dosing risdiplam: a similar dose‐dependent increase in SMN protein levels in brain and muscle (0.1 mg/kg/day: brain 28%, muscle 32%; 1 mg/kg/day, brain 206%, muscle 210%) was observed after 7 days of once daily dosing in SMNΔ7 mice (Study 4, n = 6 or 7 per dose) (Figure 7). Risdiplam also prolonged survival of SMNΔ7 mice (Study 5) when dosed once daily with risdiplam for 219 days. In Study 5, SMN protein level increases were measured relative to SMN levels in heterozygous control animals and a correlation was observed between increase in SMN protein in blood and in brain. In the same study, risdiplam was shown to improve phenotype and motor function, to prolong survival and to increase body weight gain in SMNΔ7 mice. Reference: Pharmacol Res Perspect. 2018 Dec; 6(6): e00447. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6262736/ |
Solvent | Max Conc. mg/mL | Max Conc. mM | |
---|---|---|---|
Solubility | |||
DMSO | 2.0 | 4.98 | |
Ethanol | 2.0 | 4.98 | |
Water | 1.0 | 2.49 |
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 | 1.15 mL | 5.76 mL | 11.51 mL |
5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
50 mM | 0.02 mL | 0.12 mL | 0.23 mL |
Formulation protocol: | 1. Poirier A, Weetall M, Heinig K, Bucheli F, Schoenlein K, Alsenz J, Bassett S, Ullah M, Senn C, Ratni H, Naryshkin N, Paushkin S, Mueller L. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447. doi: 10.1002/prp2.447. PMID: 30519476; PMCID: PMC6262736. |
In vitro protocol: | 1. Poirier A, Weetall M, Heinig K, Bucheli F, Schoenlein K, Alsenz J, Bassett S, Ullah M, Senn C, Ratni H, Naryshkin N, Paushkin S, Mueller L. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447. doi: 10.1002/prp2.447. PMID: 30519476; PMCID: PMC6262736. |
In vivo protocol: | 1. Poirier A, Weetall M, Heinig K, Bucheli F, Schoenlein K, Alsenz J, Bassett S, Ullah M, Senn C, Ratni H, Naryshkin N, Paushkin S, Mueller L. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447. doi: 10.1002/prp2.447. PMID: 30519476; PMCID: PMC6262736. |