RG7800
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MedKoo CAT#: 526734

CAS#: 1449598-06-4

Description: RG7800, also known as RO6885247 is a potent and selective SMN2 splicing modifier, which is currently under human clinical trials for the treatment of Spinal Muscular Atrophy (SMA). SMA is a genetic disorder caused by the mutation or deletion of the survival of motor neuron (SMN1) gene. It affects one in approximately 10,000 live births and in the most severe forms is assocaited with a high rate of childhood mortality. RG7800 exhibits excellent pharmacokinetic and in vivo efficacy and had a favorable safety profile. RG7800 corrects alternative splicing of the human SMN2 gene in the brain of transgenic SMA model mice, leading to an increase of the SMN protein in the brain.


Price and Availability

Size Price Shipping out time Quantity
10mg USD 90 Same day
25mg USD 150 Same day
50mg USD 250 Same day
100mg USD 450 Same day
200mg USD 750 Same day
500mg USD 1750 Same day
1g USD 2950 Same day
2g USD 4950 Same day
5g USD 8450 Same day
Inquire bulk and customized quantity

Pricing updated 2020-10-28. Prices are subject to change without notice.

RG7800, purity > 98%, is in stock. The same day shipping out after order is received. Note: the estimated shipping out time for order > 5g may be 2 weeks.


Chemical Structure

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Theoretical Analysis

MedKoo Cat#: 526734
Name: RG7800
CAS#: 1449598-06-4
Chemical Formula: C24H28N6O
Exact Mass: 416.2325
Molecular Weight: 416.529
Elemental Analysis: C, 69.21; H, 6.78; N, 20.18; O, 3.84


Synonym: RG7800; RG-7800; RG 7800; RO6885247; RO-6885247; RO 6885247.

IUPAC/Chemical Name: 2-(4-ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl)-9-methyl-7-(1-methylpiperidin-4-yl)-4H-pyrido[1,2-a]pyrimidin-4-one

InChi Key: GYFRQCMDLBNZSF-UHFFFAOYSA-N

InChi Code: InChI=1S/C24H28N6O/c1-5-19-22-11-21(27-30(22)13-16(3)25-19)20-12-23(31)29-14-18(10-15(2)24(29)26-20)17-6-8-28(4)9-7-17/h10-14,17H,5-9H2,1-4H3

SMILES Code: O=C1C=C(C2=NN3C(C(CC)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(C)CC5)C=C4C


Technical Data

Appearance:
Solid powder

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

Certificate of Analysis:

Safety Data Sheet (SDS):

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, not in water

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


Additional Information

Spinal muscular atrophy (SMA) is the leading genetic cause of infant and toddler mortality, and there is currently no approved therapy available. SMA is caused by mutation or deletion of the survival motor neuron 1 (SMN1) gene. These mutations or deletions result in low levels of functional SMN protein. SMN2, a paralogous gene to SMN1, undergoes alternative splicing and exclusion of exon 7, producing an unstable, truncated SMN∆7 protein.


References

1: Ratni H, Karp GM, Weetall M, Naryshkin NN, Paushkin SV, Chen KS, McCarthy KD,
Qi H, Turpoff A, Woll MG, Zhang X, Zhang N, Yang T, Dakka A, Vazirani P, Zhao X,
Pinard E, Green L, David-Pierson P, Tuerck D, Poirier A, Muster W, Kirchner S,
Mueller L, Gerlach I, Metzger F. Specific correction of alternative survival
motor neuron 2 (SMN2) splicing by small molecules: Discovery of a potential novel
medicine to treat spinal muscular atrophy. J Med Chem. 2016 Jun 14. [Epub ahead
of print] PubMed PMID: 27299419.