RG7800 (tetrahydrochloride)

RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.

Product Specifications

Product Name Alternative

RO6885247 (tetrahydrochloride)

UNSPSC

12352005

Target

DNA/RNA Synthesis

Related Pathways

Cell Cycle/DNA Damage

Applications

Neuroscience-Neuromodulation

Field of Research

Neurological Disease

Purity

99.79

Solubility

H2O : ≥ 113.5 mg/mL

Smiles

CC1=CC(C2CCN(C)CC2)=CN3C1=NC(C4=NN(C=C(C)N=C5CC)C5=C4)=CC3=O.[H]Cl.[H]Cl.[H]Cl.[H]Cl

Molecular Formula

C24H32Cl4N6O

Molecular Weight

562.36

References & Citations

[1]Ratni H, et al. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. J Med Chem. 2016 Jul 14;59 (13) :6086-100.

Shipping Conditions

Room Temperature

Storage Conditions

4°C (Powder, sealed storage, away from moisture)

Scientific Category

Reference compound1

Clinical Information

Phase 1

Citation 01

Nature. 2021 Aug;596 (7871) :291-295.|Patent. US20230340498A1.|bioRxiv. 2020 Feb.|J Mol Med (Berl) . 2019 Aug;97 (8) :1183-1193.|Life Sci Alliance. 2019 Mar 25;2 (2) :e201800268.