smn — TargetMol Chemicals

Cat No.	Product Name	Synonyms	Targets
T12935	SMN-C3	MV8T2MCK57	DNA/RNA Synthesis
	SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA).		DNA/RNA Synthesis
T73475	SMN-C2		DNA/RNA Synthesis
	SMN-C2 is a selective regulator of SMN2 gene splicing, a risdiplam analogue, a selective RNA-binding ligand that regulates pre-mRNA splicing and acts by binding to SMN2 pre-mRNA.SMN-C2 has the potential to be used in the...		DNA/RNA Synthesis
T4641	Branaplam	LMI 070,NVS-SM1	Others , Potassium Channel , DNA/RNA Synthesis
	Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.		Others , Potassium Channel , DNA/RNA Synthesis
T34664	SMN2-Stablizer-27	SMN2-Stablizer 27,SMN2 Stablizer-27
	SMN2-Stablizer-27 is a stablizer of survival of motor neuron 2 (SMN2), and a post-translationally stablizing SMN protein for the treatment of spinal muscular atrophy (SMA).
T16902	SMND-309		Others
	SMND-309 is a metabolite of salvianolic acid B. It also shows neuroprotective effects in cultured neurons and in permanent middle cerebral artery occlusion rats.		Others
T16757	Risdiplam	RO7034067,RG7916	DNA/RNA Synthesis
	Risdiplam (RO7034067) (RG7916) is orally administered. Risdiplam is a centrally and peripherally distributed SMN2 pre-mRNA splicing modifier which increases survival motor neuron (SMN) protein levels[1].		DNA/RNA Synthesis
T3336	Rg3039	PF-06687859	Others
	Rg3039 (PF-06687859) is a potent DcpS inhibitor. DcpS is a therapeutic target for spinal muscular atrophy. Spinal muscular atrophy (SMA) is caused by deletion or mutation of both copies of the SMN1 gene which produces an...		Others
T33764	NVS-SM2
	NVS-SM2 is an effective small molecule splicing enhancer of SMN2 with oral activity. NVS-SM2 showed increased exon 7 inclusion and upregulated SMN protein expression in SMA fibroblasts and SMN cells Δ7 5025 mouse myoblas...
T62381	Branaplam hydrochloride
	Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM). branaplam hydrochloride inhibits hERG (IC50: 6.3 μM). In a mouse model of severe spinal musc...
T64288	RG7800 hydrochloride
	RG7800 hydrochloride is an orally active SMN2 splicing modulator that acts on SMN2 splicing (EC1.5x: 23 nM) and SMN proteins (EC1.5x: 87 nM).
T35594	Cuspin-1
	The Survival of Motor Neurons (SMN) protein participates in RNA splicing. Decreases in SMN, typically a consequence of defects in the smn1 gene, result in the death of motor neurons and lead to the neurodegenerative dise...
T28067	ML372	ML 372,ML-372
	ML372 is a potent and selective SMN Modulator (EC50 = 12 nM, 325% increase inSMN2) with good potency, pharmacokinetics, tolerance, and CNS penetration.
T38656	Nusinersen
	Nusinersen, an antisense oligonucleotide drug, modifies the pre-messenger RNA (pre-mRNA) splicing of the SMN2 gene, thereby enhancing the production of the full-length Survival Motor Neuron (SMN) protein.
T26893	Branaplam mesylate (1562338-42-4 free base)	LMI070,Branaplam mesylate,LMI 070,LMI-070
	Branaplam is a highly potent, selective and orally active SMN2 splicing modulator. Branaplam increases the amount of functional SMN protein produced by the SMN2 gene through modifying its splicing.
T78046	RG7800 tetrahydrochloride	RO6885247 tetrahydrochloride	DNA/RNA Synthesis
	RG7800 hydrochloride is an orally active modulator of SMN2 splicing, exhibiting EC50 values of 23 nM for SMN2 splicing and 87 nM for SMN protein production. This compound holds promise as a therapeutic agent for spinal m...		DNA/RNA Synthesis
T12718	RG7800 hydrochloride (1449598-06-4 free base)	RO6885247 hydrochloride,RG7800 hydrochloride	Others
	RG7800 hydrochloride is an orally active modulator of SMN2 splicing.		Others