Select Language

SMN1

SMN1 Overview

English Name	Survival motor neuron protein	Approved Drugs	3
Drugs in Clinical Trials	9	Target Synonym	Survival Of Motor Neuron 1 Isoform D345，Survival Of Motor Neuron 1 Isoform D2A2B3457，Survival Of Motor Neuron 1 Isoform D347，SMN1 more，SMN1，Survival Of Motor Neuron 1 Isoform D3457，Survival Of Motor Neuron 1， Telomeric，Gemin-1，Tudor Domain Containing 16A，SMNT，SMN，Survival Motor Neuron 1 Protein，Survival Motor Neuron Protein，T-BCD541，TDRD16A，BCD541，GEMIN1，SMA1，SMA2，SMA3，SMA4，SMA@，SMA，SMN Complex Proteins，Component of gems 1，SMN2，SMNC，Survival Of Motor Neuron 1 Isoform D2B3457，Survival Of Motor Neuron 1 Isoform D2A3457，Spinal Muscular Atrophy (Werdnig-Hoffmann Disease， Kugelberg-Welander Disease)，Survival Of Motor Neuron 1 Isoform D2A2B345
Highest Development Stage	Approved

English Name	Survival motor neuron protein
Target Synonym	Survival Of Motor Neuron 1 Isoform D345，Survival Of Motor Neuron 1 Isoform D2A2B3457，Survival Of Motor Neuron 1 Isoform D347，SMN1 more，SMN1，Survival Of Motor Neuron 1 Isoform D3457，Survival Of Motor Neuron 1， Telomeric，Gemin-1，Tudor Domain Containing 16A，SMNT，SMN，Survival Motor Neuron 1 Protein，Survival Motor Neuron Protein，T-BCD541，TDRD16A，BCD541，GEMIN1，SMA1，SMA2，SMA3，SMA4，SMA@，SMA，SMN Complex Proteins，Component of gems 1，SMN2，SMNC，Survival Of Motor Neuron 1 Isoform D2B3457，Survival Of Motor Neuron 1 Isoform D2A3457，Spinal Muscular Atrophy (Werdnig-Hoffmann Disease， Kugelberg-Welander Disease)，Survival Of Motor Neuron 1 Isoform D2A2B345
Approved Drugs	3
Drugs in Clinical Trials	9
Highest Development Stage	Approved

SMN1 Product List

Product Category
Protein (1)
Product Status
Launched (1)
Species
Human (1)
Tag
His Tag (1)
Conjugate
Unconjugated (1)

Cat. No.	Species	Host	Product Description	Structure	Purity	Feature
SM1-H5145	Human	E. coli	Human SMN1 Protein, His Tag

Total: 1

SMN1 Background

SMN1 is produced chiefly by the SMN1 gene, located on the telomeric portion of chromosome 5q. SMN1 protein is one of a group of proteins called the SMN complex, which is important for the maintenance of specialized nerve cells called motor neurons. In cells, the SMN complex plays an important role in processing mRNA. The SMN complex helps to assemble the cellular machinery needed to process pre-mRNA. The SMN complex is also important for the development of specialized outgrowths from nerve cells called dendrites and axons. Deletion or mutation of the SMN1 gene results in a reduced level of full-length SMN protein and manifests as a range of neuromuscular phenotypes in humans as the disease spinal muscular atrophy (SMA).

SMN1 Synonyms

Survival Motor Neuron 1,SMN1

Clinical Drug Information

English Name	Research Code	Highest Development Stage	Company	Indication	Clinical Trial
vesemnogene lantuparvovec	AAV-hSMN1	Phase 3 Clinical	Lantu Biopharma Inc	Muscular Atrophy, Spinal	Details
Salanersen	BIIB-115; ION-306	Phase 3 Clinical	Ionis Pharmaceuticals Inc	Muscular Atrophy, Spinal	Details
Branaplam	LMI-070; NVP-LMI070-AAA; NVP-LMI070-NX; NVS-SM1	Phase 2 Clinical	Novartis Pharma Ag	Huntington Disease; Muscular Atrophy, Spinal	Details
GB-221 (GEMMA Biotherapeutics)	GB-221	Phase 2 Clinical	Gemma Biotherapeutics Inc	Spinal Muscular Atrophies of Childhood	Details
ANB-004	ANB-004	Phase 2 Clinical	BIOCAD JSC	Muscular Atrophy, Spinal	Details
EXG001-307	EXG001-307	Phase 2 Clinical	Exegenesis Bio Inc	Muscular Atrophy, Spinal	Details
SMN-C3	SMN-C3	Phase 1 Clinical	F. Hoffmann-La Roche Ltd, Ptc Therapeutics Inc	Muscular Atrophy, Spinal	Details
SKG-0201	SKG-0201; SKG0201	Phase 1 Clinical	Skyline Therapeutics (Hong Kong) Ltd	Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood	Details
GCB-001	GCB-001; GCB001	Clinical	Shanghai Jinke Bo Biotechnology Co Ltd	Spinal Muscular Atrophies of Childhood	Details

Marketed Drug Information

English Name	Research Code	Highest Development Stage	Company	Earliest Brand Name	Earliest Approval Country	Earliest Indication	Earliest Approval Company	Earliest Approval Date	Indication	Clinical Trial
Risdiplam	RG-7916; RO-7034067	Approved	Ptc Therapeutics Inc	EVRYSDI, 艾满欣	United States	Muscular Atrophy, Spinal	Genentech Inc	2020-08-07	Muscular Atrophy, Spinal	Details
Nusinersen sodium	BIIB-058; ISIS-396443	Approved	Ionis Pharmaceuticals Inc	Spinraza	United States	Muscular Atrophy, Spinal	Biogen Idec	2016-12-23	Muscular Atrophy, Spinal	Details
Onasemnogene abeparvovec	AVXS-101; scAAV-9.CB.SMN; OAV101; rAAV-9	Approved	Novartis Gene Therapies Inc, Novartis Pharma Ag	Zolgensma, Itvisma	United States	Muscular Atrophy, Spinal	Novartis Gene Therapies Inc	2019-05-24	Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood	Details