SMN1 Overview
Molecule Name
Survival motor neuron protein
Synonyms
Survival Motor Neuron 1,SMN1
Drug Development Progress
Drugs in Clinical Trials9
Approved Drugs3
Highest Development StageApproved
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
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 Product List
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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 | First Brand Name | First Approved Country | First Indication | First Approved Company | First Approved 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 |