Home Cart Sign in  
Chemical Structure| 1562338-42-4 Chemical Structure| 1562338-42-4

Structure of Branaplam
CAS No.: 1562338-42-4

Chemical Structure| 1562338-42-4

*Storage: {[sel_prStorage]}

*Shipping: {[sel_prShipping]}

,{[proInfo.pro_purity]}

LMI070 is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

Synonyms: LMI070; NVS-SM1

4.5 *For Research Use Only !

{[proInfo.pro_purity]}
Cat. No.: {[proInfo.prAm]} Purity: {[proInfo.pro_purity]}

Change View

Size Price VIP Price

US Stock

Global Stock

In Stock
{[ item.pr_size ]} Inquiry {[ getRatePrice(item.pr_usd,item.pr_rate,item.mem_rate,item.pr_is_large_size_no_price, item.vip_usd) ]}

US Stock: ship in 0-1 business day
Global Stock: ship in 5-7 days

  • {[ item.pr_size ]}

In Stock

- +

Please Login or Create an Account to: See VIP prices and availability

US Stock: ship in 0-1 business day
Global Stock: ship in 2 weeks

  • 1-2 Day Shipping
  • High Quality
  • Technical Support
Product Citations

Alternative Products

Product Details of Branaplam

CAS No. :1562338-42-4
Formula : C22H27N5O2
M.W : 393.48
SMILES Code : OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3
Synonyms :
LMI070; NVS-SM1
MDL No. :MFCD29472288
InChI Key :STWTUEAWRAIWJG-UHFFFAOYSA-N
Pubchem ID :135565042

Safety of Branaplam

GHS Pictogram:
Signal Word:Warning
Hazard Statements:H302-H315-H319-H335
Precautionary Statements:P261-P305+P351+P338

Isoform Comparison

Biological Activity

Description
Branaplam, identified as LMI070 or NVS-SM1, is distinguished by its potent, selective, and orally bioavailable qualities as an SMN2 splicing modulator, displaying an EC50 of 20 nM. It inhibits the hERG channel with an IC50 of 6.3 μM, enhances full-length SMN protein levels, and improves survival in a severe spinal muscular atrophy (SMA) mouse model[1].[2].

In Vitro:

Cell Line
Concentration Treated Time Description References
Lymphoblastoid cell lines (LCLs) 50 nM 24 hours To confirm HTT splice modulation, producing two splice products PMC11015039
HeLa cells 10 nM 24 hours Using minigenes to assess the effect of Branaplam on splicing of specific exons, results showed Branaplam could promote inclusion of certain exons. PMC10325915
GM03813 Type I SMA patient fibroblasts 40 nM 24 hours To evaluate the effect of Branaplam on SMN2 exon 7 splicing, results showed high concentration of Branaplam significantly promoted inclusion of SMN2 exon 7. PMC10325915
SH-SY5Y human neuroblastoma cells 100 nM 24 hours To investigate the effect of branaplam on HTT gene expression, results showed a reduction in HTT transcript levels PMC8894458
RPE1-AAVS1-CAG115 cells 25 nM, 100 nM 4-6 weeks To investigate the effect of Branaplam on CAG repeat instability PMC11015039
HD patient fibroblasts 5-125 nM 96 hours To evaluate the effect of branaplam on mHTT protein levels, results showed mHTT protein levels reduced to 70% of normal levels PMC8894458
HEK293T cells 100 nM To study the mechanism of HTT pseudoexon splice modulation PMC11015039
HeLa cells 50 nM To study the effect of Branaplam on splicing of SMN2 exon 7 PMC10904865

In Vivo:

Species
Animal Model
Administration Dosage Frequency Description References
BacHD mice Mice harboring the full-length human mHTT gene Oral 10 mg/kg or 50 mg/kg Single dose, effects assessed at 8, 24, and 48 hours To evaluate the effect of branaplam on mHTT mRNA and protein levels, results showed a 60-70% reduction in mHTT transcript levels and a 40-45% reduction in protein levels PMC8894458

Protocol

Bio Calculators
Preparing Stock Solutions 1mg 5mg 10mg

1 mM

5 mM

10 mM

2.54mL

0.51mL

0.25mL

12.71mL

2.54mL

1.27mL

25.41mL

5.08mL

2.54mL

References

 

Historical Records