1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis

LMI070 (Synonyms: NVS-SM1; Branaplam)

Cat. No.: HY-19620 Purity: 98.78%
Data Sheet SDS Handling Instructions

LMI070 (NVS-SM1) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

For research use only. We do not sell to patients.
LMI070 Chemical Structure

LMI070 Chemical Structure

CAS No. : 1562338-42-4

Size Price Stock Quantity
Free Sample (0.5-1 mg)   Apply now  
10 mM * 1 mL in DMSO USD 211 In-stock
5 mg USD 192 In-stock
10 mg USD 312 In-stock
50 mg USD 1200 In-stock
100 mg   Get quote  
200 mg   Get quote  

* Please select Quantity before adding items.

Customer Review

Featured Recommendations

  • Biological Activity

  • Protocol

  • Technical Information

  • Purity & Documentation

  • References

Description

LMI070 (NVS-SM1) is a highly potent, selective and orally active small molecule SMN2 splicing modulator. Target: RNA splicing in vitro: NVS-SM1 shows high plasma exposure, good bioavailability and, notably, good distribution to the brain, a primary target tissue NVS-SM1 exhibits efficacy at lower doses and exposures. NVS-SM1 shows robust activity across disease-relevant induced pluripotent stem cell (iPSc)-derived neurons. [1] invivo : To evaluate the efficacy of NVS-SM1, we used the SMNΔ7 mouse model, which displays a severe phenotype. In the specific colony used for this study, death typically occurs before postnatal day 15. We were pleased to observe that, in addition to a dose-dependent elevation of SMN protein in the brain, oral administration of NVS-SM1 improved body weight and extended lifespan, with 50% of the animals in the 1 mg per kg body weight group and 62% of animals in the 3 mg per kg body weight group showing increased survival. [1]

Clinical Trial
View MoreCollapse
References
Preparing Stock Solutions
Concentration Volume Mass 1 mg 5 mg 10 mg
1 mM 2.5414 mL 12.7071 mL 25.4143 mL
5 mM 0.5083 mL 2.5414 mL 5.0829 mL
10 mM 0.2541 mL 1.2707 mL 2.5414 mL
Please refer to the solubility information to select the appropriate solvent.
References
Molecular Weight

393.48

Formula

C₂₂H₂₇N₅O₂

CAS No.

1562338-42-4

Storage
Powder -20°C 3 years
  4°C 2 years
In solvent -80°C 6 months
  -20°C 1 month
Shipping

Room temperature in continental US; may vary elsewhere

Solvent & Solubility

DMSO: 6.12 mg/mL

* "<1 mg/mL" means slightly soluble or insoluble. "≥" means soluble, but saturation unknown.

Inquiry Online

Your information is safe with us. * Required Fields.

Product name

 

Salutation

Applicant name *

 

Email address *

Phone number *

 

Organization name *

Country *

 

Requested quantity *

Remarks

Bulk Inquiry

Inquiry Information

Product Name:
LMI070
Cat. No.:
HY-19620
Quantity: