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

LMI070 (Synonyms: NVS-SM1; Branaplam)

Cat. No.: HY-19620 Purity: 98.78%
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
Estimated Time of Arrival: December 31
5 mg USD 192 In-stock
Estimated Time of Arrival: December 31
10 mg USD 312 In-stock
Estimated Time of Arrival: December 31
50 mg USD 1200 In-stock
Estimated Time of Arrival: December 31
100 mg   Get quote  
200 mg   Get quote  

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Customer Review

  • Biological Activity

  • 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
Solvent & Solubility
In Vitro: 

DMSO : 6.12 mg/mL (15.55 mM; Need ultrasonic and warming)

Preparing
Stock Solutions
Concentration Solvent 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

SMILES

OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3

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

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This equation is commonly abbreviated as: C1V1 = C2V2

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× = ×
C1   V1   C2   V2

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

Cat. No.: HY-19620