NVS-SM2 - ≥99% , CAS No.1562333-92-9

CAS: 1562333-92-9 Cat. No.: N647689 Summenformel: C₂₃H₃₀N₆O Molekulargewicht: 406.52

QUALITÄT & REINHEIT ≥99%

Storage

Store at -20°C

Shipped In

Ice chest + Ice pads

Übersicht

NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC 50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research

In Vitro

For NVS-SM2, the molecular mechanism of action is via stabilization of the transient double-strand RNA structure formed by the SMN2 pre-mRNA and U1 small nuclear ribonucleic protein (snRNP) complex. The binding affinity of U1 snRNP to the 5' splice site is increased in a sequence-selective manner, discrete from constitutive recognition. MCE has not independently confirmed the accuracy of these methods. They are for reference only.

In Vivo

NVS-SM2 (0.1-1 mg/kg; s.c.; for 30 days) treatment extends survival in a severe SMA mouse model. Pharmacokinetic analysis demonstrate that NVS-SM2 is readily available in the brain after IV and oral (PO) administration in mouse and rat with T max of 3 h after PO with 3 mg/kg in mice, and NVS-SM2 treatment induces a 1.5-fold increase in SMN protein levels in the mouse brain . MCE has not independently confirmed the accuracy of these methods. They are for reference only. Animal Model: Severe SMA miceDosage: 0.1 mg/kg and 1 mg/kg Administration: Subcutaneous injection; daily from day 2 to day 15, followed by every other day until day 30 Result: Extended survival in a severe SMA mouse model.

Form:Solid

IC50& Target:EC50: 5 nM (SMN)

Spezifikationen

Spezifikationen & Reinheit

≥99%

Biochemische und physiologische Mechanismen

NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC 50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NV

Storage

Store at -20°C

Verschickt in

Ice chest + Ice pads

Dieses Produkt erfordert Kühlkettenversand. Grundversand und andere Economy-Optionen sind nicht verfügbar.

Aktionsart

AGONIST

Reinheit

≥99%

Namen und Kennungen

Molekulargewicht

406.52

Chemische und physikalische Eigenschaften

• Löslichkeit: DMSO : 75 mg/mL (184.49 mM; Need ultrasonic)

Application Protocols

No validated application protocols are provided for this specific item.

General guidance for small-molecule screening:

• Stock preparation: Prepare 10–50 mM solutions in DMSO, sterile-filter if needed (0.22 µm PTFE), and aliquot to avoid repeated freeze–thaw.
• Plate handling: Use low-bind plates/tips; equilibrate frozen plates briefly at room temperature before rapid centrifugation to collect condensate.
• Assay setup: Maintain consistent final DMSO percentage across all wells; include vehicle and positive/negative controls.
• Data quality: Incorporate dilution series (e.g., 10-point, 3-fold) for robust curve fitting; monitor for edge effects and precipitation.
• Interference controls: Employ detergent controls and orthogonal readouts (fluorescence vs luminescence vs label-free) to identify assay artifacts.

For any specialized techniques (e.g., chemoproteomics pulldown, CETSA, BRET), develop fit-for-purpose protocols based on your platform. Always confirm item-specific parameters from the CoA/Spec Sheet.

Biological Roles

Item-specific biological targets, pathways, and roles are not provided.

• Known/putative targets: Not specified for this item; refer to CoA/Spec Sheet or primary literature associated with the code name if available.
• Typical use in discovery (general guidance):
  • Employed as a candidate modulator in biochemical or cell-based assays to interrogate signaling pathways, enzyme activities, transporters, or transcriptional responses.
  • Deployed in phenotypic screens to elicit cellular phenotypes; follow-up involves target deconvolution via affinity capture, CRISPR knockouts, or transcriptomic profiling.
• Off-target risks and assay interference:
  • Without structural information, assess for pan-assay interference (PAINS) or redox cycling using standard computational and experimental filters.
  • Evaluate cytotoxicity baselines (e.g., CellTiter-Glo, resazurin) across the same concentration range used in efficacy assays to ensure selectivity.

No medical or clinical claims are made; this product is supplied strictly for research use only.

Buffer Applications

Not typically applicable. NVS-SM2 is a discrete small molecule, not a buffering agent.

• Practical note (general guidance): If the compound must be used in aqueous buffers, pre-dissolve in a compatible organic solvent (commonly DMSO), then dilute into the target buffer (e.g., PBS, HEPES, Tris) while keeping final organic content low enough to avoid biological interference.
• Solubility and precipitation checks should be performed at the working pH and ionic strength of your buffer system.

No specific buffer recipes or pKa data are available for this item.

Green Alternatives

This section is typically most relevant to solvents or reagents used in bulk processes. NVS-SM2 is a discrete small-molecule library member rather than a process solvent or commodity reagent.

• Applicability: Limited. There is no direct “green replacement” for a specific library compound, as its structure-defined activity is unique.
• Greener handling strategies (general guidance):
  • Use minimal volumes and high-concentration stocks to reduce solvent consumption during screening.
  • Prefer greener diluents when compatible with assays (e.g., ethanol or water-based buffers) and minimize DMSO carryover where feasible.
  • Implement microplate-based workflows and acoustic dispensing to reduce plastic and solvent waste.
• Waste management:
  • Segregate halogenated vs non-halogenated solvent waste per institutional policy.
  • Consider on-plate quenching and direct analysis approaches to reduce workup volumes.

If you are optimizing a process that uses NVS-SM2 as a reagent or intermediate (uncommon), apply solvent selection guides (e.g., CHEM21, ACS GCI) to choose greener media and conditions compatible with the compound’s stability.

Pharmaceutical Uses

No pharmacopeial status, excipient role, or formulation use is specified for this product.

• Intended use: For research use only; not for human or veterinary use, diagnostic procedures, or clinical applications.
• If used as a tool compound in preclinical research (general guidance):
  • Consider formulating DMSO stocks for in vitro work and characterize stability (freeze–thaw, light, and oxidative stability) before extended studies.
  • For any non-clinical in vivo explorations in research settings, ensure independent verification of identity/purity and develop fit-for-purpose formulations (e.g., PEG400/saline, 0.5% methylcellulose) only after confirming solubility and stability. This is general guidance and not item-specific instruction.
• Regulatory note: This catalog item is not supplied under GMP and carries no compendial monograph.

All uses must remain within laboratory research settings.

Physical Properties

Item-specific physical constants are not provided in the product data and should be confirmed on the CoA/Spec Sheet.

• Appearance: Not specified for this item; refer to CoA/Spec Sheet.
• Melting point (MP): Not specified for this item; refer to CoA/Spec Sheet.
• Boiling point (BP): Not specified for this item; refer to CoA/Spec Sheet.
• Density: Not specified for this item; refer to CoA/Spec Sheet.
• Solubility: Not specified for this item; refer to CoA/Spec Sheet. General practice for screening compounds is to prepare DMSO stock solutions (literature/general guidance) and dilute into assay media.
• LogP/logD, pKa, refractive index: Not specified for this item; refer to CoA/Spec Sheet.

General considerations (literature/general guidance):

• Library molecules often show good solubility in polar aprotic solvents such as DMSO, DMF, or acetonitrile; aqueous solubility depends on ionizable functionality and salt form.
• If solid, an indicative MP can aid quick identity checks; if oil, LC–MS/HPLC purity is more informative.
• For new assays, perform a small-scale solubility screen across water, buffer (pH 6–8), DMSO, ethanol, and isopropanol to define working concentrations and avoid precipitation upon dilution.

Quality and Grades

Grade/purity is not specified in the product data and should be confirmed on the CoA/Spec Sheet.

• Grade/Purity: Not specified for this item; refer to CoA/Spec Sheet.
• Typical quality metrics for small-molecule library members (general guidance):
  • HPLC/UPLC purity (UV and/or MS detection), often >95% for screening-grade materials.
  • Identity confirmation by LC–MS and 1H NMR; HRMS for exact mass.
  • Residual solvents and water content (e.g., by GC and Karl Fischer) when relevant.
• Implications for research:
  • Screening-grade material is suitable for biochemical and cell-based assays, SAR exploration, and hit triage. For mechanistic studies or in vivo models, many groups re-verify purity/identity and, if needed, perform a quick repurification.
• Stabilizers/salt form: Not specified for this item; refer to CoA/Spec Sheet. Presence of a salt counterion can strongly influence solubility and storage stability.
• Documentation: Each shipment should be accompanied by a lot-specific CoA. Contact us if you require additional analytical data (chromatograms, spectra) for your QA workflows.

Reaction and Applications

Manufacturer Application details are not provided for this item. As a member of a small-molecule library, NVS-SM2 is typically used in discovery biology and chemistry workflows (general guidance):

• Primary application domains:
  • High-throughput or high-content screening (HTS/HCS) for target/pathway modulation.
  • Hit validation, orthogonal assay confirmation, and preliminary structure–activity relationship (SAR) development.
  • Phenotypic screening followed by chemoproteomic deconvolution to identify targets (when applicable).
• Reference standards and controls:
  • Can serve as an internal control or comparator in assays if its behavior is known in your platform (user-established).
• Practical tips:
  • Prepare concentrated DMSO stocks (e.g., 10–20 mM), aliquot to minimize freeze–thaw, and record exact lot concentration by qNMR or UV if chromophore is known (general practice).
  • Validate solubility/aggregation using dynamic light scattering (DLS) or detergent controls to rule out colloidal interference.
  • Employ counterscreens for redox cycling, PAINS motifs, or assay interference (e.g., luciferase, fluorescence quenchers) as part of hit triage.
• Synthetic chemistry use:
  • If used as a benchmarking substrate or probe in methods development, ensure identity/purity are independently verified for reproducibility.

Note: No specific reaction chemistry or mechanistic role can be assigned without the compound structure; consult the CoA/Spec Sheet for further details.

Reaction Conditions

Specific reaction conditions cannot be advised for NVS-SM2 absent structural information.

General guidance if using this compound as a substrate or probe in chemical reactions:

• Solvent: Start with DMSO, DMF, or acetonitrile for polar reactions; use toluene, THF, or 2-MeTHF for less polar contexts, conditional on solubility and stability.
• Temperature: Screen from 0–80°C as compatible with thermal stability; monitor by TLC/UPLC–MS.
• Atmosphere: Use inert atmosphere (N2/Ar) if the compound shows oxidation sensitivity (unknown here); default to inert for exploratory work.
• Catalysts/additives: Choose based on hypothesized functional groups; perform small-scale scouts first.
• Workup: Quench cautiously, extract with appropriate solvents, and avoid pH extremes until compatibility is known.
• Analytics: Track conversion/purity by LC–MS and orthogonal HPLC/UV; verify identity by NMR when feasible.

Expected yields, kinetics, and selectivity are inherently structure-dependent and cannot be provided for this item.

Safety and Handling

GHS and hazard statements are not provided for this specific item. Handle as a research chemical of unknown hazards.

• GHS classification, pictograms, signal word, H-statements: Not specified for this item; refer to SDS.
• Primary hazards (general): With unknown toxicological profile, avoid inhalation, ingestion, and skin/eye contact. Use in a chemical fume hood.
• PPE: Lab coat, safety glasses, and appropriate chemically resistant gloves (e.g., nitrile). For weighing fine powders, consider respiratory protection per institutional policy.
• First aid (general guidance; defer to SDS):
  • Inhalation: Move to fresh air; seek medical attention if symptoms occur.
  • Skin contact: Wash with soap and water; remove contaminated clothing.
  • Eye contact: Rinse cautiously with water for several minutes; seek medical advice.
  • Ingestion: Rinse mouth; seek medical attention.
• Incompatibilities: Unknown for this item. Keep away from strong oxidizers and acids/bases until compatibility is assessed.
• Environmental precautions: Prevent release to the environment; collect waste in appropriate, labeled containers.
• Thermal/chemical stability: Unknown for this item. Avoid prolonged exposure to heat, light, and moisture until stability is established.
• Always consult the Safety Data Sheet (SDS) for authoritative, item-specific safety measures.

Solvent Selection

No compound-specific solvation data are provided; selection should follow general best practices for small-molecule library compounds (literature/general guidance).

• Primary stock solvent: DMSO is typically preferred for 10–50 mM master stocks due to broad solvency and assay compatibility at ≤0.5% v/v.
• Alternative solvents: DMF, acetonitrile, methanol, ethanol, and isopropanol. For hydrophobic compounds, co-solvent blends (e.g., DMSO+acetonitrile) or gentle warming/sonication can help dissolution.
• Aqueous use: Pre-dissolve in an organic solvent before dilution into buffer/media. Maintain final organic content low (commonly ≤0.1–1% v/v) to protect biological assay integrity.
• Polarity and ionization: If a basic or acidic center is present (unknown here), pH adjustment or salt formation can enhance solubility. Evaluate solubility across pH 3–9 where compatible with your assay.
• Adsorption minimization: For low nM work, include inert carriers (e.g., 0.01–0.1% Tween 80 or 0.1% BSA where compatible) and use low-bind plastics or silanized glass to reduce loss to surfaces.
• Filtration: If particulates appear after dilution, filter through PTFE or PES (0.22 µm). Avoid cellulose esters if the compound is highly hydrophobic.
• Light/air sensitivity: Unknown for this item; prepare fresh aliquots, minimize air/light exposure, and consider amber vials.

Storage and Reconstitution

Item-specific storage guidance provided in the product data should be followed strictly.

• Storage conditions (from Product Data): Store at −20°C.
• Shipping (from Product Data): Shipped in an ice chest with ice pads.
• Container: Keep tightly closed in original, chemically compatible container. Protect from moisture and light until stability is known.
• Stability: Not specified for this item; refer to CoA/Spec Sheet. As general practice, limit freeze–thaw cycles by preparing single-use aliquots.
• Reconstitution (general guidance for small molecules):
  • Prepare concentrated stock solutions in anhydrous DMSO (e.g., 10–50 mM) unless compound-specific solubility dictates otherwise.
  • Warm gently (room temperature) and vortex/sonicate to aid dissolution; avoid excessive heat.
  • If aqueous use is required, dilute the organic stock into buffer/media with thorough mixing; keep final organic content as low as assay permits.
• Handling after reconstitution: Store aliquots at −20°C or −80°C as compatible with your workflow; minimize headspace and use amber vials to mitigate light exposure.
• Expiry/retention: Not specified for this item; refer to CoA/Spec Sheet. Reassess purity periodically by LC–MS/HPLC, especially after extended storage.

Structure and Identity

Brief overview: NVS-SM2 is listed as a small-molecule library member. Item-specific identifiers beyond CAS are not provided in the product data.

• CAS: 1562333-92-9 (provided)
• Chemical name: Not specified for this item; refer to CoA/Spec Sheet.
• Molecular formula: Not specified for this item; refer to CoA/Spec Sheet.
• Molecular weight: Not specified for this item; refer to CoA/Spec Sheet.
• SMILES: Not specified for this item; refer to CoA/Spec Sheet.
• InChI / InChIKey: Not specified for this item; refer to CoA/Spec Sheet.
• Synonyms/Code names: NVS-SM2 (catalog/designation)

Structural features (general guidance):

• Without a structure, functional groups, ring systems, and stereochemistry cannot be confirmed for this item.
• For identity confirmation, consult the product’s Certificate of Analysis (CoA) and accompanying spectral data (e.g., 1H/13C NMR, HRMS, LC–MS, HPLC purity).

Synthetic Utility

Without a disclosed structure, NVS-SM2 should be viewed primarily as a screening/test article rather than a synthetic building block.

• Building-block use: Not established for this item; functional handles and reactivity are unknown without the structure. Refer to CoA/Spec Sheet.
• Typical roles for analogous items (general guidance):
  • Analytical standard or internal reference in method development if the analyte is relevant to your workflow.
  • Benchmark comparator in reaction discovery if it presents a known reactive motif (user-verified).
• If derivatization is intended:
  • Confirm presence of modifiable functional groups via NMR/MS and, if available, single-crystal or computational assignment.
  • Establish protective group strategy based on observed acid/base sensitivity and oxidation profile.

In summary, treat this material as a research probe; no specific synthetic transformations can be recommended without structural information.

Target Specificity

No target, epitope, or specificity information is provided for NVS-SM2.

• Intended scope: Small-molecule library member for general research.
• Target/Pathway: Not specified for this item; refer to CoA/Spec Sheet or primary literature if the code name is associated with a published tool compound.

No antibody/biologic attributes (clone, isotype, species reactivity) apply to this product.