Ulefnersen sodium , CAS No.2589926-27-0

CAS: 2589926-27-0 Cat. No.: U1426641 Formula: C₂₃₀H₃₀₂N₆₈O₁₂₆P₁₉S₁₃Na₁₉ Molecular Weight: 7477.40

Storage

Store at -20°C,Desiccated

Shipped In

Ice chest + Ice pads

Overview

Ulefnersen sodium (ION363) is an Antisense Oligonucleotide ( ASO ) directed against the 6th intron of the fused-in sarcoma ( FUS ) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS).

Specifications

Storage

Store at -20°C,Desiccated

Shipped In

Ice chest + Ice pads

This product requires cold chain shipping. Ground and other economy services are not available.

Names and Identifiers

Molecular Weight

7477.40

Application Protocols

Item-specific facts (from Product Data)

• No tested applications or recommended dilutions are provided.

General protocols (not item-specific; adapt after confirming solubility/stability)

• Stock preparation for screening
  • Weigh accurately in a dry environment.
  • Dissolve to 10–50 mM in dry DMSO (or water/buffer if freely soluble). Vortex and, if needed, sonicate briefly.
  • Filter if required and aliquot into low‑binding microtubes; store at −20°C.
• Working solution
  • Dilute into assay buffer immediately before use to the desired concentration. Maintain final co‑solvent ≤0.5–1% v/v unless the assay tolerates more.
• Analytical QC prior to use
  • Verify identity and purity by LC‑MS/HPLC each time a new lot is opened. Confirm concentration by UV (if chromophore known) or quantitative NMR/gravimetry.
• Stability checks
  • Avoid repeated freeze–thaw cycles; thaw once, subdivide into single‑use aliquots. Track time on bench and in autosampler; discard after pre‑set stability window.

Please refer to the CoA/Spec Sheet for any item‑specific instructions if available.

Biological Roles

Item-specific facts (from Product Data)

• No target, pathway, or biological role information is provided for Ulefnersen sodium.

General considerations (not item-specific; research only)

• Compounds supplied as sodium salts often carry anionic functionality that can influence membrane permeability, protein binding, and distribution in biochemical assays. These properties can affect observed potency and selectivity profiles.
• In vitro evaluation commonly includes:
  • Enzyme or receptor binding assays (biochemical or biophysical),
  • Cell‑free functional readouts (e.g., reporter systems), and
  • ADME‑relevant screens such as plasma protein binding and microsomal stability.
• If the compound is intended as a tool molecule, establish on‑target engagement using orthogonal assays (e.g., thermal shift, SPR/ITC, or activity rescue experiments) once a putative target is known.

Caveats

• Without validated structural and target data, any assignment of mechanism of action or biological pathway would be speculative. Please consult the CoA/Spec Sheet or primary literature for definitive biological characterization, if available.

Buffer Applications

Item-specific facts (from Product Data)

• None provided.

General guidance for preparing stocks (not item-specific)

• If water‑soluble (common for sodium salts), prepare concentrated stocks in:
  • Neutral to mildly basic buffers (e.g., 10–50 mM HEPES, pH 7.4–8.0; or PBS) to maintain ionized state and minimize precipitation upon dilution.
  • For limited solubility, dissolve first in a minimal volume of DMSO, then dilute into buffer with vigorous mixing; keep final DMSO ≤0.5–1% v/v in sensitive assays.
• Filter through 0.22 µm low‑binding membranes to ensure clarity for optical assays.
• Check stability by LC‑MS or HPLC over intended assay time; some salts may undergo pH‑dependent degradation.

Not typically applicable

• This product is not a classical buffering agent. It is generally used as an analyte or test article within buffered systems rather than to impart buffering capacity.

Green Alternatives

Context

• Structural and hazard details for Ulefnersen sodium are not provided. Therefore, green‑chemistry evaluation is limited to general best practices for handling ionic organic salts used in discovery.

General greener practice suggestions (not item-specific)

• Prefer water or water‑miscible green solvents (e.g., ethanol, isopropanol, ethyl lactate) for solution prep when compatible with stability and assay design.
• Minimize DMSO volumes by using the highest feasible aqueous fraction; consider glycerol or PEG‑400 as co‑solvents when appropriate for biophysical assays.
• Apply microscale experimentation and parallel screening to reduce solvent use and waste per data point.
• Choose energy‑efficient storage and shipping: consolidate shipments on cold packs, and avoid dry ice where −20°C stability is adequate (per Product Data, −20°C storage is suitable).

Comparison notes (illustrative, not item-specific)

• DMSO vs ethanol/water: DMSO offers broad solvency but is less preferred environmentally; ethanol/water mixes are greener if the compound remains soluble and stable.
• Acetonitrile vs methanol: ACN provides LC compatibility but is higher in environmental impact; methanol or water‑rich gradients may be acceptable if chromatographic resolution is maintained.

Waste handling

• Segregate halogenated vs non‑halogenated organic waste. For aqueous salt solutions, neutralize and dispose according to local regulations and SDS guidance.

Pharmaceutical Uses

Compliance note

• Research Use Only (per Product Data). No clinical or therapeutic claims are made or implied.

General formulation/manufacturing context (not item-specific)

• For discovery and preformulation studies, sodium salts are often evaluated due to improved aqueous solubility, crystallinity, or solid‑state stability relative to the free acid. Typical activities include:
  • Salt screening and polymorph assessment (XRPD, DSC, TGA, moisture sorption),
  • pH–solubility profiling and pKa determination to inform enabling formulations,
  • Development of analytical methods (stability‑indicating HPLC/UPLC) and forced degradation mapping.
• If used as a reference standard, document salt stoichiometry and water content to ensure accurate potency assignment (assay on anhydrous vs as‑is basis).

Regulatory references

• Pharmacopeial monographs are not indicated for this item. Any cGMP or GLP use would require additional qualification and documentation beyond the current Product Data.

Physical Properties

Item-specific facts (from Product Data)

• Appearance: Not specified for this item; refer to CoA/Spec Sheet.

Literature/general properties guidance (not item-specific)

• Exact melting point, boiling point, density, refractive index, pKa, logP/logD, and aqueous/organic solubility are not provided. For method development, determine:
  • Aqueous solubility across pH 2–10 (shake‑flask or kinetic solubility).
  • Partitioning (logD at pH 7.4) to estimate permeability and extraction behavior.
  • Thermal profile (DSC/TGA) to inform drying, storage, and formulation.
  • Hygroscopicity (dynamic vapor sorption, DVS), as sodium salts often exhibit moisture uptake.
• If an anionic sodium salt, expect higher water compatibility and reduced solubility in nonpolar solvents relative to the corresponding free acid; confirm experimentally.

Specifications note

• Where a numerical value is required for QC or process design (e.g., water content, residual solvents, UV cutoff), this product’s values are Not specified for this item; refer to CoA/Spec Sheet.

Quality & Grades

Item-specific facts (from Product Data)

• Grade/Purity: Not specified for this item; refer to CoA/Spec Sheet.

Interpreting grade (general guidance; not item-specific)

• Research grade materials typically support discovery, screening, and method development. If HPLC/LC‑MS applications are planned, request or verify:
  • Purity basis (area% by HPLC/UPLC vs. NMR assay) and reporting wavelength(s).
  • Counter‑ion content (e.g., sodium assay), residual solvents, inorganic salts, and water (Karl Fischer) where applicable.
  • Identity confirmation by HRMS and orthogonal methods (1H/13C NMR, IR).
  • For salts, report of assay on an anhydrous basis vs. as-is is important for accurate molarity.

Stabilizers and additives

• No stabilizers are declared in the Product Data. If stabilizers, antioxidants, or bulking agents are present, they will be stated on the CoA/Spec Sheet. Absence/presence of stabilizers impacts UV background, MS ionization, and bioassay interference.

Documentation

• For regulated workflows, request a batch-specific CoA including test methods, acceptance criteria, and analytical chromatograms. Where needed, inquire about lot-to-lot consistency data.

Reaction & Applications

Item-specific facts (from Product Data)

• Manufacturer Applications: Not provided.

General research applications (not item-specific)

• As part of a small‑molecule/compound library, materials like Ulefnersen sodium are commonly used for:
  • High‑throughput or targeted screening in biochemical or biophysical assays.
  • Reference standard development for analytical methods (HPLC/UPLC‑MS quantification, stability‑indicating methods).
  • Preliminary SAR exploration when analog series are available.
• If the substance is employed as a sodium salt, its ionic form may enhance aqueous assay compatibility, reduce nonspecific binding to plastics, and affect chromatographic retention (general considerations).

Chemical reactivity/use

• Without a disclosed structure, specific reaction chemistry (e.g., cross‑coupling, acylations) cannot be assigned. If derivatization or prodrug studies are intended, obtain structural details and functional group map from the CoA/Spec Sheet and verify reactivity with small‑scale scouting experiments.

Assay design tips

• Normalize dosing by molarity using the salt‑corrected molecular weight (request from CoA). Track pH of assay media upon dosing concentrated stock solutions to avoid activity artifacts.

Reaction Conditions

Item-specific facts (from Product Data)

• None provided.

Not typically applicable

• In the absence of a disclosed structure and functional groups, there are no recommended synthetic reaction conditions specific to Ulefnersen sodium.

General guidance for handling in solution (not item-specific)

• Prepare small trial solutions across candidate solvents (water, buffer, DMSO, methanol, acetonitrile) and monitor by HPLC/LC‑MS at room temperature and 4°C over 24–72 hours to establish chemical stability.
• If base/acid sensitivity is suspected, bracket pH (e.g., pH 3, 7, 9) and monitor degradation pathways; adjust storage solvent accordingly.
• For any transformation or salt‑exchange step, start at 0–25°C and scale cautiously after establishing mass balance and impurity profiles.

Safety & Handling

Item-specific facts (from Product Data)

• GHS classification, signal word, hazard statements, and pictograms: Not specified for this item; refer to SDS.
• Storage: Store at −20°C, desiccated (per Product Data). Avoid moisture ingress.

General safety guidance (not item-specific; defer to SDS)

• PPE: Wear lab coat, safety glasses, and appropriate chemically resistant gloves. Handle in a fume hood to avoid inhalation of dust/aerosol.
• Incompatibilities: As a sodium salt of an acidic functionality (general), avoid contact with strong acids (may liberate free acid) and strong oxidizers until compatibility is established.
• First aid (overview): If inhaled, move to fresh air; if on skin/eyes, rinse with water for ≥15 minutes; if ingested, rinse mouth. Seek medical attention in all cases. Follow institutional SOPs.
• Spill response: Avoid dust generation; collect with inert absorbent, place in compatible container for disposal according to local regulations.
• Thermal and moisture considerations: Maintain cold, dry storage. Warm to room temperature in a desiccator before opening to minimize condensation. Reseal promptly; consider handling under inert gas if the material proves moisture‑sensitive.

Authoritative source

• Always consult the product‑specific SDS and CoA for definitive hazard classification, exposure controls, and waste disposal.

Solvent Selection

Item-specific facts (from Product Data)

• No solvent preferences or solubility data are provided. Values are Not specified for this item; refer to CoA/Spec Sheet.

General selection strategy (literature/general; not item-specific)

• Start with water and common biological buffers (e.g., PBS, HEPES) given the sodium salt form often implies water compatibility. If solubility is limited:
  • Adjust pH to favor the ionized state (for acidic salts, mildly basic buffer can increase solubility) while staying within the compound’s stability window.
  • Use cosolvents: methanol, ethanol, isopropanol, acetonitrile, or DMSO. Prepare concentrated DMSO stocks and dilute into aqueous media last to minimize precipitation.
• For purely chemical workflows, polar aprotic solvents (DMSO, DMF, NMP) commonly dissolve ionic organics; confirm stability by test LC‑MS after 24–48 h.
• Avoid nonpolar solvents (hexanes, toluene) unless the free acid/base form is generated or an ion‑pairing strategy is used.

Practical tips

• Establish a solvent compatibility matrix (qualitative: clear/turbid/precipitation) at room temperature and at working concentrations.
• Filter sterilization for cell‑free assays should use low‑binding membranes (PTFE for organic, PES/RC for aqueous). Confirm absence of adsorption losses.

Storage & Reconstitution

Item-specific facts (from Product Data)

• Storage conditions: Store at −20°C, desiccated.
• Shipping: Ice chest + ice pads.

General, non‑item‑specific guidance

• Minimize moisture exposure: allow vials to equilibrate to room temperature inside a desiccator before opening to prevent condensation. Reseal promptly; consider handling under dry nitrogen if hygroscopic behavior is observed.
• Aliquoting
  • Upon first opening, partition into single‑use aliquots in low‑binding, airtight containers. For solutions, use dry, oxygen‑free solvents if stability requires.
• Reconstitution
  • Solvent choice should be based on small‑scale solubility screening. For sodium salts, start with water or appropriate buffer; alternatively, dissolve in DMSO then dilute with aqueous media.
  • Record exact solvent, concentration, and date on each aliquot.
• Freeze–thaw
  • Avoid repeated freeze–thaw cycles. Store aliquots at −20°C (or as otherwise validated) and keep a working aliquot at 2–8°C during the active use window.
• Stability monitoring
  • Periodically verify integrity by HPLC/LC‑MS or other suitable analytical methods, especially after prolonged storage or shipment.

Specifications note

• Any item‑specific limits (e.g., water content, residual solvents, assay) are Not specified for this item; refer to CoA/Spec Sheet.

Research use

• For research use only (per Product Data).

Structure & Identity

Item-specific facts (from Product Data)

• Product name: Ulefnersen sodium (SKU: U1426641)
• CAS: 2589926-27-0
• 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.
• InChIKey: Not specified for this item; refer to CoA/Spec Sheet.

General chemistry notes (literature/general)

• “Sodium” in the name indicates an anionic functional group is present in its salt form (e.g., carboxylate, sulfonate, or phosphate). The sodium counter‑ion generally increases aqueous compatibility and can influence solid-state hygroscopicity. This is general information and not item-specific without a disclosed structure.
• Without a provided structure, stereochemistry, ring systems, and functional group assignments cannot be confirmed. Please consult the CoA/Spec Sheet for definitive identifiers (full IUPAC name, exact structure diagram) prior to method development or regulatory documentation.

2D structure description

• Not available for this item; refer to CoA/Spec Sheet for a definitive 2D structure and atom connectivity.

Synthetic Utility

Item-specific facts (from Product Data)

• Structural details enabling synthetic transformations are not provided.

Applicability

• Without confirmed functional groups, Ulefnersen sodium is not typically positioned as a general synthetic reagent. Its primary use is expected to be as a test article or reference compound in analytical and biological studies.

General guidance (not item-specific)

• If derivatization is of interest (e.g., prodrug formation, isotopic labeling, bioconjugation), obtain the complete structure and functional group map from the CoA/Spec Sheet. Then:
  • Assess chemoselectivity via small‑scale scouting reactions.
  • Verify salt effects on reactivity (counter‑ion exchange to the free acid/base may be required).
  • Confirm product identity and purity with orthogonal analytics (NMR, HRMS, HPLC) due to potential ion‑pair or counter‑ion artifacts.

Retrosynthetic note

• Any discussion of named reactions or protecting‑group strategy would be speculative without a disclosed scaffold. Proceed only after structural confirmation.

Target Specificity

Item-specific facts (from Product Data)

• Target/epitope, species reactivity, clone/isotype: Not applicable and not specified for this item.

Note

• This product is not an antibody or affinity reagent. No target specificity data are available in the Product Data. If the compound has a known molecular target, consult the CoA/Spec Sheet or primary literature before designing target‑engagement assays.