| Size | Price | Stock | Qty |
|---|---|---|---|
| 1mg |
|
||
| Other Sizes |
| Targets |
Avacincaptad pegol targets complement factor C5 (C5), a key protein in the complement system. By binding to C5, it prevents its proteolytic cleavage into the pro-inflammatory anaphylatoxin C5a and the membrane attack complex-initiating fragment C5b, thereby inhibiting downstream complement activation.
|
|---|---|
| ln Vitro |
Avacincaptad pegol is a highly specific RNA aptamer that binds to human C5 with high affinity, inhibiting its cleavage. This blocks the generation of C5a and C5b, reducing inflammation and cell lysis. It represents a novel mechanism for complement inhibition distinct from small-molecule or antibody-based approaches.
|
| ln Vivo |
In animal models and human patients, avacincaptad pegol reduces complement-mediated inflammation and cell death. It has been shown to slow the progression of geographic atrophy, an advanced form of age-related macular degeneration, by reducing photoreceptor and retinal pigment epithelium cell loss. Detailed in vivo data come from clinical trials.
|
| Enzyme Assay |
The binding affinity of avacincaptad pegol to C5 is measured by Surface Plasmon Resonance (SPR). The inhibition of C5 cleavage is assessed using an in vitro hemolysis assay. Human red blood cells are incubated with C5 and complement components in the presence of the aptamer, and the percentage of hemolysis is quantified.
|
| Cell Assay |
The primary “cell-based” assays for avacincaptad pegol are hemolysis assays using human red blood cells. The compound's ability to inhibit complement-mediated cell lysis is assessed. For more detailed cellular studies, the effect on C5a-induced inflammatory responses in immune cells can be measured by ELISA for downstream cytokines.
|
| Animal Protocol |
In vivo protocols involve intravitreal injection of avacincaptad pegol into the eye. In animal models of AMD, retinal structure and function are assessed by optical coherence tomography (OCT) and electroretinography. In clinical trials, patients receive monthly intravitreal injections, and geographic atrophy lesion area is measured by fundus autofluorescence imaging.
|
| ADME/Pharmacokinetics |
For ocular administration, avacincaptad pegol is administered via intravitreal injection, providing high local concentrations in the eye with minimal systemic exposure. The PEGylated aptamer has a prolonged half-life in the vitreous, supporting monthly dosing intervals. Systemic PK is limited due to local administration.
|
| Toxicity/Toxicokinetics |
Toxicological data for avacincaptad pegol have been evaluated in clinical trials. The most common adverse events include endophthalmitis, conjunctival hemorrhage, and increased intraocular pressure. Systemic complement inhibition carries a risk of increased infection, but this risk is minimized by local ocular administration.
|
| References |
|
| Additional Infomation |
Avacincaptad pegol is the first approved therapy for geographic atrophy secondary to age-related macular degeneration, receiving FDA approval in August 2023. It is marketed under the brand name Izervay. It is a landmark drug as an RNA aptamer therapeutic.
|
| CAS # |
1491144-00-3
|
|---|---|
| Related CAS # |
Avacincaptad pegol;1613641-69-2
|
| Appearance |
White to off-white solid powder
|
| Synonyms |
Izervay
|
| HS Tariff Code |
2934.99.9001
|
| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment, avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
|
| Solubility (In Vitro) |
H2O : ~100 mg/mL
|
|---|---|
| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
Products are for research use only; We do not sell to patients
Copyright 2020 InvivoChem LLC | All Rights Reserved
