%5d)
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| Targets |
Rovanersen targets the UBE3A-ATS (UBE3A antisense transcript), a long non-coding RNA that silences the paternal UBE3A allele in neurons. By binding to the UBE3A-ATS, the antisense oligonucleotide inhibits its expression, thereby allowing the normally silenced paternal UBE3A gene to be expressed and produce functional UBE3A protein. This approach restores UBE3A protein levels in the brain, addressing the root cause of Angelman syndrome.
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| ln Vitro |
In vitro, Rovanersen has been tested in patient-derived cells and neuronal models. The ASO leads to dose-dependent reduction of the UBE3A-ATS transcript and corresponding increase in UBE3A protein levels. No specific IC50 or EC50 values are reported in the search results. The compound demonstrates specificity for the UBE3A-ATS with minimal off-target effects. Cellular efficacy is typically assessed by qPCR for UBE3A-ATS and RT-PCR or Western blot for UBE3A protein.
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| ln Vivo |
In vivo, Rovanersen has demonstrated dose-dependent improvements in brain activity biomarkers and developmental abilities in preclinical models and in Phase 1 clinical trial patients. The Phase 1 TANGELO trial results (published in Nature Medicine) showed that Rovanersen treatment led to dose-dependent improvements in EEG biomarkers and developmental abilities compared to natural history. The compound is expected to enter a pivotal Phase 3 clinical trial in mid-2026. Preclinical in vivo data in ASO mouse models are not detailed in the search results.
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| Enzyme Assay |
Binding of Rovanersen to the UBE3A-ATS RNA target is measured by standard antisense oligonucleotide hybridization assays, including electrophoretic mobility shift assay (EMSA) or surface plasmon resonance (SPR) using a complementary oligonucleotide. The thermal stability (Tm) of the ASO-RNA duplex is assessed by UV melting curve analysis. Specific binding affinity (KD) is not reported in the search results. RNase H-mediated cleavage activity is confirmed in cell-free systems using fluorescently labeled target RNA.
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| Cell Assay |
For in vitro cellular assays, patient-derived fibroblasts or induced pluripotent stem cell (iPSC)-derived neurons are treated with Rovanersen (0.1-10 microM) for 48-96 h. Free uptake (gymnosis) in serum-containing medium is used to mimic in vivo conditions without transfection reagents. UBE3A-ATS levels are measured by qRT-PCR, and UBE3A protein levels are quantified by Western blot or ELISA. Neuronal maturation and synaptic protein expression (e.g., synapsin, PSD-95) are assessed by immunostaining. Cell viability is assessed by MTT or CellTiter-Glo assays.
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| Animal Protocol |
In vivo efficacy studies are performed in Angelman syndrome mouse models (e.g., Ube3a maternal deficiency mice, which lack maternal Ube3a expression). Rovanersen is administered intracerebroventricularly (ICV) or intrathecally (IT) at doses of 10-100 microg. At various time points (1-4 weeks post-dose), brains are collected, and UBE3A-ATS and UBE3A mRNA/protein levels are quantified by qPCR and Western blot. Behavioral tests including open field, rotarod, and novel object recognition are performed to assess motor coordination, anxiety, and learning/memory. EEG recordings are conducted to measure brain activity patterns.
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| ADME/Pharmacokinetics |
Rovanersen sodium is a 2‘-O-methoxyethyl (2‘-MOE) modified antisense oligonucleotide with a phosphorothioate backbone for improved stability and CNS penetration. The molecular weight is approximately 7,000-8,000 Da. For in vivo administration, the ASO is formulated in saline or artificial cerebrospinal fluid (aCSF) and delivered by intrathecal (IT) or intracerebroventricular (ICV) injection. In humans, the ASO is administered via lumbar puncture. No detailed PK parameters (CSF half-life, distribution volume, brain penetration) are reported in the search results. The ASO is expected to have a prolonged CSF half-life of 2-4 weeks, similar to other 2‘-MOE ASOs.
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| Toxicity/Toxicokinetics |
In Phase 1 clinical trials (TANGELO), Rovanersen was generally well-tolerated with no serious treatment-related adverse events reported. The most common adverse events were those typical of lumbar puncture procedures (headache, back pain). No dose-limiting toxicities were observed. In preclinical studies, no significant neurotoxicity or systemic toxicity has been reported at therapeutically relevant doses. No formal toxicology data (LD50, target organ toxicity) are provided in the search results.
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| References | |
| Additional Infomation |
Rovanersen sodium (rugonersen, OHB-724, formerly RO7248824) is an investigational antisense oligonucleotide for Angelman syndrome developed originally by Roche and subsequently licensed to Oak Hill Bio in 2025. Oak Hill Bio plans to initiate a pivotal Phase 3 clinical trial in mid-2026. The compound has received regulatory designations (e.g., Orphan Drug Designation, Rare Pediatric Disease Designation) from the FDA. Rovanersen is for research use only and has not received regulatory approval for any indication. The compound is not available for human use in its research grade. Angelman syndrome is a rare neurodevelopmental disorder caused by loss of function of the maternal UBE3A allele, affecting approximately 1 in 12,000-20,000 individuals.
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| Molecular Formula |
C202H240N82NA19O111P19S13
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| Molecular Weight |
7034.70
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| CAS # |
2231347-41-2
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| Related CAS # |
Rovanersen; 2072901-32-5
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| Appearance |
White to off-white solid powder
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| SMILES |
[Rovanersen (sodium)]
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| Synonyms |
WVE-120101 sodium
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| HS Tariff Code |
2934.99.9001
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| 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 (e.g. under nitrogen), 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)
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| Solubility (In Vitro) |
H2O: ~100 mg/mL (with ultrasonication)
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| 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.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 0.1422 mL | 0.7108 mL | 1.4215 mL | |
| 5 mM | 0.0284 mL | 0.1422 mL | 0.2843 mL | |
| 10 mM | 0.0142 mL | 0.0711 mL | 0.1422 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
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.
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