Small Nucleic Acid Drugs Development Services

siRNA & ASO Sequence Design / Synthesis / Delivery / Screening

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Overview

Accelerate siRNA and ASO therapeutic development with end-to-end solutions encompassing design, synthesis, screening, and performance evaluation in a single integrated workflow. Our customizable service packages support both unmodified and modified siRNA discovery, offering optimized solutions for design, synthesis, preliminary screening, in-depth validation, and comprehensive candidate evaluation.

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Oligo Synthesis

Advantages

One-stop service,
faster delivery

From design to therapeutic candidate in as fast as 6 weeks
Over 22 years of expertise in oligonucleotide synthesis
Scalable production from RUO to cGMP, up to kg levels
Optimized conjugation technology for targeted delivery

Flexible, diversified packages

Choose from full-process solutions or individual services tailored to your specific needs
Obtain candidate molecules with high knockdown efficiency

AI-enhanced design for greater precision

AI-driven design combined with experimental validation to optimize logic, achieving knockdown efficiency of up to 98%
Predictive modeling for modified siRNA knockdown efficiency to reduce screening costs and time

Workflow

Sequence Design

Starting from 3 days

siRNA or ASO sequence designed based on target gene, with predicted knockdown efficiency for modified siRNA.

Advantages

Optimize design logic validated by experimental results, ensuring higher gene knockdown efficiency
Cross-species design to support animal and clinical studies
Customizable design to meet specific needs

Synthesis

Starting from 4 days

High-throughput, flexible-scale synthesis (5 nmol to kg level) for siRNA & ASO, from RUO to cGMP.

Advantages

Standard HPLC purification and MS/HPLC QC with purity ≥90%
Customized modifications, purification & QC options
Proprietary modifications enhance stability and minimize off-target effects

Delivery

Starting from 2 weeks

Site-specific conjugation (antibody, peptide, lipid, etc.), GalNAc conjugation, and LNP delivery.

Advantages

Proprietary methodology for antibody-oligo conjugation (AOC)
Targeted LNP for liver, lung, spleen, T cells, and more

Screening & Evaluation

Starting from 2 weeks

Detection of mRNA or protein-level knockdown efficiency, stability, off-target effects, immunogenicity, cytotoxicity, and more.

Advantages

High-throughput screening and IC₅₀ detection for consistent and reproducible results
Optimized detection for target genes to enhance precision

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Case Studies

Design platform: : Enhancing knock down efficiency to identify candidates with ≥80% efficiency
- siRNA designed with our tool achieved ≥80% knockdown efficiency for four genes, offering strong development potential.
- Our siRNA demonstrated up to 98% knockdown efficiency, surpassing other platforms.
Synthesis Platform: Accurate MW, high purity and batch-to-batch consistency
Delivery Platform: Optimized site-specific conjugation, GalNAc conjugation, and LNP delivery ensure targeted knockdown efficiency
Site-specific conjugation
- Effective site-specific conjugation between siRNA and antibody (ARC, Antibody-RNA Conjugation) not only ensures knockdown efficiency but also enables targeted delivery
LNP packaging
- LNP packaged siRNA: 10nM, knockdown efficiency around 96%
- Various targeted LNPs available (for liver, lungs, spleen, T cells, and more) Review validation data here.
Screening Platform: High-throughput screening to identify the best candidates

With GenScript's optimized design and superior synthesis capabilities, among the 50 designed siRNA, knockdown efficiency reached up to 91%, and 13 siRNA groups have knockdown efficiency higher than the positive control.

Among 150 designed ASO candidates, 9 ASOs achieves a knockdown efficiency of over 90%.
Screening Platform: Protein expression was evaluated through various methods to verify siRNA activity

ELISA assay: suitable for quantification of secreted proteins

Qualitative as well as quantitative (Microplate reader)

Immunofluorescence Staining: suitable for quantification of membrane proteins

Qualitative as well as quantitative (Flow cytometry)

Western blot: suitable for quantification of all kinds of proteins

Qualitative, sometimes semi-quantitative (Gel electrophoresis)