Small Nucleic Acid Drugs Discovery Services

From Design to Therapeutic Candidate in 6 Weeks

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Overview

Expedite your drug development pipeline with screening solutions tailored for your screening needs, from simple unmodified design and preliminary screening, to complex modified design and functional analysis.

Advantages

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

Diversified packages, one stop solutions

One vendor covers design, synthesis to screening to save your time
Diversified packages meet the needs of different stages in candidate discovery, optimization, and bioanalysis

Oligo Synthesis

Service Details

Basic Package

Starting at 6 weeks, $11K

Designed naked siRNA/simple modified ASO^* with knockdown efficiency

Economy Package

Starting at 8 weeks, $22K

Designed naked siRNA/simple modified ASO^* with knockdown efficiency & IC₅₀

Premium Package

Starting at 12 weeks, $38K

Designed modified siRNA/complex modified ASO^* with knockdown efficiency & IC₅₀
(Recommend stability & off target analysis)

All packages offer screening volumes of 20, 50, 100, or 200 sequences.

* Simple modified ASO: Sequences designed with standardized Gapmer architectures (PS, MOE, OMe) with fixed modification patterns.
Complex modified ASO: Customized modifications available.

Package Name	Service Content	Basic	Economy	Premium
Design & synthesis	Naked siRNA/simple modified ASO
Design & synthesis	Modified siRNA/complex modified ASO Predicts knockdown efficiency of modified siRNA
In Vitro Screening (mRNA-level)	Pilot experiment: detection plan confirmed for target gene
	Round 1 screening (naked siRNA/simple modified ASO): screen all, single concentration
	Round 2 screening (naked siRNA/simple modified ASO): select 10 candidates for IC₅₀ testing
	Round 3 &4 screening (modified siRNA/complex modified ASO): screen all, single concentration. Select 5 candidates for IC₅₀ testing.
	Knockdown efficiency (protein Level) Free uptake experiment	Optional	Optional	Optional
Functional Assays	Off-target analysis (RNA-seq) Cytotoxicity assay Immunogenicity (ELISA) Stability (LC-MS/MS)	Not applicable	Optional	Recommend
Deliverable	Design report: sequences, knockdown efficiency, off-target score, GC% content (modification plan for Premium package)
	In vitro screening report: knockdown efficiency (IC₅₀ for Economy & Premium package)
	Functional assay report: based on selected projects	Not applicable

Add-on Service	Service Content	Price
Patent search	Verify if candidate sequences are covered by existing patents and provide risk levels for client evaluation and selection for further development.	Additional evaluation fees required

^# Available cell line models for function testing >>

Disease State or Tissue Type	Cell Line
Bladder cancer	RT-4
Cervical cancer	Hela
Fibroblast cancer	SJCRH30
Leukemia	THP1, Jurkat, SUP-T1
Liver cancer	HepG2, Huh-7, Hep3B
Lung cancer	A549, H1975
Neuroblastoma	SH-SY5Y, SK-N-BE(2)
Ovarian cancer	RMG-1
Skin cancer	A375, A431
Non-cancerous	HEK293T, HUVECS
Mouse	L929S, CHO, Raw264
Primary cells	PBMC, Primary Cynomolgus Monkey Hepatocytes

GenScript also support customized cell mode

Ready to order? Download the quote form and fill in the necessary information for your request. Send the quote form to your Technical Account Manager or to [email protected].

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Case Studies

Advanced AI design ensures higher knockdown efficiency

Genscript's Small Nucleic Acid Drugs AI Design Platform

siRNA sequences designed for Gene B with GenScript's bioinformatic design platform achieved higher knockdown efficiency (KD%) than those created with other vendors' tools.
High-throughput screening for 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%.
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)