Antisense Oligonucleotide Service

In the increasingly competitive field of antisense oligonucleotide (ASO) drug development, selecting a partner with strong technical foundations, comprehensive platform capabilities, and extensive project experience is essential for accelerating the progression from concept to clinic and minimizing development risks. Since its establishment, Creative Biogene has been dedicated to nucleic-acid–based technologies as its core focus. From fundamental DNA primer synthesis to complex functional RNA preparation and, more recently, small-nucleic-acid drug development, we have accumulated unparalleled technical expertise and practical know-how. Our team comprises senior scientists with deep backgrounds in chemistry, biology, bioinformatics, and pharmaceutical science, all of whom combine rigorous theoretical knowledge with exceptional problem-solving capabilities in real-world R&D scenarios.

Background

Antisense oligonucleotides are short, chemically synthesized DNA or RNA molecules, typically 15–30 nucleotides in length. ASOs bind precisely to their target mRNA via canonical Watson–Crick base pairing, thereby enabling targeted modulation of gene expression.

ASO Mechanisms of Action

Figure 1. Common ASOs Mechanisms of Action. (Schoch KM, et al., 2017)

RNase H1-mediated mRNA degradation

Once an ASO hybridizes with its target mRNA, a DNA–RNA heteroduplex is formed, which recruits cellular RNase H1. This enzyme selectively cleaves the RNA strand, leading to mRNA degradation and subsequent inhibition of protein synthesis.

Steric blocking

By binding to key functional regions on the mRNA—such as splice junctions, the 5' cap-proximal region, or ribosome-binding sites—ASOs can physically interfere with splicing, translation initiation, or other regulatory processes.

miRNA mimicry or inhibition

Specially engineered ASOs can mimic endogenous microRNAs or inhibit their functions, allowing them to participate in intricate regulatory networks.

With their high specificity and programmability, ASO technologies have demonstrated remarkable therapeutic potential across various conditions, including oncology, neurodegenerative disorders, inherited diseases, metabolic disorders, and numerous rare diseases. Several ASO therapeutics—including nusinersen and inotersen—have already gained FDA approval, marking the successful translation of this technology from bench to bedside.

Creative Biogene's Integrated ASO Service Solution

We recognize that each stage of drug development imposes distinct requirements for material quality. Accordingly, we have established a complete production and quality-control system spanning research-grade to cGMP-grade manufacturing. Whether you are in early target-discovery research, preclinical development, or progressing into clinical trials, Creative Biogene can supply high-quality ASO materials tailored to your needs—ensuring data reliability, regulatory compliance, and seamless project continuity.

Our Core Service Modules

Antisense Oligonucleotide Synthesis Service

A fully developed production system from RUO to cGMP ensures every batch of ASO manufactured meets high standards of consistency, stability, and reliability.

ASO In Vitro Screening Service

By integrating our proprietary AI-driven design platform with robust experimental validation, we significantly increase screening efficiency and hit rates.

ASO In Vivo Testing Service

Lead candidates identified in vitro undergo rigorous evaluation in vivo. Our in vivo service provides dependable pharmacodynamic, pharmacokinetic, and preliminary safety data to support IND submissions.

A Four-Pillar Integrated Support System for ASO R&D

Our strengths stem not from a single capability, but from a synergistic ecosystem of complementary platforms.

01 Oligonucleotide Chemistry and Manufacturing Platform

A cornerstone of our technical portfolio, this platform provides:

• Scalability and Flexibility: Production ranging from nmol-scale for discovery research to kg-scale for commercial needs.
• Advanced Modification and Conjugation Technologies: Expertise in over 200 chemical modifications—including phosphorothioates, 2'-O-Me, 2'-MOE, LNA, and more—and precise conjugation with GalNAc, cholesterol, peptides, antibodies, and other ligands to enhance PK and targeting profiles.
• Stringent Quality Standards: ASO purity consistently ≥90%, supported by advanced synthesis and purification technologies such as RP-HPLC and IE-HPLC, ensuring superior batch-to-batch reproducibility.

02 Bioinformatics

In the era of data-driven drug discovery, we have built a next-generation intelligent ASO design system.

• Beyond Rule-Based Design: Our platform integrates extensive internal experimental datasets with public databases, applying machine learning and deep learning algorithms to predict silencing potency, molecular stability, and potential off-target effects with high accuracy.
• Rational Design for Higher Efficiency: This dramatically increases the success rate of lead identification and shortens optimization cycles. Validated ASOs designed using our platform have achieved silencing efficiencies as high as 98%.

03 Proprietary Chemical-Modification Strategies

Chemical modifications play a decisive role in determining drug-like properties. Based on comprehensive bioinformatic and experimental evidence, we have developed multiple proprietary modification suites (e.g., GS1–GS7), each optimized for specific objectives such as enhanced stability, reduced off-target activity, or maximal potency.

04 Rigorous Analytical and Quality Control Systems

Quality begins at the design stage and is ensured through uncompromising analytical rigor.

• Comprehensive Analytical Capabilities: Mass spectrometry, HPLC, UV spectrophotometry, sterility testing, endotoxin assessment, and more—supporting full characterization of molecular identity, purity, potency, and safety.
• Data Integrity and Traceability: All production and QC data meet regulatory requirements for completeness and traceability, supporting smooth regulatory submissions.

Client Case Studies & Research Outcomes

Case Study 1

The researchers conducted a preclinical evaluation of antagomiR-218 in myotonic dystrophy type 1 (DM1) using the HSALR mouse model and patient-derived myotubes. They quantified antagomiR-218 levels at 40–60 pM two weeks after injection in HSALR mice and documented dose- and time-dependent molecular and functional responses, along with a favorable toxicity profile after a single subcutaneous administration. In muscle tissue, they observed restoration of normal Mbnl1 subcellular distribution without changes in the proportion of myonuclei containing CUG foci. At the same time, in patient-derived cells, antagomiR-218 improved fusion and differentiation and reversed up to 34% of transcriptomic alterations. For these experiments, the researchers used antagomiR-218 and agomiR-218, synthesized by Creative Biogene, incorporating 2′-O-methyl modifications (m), phosphorothioate linkages (∗), and cholesterol conjugation (chol).

Figure 1. Target expression patterns across antagomiR-218 doses, including reporter responses in C2C12 cells and the in vivo dose-response study design. (Cerro-Herreros E, et al., 2021)

Case Study 2

The researchers induced colitis in male C57BL/6 mice with 3% DSS and administered antagomiR-200c or a nonspecific control, synthesized by Creative Biogene, by daily oral gavage starting two days before DSS treatment. They found that MIR200C-3p was upregulated by IL1B in Caco-2 cells and mouse enterocytes, and that antagomiR-200c prevented IL1B- and DSS-induced decreases in occludin mRNA and protein, reducing tight junction permeability. Patient colon tissues and organoids from ulcerative colitis cases showed elevated IL1B and MIR200C-3p, and three-dimensional modeling identified interacting sites in the occludin 3′UTR.

Figure 2. The effects of DSS-induced colitis and antagomiR-200c on IL-1β, miR-200c-3p, occludin expression, intestinal permeability, body weight, and colon histology in mice.(Rawat M, et al., 2020)

Delivering Real Value to Your ASO Program

By integrating these four major platforms, Creative Biogene offers unmatched benefits:

A Seamless, End-to-End Solution

From sequence design and chemical modification to in vitro screening and in vivo validation, we deliver a fully connected workflow without the risks associated with multi-vendor transitions.

Enhanced Efficiency and Higher Success Rates

The combination of AI-assisted design with proprietary modification systems enables rapid identification of potent, stable, and low-off-target candidates at reduced cost.

Effective Risk Control

A unified production pipeline spanning RUO to GMP ensures quality consistency and regulatory compliance throughout development, reducing delays and failure risks stemming from material variability.

Contact Us

Creative Biogene is more than a service vendor—we are your trusted partner in therapeutic innovation. With advanced technologies, industry-leading expertise, and a commitment to scientific excellence, we provide high-value, high-quality ASO services designed to ensure your success.

If you have specific requirements or would like to discuss your project in greater detail, please feel free to contact us. We look forward to collaborating with you to bring the next breakthrough in gene therapy to life.