Compared to traditional small molecule drugs and antibody drugs, small nucleic acid drugs offer the advantage of intervening at the source. They also provide benefits such as rapid target screening, high therapeutic efficiency, low drug toxicity, high specificity, and a high success rate in research and development. CD Formulation has extensive experience in custom synthesis of small nucleic acids, and we use the latest nucleic acid preparation technologies and optimization strategies to guarantee our customers receive products with high yield and purity.
Small nucleic acids, consisting of nucleotide sequences with a maximum length of 30 base pairs, function by suppressing the expression of target proteins through specific interactions with mRNA molecules, thereby leading to therapeutic outcomes. The advantages of using small nucleic acids in disease treatment are outlined as follows.
By targeting mRNA, small nucleic acid drugs have the potential to make significant advancements in addressing protein targets that are challenging to target with traditional drugs, thereby significantly broadening the range of target actions.
In contrast to small molecule drugs that require extensive compound screening, small nucleic acid drugs only involve targeting the disease-causing gene sequence, designing and synthesizing the corresponding RNA fragment for the gene sequence, and subsequently screening and validating the candidate compounds.
Small nucleic acid drugs can exert precise and efficient effects by binding to the target gene mRNA in a sequence-specific manner through base complementary pairing.
Small molecule drugs only require DNA or RNA synthesis, thereby expediting the production of small nucleic acid drugs.
Fig.1 Structures of various types of nucleic acids. (CD Formulation)
As a leading biopharmaceutical contract manufacturing organization worldwide, CD Formulation has extensive experience in providing tailored services for synthesizing small nucleic acid drugs. The company focuses on delivering high-quality manufacturing services to clients globally, while complying with drug quality standards and regulatory requirements.
CD Formulation's cutting-edge drug manufacturing facilities are designed specifically for producing small nucleic acid drugs, offering comprehensive support from nucleic acid drug discovery, formulation type development, and scale-up, covering all aspects of nucleic acid design, modification, bioanalysis, and stability assessment.
We offer customized synthesis services for various types of small nucleic acids, such as antisense oligonucleotide (ASO), small interfering RNA (siRNA), RNA (miRNA), small activating RNA (saRNA), aptamers and so on. Available small nucleic acids custom synthesis projects include:
Fig.2 Flow chart of small nucleic acid preparation. (CD Formulation)
Target analysis and sequence design: According to the specific disease target, design small nucleic acid sequences with high relevance and specificity, such as siRNA, miRNA, and others.
Oligonucleotide synthesis: Prepare the designed oligonucleotide sequence through chemical synthesis.
Chemical modification: In order to enhance the bioavailability and stability of the mini-nucleic acids, various chemical modifications are required during the synthesis process.
Purification and characterization: The synthesized mini-nucleic acids need to be purified and structurally characterized to ensure their purity and quality.
Formulation development: Suitable routes of administration and forms of formulation need to be developed and optimized to facilitate efficient drug delivery.
Activity and safety assessment: Evaluate the targeting, pharmacodynamic properties, and safety indicators of the mini-nucleic acid drug, providing a crucial foundation for preclinical and clinical studies.
We have advanced equipment and rich experience in small nucleic acid preparation, and can provide a variety of customized nucleic acid synthesis service solutions according to customers' existing research and development progress.
Synthesis Method | Length | Quantity | Safety |
---|---|---|---|
Chemical synthesis | 4-200 nt | Up to kg | May have some immunogenicity, which correlates with RNA size, modification, etc. |
In vitro transcription synthesis | 4-200 nt | - | Some immunogenicity is present and may be associated with double-stranded RNA (dsRNA) formation due to the increased length of transcribed RNA in vitro. |
In vivo biosynthesis | <400 nt | Up to kg | Low immunogenicity due to natural modifications. |
Technology: RNAi technology for preparation of small nucleic acid drugs
Journal: Acta Pharmaceutica Sinica B
IF: 14.5
Published: 2024
Results:
The authors provide an overview of the current status and future trends in the development of mini-nucleic acid drugs. Beginning with a historical background, the authors describe the major classifications and mechanisms of mini-nucleic acid drugs. The authors then delve into the advantages of U.S. Food and Drug Administration (FDA)-approved drugs and focus on the challenges encountered during their development. Future directions in the field will prioritize addressing these challenges to facilitate the clinical translation of small nucleic acid drugs.
Fig. 3 The key milestones in the small nucleic acid drug industry. (Miao Y. et al., 2024)
Customized services for small nucleic acids involve synthesizing based on the customer's target sequence or design requirements to obtain small nucleic acid drugs with high yield, purity and quality. Please feel free to contact us if you are interested in our services. CD Formulation will collaborate closely with you to ensure the smooth progress of your project and will customize and optimize the solution according to the customer's needs.
References