Small nucleic acid drugs, by directing gene expression in a precise manner, provide personalised therapies for various genetic and acquired diseases. Small nucleic acids are referred to as antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs) and microRNAs (miRNAs).

ASOs

ASOs are small synthetic nucleic acids that attach to complementary mRNA molecules to manipulate gene expression in several ways.

1. Exon Skipping: ASOs can induce skipping of specific exons when mRNA is spliced. The strategy can be employed for disorders caused by mutations preventing splicing (such as some muscle dystrophy).

2. mRNA Degradation: ASOs can also employ RNase H that digests the mRNA target and mutates the protein encoded. This is handy in the case of gene overexpression leading to disease.

3.Translation Blocking: By helping to bind to the mRNA, ASOs will prevent translation and so protein will not be produced. It is administered in diseases where a protein is damaged.

ASOs have been promising in genetic conditions such as spinal muscular atrophy (SMA) and some cancers too.

siRNAs

siRNAs are double-stranded RNA molecules that enact gene silencing through RNAi. Their mechanism includes:

1. RISC Loading: siRNAs are packed inside the RNA-induced silencing complex (RISC), and navigate the complex to complementary mRNA sequences.

2. mRNA Breaking: RISC complex snips the mRNA of interest and breaks it down. This is an easy way to turn down target gene expression.

siRNA has the potential to treat viral infections, cancers, and genetic diseases by specifically targeting and silencing disease-causing genes.

miRNAs

As endogenous small RNA molecules, miRNAs play important regulatory roles in the cell by binding to complementary sequences on target mRNAs.

1. mRNA Degradation: Promote target mRNA degradation and lower expression.

2. Translation Inhibition: Repression of the translation of mRNA into protein.

miRNAs affect the way cells work and what diseases do by controlling gene expression at a cellular level. Studies have found that a variety of diseases, such as cancer, cardiovascular disease and neurodegenerative disease, are associated with abnormal expression of miRNAs.

Contract Development and Manufacturing Organization (CDMO) Solutions

We specialize in the development and manufacture of small nucleic acid drugs, and provide related CDMO services including but not limited to:

• Customized development

We understand that each client's project is unique. We tailor nucleic acid drug development to your specific needs, providing technical support at all stages of sequence design, synthesis, and optimization.

• Advanced manufacturing

We utilize state of the art equipment for large-scale production of ASOs, sirnas, and mirnas, while ensuring that the entire production process complies with international regulatory standards.

• Quality control

To ensure the safety and effectiveness of the end product, we have strict quality control at all times.

• Regulatory support

We assist our clients in the preparation and submission of all necessary regulatory documents to accelerate the commercialization of their products.

At Protheragen MedAI, our commitment is to take your small nucleic acid drug from concept to clinical application through a comprehensive service. We invite you to experience our efficient AI technology, and if you are interested in our services, please contact us.