Oct 16, 2024
AstraZeneca and Ionis’s Wainzua (eplontersen) approved by UK MHRA for ATTRv polyneuropathy. Antisense oligonucleotide, polyneuropathy associated with hereditary transthyretin amyloidosis (Source)
Takeda has decided to terminate its partnership with Wave Life Sciences after investing a significant sum of $260 million in the collaborative effort. Antisense oligonucleotide, Huntington’s disease (Source)
Oct 10, 2024
Vico Therapeutics’ antisense oligonucleotide, VO659, demonstrates positive results in early-stage Huntington’s disease trials. (Source)
About “Antisense Oligonucleotides (ASOs)”
Antisense oligonucleotides (ASOs) are short, synthetic single-stranded DNA or RNA molecules designed to bind to specific mRNA sequences in cells. By binding to mRNA, ASOs can interfere with gene expression, either by blocking translation or by triggering mRNA degradation. This technology has significant potential applications in biotechnology and medicine.
Mechanisms of Action:
- Translation inhibition: ASOs can bind to mRNA sequences that are complementary to their own sequence, preventing ribosomes from translating the mRNA into a protein.
- RNA degradation: ASOs can also recruit cellular enzymes, such as RNase H, to degrade the target mRNA.
Applications of Antisense Oligonucleotides:
- Gene knockdown: ASOs can be used to reduce the expression of specific genes, allowing researchers to study the function of those genes.
- Disease treatment: ASOs are being developed as potential therapies for a variety of diseases, including genetic disorders, viral infections, and cancers.
- Drug discovery: ASOs can be used to identify new drug targets and to validate potential drug candidates.
Advantages of Antisense Oligonucleotides:
- Specificity: ASOs can be designed to target specific mRNA sequences with high specificity, minimizing off-target effects.
- Potency: ASOs can be highly potent, requiring only small doses to achieve therapeutic effects.
- Versatility: ASOs can be used to target a wide range of genes, making them applicable to a variety of diseases.
Challenges and Limitations:
- Delivery: Delivering ASOs to their target cells in vivo can be challenging, as they may be degraded or cleared from the body before reaching their destination.
- Off-target effects: While ASOs are generally highly specific, there is a risk of off-target effects, where ASOs bind to unintended mRNA sequences.
- Manufacturing costs: The synthesis of ASOs can be expensive, particularly for long sequences.
Despite these challenges, antisense oligonucleotide technology has shown great promise and continues to be an active area of research and development in biotechnology. As delivery methods improve and manufacturing costs decrease, ASOs are expected to play an increasingly important role in the treatment of various diseases.