Pharmaceutical perspectives on oligonucleotide therapeutics and delivery systems

Area of Science:

• Biochemistry
• Molecular Biology
• Genetics

Background:

• Gene therapy is crucial for treating emerging and genetic diseases.
• Recent mRNA vaccines (Pfizer-BioNTech, Moderna) and gene therapies for disorders like Duchenne's muscular dystrophy and hemophilia demonstrate significant progress.
• RNA therapeutics are rapidly advancing in clinical translation.

Purpose of the Study:

• To provide novel insights into RNA therapeutics, focusing on endogenous RNA species.
• To explore RNA structure, function, and chemical modifications enhancing RNA stability and distribution.
• To offer an updated understanding of clinically approved gene therapies, their biochemical basis, and clinical applications.

Main Methods:

• Review of endogenous RNA species and their roles.
• Analysis of RNA structure and function.
• Examination of chemical modifications for RNA stability and delivery.
• Compilation of data on clinically approved RNA therapeutics.

Main Results:

• Significant advances in the clinical translation of RNA therapeutics have been observed.
• Diverse RNA species, their structures, and functions are critical for therapeutic development.
• Chemical modifications significantly improve RNA stability and biodistribution.
• Numerous gene therapies are now approved for treating fatal genetic disorders.

Conclusions:

• RNA therapeutics represent a powerful and evolving class of treatments for various diseases.
• Understanding RNA biology, chemistry, and clinical applications is essential for future therapeutic development.
• The reviewed gene therapies offer effective treatment options for previously intractable genetic conditions.