A perspective on oligonucleotide therapy: Approaches to patient customization

Area of Science:

• Biotechnology
• Molecular Biology
• Genomics

Background:

• The human genome encodes numerous disease-modifying proteins, but only a small fraction are druggable with current therapeutics.
• A significant gap exists in treating diseases caused by non-druggable targets, necessitating novel therapeutic strategies.
• Oligonucleotide therapeutics (OTs) are emerging as a powerful class of drugs with high target selectivity.

Purpose of the Study:

• To review the current landscape of oligonucleotide therapeutics (OTs), including their historical development and future potential.
• To highlight the advantages of OTs, such as improved selectivity and reduced off-target effects compared to small molecules.
• To discuss the application of OTs in treating various diseases, including neurodegenerative disorders, cancer, and rare genetic conditions.

Main Methods:

• Comprehensive literature review of approved and investigational oligonucleotide therapeutics.
• Analysis of current challenges and bottlenecks in OT development and drug delivery.
• Exploration of recent advancements in OT delivery systems and personalized medicine approaches.

Main Results:

• Oligonucleotide therapeutics (including Antisense RNAs, microRNA, siRNA, and aptamers) demonstrate significant potential across diverse therapeutic areas.
• Advancements in drug delivery are crucial for overcoming challenges and enhancing the efficacy of OTs.
• Emerging personalized treatment strategies using OTs show promise for rare and fatal diseases.

Conclusions:

• Oligonucleotide therapeutics represent a rapidly advancing field with the potential to address unmet medical needs.
• Continued research into OT delivery and personalized medicine will be key to realizing their full therapeutic potential.
• OTs offer a viable alternative for targeting both druggable and non-druggable proteins, expanding therapeutic options.