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Related Experiment Videos

FDA-Approved Oligonucleotide Therapies in 2017.

Cy A Stein1, Daniela Castanotto1

  • 1Department of Medical Oncology and Experimental Therapeutics, City of Hope Medical Center, 1500 East Duarte Road, Duarte, CA 91010, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|April 4, 2017
PubMed
Summary

Antisense oligonucleotides (ASOs), aptamers, and small interfering RNAs (siRNAs) have undergone extensive clinical trials. Six oligonucleotide therapeutics, evaluated by January 2017, demonstrated clear clinical benefit in rigorous studies.

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Area of Science:

  • Nucleic acid therapeutics
  • Oligonucleotide drug development
  • Clinical pharmacology

Background:

  • Oligonucleotides, including antisense oligonucleotides (ASOs), aptamers, and small interfering RNAs (siRNAs), have been in clinical development for decades.
  • Thousands of participants have been enrolled in numerous clinical trials evaluating these molecules.
  • Regulatory assessment identifies specific oligonucleotide therapeutics with proven clinical benefit.

Purpose of the Study:

  • To review the development and clinical success of oligonucleotide therapeutics.
  • To highlight the six oligonucleotide drugs assessed by regulatory authorities as of January 2017 for providing clear clinical benefit.
  • To provide a historical perspective on oligonucleotide-based therapies.

Main Methods:

Keywords:
DefitelioEteplirsenKynamroMacugenSpinrazaVitraveneaptameroligonucleotidephosphorothioatetherapeutics

Related Experiment Videos

  • Review of clinical trial data and regulatory assessments of oligonucleotide therapeutics.
  • Analysis of molecules that have reached advanced stages of clinical development.
  • Identification of drugs demonstrating clear clinical benefit in controlled trials.

    • Main Results:

    • Six oligonucleotide therapeutics were identified as providing clear clinical benefit based on rigorous trials as of January 2017.
    • The review details the clinical journey of these successful oligonucleotide drugs.
    • Significant investment in oligonucleotide research has led to approved therapies.

    Conclusions:

    • Oligonucleotide therapeutics have evolved significantly over 30 years of clinical development.
    • A select group of oligonucleotide drugs has proven effective and beneficial in clinical settings.
    • Continued research and development in oligonucleotide technology holds promise for future therapies.