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Microorganisms in Medicine and Therapeutics01:29

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Nucleic Acid-Based Therapeutics in Orphan Neurological Disorders: Recent Developments.

Olga Khorkova1, Jane Hsiao1, Claes Wahlestedt2

  • 1OPKO Health, Miami, FL, United States.

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|May 17, 2021
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Nucleic acid-based therapeutics (NBTs) show promise for rare diseases, including neurological disorders. Advances in NBTs for orphan diseases are paving the way for personalized medicine and broader therapeutic applications.

Keywords:
antisense oligonucleotidegene therapyneurological disordernoncoding RNAorphan disordersiRNA

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Area of Science:

  • Biotechnology
  • Genetics
  • Neurology

Background:

  • Nucleic acid-based therapeutics (NBTs) offer rational design for faster, cost-efficient development, driving orphan disease therapy research.
  • Neurological disorders are key targets for NBTs, despite challenges in crossing the blood-brain barrier.
  • Orphan disease NBTs are advancing to clinical stages, necessitating infrastructure for design, testing, manufacturing, and regulatory support.

Purpose of the Study:

  • To review recent achievements in developing NBTs for orphan neurological disorders.
  • To highlight the broader impact of orphan disease NBT development on personalized medicine and common diseases.

Main Methods:

  • Review of current literature on NBTs for orphan neurological diseases.
  • Analysis of advancements in NBT design, delivery, and clinical application.
  • Synthesis of the infrastructure requirements for NBT development and implementation.

Main Results:

  • Significant progress in NBT development for rare neurological conditions.
  • Identification of key areas requiring further development, including genetic testing and manufacturing.
  • Recognition of the potential for NBTs to benefit both rare and common diseases.

Conclusions:

  • Developments in orphan neurological disease therapies are foundational for personalized medicine.
  • NBT technology platforms and understanding of disease mechanisms will advance treatment for a wider range of conditions.
  • Continued investment in infrastructure is crucial for realizing the full potential of NBTs.