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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Advances in Cell and Gene Therapy for Rare Disease Treatment.

Hyun Jin Baek1, Yoojun Nam1,2, Yeri Alice Rim3

  • 1YiPSCELL Inc., Seoul, Korea.

International Journal of Stem Cells
|August 31, 2025
PubMed
Summary

Rare disease treatments are advancing rapidly, driven by government support and innovative therapies like cell and gene therapies. The orphan drug market shows significant growth, exceeding the overall pharmaceutical industry.

Keywords:
Advanced therapyGenetic therapyOrphan drug productionRare diseasesRegulationStem cell therapy

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Area of Science:

  • Biotechnology
  • Pharmacology
  • Medical Research

Background:

  • Rare diseases affect small populations, often lacking effective treatments and facing research neglect due to low commercial viability.
  • Government initiatives since the 1990s have spurred rare disease research and development, leading to market growth exceeding the broader pharmaceutical sector.

Purpose of the Study:

  • To review orphan drug development within the rare disease market, focusing on advanced therapy medicinal products (ATMPs).
  • To explore the strategic use of cell and gene therapies as ATMPs to boost the competitiveness of the orphan drug industry.

Main Methods:

  • Literature review of orphan drug development trends.
  • Analysis of advanced therapy medicinal products (ATMPs), specifically cell and gene therapies, in the rare disease context.

Main Results:

  • The rare disease market is experiencing substantial growth, surpassing the overall pharmaceutical industry.
  • Cell and gene therapies are leading categories within ATMPs for rare diseases, showing active development and clinical application.

Conclusions:

  • ATMPs, particularly cell and gene therapies, offer significant opportunities for advancing rare disease treatments.
  • Strategic utilization of these advanced therapies can enhance the competitiveness of the orphan drug industry.