Cell and Gene Therapies in Rare Disorders Market Outlook, Trends And Future Opportunities (2024-2031)

Cell and Gene Therapies in Rare Disorders Market is Forecasted to Hit US$ 17.3 Bn By 2031 | CAGR 25.6%

  • Date: 25 Mar, 2024
  • Author(s): Sagar Karlekar

Cell and Gene Therapies in Rare Disorders Market Expected to Achieve US$ 17.3 Bn by 2031 with a CAGR 25.7% from 2023 to 2031. The Cell and Gene Therapies in Rare Disorders Market is a segment of the broader biopharmaceutical industry, focusing on the development and commercialization of advanced therapeutic modalities for the treatment of rare genetic disorders. These innovative therapies hold the potential to address the underlying causes of various rare diseases, offering transformative treatment options for patients with limited or no existing therapeutic alternatives.

The growth of the Cell and Gene Therapies in Rare Disorders Market is driven by several factors, including the increasing prevalence of rare diseases, advancements in genomic research and gene editing technologies, and a supportive regulatory environment that facilitates the development and approval of these novel therapies. Additionally, the unmet medical needs in rare disease areas and the potential for personalized medicine have sparked significant interest from pharmaceutical companies and investors alike.

The market is witnessing a surge in investments and strategic collaborations between biotechnology companies, academic institutions, and pharmaceutical giants. The advent of gene therapy platforms, such as viral vectors and gene editing tools like CRISPR-Cas9, has opened up new avenues for developing targeted and potentially curative treatments for a wide range of rare disorders.

The Cell and Gene Therapies in Rare Disorders Market is poised for substantial growth in the coming years, driven by several key factors. The increasing understanding of genetic mechanisms underlying rare diseases, coupled with advancements in gene editing and cell therapy technologies, has paved the way for the development of innovative and targeted treatments. Additionally, the growing awareness and advocacy efforts surrounding rare diseases have prompted healthcare authorities and regulatory bodies to establish supportive frameworks and expedited approval pathways for these therapies.

The market dynamics are shaped by the collaboration between pharmaceutical companies, biotechnology firms, and academic research institutions. Strategic partnerships and licensing agreements have become pivotal in leveraging expertise and resources to accelerate the development and commercialization of cell and gene therapies for rare disorders.

Furthermore, the market presents significant opportunities for personalized medicine, as many rare diseases have a distinct genetic profile. By tailoring treatments to individual patients' genetic characteristics, cell and gene therapies offer the potential for more effective and targeted interventions.

The market opportunity assessment is also influenced by the growing recognition of the economic burden associated with rare diseases. As these therapies have the potential to address the underlying causes of rare disorders, they could lead to significant cost savings in long-term healthcare expenditures and improved patient outcomes.

However, challenges such as manufacturing complexities, logistical hurdles, and the high costs associated with these advanced therapies remain significant barriers to widespread adoption. Nonetheless, the Cell and Gene Therapies in Rare Disorders Market is expected to witness substantial growth, driven by the unmet clinical needs, technological advancements, and increasing investments in this rapidly evolving field.

Major market drivers:

  1. Increasing prevalence of rare diseases and unmet medical needs: The growing number of individuals affected by rare genetic disorders, coupled with a lack of effective treatment options, has fueled the demand for innovative cell and gene therapies. Supporting statistics: According to the National Institutes of Health (NIH), there are approximately 7,000 known rare diseases affecting an estimated 30 million people in the United States alone.
  2. Advancements in genomic research and gene editing technologies: Breakthroughs in genomic research, such as the development of gene editing tools like CRISPR-Cas9, have revolutionized the field of cell and gene therapies. These technologies enable precise genetic modifications, paving the way for more effective and targeted treatments for rare disorders. Supporting statistics:
  3. Supportive regulatory environment and accelerated approval pathways: Regulatory authorities, such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have established expedited approval pathways and incentives for the development of therapies targeting rare diseases. These initiatives have facilitated the rapid advancement of cell and gene therapies in the market. Supporting statistics: As of 2023, the FDA has approved over 20 gene therapy products for various indications, including rare genetic disorders (FDA).


  1. Emergence of personalized medicine approaches for rare diseases.
  2. Increasing adoption of advanced delivery systems for gene therapies.

Market opportunity:

The development of off-the-shelf, allogeneic cell therapies for rare disorders.

Key Report Insights:

  • Largest Region: North America, with a market share of 42%. Prominent companies in the region include Novartis, Bluebird Bio, and Spark Therapeutics. The region's strong market position is driven by factors such as robust healthcare infrastructure, favorable reimbursement policies, and a well-established biopharmaceutical industry.
  • Second Largest Region: Europe, with a market share of 32%. Major players in the region include GlaxoSmithKline, Orchard Therapeutics, and Sanofi. The region's growth is fueled by supportive regulatory frameworks, investments in research and development, and a growing focus on personalized medicine.
  • Prominent companies in the Cell and Gene Therapies in Rare Disorders Market include Novartis, Bluebird Bio, Spark Therapeutics, GlaxoSmithKline, Orchard Therapeutics, Sanofi, Celgene (a subsidiary of Bristol-Myers Squibb), Biomarin Pharmaceutical, Sangamo Therapeutics, and Allergan (now part of AbbVie).

Market Segmentation:

  • By Type of Therapy
    • Cell Therapy
    • Gene Therapy
    • Gene-Modified Cell Therapy
  • By Disease Indication
    • Neurological Disorders (e.g., Spinal Muscular Atrophy, Parkinson's Disease)
    • Hematological Disorders (e.g., Sickle Cell Disease, Hemophilia)
    • Metabolic Disorders (e.g., Lysosomal Storage Disorders, Fabry Disease)
    • Ophthalmological Disorders (e.g., Inherited Retinal Disorders)
    • Immunological Disorders (e.g., Primary Immunodeficiencies, Severe Combined Immunodeficiency)
    • Musculoskeletal Disorders (e.g., Duchenne Muscular Dystrophy)
    • Others (e.g., Cystic Fibrosis, Huntington's Disease)
  • By Vector Type
    • Viral Vectors (e.g., Lentivirus, Adeno-Associated Virus)
    • Non-Viral Vectors (e.g., Plasmid DNA, Oligonucleotides)
  • By Cell Type
    • Hematopoietic Stem Cells
    • Mesenchymal Stem Cells
    • Induced Pluripotent Stem Cells
    • Others (e.g., T-cells, NK cells)
  • By Route of Administration
    • Intravenous
    • Intramuscular
    • Subretinal
    • Others (e.g., Intranasal, Intrathecal)
  • By End-User
    • Hospitals
    • Specialty Clinics
    • Academic and Research Institutes
    • Others (e.g., Biotechnology Companies, Contract Research Organizations)
  • By Region
    • North America
      • U.S.
      • Canada
    • Europe
      • Germany
      • U.K.
      • Spain
      • France
      • Italy
      • Russia
      • Rest of Europe
    • Asia Pacific
      • China
      • India
      • Japan
      • Australia
      • South Korea
      • ASEAN
      • Rest of Asia Pacific
    • Latin America
      • Brazil
      • Argentina
      • Mexico
      • Rest of Latin America
    • Middle East & Africa
      • GCC Countries
      • Israel
      • South Africa
      • North Africa
      • Central Africa
      • Rest of the Middle East


“The Cell and Gene Therapies in Rare Disorders Market refers to the industry focused on developing and commercializing innovative treatments for rare diseases using advanced cellular and genetic technologies. Cell therapies involve transplanting healthy cells into patients to replace or repair damaged or dysfunctional cells, while gene therapies aim to introduce functional genes or correct genetic defects by modifying the patient's cells. These cutting-edge therapies offer the potential to address the underlying causes of rare disorders, many of which have a genetic basis, by targeting the root cause rather than just managing symptoms. This market encompasses various types of therapies, disease indications, delivery methods, and stakeholders working towards providing personalized and potentially curative treatments for patients with rare and often debilitating conditions.”

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