CDMOs provide affordable services by offering access to specialized expertise and infrastructure without requiring organizations to make noteworthy capital investments, driving the growth of the market.
What is Rare Disease-Focused Cell & Gene Therapy CDMO?
CDMOs provide a wide range of solutions that streamline the path from discovery to commercialization, eventually helping patients with rare diseases who often have limited treatment options. CDMOs own the infrastructure and expertise to handle these unique needs, adapting their amenities and processes to accommodate the particular requirements of each therapy. CDMOs provide an affordable solution by offering access to specialized expertise and infrastructure without requiring companies to make significant capital spending. CDMOs empower pharmaceutical organizations and biotech start-ups to overcome the unique trials of rare disease drug development and bring life-changing therapies to patients more quickly. Cell and gene therapies offer novel clinical promises for treating rare diseases via their unique mechanisms of action.
Market Growth
The global rare disease-focused cell & gene therapy CDMO market size was estimated at USD 6.8 billion in 2025 and is predicted to increase from USD 8.07 billion in 2026 to approximately USD 37.76 billion by 2035, expanding at a CAGR of 18.7% from 2026 to 2035.
Recent Advancements in Biomedical Robots:
Biomedical robots consist of algorithms or classifiers that analyze data, generate advanced knowledge, and function as healthcare decision support systems (DSS). AI-powered robotics improve diagnostic capabilities by leveraging extensive statistics, including rare disease cases, and making informed predictions based on trends that might be difficult for human doctors to detect. These robots are employed in complex surgeries, clinical training, medication dispensing, personal care, and various other applications. Platforms such as XtalPi and RareLabs merge AI-driven computational modeling with robotic laboratory automation to facilitate the design, screening, and validation of therapies.
Number of patients based on country-specific orphan drug designation criteria
| Region |
Number of patients with rare diseases |
| United States |
200,000 |
| Japan |
50,000 |
| Korea |
20,000 |
| Taiwan |
10,000 |
| Australia |
2,000 |
Market Segmentation Overview
- By service type, the manufacturing services segment contributed the largest U.S. rare disease-focused cell & gene therapy CDMO market share of 46% in 2025, as cell therapy manufacturing services for different autologous and allogeneic cell types and preliminary materials. Cell & gene therapy production services encompass a range of specialized processes and technologies aimed at producing cell-based and gene-based therapies. They enable comprehensive cell & gene therapy services (CDMO) from research to commercialization.
- By therapy type, the gene therapy segment contributed the largest market share of 52%, as gene therapy is specifically auspicious because a single genetic mutation causes major rare diseases. Gene therapy is developing as an auspicious treatment. Gene therapy has the strength to be a one-time, long-term management that directly addresses the underlying cause of some rare diseases.
- By indication, the oncology rare diseases segment contributed the largest U.S. rare disease-focused cell & gene therapy CDMO market share of 30%, as increasing demand for emerging rare drugs using advanced biopharmaceuticals is growing because around 80% of rare diseases have genetic components. CDMOs provide an affordable solution by offering access to specialized expertise and structure without requiring companies to make significant capital spending.
- By end user, the biopharmaceutical companies segment contributed the largest market share of 68%, as CDMOs experience working with regulatory agencies and provide significant guidance on government strategy, clinical trial design, and data management, which significantly quickens the approval process. CDMOs empower pharmaceutical organizations and biotech start-ups to overcome the unique complexities of rare disease drug development.
Top Companies in the U.S. Rare Disease-Focused Cell & Gene Therapy CDMO Market
The top four organizations in this market dominate the U.S. rare disease-focused cell & gene therapy CDMO market. Meanwhile, Lonza has aggressively positioned itself in the cell and gene therapy space. Catalent Biologics offers an end-to-end solution for AAV, lentiviral vectors, and mRNA synthesis, important for rare disease treatments. Thermo Fisher Scientific has now expanded its network with a novel advanced therapy collaboration center in Philadelphia. Charles River Laboratories offers end-to-end gene-modified cell therapy services in the U.S., particularly targeting early-stage clinical advancement.
Segments Covered in the Report
By Service Type
- Process Development
- Upstream Process Development
- Downstream Process Development
- Analytical Method Development
- Manufacturing Services
- Viral Vector Manufacturing
- AAV Manufacturing
- Lentiviral Vector Manufacturing
- Retroviral Vector Manufacturing
- Cell Therapy Manufacturing
- Autologous Cell Therapy
- Allogeneic Cell Therapy
- Plasmid DNA Manufacturing
- Fill & Finish Services
- Quality Control & Testing
- Potency Testing
- Safety Testing
- Stability Testing
By Therapy Type
- Gene Therapy
- In vivo Gene Therapy
- Ex vivo Gene Therapy
- Cell Therapy
- CAR-T Therapy
- Stem Cell Therapy
- TCR Therapy
- Gene-Modified Cell Therapy
By Indication
- Oncology Rare Diseases
- Neurological Disorders
- Genetic Disorders
- Hematological Disorders
- Metabolic Disorders
- Immunological Disorders
By End User
- Biopharmaceutical Companies
- Large Pharma
- Emerging Biotech
- Academic & Research Institutes
- Hospitals & Specialty Clinics
By Scale of Operation
- Preclinical Scale
- Clinical Scale
- Phase I
- Phase II
- Phase III
- Commercial Scale
