Historic market growth has been ... rare disease research pipelines, and the growing regulatory complexity associated with advanced therapies. Increased reliance on external scientific, clinical, and regulatory expertise has also strengthened demand for specialized contract research services. The cell and gene therapy CRO market is forecast to reach $12.36 billion by 2030, expanding at a CAGR of 12.3%. Key growth drivers include increasing clinical trial activity in ... Historic market growth has been supported by the rising number of early-stage cell and gene therapy programs, limited in-house development capabilities among therapy sponsors, expanding rare disease research pipelines, and the growing regulatory complexity associated with advanced therapies. Increased reliance on external scientific, clinical, and regulatory expertise has also strengthened demand for specialized contract research services. The cell and gene therapy CRO market is forecast to reach $12.36 billion by 2030, expanding at a CAGR of 12.3%. Key growth drivers include increasing clinical trial activity in gene therapy, greater investment in oncology and genetic disorder research, expansion of personalized medicine programs, and demand for faster development timelines. Regulators and healthcare systems are prioritizing precision-based treatments designed around genetic, molecular, and cellular characteristics, particularly for complex diseases with limited therapeutic options. Cell and gene therapy CROs support these programs through specialized preclinical studies, clinical trial management, regulatory strategy, safety monitoring, quality management, and analytical testing. Boost ROI by outsourcing R&D, integrating preclinical and clinical services, leveraging data-driven trials, strengthening compliance, and localizing labs... It also enhanced the organization's ability to manage global clinical trial programs involving advanced cell and gene therapies. The market supports a broad range of therapeutic areas, including oncology, central nervous system disorders, infectious diseases, immunological conditions, cardiovascular diseases, respiratory diseases, diabetes, ophthalmology, and pain management.

Major trends: Commercialization ... sickle cell disease, Shift from on-demand to prophylactic treatment regimens, Growth of bispecific antibodies in rare hematologic conditions, and Expansion of carrier screening and genetic diagnosis. Representative participants: Roche, Sanofi, CSL Behring, Novartis, and Pfizer. Immunology and genetic disorders form a diverse end-use sector covering rare primary immunodeficiencies, complement-mediated disorders, and inherited genetic ... Major trends: Commercialization of gene therapies for hemophilia and sickle cell disease, Shift from on-demand to prophylactic treatment regimens, Growth of bispecific antibodies in rare hematologic conditions, and Expansion of carrier screening and genetic diagnosis. Representative participants: Roche, Sanofi, CSL Behring, Novartis, and Pfizer. Immunology and genetic disorders form a diverse end-use sector covering rare primary immunodeficiencies, complement-mediated disorders, and inherited genetic conditions. Neurology is a high-value end-use sector for rare disease drugs, covering rare genetic neurological disorders, neuromuscular diseases, and selected ultra-rare central nervous system conditions. Currently, demand is anchored by enzyme replacement and biologic therapies for lysosomal storage disorders with neurological involvement, alongside emerging gene therapies for inherited retinal and neuromuscular conditions. Major trends: Maturation of gene therapies for inherited neurological disorders, Expansion of newborn screening for rare genetic conditions, Growth of antisense and RNA-targeted therapies for rare neurology, and Increasing focus on neuromuscular and neurodegenerative orphan indications. Representative participants: Biogen, Sarepta Therapeutics, Roche, Takeda Pharmaceutical, and Sanofi. Metabolic disorders constitute a foundational end-use sector for rare disease drugs, dominated by enzyme replacement therapies and substrate reduction approaches for lysosomal storage diseases and other inherited metabolic conditions. Hematology is a significant and expanding end-use sector for rare disease drugs, encompassing rare bleeding disorders, hemoglobinopathies, and other orphan blood conditions. Currently, demand is anchored by factor replacement therapies, monoclonal antibodies, and emerging gene therapies for hemophilia and sickle cell disease.

Washington, D.C. —October 2nd… Congressman Nick Langworthy today announced the introduction of the Biosimilars Access and Affordability Act, bipartisan legislation to expand access to lower-cost biosimilar prescription drugs and help bring them to market. Red tape should not stand between patients and affordable access to the cutting-edge treatments they need.” · The Biosimilars Access and Affordability Act is supported by the Alliance for Aging Research, Arthritis Foundation, Eosinophilic & Rare Disease Cooperative, Community Access National Network, Global Healthy Living Network, International Cancer Advocacy Network, International Pemphigus and Pemphigoid Foundation, Lupus and Allied Diseases Association, Inc., and Roswell Park. –Sarah Jones, Eosinophilic Rare Disease Coalition · Casey McPherson, Executive Director of the Alliance for Patient Access said, “Biosimilars give patients and physicians more affordable options for treating serious and chronic conditions. But unclear rules around Medicare's biosimilar delay provision are discouraging the investment needed to bring these treatments to market. The Biosimilars Access and Affordability Act restores the law's original intent with clear, predictable standards. This legislation is a pro-patient and pro-innovation solution to a harmful consequence of the IRA drug pricing negotiation program.” · Congressman Scott Peters said, “Biologics are critical to treating serious illnesses like cancer, autoimmune diseases and genetic disorders, but they are also some of the most expensive drugs on the market. Congressman Nick Langworthy today announced the introduction of the Biosimilars Access and Affordability Act, bipartisan legislation to expand access to lower-cost biosimilar prescription drugs and help bring them to market. The legislation updates Medicare policy to provide a clearer pathway for lower-cost biosimilar medicines that are actively moving toward the market, while protecting Medicare if those anticipated competitors never reach patients.