Authoritative market research on cell therapy, gene therapy, and RNA therapy analyzing markets by disease type
Cell therapy and gene therapy represent a frontier in modern medicine, promising revolutionary treatments by treating the underlying genetic causes of disease and by genetically controlling the body's immune system. Recent years have seen an increase in new therapies and the pipeline indicates this trend will continue. A large range of therapeutic areas from oncology to cardiology have seen rapidly proliferating clinical trials. "Cell Therapy and Gene Therapy Markets (Markets by Disease Type), 2023-2029" from Kalorama Information presents the market in segments that cover market estimates and forecasts, the development pipeline, and the competitive landscape of leading providers for each therapeutic area.
The report examines global developments in cell, gene, and RNA therapy markets for the select diseases and conditions in respective chapters, including principal products, trends in research and development, market breakdown of therapies, regional market revenues summary, and competitor summary.
The following therapeutic areas are covered in individual chapters with accompanying tables and figures:
Oncology
Neurology
Musculoskeletal
Dermatology
Cardiology/non-oncology blood
Other
Scope
The report covers markets for cell therapies, gene therapies, and other various forms of RNA therapies. This report does not include RNA therapies for COVID-19 or mRNA vaccines for infectious diseases in its scope.
We are excluding mRNA-based products for infectious disease in this report because that area has largely been related to vaccines which is distinct; and partly due to the obscurity of the definition of cell and gene therapy. It will, however, be addressed in other Kalorama Information vaccine reports. RNA, antisense, and related types of therapies for other diseases are included due to the broad definition of gene therapy, including the use of nucleic acids to influence gene expression.
Note that this report also does not focus on cord blood products in the scope; these have technically been approved as cell therapy products by the FDA. There is some debate as to whether these are more akin to transplants.
Diseases are categorized into therapeutic areas primarily according to the general consensus but this is often an over-simplification; genetic diseases can become complicated to classify due to many systems being affected. In some cases, there may be inconsistencies. For example, a genetic mutation may affect the liver which results in excessive amounts of a certain molecule. This molecule then causes harm to the brain, and the condition is categorized as neurological despite the origin being in the liver.
Generally, the primary effects will first dictate the category, in combination with the condition's cause. It can be case-by-case.
Information for this report was gathered using both primary and secondary research including comprehensive research of secondary sources such as company reports and literature; government documents and databases; research journals and reports; and general medical and business journals. Primary research methods include telephone interviews and email correspondence with company representatives, researchers and physicians. These interviews were conducted with the purpose of capturing the perspectives from industry participants on the market opportunity and current trends. This was the basis of the formulation of forecasting models and for confirming early findings related to market potential.
The market segments provide highlights and overviews of therapies, market estimates and forecasts, and competitive situations of leading providers. The report also includes information on cell and gene therapies in development and the impact these are expected to have on the market. Throughout the report, several market indicators, trends, and barriers are discussed.
Because gene therapies are currently not available in any wide capacity, there is little precedent upon which to base forecasts. Dollar figures represent the estimated global market for 2023 and expected market for 2029. Forecasts are provided through 2029 ,and the size of each market segment refers to manufacturers' revenues.
Table of Contents
Chapter 1: Executive Summary
Overview
Scope and Methodology
Market Overview and Potential
Figure 1-1: Global Cell and Gene Therapy Market, by Product Segment (Cardio/Non-Oncology Blood, Dermatology, Musculoskeletal, Neurology, Oncology, Other), 2023-2029 ($ millions)
Table 1-1: Global Cell and Gene Therapy Market, by Product Segment (Cardio/Non-Oncology Blood, Dermatology, Musculoskeletal, Neurology, Oncology, Other), 2023-2029 ($ millions)
Chapter 2: Introduction to Cell and Gene Therapy
Introduction
Table 2-1: Total Numbers of Approved Cell, Gene, & RNA Therapies, by Type, Q4 2022-Q1 2024 (ending March 2024), Global (count, %)
Figure 2-1: Share of Total Global Numbers of Approved Cell, Gene, & RNA Therapies, by Type, March 2024 (%)
Cell Therapy
CAR T
Stem Cells
Adult Stem Cells
Human Embryonic Stem Cells
Table 2-2: Adult vs. Embryonic Stem Cells
Gene Therapy
Table 2-3: Somatic vs. Germ Line Gene Therapy
Viral Vector System and New Developments
Table 2-4: Selected Examples of Viruses in Gene Therapy
Figure 2-2: Estimated Viral Vector Use in Commercialized Cell and Gene Therapies, by Type, 2022 (%)
Non-viral Systems for Transporting Genes
Gene Editing
Cell and Gene Therapy: Characteristics
Table 2-5: Cell Therapy vs Gene Therapy
Staffing
Manufacturing Process for CAR-T
Leukapheresis
Activation
Transduction
Expansion
Cell and Gene Therapy Manufacturing Processes
Table 2-6: Manufacturing Process Time for Select Autologous Cell and Gene Therapies (current and discontinued therapies)
Figure 2-3: Manufacturing Process Time for Select Autologous Cell and Gene Therapies (current and discontinued therapies)
Cost
Reimbursement
Logistics
Mergers and Acquisitions
Table 2-7: Selected Company Mergers and Acquisitions in the Cell and Gene Therapy Market
Deals, Agreements, and Collaborations
Regulatory Developments
Fast Track
Orphan Drug
Breakthrough Therapy
Accelerated Approval
Priority Review
Table 2-8: Regulatory Actions and Guidelines for Cell and Gene Therapy, by Select Country
Individualized Gene Therapy Approval Process
Pipeline, Development Overview
Pipeline, Development Progress
Table 2-9: Global Cell, Gene, & RNA Therapy Clinical Trial Growth (pre-clinical I through phase III), Q4 2022-Q1 2024 ($ millions, %)
Figure 2-4: Global Cell, Gene, & RNA Therapy Development Progress (pre-clinical through phase III), as of March 2024
Table 2-10: Global Gene Therapy Pipeline, by Phase, Q4 '22-Q1 '24 (count, %)
Figure 2-5: Global Gene Therapy Pipeline Shares by Phase, Q1 '24 (%)
Pipeline by Condition and Therapeutic Area
Table 2-11: Global Cell, Gene, & RNA Therapy Pipeline, by Therapeutic Area (Alimentary/Metabolic, Anticancer, Anti-infective, Blood and Clotting, Cardiovascular, Dermatological, Genitourinary [including sex hormones], Hormonal [excluding sex hormones], Immunological, Musculoskeletal, Neurological, Rare Diseases, Respiratory, Sensory, Miscellaneous, Other), Q4 '22-Q1 '24 (count, %)
Figure 2-6: Global Gene Therapy Pipeline Shares, by Therapeutic Area (Alimentary/Metabolic, Anticancer, Anti-infective, Blood and Clotting, Cardiovascular, Dermatological, Genitourinary [including sex hormones], Hormonal [excluding sex hormones], Immunological, Musculoskeletal, Neurological, Rare Diseases, Respiratory, Sensory, Miscellaneous), Q1 '24 (%)
Table 2-12: Global Cell, Gene, & RNA Therapy Pipeline Excluding Pre-clinical, by Therapeutic Area (Alimentary/Metabolic, Anticancer, Anti-infective, Blood and Clotting, Cardiovascular, Dermatological, Genitourinary [including sex hormones], Hormonal [excluding sex hormones], Immunological, Musculoskeletal, Neurological, Rare Diseases, Respiratory, Sensory, Miscellaneous), Q4 '22-Q1 '24 (count, %)
Table 3-4: Select Oncology Phase III Cell, Gene, & RNA Therapy Developments, through March 2024
Market Breakdown of Cell and Gene Therapies
Table 3-5: Oncology Cell and Gene Therapy Markets, by Product Type (CAR-T, Cell Immunotherapy, Gene Therapy), Estimated 2023 and Projected 2029 ($ millions)
Figure 3-5: Oncology Cell and Gene Therapy Market Distribution, by Product Type (CAR-T, Cell Immunotherapy, Gene Therapy), 2023 (%)
Oncology Cell and Gene Therapy Market by Cancer Type
Table 3-6: Oncology Cell, Gene, & RNA Therapy Market, by Cancer Type (Leukemia, Lymphoma, Melanoma, Multiple Myeloma, Prostate Cancer, Undefined Blood Cancer, Other Cancers), Estimated 2023 and Projected 2029 ($ millions)
Table 3-7: Oncology Cell, Gene, & RNA Therapy Market, by General Indication (Leukemia, Lymphoma, Melanoma, Multiple Myeloma, Prostate Cancer, Other Cancers), 2023 (%)
Figure 3-6: Oncology Cell, Gene, & RNA Therapy Market, by General Indication (Leukemia, Lymphoma, Melanoma, Multiple Myeloma, Prostate Cancer, Other Cancers), 2023 (%)
Regional Market Summary
Table 3-8: Oncology Cell and Gene Therapy Market, by Country/Region (Europe, United States, Other Countries), Estimated 2023 and Projected 2029 ($ millions)
Figure 3-7: Oncology Cell and Gene Therapy Market Summary, by Geographic Region (Europe, United States, Other Countries), Estimated 2023 and Projected 2029 ($ millions)
Table 4-5: Neurological Conditions Cell and Gene Therapy Market, by Country/Region (Europe, United States, Other Countries), Estimated 2023 and Projected 2029 ($ millions)
Figure 4-3: Neurological Conditions Cell, Gene, & RNA Therapy Market Summary, by Geographic Region (Europe, United States, Other Countries), Estimated 2023 and Projected 2029 ($ millions)
Competitor Summary
Table 4-6: Neurological Conditions Cell and Gene Therapy Competitor Market Share, Reported and Estimated 2023 ($ millions)
Figure 5-3: Musculoskeletal Cell, Gene, & RNA Therapy Market, by Country/Region (Europe, United States, South Korea, ROW), Estimated 2023 (%)
Table 5-6: Musculoskeletal Cell, Gene, & RNA Therapy Market, by Country/Region (Europe, United States, South Korea, ROW), Estimated 2023 and Projected 2029 ($ millions)
Figure: 5-4: Musculoskeletal Cell and Gene Therapy Market Summary, by Geographic Region (Europe, United States, South Korea, ROW), Estimated 2023 and Projected 2029 ($ millions)
Chapter 6: Cell and Gene Therapy Markets in Dermatology
Overview
Table 6-1: Wound Prevalence, by Type (Skin Ulcers: Pressure Injuries, Venous Statis Ulcers, Diabetic Ulcers and Burns: Outpatient Burns, Hospitalized Burns, and Medically Treated Burns)
Market Outlook
Figure 6-1: Cell and Gene Therapy Markets in Dermatology, 2023-2029 ($ millions)
Principal Products
Adipocel
Apligraf
CureSkin
Dermagraft
Epicel
Grafix
Holoderm
Kaloderm
KeraHeal/KeraHeal-Allo
LaViv
Orcel
Quencell
RECELL
Rosmir
Stempeutics/Cutisera
StrataGraft
Stravix
TransCyte
Vyjuvek
Table 6-2: Cell and Gene Therapy in Dermatological Diseases and Disorders
Trends in Research and Development
Phase III Development
Table 6-3: Select Dermatology Phase III Cell and Gene Therapy Developments, through March 2024
Market Breakdown of Cell and Gene Therapies
Table 6-4: Dermatology Cell and Gene Therapy Markets, by Product Type (Stem Cells, Skin Cells, Gene/Gene-Modified Cell Therapy), Estimated 2023 and Projected 2029 ($ millions)
Figure 6-2: Dermatology Cell and Gene Therapy Market Distribution, by Product Type (Stem Cells, Skin Cells, Gene/Gene-Modified Cell Therapy), 2023 (%)
Dermatology Cell and Gene Therapy Market by Indication
Regional Market Summary
Table 6-5: Dermatology Cell and Gene Therapy Market, by Country/Region (Asia, Europe, United States, Other Countries), Estimated 2023, Projected 2029 ($ millions)
Figure: 6-3: Dermatology Cell and Gene Therapy Market Summary, by Geographic Region (Asia, Europe, United States, ROW), Estimated 2023 and Projected 2029 ($ millions)
Competitor Summary
Table 6-6: Dermatology Cell and Gene Therapy Competitor Market Share, Reported and Estimated 2023 ($ millions)
Figure: 6-4: Dermatology Cell and Gene Therapy Market Share, by Company, Estimated 2023 (%)
Chapter 7: Cell and Gene Therapy Markets in Cardiovascular and Blood Disorders
Overview
Cardiovascular Disease
Selected Blood Disorders
Market Outlook
Table 7-1: Cell, Gene, and RNA Therapy Markets in Cardiovascular and Blood Disorders, 2023-2029 ($ millions)
Figure 7-1: Cell, Gene, and RNA Therapy Markets in Cardiovascular and Blood Disorders, 2023-2029 ($ millions)
Principal Products
Collategene
Hearticellgram
HeartSheet
Leqvio
Neovasculgen
Stempeucel
Zynteglo
Table 7-2: Selected Cell and Gene Therapy in Cardiovascular and Blood Disorders
Phase III Development
Table 7-3: Select Cardiovascular and Blood Disorder Phase III Cell and Gene Therapy Developments, through Mar 2023
Market Breakdown of Cell, Gene, and RNA Therapies
Table 7-4: Cardiovascular and Blood Disorders Cell, Gene, & RNA Therapy Markets, by Product Type (Cell-based Gene Therapy, Non-GM Cell, RNA, Other Gene Therapies), Estimated 2023 and Projected 2029 ($ millions)
Figure 7-2: Cardiovascular and Blood Disorders Cell and Gene Therapy Market Distribution, by Product Type (Cell-based Gene Therapy, Non-GM Cell, RNA, Other Gene Therapies), 2023 (%)
Cardiovascular and Blood Disorders Cell and Gene Therapy Market by Indication
Table 7-5: Cardiovascular and Blood Disorders Cell and Gene Therapy Market, by General Indication (Blood, Cardiac, Hypercholesterolemia, Vascular), Estimated 2023 and Projected 2029 ($ millions)
Regional Market Summary
Table 7-6: Cardiovascular and Blood Disorders Cell and Gene Therapy Market, by Country/Region (APAC, Europe, United States, ROW), Estimated 2023 and Projected 2029 ($ millions)
Figure 7-3: Cardiovascular and Blood Disorders Cell and Gene Therapy Market Summary, by Geographic Region (APAC, Europe, United States, ROW), Estimated 2023 and Projected 2029 ($ millions)
Competitor Summary
Chapter 8: Cell and Gene Therapy Markets in Other Conditions
Overview
Market Outlook
Figure 8-1: Cell and Gene Therapy Markets in Other Conditions, 2023-2029 ($ millions)
Table 8-1: Cell and Gene Therapy Markets in Other Conditions, 2023-2029 ($ millions)
Principal Products
Alofisel
Cupistem
Givlaari
Glybera
Holoclar
Luxturna
Oxlumo
Rethymic
Strimvelis
Temcell/Prochymal/Ryoncil
Table 8-2: Cell and Gene Therapy in Other Diseases and Disorders
Trends in Research and Development
Market Breakdown of Cell and Gene Therapies
Table 8-3: Other Conditions Cell and Gene Therapy Markets, by Product Type (Cell-based Gene Therapy; RNA, Antisense Etc; Stem Cell & Other Cell Therapy; Other Gene Therapy), Estimated 2023 and Projected 2029 ($ millions)
Figure 8-2: Other Conditions Cell and Gene Therapy Market Distribution, by Product Type (Cell-based Gene Therapy; RNA, Antisense Etc; Stem Cell & Other Cell Therapy; Other Gene Therapy), 2023 (%)
Regional Market Summary
Table 8-4: Other Conditions Cell and Gene Therapy Market, by Country/Region (Europe, Japan, South Korea, United States, Other Countries), Estimated 2023 and Projected 2029 ($ millions)
Figure 8-3: Other Conditions Cell and Gene Therapy Market Summary, by Geographic Region (Europe, Japan, South Korea, United States, Other Countries), Estimated 2023 and Projected 2029 ($ millions)
Competitor Summary
Chapter 9: Cell and Gene Therapy Market Review
Market Influences
Global Demographics
Table 9-1: Global Population, through 2050 (population in millions)
Aging Population
Figure 9-1: Global Population Trends, Total Population vs. Age 65+ Population, 2000-2050
Cost Containment
Table 9-2: Cost Comparisons for Selected Commercialized Cell and Gene Therapies