Gene Therapy Statistics and Facts (2025)
Updated · Mar 18, 2025
WHAT WE HAVE ON THIS PAGE
- Introduction
- Editor’s Choice
- Number Of Gene Therapies In Pipeline By Phase
- Cancer Gene Therapy Market Forecast
- Market Capitalization Of Gene And Cell Therapy
- Market Share By Region
- Gene Therapy Clinical Trials By Indications
- Distribution Of Active Gene Therapy Clinical Trials By Vector
- Number Of Gene Therapy Approved
- Number Of Oncology Gene Therapy Pipeline
- Gene Therapy Cost
- Funding And Strategic Importance Of Gene Therapy Companies
- Conclusion
Introduction
Gene Therapy Statistics: Leading the charge in modern medicine, gene therapy presents hopeful solutions to some previously incurable genetic disorders. By altering or transplanting defective genes, this revolution in healthcare could potentially change the face of such treatment. As we move into the 2025 arena, gene therapy is fast becoming the most talked about thing in medicine, showing very high and significant developments with increasing market value along with more activity in clinical trials.
The article will detail some of the industry-shaping gene therapy statistics and trends applicable to the current year.
Editor’s Choice
- According to gene therapy statistics, around 1,400 gene therapies under consideration in preclinical studies as of Q3 2024 reflect the evolution of the research and solid approaches made in gene therapy development.
- The market for gene therapy for cancer was estimated at roughly many billion in 2023 and predicted by some forecasts to approach almost USD 15 billion by 2032 because of growing identification and methodologies of progress in therapeutic approaches.
- The current gene therapy market capitalisation, represented by firms such as Bluebird Bio-oncology gene therapy, is USD 320 million, while smaller companies are witnessing capital shrinkage due to the industry’s adjusting challenges.
- Gene therapy statistics reveal that North America accounted for nearly 56% of the overall market in cell and gene therapy in 2020, mainly due to well-developed health infrastructure, large investments in biotechnology, and very many trials and approvals.
- Thus, from 2021 to 2027, the cell and gene therapy market is expected to yield a compound annual growth rate of approximately 33.8%, which demonstrates that the market will expand considerably.
- In 2022, there were more than 1,200 ongoing clinical trials for cell and gene therapies worldwide, of which around 64% focus on oncology, thus affirming the significance of gene therapy in cancer care.
- Gene therapy statistics state that until June 2022, lentiviruses were the most popular gene therapy vectors in clinical trials, accounting for nearly 50 % of all trials.
- After undergoing modification, 31 gene therapies, including genetically modified cell therapies and 30 RNA therapies, had been approved globally by as much as Q2 2024, with Rytelo for myelodysplastic syndrome and Moderna’s mRESVIA for RSV noteworthy among such approvals.
- The oncology gene therapy pipeline is very promising as it currently includes 131 clinical trials targeting the CD19 molecule, with other relevant targets like TNF receptor superfamily member 17 and CD22 molecules still within the ambit of ongoing research.
- Gene therapy statistics show that it is indeed expensive; for example, treatments such as Hemgenix® for haemophilia B at USD 3.5 million per injection and new therapies for sickle cell disease at USD 2.2 million to USD 3.1 million altogether make it impossible for many healthcare providers to afford them.
- Gene therapy has been responsible for 19% of the total number of licensing and collaboration deals in 2023, despite the fact that it represents only 10% of new approvals, underscoring the future potential for their growth and strategic importance.
Number Of Gene Therapies In Pipeline By Phase
(Reference: statista.com)
- Gene therapy statistics reveal that there are almost 1,400 gene therapies in the preclinical phases of research as of the third quarter of 2024, which illustrates the research and development work currently being conducted.
- This includes the steady progress of gene therapy products through various development stages from late 2021 onward.
- The number of therapies in the pipeline has grown consistently over the past three years, with most still in the early phases of testing and refinement.
- The quarterly data from Q4 2021 to Q3 2024 represent the increasing emphasis on moving therapies from the preclinical stage toward clinical trials, embodying the industry’s advanced innovation and fulfilment of unmet needs.
Cancer Gene Therapy Market Forecast
(Reference: statista.com)
- Gene therapy statistics state that the world market for cancer gene therapy was valued at about USD 3 billion in 2023, according to estimates.
- This market will grow rapidly over the next few years and is predicted to be worth almost USD 15 billion in 2032.
- This figure underscores the phenomenal expansion of the cancer gene therapy market over the decade as demands increase, treatment methods evolve, and the application of gene therapy to curing different types of cancer becomes possible.
- It states that growth is expected to be about five times that amount, indicating how much higher investments and attention are directed in this area as newer technologies and therapies emerge.
Market Capitalization Of Gene And Cell Therapy
(Reference: statista.com)
- Gene therapy statistics show that the last assessment of the market capitalisation of certain major gene and cell therapy businesses, excluding those of much larger companies active in the sector, took place on March 31, 2023.
- A case in point is Bluebird Bio-oncology gene therapy, which has approximately USD 320 million in capital.
- Several smaller gene and cell therapy organisations have seen their capital contracts over the past few years due to the challenges facing the sector.
- The gene therapy field is believed to be bright, but the financial futures of some companies have not been as great as predicted.
(Reference: statista.com)
- As of 2020, North America continued to dominate the worldwide cell and gene therapy market in almost every aspect, as the completed region contributed almost 56% to the overall market share.
- With advanced healthcare infrastructure, very significant biotech investments, and a considerable number of clinical trials and approvals, it’s clear that this region has its claim to fame.
- The global cell and gene therapy market is set to bubble over with an anticipated surge in significant growth potential.
- The projections rate this market at a compound annual growth rate of 33.8% from 2021 to 2027, indicating that a rapid expansion may take place during this period.
- Global expansion is expected due to continued advancement in gene therapy technologies, increasing investments, and a rising number of treatment approvals.
Gene Therapy Clinical Trials By Indications
(Reference: statista.com)
- Gene therapy statistics indicate that in 2022, over 1,200 clinical trials on cell and gene therapies were in progress worldwide.
- Out of the total number of trials, almost 770 were particularly for oncology, representing about 64% of the entire number devoted to this field in terms of cancer treatments.
- This significant distribution shows how critical cell and gene therapies are in combating different types of cancers and that researchers and industries are increasingly focusing on developing new directed therapies for cancer patients.
- The high number of ongoing trials in oncology is indicative of the increasing attention and research investment being placed into finding innovative strategies for cancer treatments through advanced gene editing and personalised therapies.
Distribution Of Active Gene Therapy Clinical Trials By Vector
(Reference: statista.com)
- By the end of June 2022, existing clinical trials for gene and cell-based immuno-oncology therapeutic interventions were deployable according to the therapeutic vectors used.
- Lentivirus therapies were the most widely employed, comprising almost 50% of all therapies.
- This strongly suggests that the prenaturation of lentiviruses makes gene therapy and immuno-oncology possible.
- Other kinds of vectors were used for the rest of the trials, but lentiviruses predominated, as they provided the best delivery of genetic material into target cells.
Number Of Gene Therapy Approved
(Reference: asgct.org)
- Gene therapy statistics state that, from a global perspective, 31 gene therapies, including genetically modified cell therapies, have been given the green light for use at the clinical level. There are also 30 RNA therapies on the approval list.
- The most exciting approvals include Rytelo, the oligonucleotide telomerase inhibitor developed by Geron, which was approved for therapeutic application in cases of myelodysplastic syndrome in the U.S.
- Also, mRESVIA, the mRNA-based SRV vaccine developed by Moderna, received approval in the U.S., and 68 more non-genetically modified cell therapies have also been passed for clinical use.
Number Of Oncology Gene Therapy Pipeline
(Reference: asgct.org)
- Oncogenics refers to the pipeline from gene therapy for the cure of malignancies, and each target represents a different molecule or protein involved in the development process and cancer treatment.
- According to gene therapy statistics, the most commonly used molecule is CD19, and there are 131 active gene therapy clinical trials associated with it. Next, with 56 clinical trials, the TNF receptor superfamily member 17 is also the target.
- Other important targets include: CD22 molecule (31 trials), “membrane spanning 4 -domains” A1 (27 trials), KRAS proto-oncogene, GTPase (24 trials), glypican 3 (23 trials), claudin 18 (22 trials), mesothelin (21 trials), erb-b2 receptor tyrosine kinase 2 (20 trials), CD70 molecule (17 trials), and CD7 molecule (16 trials).
- Other targets include cancer/testis antigen 1B (15 trials), CD33 molecule (14 trials), and programmed cell death 1 (12 trials). The molecule was assigned to CD276: 11 trials, and C-type lectin domain family 12 member A also has 11 trials.
- Gene therapy statistics show the incredible breadth of targets available in genetic research aimed at the treatment of cancer, particularly what has to do with molecules involved in immune systems and tumour-specific antigens.
Gene Therapy Cost
- Gene therapy typically consists of replacing abnormal genes within human cells with normal ones, adding genes to assist in overcoming disease or turning off functionally abnormal genes.
- The administration of gene therapy is complex and usually requires lodging in the facility itself with other modalities and medicines.
- The price tag on gene therapy is eye-poppingly expensive. Although not popularly available to the patient population, these therapies carry lofty price tags.
- A single injection of Hemgenix for adult patients with haemophilia B costs USD 3.5 million.
- Gene therapy statistics reveal that in December 2023, therapies called Casgevy and Lyfgenia for sickle cell disease were approved, with treatment costs from USD 2.2 million to USD 3.1 million.
- Then, such exorbitantly high prices put gene therapies out of reach for many healthcare providers and plan sponsors for therapy.
Funding And Strategic Importance Of Gene Therapy Companies
- Funding is necessary for gene therapy companies, as most of them are still in the research stage and need huge investments to market their revolutionary therapies.
- At this point, these companies are facing high interest rates that raise investment costs and devalue them, among many other problems. Investors who entered the market during the pandemic’s high-valuation period are now evaluating losses.
- This scenario has made private equity (PE) and venture capital (VC) investors- those traditional supporters of Gene Therapy Companies- cautious about taking risks. Investors have consequently become more limited in their choices owing to apprehensions regarding securing a suitable price tag, finding a way out into efficient cash exit strategies, and many new investors coming into the fold to support the next funding.
- Despite such conditions, funding has managed to be pretty strong, keeping up with investments during the year 2023. PE and VC, however, seem to be limiting themselves to companies with longer financial runways, proven success in early phase trials, and other means of revenue generation, such as licensing and collaboration deals.
- Diversification of revenue, especially from licensing and collaborations, can be a huge plus for gene therapy companies.
- Gene therapy statistics stated that in 2023, Gene Therapy Companies formed 19% of all licensing and collaboration agreements, even though they make up just 10% of all new approvals and PE/VC investments.
- This indicates that though such companies are just starting their development, they are seen as potential partners due to their innovative technologies and potential for turnaround treatments in the future.
- 2022 and 2023 were indeed tough years for private investments in biopharma, as investors were careful about risk exposure.
- At the same time, Gene Therapy Companies, which were seen as one of the riskiest bets taken up by the conglomerates, kept pace along the way, growing with their counterparts in biopharma in terms of long-haul investment.
- “The licensing deal for Gene Therapy Companies grew by 29% in value in the year 2022; it reached USD 62 billion,” according to Evaluate.
- Although data lags in reporting, early signs suggest that 2023 will go this way, too. It has more to do than financial investment: it reflects confidence in Gene Therapy’s ability to deliver game-changing treatments.
- These licensing contracts comprise money and access to certain resources and expertise, which may spike speed toward development and expand market reach.
- Overall, Gene Therapy Companies’ licensing arrangements made up 19% of all deals reported in 2023, even though they constituted only 10% of novel approvals, emphasising the industry’s strategic importance and the value placed on its technologies.
Conclusion
Gene therapy statistics have come into a new-age medicine that brings hope to millions of patients across the globe. 2024 could be considered a historic year for this new field through exciting, promising markets, tremendous clinical trial activities, and growing investments in gene therapy.
Challenges still remain, but as indicated, the coming days of the future seem bright for the industry, which is expected to become a mainstream therapy for most genetic disorders.
Sources
FAQ.
As of Q3 2024, nearly 1,400 gene therapies are in the preclinical research phase, and sufficient developments in gene therapy treatment-related developments have made progress. Many of these gene therapies are still in the early stages of testing, but advancement in research is always pushing therapies forward for clinical studies.
In 2023, the worldwide cancer gene therapy market was estimated to be around US$3 billion, and by 2032, it is expected to grow around US$15 billion. This will spur on the fast and rising demand, evolving technologies in treatment, and wide-ranging scope of gene therapies to treat various cancers.
Gene therapies are very costly, such as haemophilia B, for example, where the treatment is with Hemgenix at US$3.5 million per injection. Other figures on sickle cell treatments can be from US$2.2 million to US$3.1 million, many of which cannot afford to be treated by many healthcare services or by patients.
According to estimates made in 2020, North America was the greatest contributor to the global gene therapy market, comprising around 56% of the overall market share. The validity of the region is further reflected by its socio-economical advantages, such as a robust healthcare infrastructure, significant investments in biotechnology, and a high number of clinical trials and approvals.
In 2023, only 10% of new approvals were made; nonetheless, gene therapy companies received 19% of all licensing and collaboration agreements that year. This reveals how strategically valuable gene therapies are for the pharmaceutical and biotechnology sectors, as the promising growth potential and the worth of these technologies for future treatment are shown.
Maitrayee, after completing her graduation in Electrical Engineering, transitioned into the world of writing following a series of technical roles. She specializes in technology and Artificial Intelligence, bringing her experience as an Academic Research Analyst and Freelance Writer, with a focus on education and healthcare under the Australian system. From an early age, writing and painting have been her passions, leading her to pursue a full-time career in writing. In addition to her professional endeavors, Maitrayee also manages a YouTube channel dedicated to cooking.
