Gene Therapy Market Report

Demand for gene therapy is growing attributed to the increasing diagnosis and treatments for a range of chronic diseases including spinal muscular atrophy, inherited retinal disease, B-cell lymphoma, and more, these are generally treated with surgery, medication, chemotherapy and radiation resulting in any side effects. For instance, according to the National Institute of Health (NIH), over 2276.27 million cases of chronic disease and 2.56 million deaths are accounted for globally. In addition, according 2022 report of NIH, inherited retinal disease prevalence accounted for 1 in 1380 with 5.5 million people worldwide. To overcome the problems of side effects and serious many non-profitable organisations are giving funds to gene therapy companies for new research and driving the market growth.

For instance, in June 2024, Opus Genetics announced that received funding of US$ 1.7 million from a foundation fighting for blindness for treatment of inherited retinal disease. Consequently, the rise in the prevalence of sickle cell disease is propelling the market growth. According to the report published by the American Society of Haematology, 1 to 3 million individuals of the United States carry traits of sickle cell and approximately 100,000 individuals have sickle cell disease with an estimated life expectancy of 20 years, continuous, clinical trials and novel gene therapies are on-going on the defected gene responsible for blood inherited disorders, around 3000 clinical trials of gene therapy have been performed worldwide.

For instance, in December 2023, the FDA approved Casgevy the first gene-based therapy to cure sickle cell using CRISPR/Cas 9 gene editing tool. CRISPR act by modifying patients' hematopoietic blood cells by making a cut in the targeted DNA and editing it to new genome. Increasing numbers of novel therapies and progressive results of the latest gene editing technology with no side effects, expand the market and increase new opportunities for market players.

Growth in gene therapy is attributed to the successful results of gene therapies in chronic illnesses that are mostly uncurable or patients face severe side effects after treatment such as nerve damage, lung tissue damage, and many more. For instance, according to the report of NIH published in 2023, around 60% of the worldwide deaths is because of chronic diseases, to reduce chronic disease death numbers, many government and non-profitable organizations are funding to research institutes and market players for developing novel cell-based gene therapies. For instance, in 2023, Scribes Therapeutics announced two separate collaborations one with Prevail Therapeutics and another with Sanofi to accelerate Vivo CRISPR-based genetic medicine for neurological and neuromuscular diseases such as Parkinson’s and Gaucher disease and sickle cell disease, resulting in availability of newer genetically modified medicines by using CRISPR, ex vivo natural killer cells therapies and in vivo therapies for neurological and neuromuscular diseases and accelerating genome editing technology to improve patients’ lives and boosting market growth.

In addition, many non-profitable government organizations and pharmaceuticals are partnered together to develop new gene therapies for rare diseases to ensure superior treatment and optimize the high cost off gene therapies for patients. For instance, in 2021, NIH, FDA, 10 pharmaceutical companies and 5 non-profit organizations united to develop gene therapies to 30 million Americans who suffer from approximately 7000 rare diseases, by organizing medicine partnership programs such as the Bespoke Gene Therapy Consortium (BGTC).  It is a part of the NIH Accelerating Medicine Partnership (AMP) program, purpose of increasing the research related to AAV, hence, improving the efficiency of vector development and enhancing the overall therapeutic benefits of AAV gene therapy. These steps by the government and other organizations will result in the expansion of the Gene therapy field.

• Increasing advancement in gene editing techniques.
• Rising adoption of novel gene therapies in chronic diseases.
• Increasing application and discoveries of cross-evolved vectors.
• Growing use in hospitals, clinical trials and research institutes.

High gene therapy cost to treat rare diseases is one of the major challenging factors in the growth of Gene therapy market. For instance, in March 2024, FDA approved the drug Lenmeldy for the treatment of a disease in children called as metachromatic leukodystrophy, the one-time cost of the treatment was US$4.2 million, making it difficult to afford by everyone. Exceeding cost of research and development acts as another barrier, as gene therapy requires extensive laboratory research and large investments for preclinical testing, and clinical trials for gene therapy products. Therefore, providing access to these costly therapies to everyone is still a major challenge.

Many complications have been observed in patients after receiving gene therapies including unwanted immune system reactions, infection caused by viruses and the possibility of causing errors in genes hinder market growth. For instance, according to the FDA, KYMRIAH has the potential to lower the level of B-cells in patients resulting in hypogammaglobulinemia, which can result in severe infection if untreated. In addition, adverse side effects of CRISPR-Cas9 are also observed such as DNA damage with cytotoxicity, genomic deletion and chromosome rearrangements resulting in the death of patients, However, to prevent these market challenges, regulatory agencies are creating new pathways for easy access to new therapies to manufacturers, patients and healthcare professionals.

Rise in FDA approval for gene therapies for chronic disease is significantly driving the gene therapy market owing to an increase in cell-based therapies research and breaking the cycle of medication. For instance, in March 2022, FDA approved Kymriah CAR T-cell therapy for refractory or relapsed follicular lymphoma patients, it is a one-time infusion process prescription-based cancer therapy for adults and pediatrics patients based on immunology, unlike traditional chemotherapy or stem cell transplant treatment, in this patient own natural killer cells are modified with specific proteins to kill leukemia cells, natural killer cells have natural tendency of fighting against foreign cells, serving breaking of continuous cycle of daily medication.

Furthermore, to increase the success rate of gene therapies using advanced gene editing tools, many market players along with research institutes are developing novel vectors, that help in the accurate and precise delivery of gene editing tools into the target cell to minimize damage to cell such as DNA damage, chromosome rearrangement and more.  For instance, in June 2023, StrideBio and Duke University received FDA approval for the use of Adeno-Associated Virus (AAV) in gene therapy, this enables the use of enzyme and Adeno-associated virus vectors for neutralizing antibody evasion, resulting in increasing use of medicated Gene therapy products and worldwide gene therapy market expansion for wide range of chronic diseases.

The United States has one of the highest healthcare budgets in the world and it is one of the leading regions for the gene therapy market owing its proficiency in advance technology-driven healthcare facilities, with increased national healthcare per capita expenditure and GDP. Advanced hospital care, physician, and clinical services contribute to a large proportion of budget spending in the United States resulting in dramatically increased spending in per person in therapies and prescribed drugs. Therefore, continuous expediting of market growth is seen with the introduction of novel and innovative advancement of delivering therapies and techniques such as CRISPR-Cas9, and CAR-T cell therapies are expected to increase the efficiency of gene therapy.

Although, it has the highest death rate for chronic or uncurable diseases and around 17 million individuals die before the age and 86% of premature deaths occur in low and middle-income countries stated by WHO in 2023, because of the increasing prevalence of chronic diseases many researches advancements are increasing, allowing market players in boosting market growth. Companies like Dynavax Technologies, Spotlight Therapeutics, and Seragaon Biosciences are some of the top gene therapy providers of the US.

For instance, in March 2021, Abecma was approved by FDA for the treatment of relapsed and refractory in adults with multiple myeloma after four or more therapies including anti-CD38 monoclonal antibodies, immunomodulatory and proteasome inhibitors. Abecma results in a reduction of disease progression risk to about 51% and 13.8 months compared to standard treatment with a continued improvement response rate of 44% compared to 5% of standard regimes, in addition, more than 60% of oncology medicine receives approval, providing more treatment options for cancer patients. The Innovative Genomics Institute are exploring innovative ideas for genetic engineering of cell, working on managing the target manner for gene editing tool by controlling the predictability of antigen-antibody interaction, these factors boost the gene therapy market in the United States.

Europe is accounted for the second most leading market for gene therapy with 23.7% trials owing to rise in the prevalence of chronic diseases, in addition, the market of gene therapy is projected to grow highest from 2025 due to policy shift in Europe for the review of drug effectiveness of health technology assessment, set new cost-effective price for drugs, resulting in more trials and affordable healthcare. For instance, in May 2024, Pfizer Inc. announced BEQVEZ (fidanacogene elaparvovec) a one-time gene therapy for hemophilia B treatment in adult patients, using antibodies to AAV serotype RH74 variant, reducing post-treatment bleeding and long-term continuous medical and treatment burden with prophylaxis in patients.

Gene therapy market in Asia-Pacific is expected to grow in countries such as Singapore, China, South Korea, and Japan by the initiative taken by the government towards the awareness of chronic diseases. Asia-Pacific has the lowest per capita national healthcare expenditure, resulting in fewer advancements, and innovations and lack of skilled professionals in healthcare and innovations. However, with the increasing adoption of advanced therapies, investments between research and the healthcare sector are fuelling the growth of market. For instance, in April 2024, IIT Bombay in collaboration with Tata Memorial Hospital launched CAR T-cell, first home-grow gene therapy for cancer patients, is expected to create ideal conditions for expansion in the Asia-Pacific market in gene therapy.

The report provides a detailed overview of the gene therapy market insights in regions including North America, Latin America, Europe, Asia-Pacific, Oceania, and the Middle East and Africa. The country-specific assessment for gene therapy market has been offered for all regional market share, along with forecasts, market scope estimates, price point assessment, and impact analysis of prominent countries and regions. Throughout this market research report, Y-o-Y growth and CAGR estimates are also incorporated for every country and region, to provide a detailed view of the gene therapy market. These Y-o-Y projections on regional and country-level markets brighten the political, economic and business environment outlook, which are anticipated to have a substantial impact on the growth of the gene therapy market. Some key country and region included in the gene therapy market report as follows:

• Gene therapy market detailed segments and segment-wise market breakdown
• Gene therapy market dynamics (Recent industry trends, drivers, restraints, growth potential, opportunities in gene therapy industry)
• Current and forthcoming 10 years' market valuation in terms of gene therapy market size, revenue (USD), growth rate, CAGR (%) analysis
• Gene therapy market demand analysis
• Gene therapy market regional insights with a region-wise market breakdown
• Competitive analysis – key companies profiling including their market share, product offerings, and competitive strategies.
• Latest developments and innovations in the gene therapy market
• Regulatory landscape by key regions and key countries
• Gene therapy market sales and distribution strategies
• A detailed viewpoint on the gene therapy market forecast by countries
• Essential information to enhance market position
• Robust research methodology