Posts Tagged ‘government’

The global gene therapy market size was valued at USD 8.75 billion in 2023 and is poised to grow from USD 10.47 billion in 2024 to USD 52.40 billion by 2033, growing at a CAGR of 19.6% in the forecast period (2024-2033).

Gene therapy is a technique that uses a gene to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patients cells with a healthy version of that gene. Both inherited genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia) have been treated with gene therapy.

Get Sample Copy of Report@ https://www.novaoneadvisor.com/report/sample/7819

The development of the market is owing to an increase in the number of gene therapy-based discoveries, increasing investment in this sector, and rising approval of gene therapy products. According to the WHO, 10 to 20 new cell and gene therapies are expected to be approved each year by 2025.

Continuous developments in recombinant DNA technology are anticipated to enhance the efficiency of gene therapy in the coming years. Hence, ongoing progresses in recombinant DNA technology are anticipated to expand the number of ongoing clinical trials for gene therapy. Primarily, these advancements are taking place in the context of various gene-editing tools and expression systems to augment the R&D for products. The advent of CRISPR/Cas9 nuclease, ZFN, and TALEN allows easy & precise genome editing. As a result, in recent times, the gene-editing space has witnessed a substantial number of research activities, which, in turn, is expected to influence the growth of the gene therapy market.

The growth of the gene therapy market is expected to be majorly benefitted from the increasing prevalence of cancer. The ongoing increase in cancer patients and related death per year emphasizes the essential for the development of robust treatment solutions. In 2020, there were around 18.1 million new cases of cancer worldwide. 9.3 million of these cases involved men, while 8.8 million involved women. Continuing developments in tumor genetic studies have delivered substantial information about cancer-related molecular signatures, which in turn, is expected to support ongoing clinical trials for cancer therapeutics.

With rising demand for robust disease treatment therapies, companies have focused their efforts to accelerate R&D for effective genetic therapies that target the cause of disease at a genomic level. . Furthermore, the U.S. FDA provides constant support for innovations in this sector via a number of policies with regard to product manufacturing. In January 2020, the agency released six final guidelines on the manufacturing and clinical development of safe and efficient products.

Furthermore, facility expansion for cell and gene therapies is one of the major factors driving the gene therapy market growth. Several in-house facilities and CDMOs for gene therapy manufacturing have begun investing to enhance their production capacity, which, in turn, is anticipated to create lucrative opportunities for market players. For instance, in April 2022, the FDA approved commercial licensure approval to Novartis for its Durham, N.C. site. This approval permits the 170,000 square-foot facility to make, test, and issue commercial Zolgensma, as well as manufacture therapy products for current & upcoming clinical trials.

Cell and Gene Therapy Market :https://www.biospace.com/article/releases/u-s-cell-and-gene-therapy-clinical-trial-services-industry-is-rising-rapidly/

Gene Therapy Market Report Highlights

U.S. Gene Therapy Market Size in U.S. 2024 to 2033

The U.S. gene therapy market size was estimated at USD 3.19 billion in 2023 and is projected to surpass around USD 18.50 billion by 2033 at a CAGR of 19.22 % from 2024 to 2033.

North America dominated the market in 2023 with the largest revenue share of 65.12% in 2023. This region is expected to become the largest routine manufacturer of gene therapy in terms of the number of approvals and revenue generated during the forecast period. Increasing investments in R&D from large and small companies in the development of ideal therapy drugs are anticipated to further boost the market.

Furthermore, the increasing number of investments by the governments and the growing prevalence of targeted diseases are the factors fueling the market. According to the Spinal Muscular Atrophy Foundation, in 2020, around 10,000 to 25,000 children and adults in the U.S. were affected by spinal muscular atrophy, making it a fairly common disease among rare diseases.

Europe is estimated to be the fastest-growing regional segment from 2024 to 2030. This is attributed to its large population with unmet medical needs and increasing demand for novel technologies in the treatment of rare but increasingly prevalent diseases. Asia Pacific market for commercial application of genetic therapies is anticipated to witness significant growth in the forecast period, which can be attributed to the easy availability of resources, local presence of major companies, and increased investment, by the governments.

UK Gene Therapy Market

The UK gene therapy market is anticipated to witness accelerated growth over the forecast period, due to increased investments by various big companies and governments, including the NHS & research laboratories. For instance, in March 2022, the UK government invested USD 326.45 million to accelerate healthcare research and manufacturing. Under this investment, additional $80 million of the fund will help companies at the forefront of invention with their commercial-scale manufacturing investments in areas like gene and cell therapies, as well as improved diagnostic technologies, among others. Various mergers & partnerships between manufacturers, universities, and other government bodies are expected to boost the market over the forecast period.

Immediate Delivery Available, Get Full Access@

https://www.novaoneadvisor.com/report/checkout/7819

What is gene therapy used for?

Most gene therapies are still in the clinical trial phase. Clinical trials play an important role in finding treatments that are safe and effective. Clinical trials are investigating gene therapy for the treatment ofcancer,macular degenerationand other eye diseases, certaingenetic conditionsandHIV/AIDS.

The U.S. Food and Drug Administration (FDA) has approved two gene therapies for use in the U.S.:

Is gene therapy safe?

The first gene therapy trial was run more than thirty years ago. The earliest studies showed that gene therapy could have very serious health risks, such as toxicity, inflammation, and cancer. Since then, researchers have studied the mechanisms and developed improved techniques that are less likely to cause dangerous immune reactions or cancer. Because gene therapy techniques are relatively new, some risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research, clinical trials, and approved treatments are as safe as possible.

Comprehensive federal laws, regulations, and guidelines help protect people who participate in research studies (called clinical trials). The U.S. Food and Drug Administration (FDA) regulates all gene therapy products in the United States and oversees research in this area. Researchers who wish to test an approach in a clinical trial must first obtain permission from the FDA. The FDA has the authority to reject or suspend clinical trials that are suspected of being unsafe for participants.

The National Institutes of Health (NIH) also plays an important role in ensuring the safety of gene therapy research. NIH provides guidelines for investigators and institutions (such as universities and hospitals) to follow when conducting clinical trials with gene therapy. These guidelines state that clinical trials at institutions receiving NIH funding for this type of research must be registered with the NIH Office of Biotechnology Activities. The protocol, or plan, for each clinical trial is then reviewed by the NIH Recombinant DNA Advisory Committee (RAC) to determine whether it raises medical, ethical, or safety issues that warrant further discussion at a RAC public meeting.

An Institutional Review Board (IRB) and an Institutional Biosafety Committee (IBC) must approve each gene therapy clinical trial before it can be carried out. An IRB is a committee of scientific and medical advisors and consumers that reviews all research within an institution. An IBC is a group that reviews and approves an institution's potentially hazardous research studies. Multiple levels of evaluation and oversight ensure that safety concerns are a top priority in the planning and carrying out of gene therapy research.

The clinical trial process occurs in three phases. Phase I studies determine if a treatment is safe for people and identify its side effects. Phase II studies determine if the treatment is effective, meaning whether it works. Phase III studies compare the new treatment to the current treatments available. Doctors want to know whether the new treatment works better or has fewer side effects than the standard treatment. The FDA reviews the results of the clinical trial. If it determines that the benefits of the new treatment outweigh the side effects, it approves the therapy, and doctors can use it to treat a disorder.

What are CAR T cell therapy, RNA therapy, and other genetic therapies?

Several treatments have been developed that involve genetic material but are typically not considered gene therapy. Some of these methods alter DNA for a slightly different use than gene therapy. Others do not alter genes themselves, but they change whether or how a genes instructions are carried out to make proteins.

Cell-based gene therapy

CAR T cell therapy (or chimeric antigen receptor T cell therapy) is an example of cell-based gene therapy. This type of treatment combines the technologies of gene therapy and cell therapy. Cell therapy introduces cells to the body that have a particular function to help treat a disease. In cell-based gene therapy, the cells have been genetically altered to give them the special function. CAR T cell therapy introduces a gene to a persons T cells, which are a type of immune cell. This gene provides instructions for making a protein, called the chimeric antigen receptor (CAR), that attaches to cancer cells. The modified immune cells can specifically attack cancer cells.

RNA therapy

Several techniques, called RNA therapies, use pieces of RNA, which is a type of genetic material similar to DNA, to help treat a disorder. In many of these techniques, the pieces of RNA interact with a molecule calledmessenger RNA(or mRNA for short). In cells, mRNA uses the information in genes to create a blueprint for making proteins. By interacting with mRNA, these therapies influence how much protein is produced from a gene, which can compensate for the effects of a genetic alteration. Examples of these RNA therapies include antisense oligonucleotide (ASO), small interfering RNA (siRNA), and microRNA (miRNA) therapies. An RNA therapy called RNA aptamer therapy introduces small pieces of RNA that attach directly to proteins to alter their function.

Epigenetic therapy

Another gene-related therapy, called epigenetic therapy, affectsepigenetic changesin cells. Epigenetic changes are specific modifications (often called tags) attached to DNA that control whether genes are turned on or off. Abnormal patterns of epigenetic modifications alter gene activity and, subsequently, protein production. Epigenetic therapies are used to correct epigenetic errors that underlie genetic disorders.

Immediate Delivery Available, Get Full Access@

https://www.novaoneadvisor.com/report/checkout/7819

Vector Insights

The AAV segment shows a significant revenue contribution of 22.9% in 2023. Several biopharma companies are offering their viral vector platform for the development of AAV-based gene therapy product. For instance, in September 2016, Lonza signed an exclusive agreement with Massachusetts Eye and Ear to support its novel Anc-AAV gene therapy platform for development and commercialization of next-generation gene therapies based on their AAV platform. Similarly, RegenxBio had made an agreement with companies AveXis & Biogen in March 2014 and May 2016, respectively, which would allow both companies to use RegenxBios AAV vector platform for development of gene therapy molecules. Furthermore, in May 2021, Biogen Inc. and Capsigen Inc. entered into a strategic research partnership to engineer novel AAV capsids that have the possibility to deliver transformative gene therapies, which can address the fundamental genetic causes of numerous neuromuscular and CNS disorders. In July 2021, the U.S. Department of Commerces National Institute of Standards and Technology (NIST), National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and United States Pharmacopeia (USP) announced a collaboration to evaluate analytical methods and develop standards for AAV. As part of this partnership, NIST and USP will be conducting an interlaboratory study in which several laboratories will measure these serious quality attributes, and their results will be linked and examined. This collaboration will support the development of new promising gene therapies that will significantly advance peoples lives.

Indication Insights

The spinal muscular atrophy (SMA) segment dominated the market in 2023. Although SMA is a rare disorder, it is one of the most common fatal inherited diseases of infancy. The development of Zolgensma (AVXS-101), has proven its effectiveness in treating SMA and altering the phenotype of the illness. The FDA approved Novartis' Zolgensma approval in May 2019, which is aimed at treating the underlying cause of SMA. As of now, Zolgensma is the only gene treatment in this field to have been approved. The approval of this gene therapy is evidence of the growing use of therapies to treat serious hereditary illnesses like SMA.

The Beta-Thalassemia Major/SCD segment is anticipated to register the fastest CAGR over the forecast period. Gene therapy for SCD and -thalassemia is based on transplantation of gene-modified hematopoietic stem cells. Clinical and preclinical studies have shown the efficacy and safety of this therapeutic modality. However, several other factors, such as suboptimal gene expression levels & gene transfer efficiency, limited stem-cell dose and quality, and toxicity of myeloablative regimens are still hampering its efficacy. Despite these challenges, in June 2019, bluebird Bios Zynteglo (formerly LentiGlobin) received conditional approval in Europe for the treatment of -thalassemia and is expected to receive U.S. FDA approval in August 2022. Moreover, the product has already received Orphan Drug status by the U.S. FDA for treatment of patients with sickle cell disease (SCD). Furthermore, in April 2021, Vertex Pharmaceuticals and CRISPR Therapeutics amended partnership for the development, production, and commercialization of CTX001 in sickle beta thalassemia and cell disease. These achievements in this segment are anticipated to significantly boost the adoption of the product in this segment.

Route of Administration Insights

The intravenous segment dominated the global gene therapy market in 2023. Large number of approved products along with strong pipeline for IV candidates is the major reason for the segment dominance. The segment is also expected to emerge as the most lucrative over the forecast period.

Recent Developments

Some of the prominent players in the Gene therapy market include:

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the global gene therapy market.

Indication

Vector Type

Route of Administration

By Region

Order the 150+ Pages Detailed Report @ https://www.novaoneadvisor.com/report/checkout/7819

Call: USA: +1 650 460 3308 | IND: +91 87933 22019 |Europe: +44 2080772818

Email: sales@novaoneadvisor.com

Read the original here:
Gene Therapy Market Size Poised to Surge USD 52.40 Billion by 2033 - BioSpace

The global cancer gene therapy market size was accounted for USD 2.95 billion in 2023 and it is increasing around USD 18.11 billion by 2033 with a CAGR of 19.9% from 2024 to 2033, according to a new report by Nova One Advisor.

Cancer Gene Therapy Market Overview

Cancer is a group of diseases that involve abnormal cell growth which can spread to respective parts of the body. Cancer can spread throughout the human body.Gene therapyis a kind of treatment in which the genes that are not normal or are missing in the patients cells are replaced with normal genes. Cancer gene therapy is a technique for treating cancers where the therapeutic DNA is introduced in the gene of the individual suffering from cancer.

Due to a high success rate in preclinical as well asclinical trials, cancer gene therapy is gaining high popularity all over the world. There are numerous techniques utilized in cancer gene therapy. In one of the gene therapy techniques, either the mutated gene is replaced with a healthy gene, or the gene is inactivated if its function is abnormal. In a newly developed technique, new genes can be introduced in the body of the patient to help fight against cancer cells.

Further, the ongoing extensive research and development (R&D) strategies implemented bybiopharmaceuticalfirms for producing novel therapeutic drugs are driving the market growth notably.

The market players can aim towards expansions, collaborations, joint ventures, acquisitions, and partnerships to advance capabilities in gene therapy. This would help in yielding effective therapeutic drugs for treating different kinds of cancers. In April 2022, GSK plc announced the acquisition of Sierra Oncology for 1.6 billion ($1.9 billion). This acquisition would help GSK plc in enhancing its capabilities with respect to targeted therapies for treating rare forms of cancer.

Biotechnologyfirms are evaluating novel gene therapy vectors for increasing levels of protein production/gene expression, reducing immunogenicity, and improving durability.

The top cancers in terms of the count of new cases in 2020 all over the world were Lung Cancer (2,206,771 cases),Breast Cancer(2,261,419 cases),Prostate Cancer(1,414,259 cases), Colorectal Cancer (1,931,590 cases), Stomach Cancer (1,089,103 cases), and Liver cancer (905,677 cases). In 2018, there were around 134,632 new cancer cases and 89,042 cancer-related fatalities. Breast and liver cancers were among the most common tumors in terms of incidence and mortality. The high prevalence of breast cancer cases enhances the scope for CRISPR/Cas9-based gene editing for breast cancer therapy and VISA-claudin4-BikDD gene therapy.

Get Sample Copy of Report@ https://www.novaoneadvisor.com/report/sample/7820

Key Takeaways:

Cancer Gene Therapy Market Size in U.S. 2024 to 2033

The U.S. cancer gene therapy market size was valued at USD 1.25 billion in 2023 and is anticipated to reach around USD 7.94 billion by 2033, growing at a CAGR of 20.31% from 2024 to 2033.

North America accounted for the largest share of over 61.15% in 2023. This is attributed to the conducive environment facilitated by the government and the National Cancer Institute that supports research and development activities to enhance cancer therapeutics. Further, the presence of key market players in the region, their research efforts in devising gene therapy for cancer treatment, and collaborative efforts among market players to enhance research are boosting the market growth in the region. For instance, in August 2022, Merck & Co., Inc., collaborated with Orna Therapeutics Inc., for discovery, development, and commercialization of multiple programs, inclusive of utilization of mRNA for cancer gene therapy.

Europe is estimated to be the fastest-growing region over the forecast period due to increase in research funding for novel therapeutics by government bodies and increasing demand for novel therapeutics that could help combat the growing incidence of cancer cases across the region. Moreover, The European Unions Horizon Europe Mission on Cancer was launched in September 2023 so as to offer funds to a broad spectrum of activities that are intended to lower Europes cancer burden by accelerating research and innovation in cancer therapeutics. The mission is anticipated to help over 3 million cancer survivors by the year 2033.

The cancer gene therapy market in the Asia Pacific (APAC) region is segmented into India, China, Japan, South Korea, and the rest of the Asia Pacific (APAC) region. China dominated the Asia Pacific region followed by Japan and India in 2023.

The Latin America, Middle East, and African (LAMEA) cancer gene therapy market is segmented into North Africa, South Africa, Saudi Arabia, Brazil, Argentina, and the Rest of LAMEA. The Middle East and the Latin America region are anticipated to have notable growth in the cancer gene therapy market during the forecast period. Brazil held the largest share in the LAMEA region in 2023. Due to low literacy, uncertainty, and civil war in African countries, the cancer gene therapy market in Africa is expected to grow at a comparatively slow rate.

What are the importance of Cancer Gene Therapy?

Immediate Delivery Available, Get Full Access@

https://www.novaoneadvisor.com/report/checkout/7820

Types of gene therapy for cancer

Gene therapy aims tocontrolthe altered genesor genetic mutationsof a cancertoprevent the cancers growth.This approach to using our own cells and genes to treat cancer is called somatic gene therapy.Thistype of gene therapydoes not impact germ-line cells in the reproductive system, meaning none of the genetic changescan bepassedon to otherfamily members.

There are four types of somatic gene therapy: gene editing; gene replacement; gene addition; and gene inhibition.

Gene editing is correcting the cells gene to fix the imbalance by snipping out the faulty part of the gene and changing the cancers DNA. This type of gene therapy may correct the alteration rather than trying to remove it. Gene replacement is just that: replacing the faulty or nonworking gene with a healthy copy of it. This type of gene therapy is another form of trying to fix the genetic change rather than trying to remove it.

Gene addition is adding novel genetic code to a different cell usually an immune system fighter cell to help it combat the protein linked to the damaged gene. CAR T-cell therapy is an example of gene addition. This form of gene therapy isnt adding a copy of an already-existing gene but rather an entirely new gene usually with the intent of killing the cancer cell via the immune system. Doctors may also add a new gene directly to the cancer cell that causes the cancer cell to commit apoptosis (kill itself).

Gene inhibition simply shuts down the faulty gene. This can either kill the cell or prevent it from acting in a cancerous manner, such as growing and replicating exponentially.

Steps of gene therapy

Gene therapy is a new and potentially curative approach to treating cancer, but researchers still have so much to learn. While the steps below may seem straightforward, each part of the process requires years of study to develop the technologies.

Researchers must first identify the gene and protein linked to the cancer. The next steps are:

Steps of CAR T-cell therapy

CAR T-cell therapy has a slightly different process than more direct forms of gene therapy. CAR T cells are lab-generated fighter cells with specific, anti-cancer genetic code. Adding this genetic code is the gene therapy component of CAR T-cell therapy. CAR stands for chimeric antigen receptor, which is the new genetic code added to the T cells.

There are six CAR T-cell therapy agents approved by the U.S. Food and Drug Administration for different blood cancers. These approvals validate CAR T cells as an effective form of cancer gene therapy to improve patient life expectancy.

Doctors first draw blood from a patient and separate the T cells, which are white blood cells leading the immune systems defense against viruses, diseases and more unwanted intruders. T cells aim to protect the body from cancer, but theyre often ineffective at doing so.

The process of drawing blood from patients and separating the T cells is called apheresis.

After removing T cells from the body, the steps of CAR T-cell therapy are:

A similar process occurs for CAR NK-cell therapy. Scientists create chimeric antigen receptors to strengthen natural killer (NK) cells, another white blood cell of the immune system.

How long does CAR T-cell therapy take?

There are six CAR T-cell therapies approved for types of three blood cancers: myeloma, leukemia and lymphoma. CAR T-cell therapy infusions can take place in an inpatient or outpatient care setting, but the patient must be closely monitored at all times.

CAR T-cell therapy can lead to side effects, most notable cytokine release syndrome.

The entire CAR T-cell process lasts approximately one month, not including the recovery time after treatment:

For the first seven days after receiving the CAR T-cell infusion, patients must remain under medical supervision. For weeks 2-4 of the post-infusion timeline, patients must remain within a short drive of their medical facility to respond to any issues.

The total recovery period from CAR T-cell therapy is usually 2-3 months following infusion, according to the Dana-Farber Cancer Institute.

There are several studies for CAR T-cell therapies for cancer. Participating in a clinical trial helps advance cell and gene therapy research and can advance much-needed therapies to more patients in need.

Therapy Insights

Gene induced immunotherapy dominated the market with a revenue share of over 41.9% in 2023. The dominance of the segment can be attributed to research studies aiming to lower the proliferation of various types of cancer by strengthening the immune system. Many gene therapies for cancers are designed on the basis of immunotherapy elements. For instance, PROVENGE (by Dendreon Corporation) is an autologous cellular immunotherapy designed to stimulate a subjects immune system against prostate cancer.

Oncolytic virotherapy is expected to grow at the fastest rate over the forecast period owing to the favorable outcomes and the level of efficacy offered by oncolytic virotherapy. Oncolytic viruses can combat cancer cells without disturbing the healthy cells in vicinity by stimulating natural killer cells. Moreover, there are lucrative research grants for the research on oncolytic virotherapy. For instance, in July 2022, the researchers at the Center for Nuclear Receptors and Cell Signaling at the University of Houston received a USD 1.8 million grant from the National Institutes of Health to work on oncolytic virotherapy.

End-use Insights

Biopharmaceutical companies led the market with a revenue share of over 50.0% in 2023. This is attributed to the increasing global prevalence of different types of cancers owing to various hereditary, environmental, and lifestyle risk factors. Moreover, the market is driven by increasing adoption of elemental gene therapy options by biopharmaceutical giants to design cancer therapeutic regimes. Many novel therapeutic drugs are under different phases of trials and firms are striving to market them in different regions across the globe. For instance, in January 2020, bluebird bio, Inc. launches its drug, ZYNTEGLO in Germany to be used as a one-time gene therapy solution for patients aged 12 years and above.

The biopharmaceutical companies segment is projected to grow at the fastest rate over the forecast period. The increasing global prevalence of malignant tumors is a key factor driving the market. Moreover, an increased interest in oncology therapeutics research and development is resulting in a rise in the number of FDA approvals of gene therapy drugs. For instance, there are 6 FDA-approved cancer gene therapy drugs with Tecratus, Abcema, and Kymriah being the recent approvals.

Recent Developments:

Some of the prominent players in the Cancer gene therapy market include:

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2023 to 2033. For this study, Nova one advisor, Inc. has segmented the global cancer gene therapy market.

Therapy

End-use

By Region

Order the 150+ Pages Detailed Report @ https://www.novaoneadvisor.com/report/checkout/7820

Call: USA: +1 650 460 3308 | IND: +91 87933 22019 |Europe: +44 2080772818

Email: sales@novaoneadvisor.com

Excerpt from:
Cancer Gene Therapy Industry is Rising Rapidly Up to USD 18.11 Bn by 2033 - BioSpace