Hemophilia Treatment Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19) – Cheshire Media

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Hemophilia Treatment market.

Trusted Business Insights presents an updated and Latest Study on Hemophilia Treatment Market 2020-2029. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Hemophilia Treatment market during the forecast period (2020-2029). It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

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Abstract, Snapshot, Market Analysis & Market Definition: Hemophilia Treatment Market Industry / Sector Trends

Hemophilia Treatment Market size was valued USD 10.5 billion in 2018 and is expected to witness 5.0% CAGR from 2019 to 2025.

U.S. hemophilia treatment market, by disease, 2018 & 2025 (USD Million)

Growing prevalence of hemophilia across the globe will be one of the major market augmenting factors over the forthcoming years. As per the recent statistics published by Centers for Disease Control and Prevention (CDC), hemophilia A affects around 1 in 5,000 live male births in the U.S. Majority of the people with hemophilia are diagnosed at a young age. According to the CDC data, average age of hemophilia diagnosis in the U.S. is 36 months for mild hemophilia, 8 months for moderate hemophilia and 1 month for severe hemophilia. Hence, rise in number of people suffering from hemophilia will enhance the hemophilia treatment market growth in the near future.

Rise in several government initiatives will augment the industry growth over the projection period. Organization including the World Hemophilia Organization along with governments of several countries carry out awareness and diagnosis programs. Government efforts to enhance public health and treat underprivileged people will result in boosting the hemophilia treatment market growth in the near future.

However, high cost of hemophilia medications will be one the major growth impeding factors. High investment, R&D, manufacturing and marketing of drugs by manufacturers is the reason for high cost of the products. High cost of products are unaffordable for the middle and lower income class families, thus resulting in restraining the business growth.

Market Segmentation, Outlook & Regional Insights: Hemophilia Treatment Market

Hemophilia Treatment Market, By Disease

Germany hemophilia treatment market share, by disease, 2018

Hemophilia A treatment market was valued at USD 6,743.7 million in the year 2018. Hemophilia A is a common blood disorder and the prevalence rate is high in developing and underdeveloped regions. As per the recent statistics, around 19,000 people in India suffer from hemophilia and only 15% of the patients are registered. Increase in number of people suffering from hemophilia A and rise in awareness among the people regarding the treatment and medications of hemophilia will result in rise in demand and adoption rate of hemophilia drugs, fueling the business growth.

Hemophilia B treatment market is estimated to grow at 4.0% over the estimation period. As per a recent research article, the prevalence of hemophilia B is quite rare as compared to hemophilia A and the incidence of hemophilia B is around 1 in 30,000 in the U.S. Recent technological advancements has led to growing medications for hemophilia B treatment, hence resulting in boosting the segmental growth.

Hemophilia Treatment Market, By Product

Recombinant factor concentrates market was valued at USD 6,626.9 million in the year 2018. Rise in production of recombinant factor concentrates has provided growth opportunity to treat hemophilia and overcome limited availability of plasma-derived concentrates. Also, increased safety of replacement therapy with the help of recombinant factor concentrates has dramatically enhanced the quality of patient life. Increase in preference of recombinant factor concentrates in treatment of hemophilia will ultimately lead to augmenting the business growth over the forecast period.

Extended half-life products market is estimated to witness a robust growth of 5.3% over the forecast period. Recent research and development has led to the production of new factor concentrates that are efficient and need less frequent injections. The reason for fewer injections are due to the increased half-life of the product. Hence, recent technological advancements and new product launches will lead to increase in demand and adoption rate of extended half-life products, thereby fueling the business growth.

Hemophilia Treatment Market, By Patient

Hemophilia treatment in adults was valued at USD 6,778.1 million in the year 2018. As per a recent research article, around 65% of the people suffering from hemophilia are adults. In some of the adults mild hemophilia is diagnosed in the later stages of life. As the age of patients increases, the need for hemophilia care also increases. There are various possibilities of developing moderate to severe hemophilia in adults. Hence, growing age results in rise in chances of developing hemophilia in adults, thereby boosting the demand for hemophilia treatment in the near future.

Hemophilia treatment in pediatrics is projected to grow at 5.2% over the estimation period. Hemophilia is inherited diseases that are passed to children from gene located on X-chromosome. In approximately one third of children, no family history of hemophilia is found, and the disease occurs due to gene mutation. Children with hemophilia gene bruise easily, bleed more with surgeries and have frequent nose bleedings. According to the National Center for Biotechnology Information, severe form of factor deficiencies including hepatitis A and hepatitis B were diagnosed in the neonates with 52% and 68% of cases respectively. Hence, increase in prevalence of hemophilia in children will augment the hemophilia treatment market growth in the near future.

Hemophilia Treatment Market, By Treatment

Prophylaxis treatment market was valued at USD 7,219.9 million in the year 2018. Long-term prophylaxis treatment is considered as standard of care to prevent chronic arthropathy and joint bleeding in patients with severe hemophilia. As per a recent research, it was observed that the prophylaxis treatment for hemophilia was quite effective and slowed the progression of joint damage. Majority of patients and healthcare providers opt for prophylaxis treatment owing to its efficiency and better end results. Hence, increase in preference of prophylaxis treatment will further augment the business growth in the near future.

On demand treatment market is estimated to grow at 5.2% over the forthcoming years. People suffering from mild to moderate hemophilia are treated with on demand treatment to prevent bleeding. Children with severe hemophilia initially receive prophylactic therapy to prevent bleeding; however, after reaching adulthood, they switch to on-demand treatment for several reasons, such as strict work schedules, inconvenience of visiting doctors or going to a clinic several times a week, along with high expenditure related to continuous prophylactic therapy. Hence, the aforementioned reasons will be responsible for the market growth of on demand treatment segment.

Hemophilia Treatment Market, By Therapy

Factor replacement therapy market was valued at USD 9,574.2 million in the year 2018. As per the Medical and Scientific Advisory Council (MASAC) of National Hemophilia Foundation (NHF), the use of recombinant factor concentrates in children is quite effective as the recombinant factor does not contain actual human blood and cannot transmit harmful viruses including hepatitis. Hence, the use of factor replacement therapy is quite common due to direct infusion of clotting factors, resulting in boosting the segmental growth.

Non-factor replacement therapy is projected to show rapid growth of 38.3% over the estimation period. One of the challenging problems in the treatment of hemophilia is the development of alloantibodies against the infused factor concentrates. Development of inhibitors render the factor replacement therapy ineffective, leading to high risk of morbidity and mortality in patients. Also, there is growing interest in non-factor replacement therapy agents that act by inhibiting anticoagulant pathways or enhancing coagulation. Hence, introduction of new therapeutic agents in hemophilia treatment will enhance the business growth.

Hemophilia Treatment Market, By Drug Class

Coagulation factors market was valued at USD 9,948.3 million in the year 2018. Hemophilia is considered as hereditary blood disease with coagulation time. The use of coagulation factors is quite common and is used mostly in the replacement therapy. Absence or improper functioning of several clotting factors in the blood such as factors VIII, XI, etc. result in causing hemophilia. Hence, use of several clotting factors to replace the absent blood clotting factors will lead to boosting the hemophilia treatment market growth.

Vasopressin drug class market is estimated to grow at 6.0% in the near future. Patients suffering from mild hemophilia use vasopressin drugs for joint and muscle bleeds, bleeding in mucous membranes of nose and mouth and pre and post-surgery. Vasopressin drugs comes in injectable and nasal spray form and is comparatively easy to use. Hence, the aforementioned factors will be responsible for the market growth of vasopressin drug class.

Hemophilia Treatment Market, By Route of Administration

Injectable route of administration for hemophilia treatment was valued at USD 9,629.3 million in the year 2018. Majority of the drugs and factor concentrate products are intravenously given to patients for better and quick results. Intravenous route of administration helps the medications to directly interact with the blood. Hence, majority of healthcare providers opt for injectable or intravenous route of drug administration to treat hemophilia for better and quick results, hence boosting the market growth.

Nasal spray or intra nasal route of administration will grow at 6.0% over the forecast period. Drugs including vasopressin are required to be taken by patients for joint and muscle bleeds, and bleeding in mucous membranes of nose and mouth. Several advantages offered by nasal spray such as ease of use and user convenience will result in growing demand, thereby fueling the business growth.

Hemophilia Treatment Market, By End-use

Hemophilia treatment centers as end-use was valued at USD 4,134.6 million in the year 2018. A substantial proportion of hemophilia treatment is carried out in such centers. During the forecast period, the hemophilia treatment center end-use market segment will continue to lead, owing to rising number of special care provided and peoples increased preference for these centers over other healthcare settings.

Clinics as end-use is estimated to grow at 5.3% in the near future. Clinics provide fast-track treatment and infusion of factor concentrates to patients. Also, immediate and short-time treatment provided during the daytime to people with busy and hectic work schedule will enhance business growth in the near future.

Hemophilia treatment Market, By Region

Europe hemophilia treatment market, by country, 2025 (USD Million)

U.S. dominated the North America hemophilia treatment market and was valued at USD 3,539.3 million in the year 2018. As per the Hemophilia Federation of America, hemophilia A affects around 1 in 5,000 people in the U.S. and approximately 400 new-borns are diagnosed with hemophilia every year. Also, presence of favorable reimbursement and insurance in the country will result in growing demand and adoption rate of hemophilia medications, expanding the hemophilia treatment market growth in the country.

India hemophilia treatment market is projected to grow at 9.2% over the estimation period. Various government initiatives has helped in building necessary infrastructure, empower healthcare providers and patients to self-administer treatment at home, hence helping patients achieve a better quality of life. Recent technological advancements, rise in awareness among people, new product launches and government initiatives will augment the business growth over the forecast period.

Key Players, Recent Developments & Sector Viewpoints: Hemophilia Treatment Market

Major industry players involved in the hemophilia treatment market include Swedish Orphan Biovitrum AB, Sanofi SA, Pfizer, Novo Nordisk, Genentech, CSL Behring, Biogen, among other market players. These players have implemented several strategic initiatives such as collaborations, merger and acquisitions, new product launches and partnerships that have enhanced their financial stability, helped them evolve as major industrial players and gain strong market position.

Hemophilia Treatment Industry Viewpoint

Hemophilia has been an extremely old and critical disease that the global population has been facing. People in ancient times have written and articulated about bleeding problems. Some of the bleeding problems identified have been different as compared to others. But little they knew about blood clots and the disease caused by their deficiency. Until before World War II, doctors learned about hemophilia A and its cause due to deficiency of factor VIII. Several other blood factors were recognized later on and Roman numeral names were given to avoid confusion. Hemophilia research has come a long way with scientists discovering human blood in different groups, hence helping blood transfusions to be more successful. In 1960s, Dr. Judith Pool discovered a procedure to freeze and thaw plasma to get a layer of factor-rich plasma called as cryoprecipitate. Use of cryoprecipitate was the best way of stopping hemophilia bleeding. Later on, the greatest breakthrough that came in hemophilia treatment was the development and introduction of factor concentrates. These clotting factors can be freeze-dried to a powder that can be easily stored and consumed. With the help of these factor concentrates, people suffering from hemophilia can be treated quickly with maximum efficiency. Also, factor concentrates have helped people treat their bleedings at home or at work, resulting in people to lead normal lives. Also, development and introduction of new medications and technologies such as non-factor replacement therapy, extended half-life products, gene therapy, etc. will augment the growth of hemophilia treatment market growth in the near future.

Key Insights Covered: Exhaustive Hemophilia Treatment Market

1. Market size (sales, revenue and growth rate) of Hemophilia Treatment industry.

2. Global major manufacturers operating situation (sales, revenue, growth rate and gross margin) of Hemophilia Treatment industry.

3. SWOT analysis, New Project Investment Feasibility Analysis, Upstream raw materials and manufacturing equipment & Industry chain analysis of Hemophilia Treatment industry.

4. Market size (sales, revenue) forecast by regions and countries from 2019 to 2025 of Hemophilia Treatment industry.

Research Methodology: Hemophilia Treatment Market

Looking for more? Check out our repository for all available reports on Hemophilia Treatment in related sectors.

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Hemophilia Treatment Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19) - Cheshire Media

Global Contract Cell and Gene Therapy Manufacturing Market Report 2020-2026: CDMO Categorization – Primed for Business Model Disruption – Benzinga

DUBLIN, Nov. 24, 2020 /PRNewswire/ -- The "Global Contract Cell and Gene Therapy Manufacturing Market 2020-2026 - Supply Chain Optimization and Decentralized Manufacturing to Expand the Industry" report has been added to ResearchAndMarkets.com's offering.

This research service focuses on the critical role being played by CDMOs in not only supporting new product research and development but also in creating standardized manufacturing protocols.

Additionally, the study explores different cross-sections of the market and discusses market dynamics for autologous and allogeneic solutions for cell and gene therapies and for products being manufactured for clinical trial use and for commercial markets. The variability in market dynamics, manufacturing protocols, and business models across cross-sections is high. Therefore, the study also covers the emergence of non-traditional CDMOs that have thrived as a result of this variability.

The cell and gene therapy segment is one of the fastest growing segments in the biopharmaceutical space. While the science behind the therapy has grown by leaps and bounds on the back of decades worth of research, manufacturing has unfortunately lagged behind.

To fully harness the curative potential of these therapies and ensure greater reach and affordability to patients, it is imperative that aggressive investments in manufacturing technology and capacity are made today. Investments in manufacturing technology advancements including automation, single-use technologies, and GMP-in-a-box, will not only enable operational efficiency gains but also reduce project costs, generating benefits which can be transferred directly to the patients.

A large part of this growth in the cell and gene therapy product pipeline is being driven by small and mid-sized biotechs that depend on CDMOs to mitigate product development risks and diffuse infrastructure investments to be able to build future products pipeline.

The study also provides a comprehensive and critical analysis of nationally coordinated efforts towards infrastructure development and the rise of academic institutes and hospitals in meeting not only the demand from clinics, but also the demand of the commercial market.

The study also analyses the growth of specialist CDMOs focusing on vectors and plasmids, for instance. The captures the regional outlook for growth based on the currently installed capacities as well as the pipeline investments being made towards expansion. Lastly, the study tracks growth opportunities across the entire product development spectrum from supply chain to upstream and downstream manufacturing.

Key Topics Covered:

1. Strategic Imperatives

2. Growth Opportunity Analysis - CGT CDMO Market

3. Growth Opportunity Universe - CGT CDMO Market

4. Next Steps

For more information about this report visit https://www.researchandmarkets.com/r/kmx4o3

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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Global Contract Cell and Gene Therapy Manufacturing Market Report 2020-2026: CDMO Categorization - Primed for Business Model Disruption - Benzinga

Internet hawkers of stem cell therapies raising red flags – Ophthalmology Times

This article was reviewed by Ajay E. Kuriyan. MD, MS

Patients have been blinded or have experienced retinal detachments after treatment with so-called stem cell therapies such as intravitreal injections of autologous stem cells that are not FDA approved for many ocular conditions across the US and that were marketed directly to patients.

Ajay Kuriyan, MD, MS, and colleagues had previously identified 40 such business with 70 clinics in the US that advertise these treatments directly to consumers.

Related: Improve patient comfort with intravitreal injections

He explained that the FDA began to issue permanent injunctions against these company, including US Stem Cell Clinic, LLC, US Stem Cell Inc., and the Cell Surgical Network Corporation.

Currently, the FDA has regulatory authority over such companies and since June 2019, has been issuing orders to prevent them from developing or distributing stromal vascular fraction products that are not FDA approved, he explained.

In light of this kind of marketing activity, Adam Ross-Hirsch, MD, first author of the study under discussion, and colleagues set out to determine the scope of U.S.-based businesses advertising and administering cell therapy for ocular conditions, by looking at the businesses public websites to see what has changed since they first published their findings in 2017.1

The investigators identified these business by searching the Internet using specific keywords, such as clinic locations, marketed ocular conditions, types of cell therapy offered, routes of administration, affiliation with credentialed physician, safety language, and treatment costs.

Related: Intravitreally injected hRPCs improve vision in retinitis pigmentosa cases

Results The results of the analysis showed that 13 companies had removed their advertisements for ocular conditions, and 6 discontinued their URLs.

However, despite these findings, the total number of companies promoting treatment remained almost the same as the first study.

A total of 39 businesses with 62 clinics were identified by August 2019; 14 of these and 20 clinics were newly identified, Dr. Ross-Hirsch reported.

Most of the clinics were in Texas (n=12), California (n=11), Florida (n=10), and Illinois (n=10). When the investigators compared these numbers of clinics to those previously identified, the number in California decreased by 48% and the ones in Texas more than doubled, he said.

Thirty of the 39 companies reported that they were affiliated with at least 1 credentialed physician. Six businesses claimed to be associated with ongoing clinical trials, however, only 2 were registered on the clinicaltrials.gov website.

Related: Stem cell transplantation: Restoring vision in AMD may be possible

Most of the identified companies did not show their treatment fees, but of the 4 that did the costs ranged from $4,000 to $12,000/treatment. Consultation fees ranged from $350 to $800 when they were listed.

All businesses marketed treatments for ocular diseases that included age-related macular degeneration [AMD], optic neuritis, retinitis pigmentosa [RP], diabetic retinopathy, and glaucoma, he said. Most claimed to be able to treat AMD followed by RP.

The cell therapies most often advertised were autologous adipose-derived stem cells followed by autologous bone marrow-derived stem cells, which was similar to the previous study.

Related: Exploring novel gene therapy approaches to treat ocular disease

Making claims In commenting on the findings, Kuriyan said, These websites contained various claims regarding the safety and the FDA, but the language contained variable and vague claims about the safety of the various cell therapies.

Examples of the language were the following: not approved or evaluated by the FDA, participation in patient funded clinical research, use of FDA or institutional review board approved equipment and/or protocols for cell isolation, and use of FDA guidelines for minimally manipulated same-day procedures intended for homologous use.

Ten companies did not mention the FDA, while other claims were that the advertised therapies were safe under FDA regulation and no knowledge in the current literature of serious harm transpiring due to the use of these products, he emphasized.

Related: Stem cells for dry AMD with GA show promise in early study

These companies identified in the study have the potential to leverage a faade of scientific legitimacy to capitalize on patients understandable desperation for effective sight-restoring therapies. Stem cell therapy is an area of active, intense, and scientifically rigorous research. In the future, it may safely and effectively treat a number of ocular conditions, Kuriyan stated.

Lacking evidence These companies persist despite the absence of high-quality evidence of the efficacy of the treatment, FDA approval of the procedures, the increasing regulatory oversight and legal action against some of these companies, and evidence of severe visual loss as a result of the procedures.

Based on these findings, the investigators concluded that these companies persist in their US-based direct-to-consumer marketing of cell therapies for ocular conditions. Related: Exploring safety of stem cells for dry AMD

Ross-Hirsch emphasized the importance of both counseling by ophthalmologists and optometrists for patients as a safeguard against these companies and as stem cell therapies enter well-designed trials and evidence-based practice and federal regulation. However, regulation remains limited.

Conclusion Direct marketing of cell therapies persists in the US. As of August 2019, at least 39 such companies are operating in the US. Current federal regulation is limited and the marketing on the internet is pervasive.

Counseling will serve as a safeguard for patients and become increasingly important as stem cell therapies enter well-designed clinical trials and possibly evidence-based practice. Read more by Lynda Charters

Reference 1. Nirwan RS, Albini TA, Sridhar J, et al. Assessing cell therapy clinics offering treatment of ocular conditions using direct-to-consumer marketing in the United States. Ophthalmology 2019;126:1350-1355.

Originally posted here:
Internet hawkers of stem cell therapies raising red flags - Ophthalmology Times

Details are everything: Where is Ottawas COVID-19 vaccination plan? – The Globe and Mail

Inside Oxford Vaccine Group's laboratories in Oxford, England, on Nov. 19, 2020.

ANDREW TESTA/The New York Times News Service

Prime Minister Justin Trudeau engaged in a strange bit of misdirection on Tuesday when he said Canada would not be among the first countries to receive COVID-19 vaccinations, because it no longer has any domestic production capacity for vaccines.

The United States, the United Kingdom and Germany are all aiming to start inoculating people in a matter of weeks, based on the fact the manufacturers of some of the leading vaccine candidates have production capacity on their shores.

Canada, on the other hand, will have to wait until the early months of 2021, Mr. Trudeau said.

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Its puzzling why he would mention something that, on the surface, is worrisome to Canadians, but in fact applies to most of the world.

The only Western country with any kind of a guarantee that it will be able to begin inoculations in December is the U.S., and thats because the Pfizer/BioNTech and Moderna vaccines two of the three closest to approval will first roll out there.

Germany has a deal, along with Italy, the Netherlands and France, with AstraZeneca that could see it share 400 million doses of the promising Oxford vaccine by the end of this year.

The U.K. could have the Oxford vaccine by then, too, but it will not have access to the Pfizer and Moderna vaccines until next year.

As well, all the leading manufacturers are busy creating non-U.S. supply chains that will begin delivery early in 2021.

Its not even true that Canada has no domestic production capacity for traditional vaccines. And the feds earlier this year funded expansions at two vaccine facilities; one in Montreal was supposed to be capable of producing 250,000 doses a month by November and two million by next year. However, there just is no immediate capacity to make the vaccines developed by Moderna and Pfizer, which use a groundbreaking gene therapy.

Ottawa probably should have spent more on a bigger domestic production facility and pushed for it to be built faster. But the federal government was aggressive in ordering close to 400 million doses from multiple manufacturers, while those vaccines were still in testing and development. By contracting for far more doses than Canada will ever need, Ottawa bought a kind of insurance policy. Assuming the manufacturers honour the contracts, there should be more than enough to go around.

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So why did Mr. Trudeau alarm the population and arm the opposition?

Its hard to explain, unless you look at the debate over Canadas ability to quickly get its hands on a new vaccine as a distraction from a more pressing issue: Ottawas glaring lack of a plan to distribute and administer those millions of doses.

In the U.K., the government has designated 1,250 local health clinics as injection sites that will give the first available vaccines to vulnerable people, such as the elderly, as early as next month.

It is also setting up at least 42 mass vaccination sites, such as conference centres, which if all goes well will begin serving the rest of the population beginning in January.

In the U.S., the federal government has hired a retired general who ran the armys supply chain to distribute vaccines to injection sites in each state on a per capita basis. The doses will come with prepackaged kits containing syringes, wipes and personal protective equipment.

And in Canada? We get platitudes from Health Minister Patty Hajdu about how the government is working around the clock to come up with a concrete plan with the provinces and territories.

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Given the poor job Ottawa and the provinces have done on testing and tracing, which have the same logistical challenges, theres reason to be concerned that when vaccines arrive, their distribution will be haphazard.

Tell us: Who is in charge of distribution at the federal level? Who are their counterparts in the provinces and territories? Who will get inoculated first? Will the vaccines be allocated to provinces on a per capita basis or by some other formula?

Where will the vaccines be delivered? In pharmacies? Hospitals? Temporary sites in hockey arenas? Who will staff them? What other infrastructure is needed?

Canadians have no clue about any of these things. And right now there is zero concrete evidence that their governments do either.

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Details are everything: Where is Ottawas COVID-19 vaccination plan? - The Globe and Mail

Impact of Covid 19 On Brain Tumor Treatment Market 2020 Industry Challenges Business Overview And Forecast Research Study 2026 – The Market Feed

Overview for Brain Tumor Treatment Market Helps in providing scope and definitions, Key Findings, Growth Drivers, and Brain Tumor Treatment Industry Various Dynamics.

The global Brain Tumor Treatment market focuses on encompassing major statistical evidence for the Brain Tumor Treatment industry as it offers our readers a value addition on guiding them in encountering the obstacles surrounding the market. A comprehensive addition of several factors such as global distribution, manufacturers, market size, and market factors that affect the global contributions are reported in the study. In addition the Brain Tumor Treatment study also shifts its attention with an in-depth competitive landscape, defined growth opportunities, market share coupled with product type and applications, key companies responsible for the production, and utilized strategies are also marked.

This intelligence and 2026 forecasts Brain Tumor Treatment industry report further exhibits a pattern of analyzing previous data sources gathered from reliable sources and sets a precedented growth trajectory for the Brain Tumor Treatment market. The report also focuses on a comprehensive market revenue streams along with growth patterns, analytics focused on market trends, and the overall volume of the market.

Moreover, the Brain Tumor Treatment report describes the market division based on various parameters and attributes that are based on geographical distribution, product types, applications, etc. The market segmentation clarifies further regional distribution for the Brain Tumor Treatment market, business trends, potential revenue sources, and upcoming market opportunities.

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Key players in the global Brain Tumor Treatment market covered in Chapter 4: Bristol Myers Squibb Antisense Pharma Dr. Reddys Laboratories Ltd Genetech U.S.A Hoffmann- La Roche AstraZeneca plc Merck & Co Mankind Pharma Novartis AG Macleods Pharmaceutical Limited Pfizer, Inc.

In Chapter 11 and 13.3, on the basis of types, the Brain Tumor Treatment market from 2015 to 2026 is primarily split into: Tissue Engineering Immunotherapy Gene Therapy Other Therapies

In Chapter 12 and 13.4, on the basis of applications, the Brain Tumor Treatment market from 2015 to 2026 covers: Hospitals and Clinics Treatment Center Others

Geographically, the detailed analysis of consumption, revenue, market share and growth rate, historic and forecast (2015-2026) of the following regions are covered in Chapter 5, 6, 7, 8, 9, 10, 13: North America (Covered in Chapter 6 and 13) United States Canada Mexico Europe (Covered in Chapter 7 and 13) Germany UK France Italy Spain Russia Others Asia-Pacific (Covered in Chapter 8 and 13) China Japan South Korea Australia India Southeast Asia Others Middle East and Africa (Covered in Chapter 9 and 13) Saudi Arabia UAE Egypt Nigeria South Africa Others South America (Covered in Chapter 10 and 13) Brazil Argentina Columbia Chile Others

The Brain Tumor Treatment market study further highlights the segmentation of the Brain Tumor Treatment industry on a global distribution. The report focuses on regions of North America, Europe, Asia, and the Rest of the World in terms of developing business trends, preferred market channels, investment feasibility, long term investments, and environmental analysis. The Brain Tumor Treatment report also calls attention to investigate product capacity, product price, profit streams, supply to demand ratio, production and market growth rate, and a projected growth forecast.

In addition, the Brain Tumor Treatment market study also covers several factors such as market status, key market trends, growth forecast, and growth opportunities. Furthermore, we analyze the challenges faced by the Brain Tumor Treatment market in terms of global and regional basis. The study also encompasses a number of opportunities and emerging trends which are considered by considering their impact on the global scale in acquiring a majority of the market share.

The study encompasses a variety of analytical resources such as SWOT analysis and Porters Five Forces analysis coupled with primary and secondary research methodologies. It covers all the bases surrounding the Brain Tumor Treatment industry as it explores the competitive nature of the market complete with a regional analysis.

Brief about Brain Tumor Treatment Market Report with [emailprotected]https://hongchunresearch.com/report/brain-tumor-treatment-market-2020-71373

Some Point of Table of Content:

Chapter One: Report Overview

Chapter Two: Global Market Growth Trends

Chapter Three: Value Chain of Brain Tumor Treatment Market

Chapter Four: Players Profiles

Chapter Five: Global Brain Tumor Treatment Market Analysis by Regions

Chapter Six: North America Brain Tumor Treatment Market Analysis by Countries

Chapter Seven: Europe Brain Tumor Treatment Market Analysis by Countries

Chapter Eight: Asia-Pacific Brain Tumor Treatment Market Analysis by Countries

Chapter Nine: Middle East and Africa Brain Tumor Treatment Market Analysis by Countries

Chapter Ten: South America Brain Tumor Treatment Market Analysis by Countries

Chapter Eleven: Global Brain Tumor Treatment Market Segment by Types

Chapter Twelve: Global Brain Tumor Treatment Market Segment by Applications 12.1 Global Brain Tumor Treatment Sales, Revenue and Market Share by Applications (2015-2020) 12.1.1 Global Brain Tumor Treatment Sales and Market Share by Applications (2015-2020) 12.1.2 Global Brain Tumor Treatment Revenue and Market Share by Applications (2015-2020) 12.2 Hospitals and Clinics Sales, Revenue and Growth Rate (2015-2020) 12.3 Treatment Center Sales, Revenue and Growth Rate (2015-2020) 12.4 Others Sales, Revenue and Growth Rate (2015-2020)

Chapter Thirteen: Brain Tumor Treatment Market Forecast by Regions (2020-2026) continued

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List of tables List of Tables and Figures Table Global Brain Tumor Treatment Market Size Growth Rate by Type (2020-2026) Figure Global Brain Tumor Treatment Market Share by Type in 2019 & 2026 Figure Tissue Engineering Features Figure Immunotherapy Features Figure Gene Therapy Features Figure Other Therapies Features Table Global Brain Tumor Treatment Market Size Growth by Application (2020-2026) Figure Global Brain Tumor Treatment Market Share by Application in 2019 & 2026 Figure Hospitals and Clinics Description Figure Treatment Center Description Figure Others Description Figure Global COVID-19 Status Overview Table Influence of COVID-19 Outbreak on Brain Tumor Treatment Industry Development Table SWOT Analysis Figure Porters Five Forces Analysis Figure Global Brain Tumor Treatment Market Size and Growth Rate 2015-2026 Table Industry News Table Industry Policies Figure Value Chain Status of Brain Tumor Treatment Figure Production Process of Brain Tumor Treatment Figure Manufacturing Cost Structure of Brain Tumor Treatment Figure Major Company Analysis (by Business Distribution Base, by Product Type) Table Downstream Major Customer Analysis (by Region) Table Bristol Myers Squibb Profile Table Bristol Myers Squibb Production, Value, Price, Gross Margin 2015-2020 Table Antisense Pharma Profile Table Antisense Pharma Production, Value, Price, Gross Margin 2015-2020 Table Dr. Reddys Laboratories Ltd Profile Table Dr. Reddys Laboratories Ltd Production, Value, Price, Gross Margin 2015-2020 Table Genetech U.S.A Profile Table Genetech U.S.A Production, Value, Price, Gross Margin 2015-2020 Table Hoffmann- La Roche Profile Table Hoffmann- La Roche Production, Value, Price, Gross Margin 2015-2020 Table AstraZeneca plc Profile Table AstraZeneca plc Production, Value, Price, Gross Margin 2015-2020 Table Merck & Co Profile Table Merck & Co Production, Value, Price, Gross Margin 2015-2020 Table Mankind Pharma Profile Table Mankind Pharma Production, Value, Price, Gross Margin 2015-2020 Table Novartis AG Profile Table Novartis AG Production, Value, Price, Gross Margin 2015-2020 Table Macleods Pharmaceutical Limited Profile Table Macleods Pharmaceutical Limited Production, Value, Price, Gross Margin 2015-2020 Table Pfizer, Inc. Profile Table Pfizer, Inc. Production, Value, Price, Gross Margin 2015-2020 Figure Global Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Global Brain Tumor Treatment Revenue ($) and Growth (2015-2020) Table Global Brain Tumor Treatment Sales by Regions (2015-2020) Table Global Brain Tumor Treatment Sales Market Share by Regions (2015-2020) Table Global Brain Tumor Treatment Revenue ($) by Regions (2015-2020) Table Global Brain Tumor Treatment Revenue Market Share by Regions (2015-2020) Table Global Brain Tumor Treatment Revenue Market Share by Regions in 2015 Table Global Brain Tumor Treatment Revenue Market Share by Regions in 2019 Figure North America Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Europe Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Asia-Pacific Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Middle East and Africa Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure South America Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure North America Brain Tumor Treatment Revenue ($) and Growth (2015-2020) Table North America Brain Tumor Treatment Sales by Countries (2015-2020) Table North America Brain Tumor Treatment Sales Market Share by Countries (2015-2020) Figure North America Brain Tumor Treatment Sales Market Share by Countries in 2015 Figure North America Brain Tumor Treatment Sales Market Share by Countries in 2019 Table North America Brain Tumor Treatment Revenue ($) by Countries (2015-2020) Table North America Brain Tumor Treatment Revenue Market Share by Countries (2015-2020) Figure North America Brain Tumor Treatment Revenue Market Share by Countries in 2015 Figure North America Brain Tumor Treatment Revenue Market Share by Countries in 2019 Figure United States Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Canada Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Mexico Brain Tumor Treatment Sales and Growth (2015-2020) Figure Europe Brain Tumor Treatment Revenue ($) Growth (2015-2020) Table Europe Brain Tumor Treatment Sales by Countries (2015-2020) Table Europe Brain Tumor Treatment Sales Market Share by Countries (2015-2020) Figure Europe Brain Tumor Treatment Sales Market Share by Countries in 2015 Figure Europe Brain Tumor Treatment Sales Market Share by Countries in 2019 Table Europe Brain Tumor Treatment Revenue ($) by Countries (2015-2020) Table Europe Brain Tumor Treatment Revenue Market Share by Countries (2015-2020) Figure Europe Brain Tumor Treatment Revenue Market Share by Countries in 2015 Figure Europe Brain Tumor Treatment Revenue Market Share by Countries in 2019 Figure Germany Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure UK Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure France Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Italy Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Spain Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Russia Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Asia-Pacific Brain Tumor Treatment Revenue ($) and Growth (2015-2020) Table Asia-Pacific Brain Tumor Treatment Sales by Countries (2015-2020) Table Asia-Pacific Brain Tumor Treatment Sales Market Share by Countries (2015-2020) Figure Asia-Pacific Brain Tumor Treatment Sales Market Share by Countries in 2015 Figure Asia-Pacific Brain Tumor Treatment Sales Market Share by Countries in 2019 Table Asia-Pacific Brain Tumor Treatment Revenue ($) by Countries (2015-2020) Table Asia-Pacific Brain Tumor Treatment Revenue Market Share by Countries (2015-2020) Figure Asia-Pacific Brain Tumor Treatment Revenue Market Share by Countries in 2015 Figure Asia-Pacific Brain Tumor Treatment Revenue Market Share by Countries in 2019 Figure China Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Japan Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure South Korea Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Australia Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure India Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Southeast Asia Brain Tumor Treatment Sales and Growth Rate (2015-2020) Figure Middle East and Africa Brain Tumor Treatment Revenue ($) and Growth (2015-2020) continued

About HongChun Research: HongChun Research main aim is to assist our clients in order to give a detailed perspective on the current market trends and build long-lasting connections with our clientele. Our studies are designed to provide solid quantitative facts combined with strategic industrial insights that are acquired from proprietary sources and an in-house model.

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Impact of Covid 19 On Brain Tumor Treatment Market 2020 Industry Challenges Business Overview And Forecast Research Study 2026 - The Market Feed

Global Gene Therapy Industry – GlobeNewswire

November 19, 2020 05:27 ET | Source: ReportLinker

New York, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Industry" - https://www.reportlinker.com/p05817594/?utm_source=GNW 6% in the year 2020 and thereafter recover and grow to reach US$3.3 billion by the year 2027, trailing a post COVID-19 CAGR of 19.5% over the analysis period 2020 through 2027. Governments worldwide are focusing all healthcare resources on fighting the global pandemic. Billions of dollars have poured into researching COVID-19 drugs, therapies and vaccines. Over US$8 billion globally excluding the U.S. has been pledged only for vaccine development. The U.S. has independently pumped billions of dollars into COVID-19 research and response. The massive reallocation of funds and reprioritization of efforts has left a glaring gap in other sectors of healthcare. Gene therapy which holds promise for treating cancer, cystic fibrosis, heart disease, diabetes, hemophilia & AIDS, is slumping due to lack of research funds & reduced footfall of patients seeking treatment. Given the complex and fragile manufacturing and delivery system along with funding models of the industry, COVID-19 has emerged as a black swan event. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites. There are concerns regarding administration of cell and gene therapies. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centres. Treatments intended to be delivered into ICUs are being impacted by bed reservations made for patients with COVID-19 infection.

R&D and preclinical activities are also affected by supply shortages as a result of strong demand for consumables like reagents and PPE from COVID-19 laboratories. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrolments for protecting participants from the risk of infection. These issues are delaying activation of new sites, prompting players to postpone new clinical trials. However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. While companies targeting paediatric diseases suspended trials, others dealing with oncology maintained the pace. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. These issues are attributed to confluence of various factors like travel ban, withdrawal of several services from healthcare sites and the risk of virus transmission. In addition, these disruptions are anticipated to threaten existence of certain cell and gene therapy companies, particularly small-scale biotech players that are in pre-commercial phase and rely on external funding. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.

In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Gene therapies hold promise to improve the condition of patients where traditional cancer treatments such as radiation and chemotherapy are not effective. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. Oncology will remain the key area of focus for gene therapy applications. Cancer therapies represent the leading category, as is gauged through robust rise in the number of molecules being tested across numerous clinical trials. Novartis which recently bagged the U.S. FDA approval for Kymriah, a gene therapy designed for the treatment of hematological cancer, is seeking to gain commercial approval in established and emerging countries. Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals. More developmental focus will also be shed on monogenic rare diseases which have clearer genomic targets and the unmet need in smaller patient populations. Majority gene therapies so far have come to market through accelerated review pathways of regulatory authorities. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed. In the coming years, there will be significant improvement in the number of approvals for new gene therapies. The growth is anticipated to emerge from different modalities including RNAi, ASOs and CRISPR gene editing based therapeutics which offer long term opportunities for growth. These technologies are generating much excitement for investors.

Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=GNW

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE I-1

II. EXECUTIVE SUMMARY II-1

1. MARKET OVERVIEW II-1 A Prelude to Gene Therapy II-1 Classification of Gene Therapies II-1 Impact of Covid-19 and a Looming Global Recession II-2 COVID-19 Causes Gene Therapy Market to Buckle & Collapse II-2 COVID-19 Impact on Different Aspects of Gene Therapy II-2 Manufacturing & Delivery II-2 Research & Clinical Development II-3 Commercial Operations & Access II-3 Managing Derailed Operations II-4 Focus on Clinical Development Programs II-4 Targeting Manufacturing & Delivery Strategies II-4 Securing Supplies II-4 Remote Working II-4 Gene Therapy Set to Witness Rapid Growth Post COVID-19 II-5 By Vector Type II-5 VIRAL VECTORS ACCOUNT FOR A MAJOR SHARE OF THE MARKET II-5 Adeno-Associated Virus Vectors II-6 Lentivirus II-6 NON-VIRAL VECTORS TO WITNESS FASTER GROWTH II-7 US and Europe Dominate the Gene Therapy Market II-8 Oncology Represents the Largest Indication for Gene Therapy II-9 Market Outlook II-9 WORLD BRANDS II-10

2. FOCUS ON SELECT PLAYERS II-16 Recent Market Activity II-18 Select Innovations II-24

3. MARKET TRENDS & DRIVERS II-25 Availability of Novel Therapies Drive Market Growth II-25 Select Approved Gene Therapy Products II-26 Adeno-associated Virus Vectors - A Leading Platform for Gene Therapy II-27 Lentiviral Vectors Witness Increased Interest II-27 Rising Cancer Incidence Worldwide Spurs Demand for Gene Therapy II-28 Exhibit 1: Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 II-28 Exhibit 2: Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 II-29 Exhibit 3: Number of New Cancer Cases and Deaths (in Million) by Region for 2018 II-30 Compelling Level of Technology & Innovation to Ignite Gene Therapy II-30 Promising Gene Therapy Innovations for Treatment of Inherited Retinal Diseases II-31 Gene Therapy Pivots M&A Activity in Dynamic Domain of Genomic Medicine II-31 M&As Rampant in Gene Therapy Space II-31 Gene Therapy Deals: 2018 and 2019 II-32 Emphasis on Formulating Robust Regulatory Framework II-33 Strong Gene Therapy Pipeline II-33 Gene Therapy: Phase III Clinical Trials II-33 OHSU Implements First-Ever LCA10 Gene Therapy Clinical Trial with CRISPR II-35 Growing Funding for Gene Therapy Research II-35 Market Issues & Challenges II-35

4. GLOBAL MARKET PERSPECTIVE II-37 Table 1: World Current & Future Analysis for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-37

Table 2: World Historic Review for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-38

Table 3: World 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2017, 2020 & 2027 II-39

Table 4: World Current & Future Analysis for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-40

Table 5: World Historic Review for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-41

Table 6: World 10-Year Perspective for Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-42

Table 7: World Current & Future Analysis for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-43

Table 8: World Historic Review for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-44

Table 9: World 10-Year Perspective for Non-Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-45

Table 10: World Current & Future Analysis for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-46

Table 11: World Historic Review for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-47

Table 12: World 10-Year Perspective for Oncological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-48

Table 13: World Current & Future Analysis for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-49

Table 14: World Historic Review for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-50

Table 15: World 10-Year Perspective for Rare Diseases by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-51

Table 16: World Current & Future Analysis for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-52

Table 17: World Historic Review for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-53

Table 18: World 10-Year Perspective for Neurological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-54

Table 19: World Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-55

Table 20: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 II-56

Table 21: World 10-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2020 & 2027 II-57

III. MARKET ANALYSIS III-1

GEOGRAPHIC MARKET ANALYSIS III-1

UNITED STATES III-1 Table 22: USA Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-1

Table 23: USA Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-2

Table 24: USA 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-3

Table 25: USA Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-4

Table 26: USA Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-5

Table 27: USA 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-6

CANADA III-7 Table 28: Canada Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-7

Table 29: Canada Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-8

Table 30: Canada 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-9

Table 31: Canada Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-10

Table 32: Canada Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-11

Table 33: Canada 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-12

JAPAN III-13 Table 34: Japan Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-13

Table 35: Japan Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-14

Table 36: Japan 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-15

Table 37: Japan Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-16

Table 38: Japan Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-17

Table 39: Japan 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-18

CHINA III-19 Table 40: China Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-19

Table 41: China Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-20

Table 42: China 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-21

Table 43: China Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-22

Table 44: China Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-23

Table 45: China 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-24

EUROPE III-25 Table 46: Europe Current & Future Analysis for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 III-25

Table 47: Europe Historic Review for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-26

Table 48: Europe 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2017, 2020 & 2027 III-27

Table 49: Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-28

Table 50: Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-29

Table 51: Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-30

Table 52: Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-31

Table 53: Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-32

Table 54: Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-33

FRANCE III-34 Table 55: France Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-34

Table 56: France Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-35

Table 57: France 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-36

Table 58: France Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-37

Table 59: France Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-38

Table 60: France 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-39

GERMANY III-40 Table 61: Germany Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-40

Table 62: Germany Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-41

Table 63: Germany 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-42

Table 64: Germany Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-43

Table 65: Germany Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-44

Table 66: Germany 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-45

ITALY III-46 Table 67: Italy Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-46

Table 68: Italy Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-47

Table 69: Italy 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-48

Table 70: Italy Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-49

Table 71: Italy Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-50

Table 72: Italy 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-51

UNITED KINGDOM III-52 Table 73: UK Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-52

Table 74: UK Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-53

Table 75: UK 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-54

Table 76: UK Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-55

Table 77: UK Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-56

Table 78: UK 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-57

REST OF EUROPE III-58 Table 79: Rest of Europe Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-58

Table 80: Rest of Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-59

Table 81: Rest of Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-60

Table 82: Rest of Europe Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-61

Table 83: Rest of Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-62

Table 84: Rest of Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2020 & 2027 III-63

ASIA-PACIFIC III-64 Table 85: Asia-Pacific Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-64

Table 86: Asia-Pacific Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 III-65

Table 87: Asia-Pacific 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2020 & 2027 III-66

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Global Gene Therapy Industry - GlobeNewswire

Children’s Hospital delivers life-saving gene replacement therapy to infant – Loma Linda University Health

Loma Linda University Childrens Hospital recently gave its first administration of a new gene replacement therapy to an infant who screened positive for spinal muscular atrophy (SMA), the top genetic killer of children under the age of two, potentially providing a symptom-free life for the patient.

Childrens Hospital physicians said the new drug therapy, Zolgensma, is a revolution in neurological pediatric care.

This therapy represents a new opportunity for us to intervene and change childrens long-term prospects, and sometimes if we can treat the disease in time, we can restore them to full health, said David Michelson, MD, chief of the division of child neurology and director of the pediatric muscular dystrophy association clinics at Childrens Hospital. This therapy is a chance to wipe a patients health slate clean.

The patient was diagnosed with SMA through the state of Californias newborn screening program. Even though the patient seemed perfectly healthy during the first weeks of life, the healthcare team was working against the clock to treat the patient before the first potential onset of symptoms. Studies have shown that infants who receive the therapy as soon after diagnosis as possible have better outcomes than those who receive delayed treatment.

SMA is a rare genetic disease that causes a persons muscles to weaken and become smaller over time. In an infant, this affects development, including crawling, walking, sitting and head control. Severe types of SMA affect the muscles used for swallowing and breathing and, untreated, lead to ventilator dependence and a high risk of mortality.

According to Michelson, the patients neurologist, there are four types of SMA. These types differ in both their severity and at what age they will manifest in a persons body. SMA type I, the most common of the severe forms, can present in infants as early as three months and is often fatal by the age of two.

The new therapy involves one of only three approved drugs to treat SMA in children under two and is the only single-dose treatment option for SMA patients. Hospitals across the country are only beginning to be able to provide this therapy to qualifying patients since the therapy received FDA approval in May 2019.

Michelson said he and his team dont yet know exactly how the treatment will affect the patient.

We may not know how much we helped since we were able to treat him before he developed symptoms, Michelson said. Best case scenario is he will never develop any symptoms of the disease.

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Children's Hospital delivers life-saving gene replacement therapy to infant - Loma Linda University Health

Global Precision Cancer Therapies Market 2020: Analysis, Industry Growth, Current Trends and Forecast till 2025 – The Daily Philadelphian

The recently updated and published report titled Global Precision Cancer Therapies Market 2020 by Company, Type and Application, Forecast to 2025, by MarketQuest.biz comprises an exhaustive study of the market that gathers important and crucial information of market size, growth rate, market possibilities, and market revenue forecast from 2020-2025. The report can assist the stakeholders to understand key trends and prospects in the global Precision Cancer Therapies market as well as identify the growth opportunities and competitive scenarios. Crucial aspects covered in the report include key market trends, key dominating players, chapter-wise segregation. Mainly, the report promotes an ambitious landscape of the market, business overview, their policies, and recent developments.

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Company Profiles:

Every market participant needs to be familiar with the competitive scenario in the global Precision Cancer Therapies industry. The industry research report layouts historical and forecast market data, demand, application details, price trends, and company shares of the leading player by geography. The industry analysts have examined the strategic activities of the competitors to help the key players increase their competitiveness. The report studies the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. Some of the major companies included in the report are: Abbott Laboratories, Aveo Pharmaceuticals, OncoGenex Pharmaceuticals, Bayer HealthCare, AstraZeneca, GlaxoSmithKline, Boehringer Ingelheim, Hospira

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The study provides an in-depth overview of country-level break-up classified as potentially high growth rate territory, countries with the highest market share in past and current scenario. Some of the regional break-up classified in the study are: North America (United States, Canada and Mexico), Europe (Germany, France, United Kingdom, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), Middle East & Africa (Saudi Arabia, UAE, Egypt and South Africa)

Market segmentation by product: Hormone Therapy, Immunotherapies, Targeted Therapy, Monoclonal Antibody Therapy, Gene Therapy

Market segmentation by application: Hospitals, Diagnostic Centers, Oncology Clinics, Research Institutes

In the regional analysis, the report highlights the prospective region, which is estimated to generate opportunities in the global Precision Cancer Therapies market in the forthcoming years. The report incorporates data regarding the supply and demand situation, the competitive scenario, and the challenges for market growth. Components such as market drivers, restraints, challenges, and opportunities for the market are explained in detail. The report additionally provides a complete assessment of the expected behavior about the future market and changing market scenario.

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Global Precision Cancer Therapies Market 2020: Analysis, Industry Growth, Current Trends and Forecast till 2025 - The Daily Philadelphian

Cutting-edge Treatment Offers Hope to Infant With Spinal Muscular Atrophy – Adventist Review

November 17, 2020

By Sheann Brandon, Loma Linda University Health

Loma Linda University Childrens Hospital in Loma Linda, California, United States, recently gave its first administration of a new gene replacement therapy to an infant who screened positive for spinal muscular atrophy(SMA). SMA is the top genetic killer of children under the age of two, and the treatment potentially provides a symptom-free life for the patient.

Childrens Hospital physicians said the new drug therapy Zolgensma is a revolution in neurological pediatric care.

This therapy represents a new opportunity for us to intervene and change childrens long-term prospects, and sometimes if we can treat the disease in time, we can restore them to full health, David Michelson, chief of the division of child neurologyand director of the pediatric muscular dystrophy association clinics at Childrens Hospital, said. This therapy is a chance to wipe a patients health slate clean.

The patient was diagnosed with SMA through the state of Californias newborn screening program. Even though the patient seemed perfectly healthy during the first weeks of life, the health-care team worked against the clock to treat the patient before the first onset of symptoms. Studies have shown that infants who receive the therapy as soon after diagnosis as possible have better outcomes than those who receive delayed treatment.

SMA is a rare genetic disease that causes a persons muscles to weaken and become smaller over time. In an infant, this affects development, including crawling, walking, sitting, and head control. Severe types of SMA affect the muscles used for swallowing and breathing and, untreated, lead to ventilator dependence and a high risk of mortality.

According to Michelson, the patients neurologist, there are four types of SMA. These types differ in their severity and at what age they will manifest in a persons body. SMA type I, the most common of the severe forms, can present in infants as early as three months and is often fatal by the age of two.

The new therapy involves one of only three approved drugs to treat SMA in children under two and is the only single-dose treatment option for SMA patients. Hospitals across the country are only beginning to provide this therapy to qualifying patients since the treatment received U.S. Food and Drug Administration (FDA) approval in May 2019.

Michelson said he and his team dont yet know exactly how the treatment will affect the patient.

We may not know how much we helped, since we were able to treat him before he developed symptoms, Michelson said. Best case scenario is he will never develop any symptoms of the disease.

The original version of this story was posted on the Loma Linda University Health news site.

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Cutting-edge Treatment Offers Hope to Infant With Spinal Muscular Atrophy - Adventist Review

Molecular Testing With Universal Method Comparable to Traditional Method in Hereditary Solid Tumors – Targeted Oncology

In multiple solid malignancies, including breast, ovarian, colorectal, and pancreatic cancers, there is a subset of patients with a hereditary predisposition for these diseases, but the current testing criteria do not mention this subset. In an effort to provide more information of genetic testing in this population, a study of Memorial Sloan Kettering Cancer Center (MSKCC) patients was conducted to test the traditional guideline-based method of testing versus universal testing of a broad cancer patient population over a 5-year period.

A total of 7235 patients were included in the analysis and tested for 76 to 88 cancers. Through this study, investigators uncovered pathogenic and likely pathogenic (P/LP) variants in 7.5% (95% CI 6.6%- 8.4%) of 3,341 patients with breast cancer, 17.4% (95% CI, 14%- 21.6%) of 384 those with ovarian cancer, 13.5% (95% CI, 9.8%- 18%) of 252 patients with colorectal, and 8.8% (95% CI, 5.1%-14.8%) of 136 patients with pancreatic cancer.

Overall, the study found that testing with universal method was comparable to the guideline-based method, implying that universal testing can expand genetic testing to patient populations who are in need but are currently underserved.

In an interview with Targeted Oncology during the 2020 Association for Molecular Pathology (AMP) Annual Meeting, Ozge Ceyhan-Birsoy, PhD, assistant directorof the Laboratoryfor Molecular Medicine, MSKCC, discussed genetic testing methods for patients with hereditary predisposition and the molecular research underway at MSKCC to improve testing in this patient population.

TARGTED ONCOLOGY: In recent years, what advances have we see in cancer genetics?

Ceyhan-Birsoy: There have been significant advances in the range of genetic testing options for cancer patients in recent years. More patients are now able to receive molecular testing on their tumors to identify optimal targeted therapies for their cancer and germline genetic testing to uncover hereditary cancer predisposition. A paired analysis of tumor and normal DNA is increasingly being adapted, which improves the interpretation of both somatic and germline mutations. Additionally, incorporation of RNA analysis has expanded the scope of mutations that can be detected and characterized. Finally, the use of cell-free DNA now allows us to profile a patients tumor using only their blood.

TARGETED ONCOLOGY: How can hereditary predisposition inform oncologist for care/treatment decisions?

Ceyhan-Birsoy: Identifying hereditary mutations that predispose patients to cancer has important implications for their treatment and management. There are established targeted therapies available now for certain germline defects. For instance, germline mutations in certain homologous recombination and mismatch repair genes can predict response to PARP inhibitor and immune-checkpoint inhibitor therapies, respectively. Some therapies may pose high risk for patients with particular gene mutations, such as radiation therapy risks for patients with germline TP53 mutations. In addition, identifying hereditary cancer predisposition is critical to allow timely surveillance and prophylactic interventions for future cancers that the patient may be at higher risk of developing. As germline mutations are heritable, this information provides the opportunity for early surveillance in the patients family members, as well.

TARGETED ONCOLOGY: Can you explain how this MSKCC study came about?

Ceyhan-Birsoy: Genetic testing for hereditary cancer predisposition is traditionally performed in a guideline-dependent and targeted manner. In current practice, only patients who meet established criteria from national and professional organizations receive genetic testing and typically get tested for a small number of genes selected based on their tumor type, age of onset, and family histories. MSKCC has been 1 of the first institutes to pilot a universal testing approach for cancer patients, providing comprehensive germline testing of all known cancer predisposition genes without pre-selection of patients based on traditional genetic testing criteria. We have been performing both targeted and universal testing for our patients since 2015.

In this study, we aimed to understand how the yields (positive rates) of these 2 testing approaches compare to each other in greater than 4000 patients who had traditional and more than 9,000 patients who had universal testing at MSKCC in the past 5 years. We also assessed whether universal testing identified additional findings that would have been missed in a targeted testing approach for any given patient.

TARGETED ONCOLOGY: What are the key results of this analysis?

Ceyhan-Birsoy: We saw that universal germline testing without preselection of patients based on current guidelines yielded comparable rates of positive results to traditional guideline-dependent testing approach, particularly in patients with breast, ovarian, and pancreatic cancers. In addition, universal testing uncovered mutations that predispose to other cancers in about 9% of patients in genes that are not routinely tested for their diagnosis. Approximately half of those conferred high to moderate risk to cancer and about 40% of them implicated early surveillance or prophylactic surgery recommendations to prevent other cancers.

TARGETED ONCOLOGY: What is a key takeaway from your AMP 2020 presentation and explain the implications of these findings?

Ceyhan-Birsoy: Our results suggest that the preselection of patients for genetic testing based on the current guidelines may not significantly increase the likelihood of identifying a germline mutation in certain patient populations. A universal and comprehensive testing approach further provides the benefit of identifying hereditary risk for other cancers, allowing early surveillance and prophylactic interventions.

TARGETED ONCOLOGY: How can this information be applied in oncology clinics?

Ceyhan-Birsoy: Our study underlies the advantages of universal and comprehensive testing for cancer patients. However, there are many challenges that may limit the application of this approach for all cancer patients, including the cost of testing, resources needed to provide pre-test and post-test genetic counseling to patients, and the potential to discover more variants of uncertain significance that may lead to higher number of inconclusive results. Future efforts should be dedicated to providing wider groups of cancer patients access to genetic testing, which can aid in their clinical care and in the care of their family members.

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Molecular Testing With Universal Method Comparable to Traditional Method in Hereditary Solid Tumors - Targeted Oncology