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Global CAR T-Cell Therapy Market Analysis Report, 2019 – Market Anticipated to Record a CAGR of 56.2% During 2018-2026 – ResearchAndMarkets.com -…

DUBLIN--(BUSINESS WIRE)--The "Global CAR T-Cell Therapy Market Analysis 2019" report has been added to ResearchAndMarkets.com's offering.

The CAR T-Cell Therapy market is expected to reach $13,617.1 million by 2026 growing at a CAGR of 56.2% from 2018 to 2026.

Factors such as technological advancement for advanced & reliable treatment for cancer, growing pharmaceutical industry, high prevalence of acute lymphoblastic lymphoma (ALL) and increase in the number of cell therapy clinical studies are fuelling the market growth. However, side-effects of CAR T-cell therapy and high cost of treatment are likely to hamper the market growth.

Based on the application, diffuse large B-cell lymphoma is a cancer of B cells, a type of white blood cell responsible for producing antibodies. It is the most common type of non-Hodgkin lymphoma among adults. This cancer occurs primarily in older individuals, with a median age of diagnosis at approximately 70 years of age, though it can also occur in children and young adults in rare cases. DLBCL is an aggressive tumour which can arise in virtually any part of the body, and the first sign of this illness is typically the observation of a rapidly growing mass, sometimes associated with B symptoms of fever, weight loss, and night sweats.

The key vendors mentioned are Mustang Bio, Inc., Celgene Corporation, Bluebird Bio, Inc., CARsgen Therapeutics, Ltd., Novartis International AG, Legend Biotech, Sorrento Therapeutics Inc., Kite Pharma, Inc., Immune Therapeutics, Bellicum Pharmaceuticals, Inc., Pfizer, Inc., Juno Therapeutics, Atara Biotherapeutics, Aurora Biopharma Inc., Eureka Therapeutics, Autolus, TILT Biotherapeutics, and Fortress Biotech.

Key Questions Answered in this Report

Key Topics Covered

1 Market Synopsis

2 Research Outline

2.1 Research Snapshot

2.2 Research Methodology

2.3 Research Sources

2.3.1 Primary Research Sources

2.3.2 Secondary Research Sources

3 Market Dynamics

3.1 Drivers

3.2 Restraints

4 Market Environment

4.1 Bargaining power of suppliers

4.2 Bargaining power of buyers

4.3 Threat of substitutes

4.4 Threat of new entrants

4.5 Competitive rivalry

5 Global CAR T-Cell Therapy Market, By Target Antigen

5.1 Introduction

5.2 CD20

5.3 EGFRV III

5.4 CD19

5.5 HER2

5.6 MESO

5.7 CD22

5.8 BCMA

5.9 GD2

5.10 CD30

5.11 HER1

5.12 CD33

6 Global CAR T-Cell Therapy Market, By Product

6.1 Introduction

6.2 Allogeneic

6.3 Autologous

7 Global CAR T-Cell Therapy Market, By Therapy

7.1 Introduction

7.2 Axicabtagene Ciloleucel (Yescarta)

7.3 Tisagenlecleucel (Kymriah)

8 Global CAR T-Cell Therapy Market, By Application

8.1 Introduction

8.2 Chronic Lymphocytic Leukemia

8.3 Diffuse Large B-Cell Lymphoma

8.4 Multiple Myeloma

8.5 Acute Lymphoblastic Leukemia

8.6 Follicular Lymphoma

8.7 Mantle Cell Lymphoma

8.8 Glioblastoma

8.9 Neuroblastoma

8.10 Breast Cancer

8.11 Sarcoma

8.12 Acute Myeloid Leukemia

8.13 Colorectal Cancer

8.14 Pancreatic Cancer

8.15 Hepatocellular Carcinoma

8.16 Non-Hodgkin Leukemia

8.17 Carcinoma

9 Global CAR T-Cell Therapy Market, By End User

9.1 Introduction

9.2 Cancer Research Centers

9.3 Hospitals

9.4 Academic and Research Institutes

9.5 Pharmaceutical Companies

9.6 Biotechnology Companies

9.7 Contract Research Organizations

10 Global CAR T-Cell Therapy Market, By Geography

10.1 Introduction

10.2 North America

10.3 Europe

10.4 Asia Pacific

10.5 South America

10.6 Middle East & Africa

11 Strategic Benchmarking

12 Vendors Landscape

12.1 Mustang Bio, Inc.

12.2 Celgene Corporation

12.3 Bluebird Bio, Inc.

12.4 CARsgen Therapeutics, Ltd.

12.5 Novartis International AG

12.6 Legend Biotech

12.7 Sorrento Therapeutics Inc.

12.8 Kite Pharma, Inc.

12.9 Immune Therapeutics

12.10 Bellicum Pharmaceuticals, Inc.

12.11 Pfizer, Inc.

12.12 Juno Therapeutics

12.13 Atara Biotherapeutics

12.14 Aurora Biopharma Inc.

12.15 Eureka Therapeutics

12.16 Autolus

12.17 TILT Biotherapeutics

12.18 Fortress Biotech

For more information about this report visit https://www.researchandmarkets.com/r/q389el

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First Myeloma Patient Dosed With UCARTCS1 CAR T-cell Therapy in Phase 1 Trial, Cellectis Announces – Myeloma Research News

The donor-derived, or allogeneic CAR T-cell therapy candidate UCARTCS1 has been administrated to the first patient in an ongoing Phase 1 trial for relapsed/refractory multiple myeloma.

The study (NCT04142619), called MELANI-01, plans to enroll up to 18 adults whose multiple myeloma relapsed after prior treatment. It is currently recruiting at MD Anderson Cancer Center, in Houston, and at Hackensack Meridian Health, in New Jersey. Another testing site at Weill Cornell Medicine, in New York, is expected to open soon. More information on clinical sites and contacts is available here.

The researchers will evaluate the safety and efficacy of multiple doses of UCARTCS1, being developed by Cellectis. The trial also will assess the transplanted cells ability to expand and survive inside the body.

After determining an optimal dose to be used in Phase 2, participants will be invited to continue treatment with this investigational treatment in an expansion study. MELANI-01 is expected to conclude in November 2022.

According to Cellectis, UCARTCS1 is the first allogeneic CAR-T cell therapy candidate cleared by the U.S. Food and Drug Administration to enter clinical trials for relapsed/refractory multiple myeloma.

In taking this next clinical step, we look forward to deepening our understanding of UCARTCS1 as a potential new treatment option for relapsed/refractory multiple myeloma patients in the future, Andr Choulika, PhD, chairman and CEO of Cellectis, said in a press release.

CAR T-cell therapies commonly use a patients own immune cells, which are harvested and modified in the lab to recognize and kill tumor cells. These cells are then expanded to several million and injected back into the patient to fight the cancer. This is called autologous, or patient-derived therapy.

Although such therapies have had positive results against multiple cancer types, scientists are not always able to collect enough cells from a patient to create the therapy. In addition, development and expansion of CAR T-cells requires time, which is in opposition to the prompt treatment needed for multiple myeloma.

To overcome these issues, Cellectis has been developing its off-the-shelf CAR T-cell products for diverse blood cancers including UCARTCS1 for multiple myeloma.

Instead of using a patients own cells, it uses immune T-cells from healthy donors. These donor cells must first be modified to avoid potential damage due to graft versus host disease the attack of the hosts cells by transplanted cells.

UCARTCS1 was specifically designed to target SLAMF7, or CS1 called signaling lymphocyte activation molecule family member 7 a protein found on the surface of myeloma cells. But as T-cells also produce SLAMF7, Cellectis used the TALEN gene editing technology to knock out or remove the protein from the surface of engineered T-cells.

This approach is intended to eliminate the patients T-cells to avoid cross reaction and create room for the transplanted cells to fight cancer.

This first patient dosing for our MELANI-01 clinical trial is an important advancement, as our team has worked tirelessly to develop and take the CS1 target from the lab to the clinic, Choulika said.

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Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

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First Myeloma Patient Dosed With UCARTCS1 CAR T-cell Therapy in Phase 1 Trial, Cellectis Announces - Myeloma Research News

Takeda sees cell, gene therapy in its future. Is it too late? – BioPharma Dive

Thanks to a $62 billion acquisition of Shire, Takeda is one of the world's largest developers of rare disease drugs.

Despite that, the 238-year-old Japanese pharmaceutical company lacks any mid- or late-stage cell or gene therapies, two technologies that figure to play a large role in how many rare cancers and inherited diseases will eventually be treated.

It's a mismatch Takedais putting substantial effort into addressing. Last week, executives made cell and gene therapy a notable focus of the company's first R&D day since closing its Shire deal.

"We have a world-class gene therapy platform," Dan Curran, head of Takeda's rare disease therapeutic area unit, told investors and Wall Street analysts gathered in New York city.

"We intend to build on that over the next five years. Because as we look to lead in the second half of [next]decade, we believe patients will demand and we can deliver transformative and curative therapies to patients globally."

But right now that's just an ambition. While Takedahas begun to explore how it can improve on current gene therapies, its candidates are early stage and lag their would-be competitors.

"Our heme A program we're behind. Our heme B program we're behind," admitted Curran in an interview. "But we're behind the first generation and when has there only been one generation of anything?"

Takeda's hemophilia A program is currently in Phase 1, with the hemophilia B candidate about to join it in human testing well back from leaders BioMarin Pharmaceutical, Spark Therapeutics and SangamoTherapeutics in hemophilia A and UniQure in hemophilia B.

Curran laid out three priorities for Takeda'spush: exploring whether gene therapy, typically pitched as a one-time treatment, can be re-dosed; lowering the doses currently used for first-generation therapies; and developing alternative gene delivery vehicles than the adeno-associatedand lentiviralvectors that are predominant today.

"We need to figure out how to re-dose AAVvectors if we want to provide functional cures for patients for the rest of their lives."

How long a gene therapy's benefit lasts is a critical question. In theory, it could last decades or potentially for life, depending on the treatment's target.

But clinical evidence presented to date suggests that benefit for some therapies could wane over time. BioMarin, for example, presented data this year that it argued is proof its gene therapy could raise Factor VIII expression levels in patients with hemophilia A above the threshold for mild disease for at least eight years a long time, to be sure, but not life-long.

Still, it's an unusual objective. Much of gene therapy's promise lies in the potential for it to be given just once and still deliver lasting benefits. And the therapies that have reached market most notably Spark Therapeutics' Luxturna, Novartis' Zolgensma and Bluebird bio's Zynteglo are among the most expensive drugs to ever reach market. Were a gene therapy to be re-dosed, the current value proposition those drugmakers describe would need to be re-evaluated.

Curran recognizes that bringing down costs substantially will be essential to any attempt to advance a multi-use gene therapy. But Takeda might have an advantage. In buying Shire, the pharma inherited a viral vector manufacturing plant, originally built by Baxalta, that Curran calls the company's "best kept secret."

"It's an enormous competitive advantage," he said, adding that Takeda believes it's among the industry's top three facilities by production capacity. "Roche trying to acquire Spark, Novartis and AveXis a significant component of value of those transactions was that these companies had actually invested in manufacturing capabilities."

Curran emphasized that Takeda's ambitions in gene therapy will require it to partner with academic leaders in the field, a playbook that it's followed over the past three years as it's worked to expand into cell therapy.

"In the cell space, there's more innovation you can bring up into proof of principle milestones in academia," said Andy Plump,Takeda'shead of R&D, in an interview.

"An academic can manipulate a cell, but it's very hard in an academic setting to optimize a small molecule," he added. "This is a space where Novartis, and now we, have been quite successful in creating those relationships."

Takeda has put partnerships in place with Japan's Center for iPS Cell Research and Application, GammaDelta, Noile-Immune Biotech, Memorial Sloan Kettering Cancer Center and, just this month, The University of Texas MD Anderson Cancer Center.

That last collaboration gives Takeda access to a chimeric antigen receptor-directed natural killer, or NK, cell therapy.The drugmaker believes NK cells could offer advantages over the T cells modified to create the currently available cell therapies Kymriah and Yescarta.

Most notably, MD Anderson's approach uses NK cells isolated from umbilical cord blood, rather than extracting T cells from each individual patient a time-consuming and expensive process that has complicated the market launch of Kymriah and Yescarta. Cord blood-derived NK cells are designed to be allogeneic, or administered "off the shelf."

Additionally, CAR NK cells haven't been associated (yet) with cytokine release syndrome or neurotoxicity, two significant side effects often associated with CAR-T cell therapies. That could help Takeda position its cell therapies as an outpatient option.

"Even if we were a company that entered a little bit later into the immuno-oncology space, we've very much tried to turn this into an advantage," said Chris Arendt, head of Takeda's oncology drug discovery unit, at the company's event.

"We believe we have a chance to establish a leadership position rather than jumping on the bandwagon and being a follower."

While Takeda's choice to pursue NK cell therapy stands out, its choice of target does not. TAK-007, a drug candidate from MD Anderson that is now Takeda's lead cell therapy program, is aimed at a cell surface protein called CD19 that's found in leukemias and lymphomas.

Both Yescarta and Kymriah target CD19, and a recent count by the Cancer Research Institute tracked 181 cell therapy projects aimed at the antigen.

Takeda is planning to advance TAK-007 into pivotal studies in two types of lymphoma and chronic lymphocytic leukemia by 2021, with a potential filing for approval in 2023.

By then, Kymriah and Yescarta will have been on the market for six years and current bottlenecks in cell therapy treatment could be solved, helping both Takeda's potential entry as well as the host of competitors it will likely face.

Next year will be a test of how productive Takeda'scell therapy unit can be. In addition to TAK-007, the pharmaexpects to have four other CAR-T and gamma delta cell therapies in the clinic, two of which will target solid tumors.

Cell and gene therapy are part of what Takeda calls its "second wave" of R&D projects, a group of early-stage drugs and programs that it sees as progressing to regulatory stages by 2025 or later.

In the nearer term, the drugmakeris advancing a "first wave" of clinical candidates that it told investors will deliver 14 new molecular entities by 2024. Five of those will come in rare disease, with the others spread across oncology, neuroscience, gastro-enterology and vaccines.

"We think the cascade of news coming forward on these programs will transform how people view Takeda," Curran said.

More importantly to the investors gathered in New York, Takeda expects these experimental drugs will eventually earn $10 billion in peak annual sales, which would represent a sizable addition to a business that generated $30 billion in sales last year.

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Takeda sees cell, gene therapy in its future. Is it too late? - BioPharma Dive

Global Cell & Gene Therapy Market Outlook and Forecast 2019-2024 – Robust Pipeline Products & Increasing Funding for R&D Activities Trigger Massive…

DUBLIN--(BUSINESS WIRE)--The "Cell and Gene Therapy Market - Global Outlook and Forecast 2019-2024" report has been added to ResearchAndMarkets.com's offering.

The global cell and gene therapy market is growing at a CAGR of over 24% during the forecast period 2018-2024.

Key Market Insights

The major drivers contributing to the growth of the global cell and gene therapy market are the growing incidence of several chronic and terminal diseases, including cancer, the launch of new products, the increasing availability in clinical evidences of these products in terms of safety and efficacy, the rapid adoption of CAR T-cell therapy, favorable regulatory support in the development of these treatment, and improved manufacturing expertise in these products.

Market Dynamics

Market Growth Enablers

Market Growth Restraints

Market Opportunities and Trends

Cell and Gene Therapy Market: Segmentation

This research report includes detailed market segmentation by product, application, end-user, and geography.

The global cell therapy market is growing at a steady rate, and this trend is expected to continue during the forecast period due to the increased patient base with a wide range of diseases/ailments. The segment is likely to witness upward growth on account of expanded expertise in the manufacturing of stem cell-based products.

The gene therapy segment is expected to witness faster growth as the penetration of these products is increasing at a significant rate, especially in developed economies. The market is expected to grow during the forecast period due to the increased patient base for the existing gene remedy products, expected the launch of other gene therapy-based products for several indications, and expanded indication approvals for existing commercially available products.

The oncology segment accounts for the highest share of the global market. The growth of the oncology segment is increasing at a fast rate on account of the growing prevalence of several types of cancers. Currently, the available products not only modify the disease but also improve the quality of the patient's life, thereby decreasing the mortality rate. The market in the dermatology segment is increasing at a steady rate. This segment owns its growth to the increasing incidence and prevalence rate of several types of wounds, which are difficult to treat under normal conditions and the launch of innovative products. The dermatology segment is likely to showcase growth due to the high product availability of wound care products in the market.

Hospitals are the leading end-user segment. The segment is growing mainly due to the increasing incidence/prevalence of chronic diseases such as cancer, cardiovascular diseases, diabetes, and chronic wound on account of diabetes feet, pressure ulcers, and other injuries.

Market Segmentation by Products

Market Segmentation by Distribution Channel Type

Market Segmentation by End-users

Geographical Insights

The US market dominates the cell and gene therapy market in North America due to the high prevalence of chronic diseases and other conditions, which require these treatment methods. There is also comparably high utilization and wide accessibility of these therapies. The oncology segment is likely to witness significant growth in North America.

The market in Europe is expected to witness upward growth in the near future on account of the growing prevalence of chronic diseases and rising elderly population. In Europe, cell and gene therapy products are considered to be part of the Advanced Therapy Medicinal Products (ATMPs), which are commonly known as regenerative medicine globally.

Key Vendor Analysis

The global market is characterized by the presence of a few global, large-scale companies and several small to medium-scale companies offering one or two cell and gene therapy products. Global players are majorly offering innovative products with the potential of disease-modifying characteristics that are generating significant revenues, especially in Europe and US regions. Most innovative and breakthrough products are approved in the European countries and the US.

Vendors are targeting mostly developed economies such as the US, Germany, France, the UK, Spain, and Japan as the uptake of these products is higher in these countries than low and middle-income countries. However, the market in these regions is at the nascent stage.

Key Vendors

Other Prominent Vendors

For more information about this report visit https://www.researchandmarkets.com/r/n758z0

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Global Cell & Gene Therapy Market Outlook and Forecast 2019-2024 - Robust Pipeline Products & Increasing Funding for R&D Activities Trigger Massive...

CAR-T therapies should be made by academic medical centers – STAT

Draw blood from someone with cancer. Engineer their blood cells to seek and destroy cancer. Reinfuse the cells and watch the cancer melt away. Chimeric antigen receptor T cell therapy (CAR-T) sounds like science fiction. But its the next frontier in cancer therapy.

Were weaponizing individuals immune systems to destroy cancer and add years to their lives. Its incredibly exciting. But at hundreds of thousands of dollars per dose, insurance companies and the U.S. government are struggling to figure out how to pay for these breakthrough treatments.

High prices not only pose a challenge to patient access, but they also raise a fundamental question: Are we creating these therapies the wrong way?

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CAR-T immunotherapy is being tested for a range of cancers, and now holds the potential to benefit more than 1 million Americans who live with or are in remission from blood cancers. A single dose typically costs around $400,000. What makes the price so high? Having drug corporations make the cells.

Under the current system, a hospital extracts blood cells from a patient and sends them to a drug companys manufacturing plant to be genetically engineered. It takes the company two to six weeks to engineer the cells, increase their number, perform quality and safety tests, and ship them back to the hospital to be reinfused into the patient. Under this system, there have been well-documented problems with the engineering process, as well as with shipping and handling.

Theres a better way, one that will lower the price, enable more precise and individualized targeting for specific patients, and allow for a faster process: Let medical centers do this.

Long before pharmaceutical companies took control of CAR-T, medical centers made these treatments. Cancer centers like the University of Pennsylvania, the National Cancer Institute, Memorial Sloan Kettering Cancer Center, Fred Hutchinson Cancer Center, Baylor University, and others figured out how to engineer CAR-T cells and ran the initial trials to test them. Drug companies were later involved mainly as a means to scale up production.

There are clear advantages to keeping CAR-T treatment in medical centers, closer to patients.

First, academic medical centers are well-equipped to make these therapies. They already safely handle stem cells every day. They also routinely perform autologous stem cell transplants (these use a patients own stem cells), which require in-house doctors and specialists to preserve and protect stored blood cells, wash and radiate them if needed, and sometimes select cell populations for clinical use.

Hospitals and academic medical centers can accomplish the CAR-T process more quickly because they do not need to ship cells to and from a drug company plant. This is a critical issue, because most patients currently treated with CAR-T have not responded to other treatments and their health is seriously compromised; some patients die between the time stem cells are removed from them and the time theyre supposed to come back to the hospital for re-infusion.

Second, there are different regulations around stem cells than there are for drugs. That means CAR-T therapies created at hospitals and academic medical centers would not have to go through the yearslong FDA regulatory process. And the treatments developed would allow hospitals to target more effectively and quickly each individuals specific cancer, which is off the table in the current system. Patients with advanced cancer do not have the luxury of time. Although this paradigm shift would require greater regulatory flexibility from the FDA, it would make CAR-T therapy far more effective.

Experience making CAR-T in leading medical centers show that locally engineered CAR-T cells can be made for less than half of what they are currently priced by pharmaceutical companies. We know this is possible because Switzerland is already doing it this way, and pricing CAR-T therapy between $150,000 and $200,000. Multiply that by the 10,000 individuals in the U.S. with the types of cancer for which CAR-T therapy is currently approved and we would save almost $2 billion a year. Factor in future CAR-T approvals and we will save many times that amount.

Making this change requires bucking the system. It means the FDA must redefine CAR-T from a drug to the autologous blood product it is. Its a move that would save not only money but lives because it can target cancer better and destroy it faster.

We took a fork in the CAR-T road a few years back and went the wrong way. There is still time to change course for the good of the many Americans who need this lifesaving treatment.

David Mitchell, who is living with incurable blood cancer, is the founder of Patients for Affordable Drugs. Saad Kenderian, M.D., is a physician-scientist and assistant professor of medicine and immunology at the Mayo Clinic in Rochester, Minn. S. Vincent Rajkumar, M.D., is a hematologist and professor of medicine at the Mayo Clinic. The views expressed are the authors personal views and do not necessarily reflect the policy or position of the Mayo Clinic.

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CAR-T therapies should be made by academic medical centers - STAT

Global Cell Therapies & Oncolytic Virus Markets, 2016-2019 & 2026 – Stimulation of the Intrinsic Vascular Cell to Provide Ample Industry…

DUBLIN--(BUSINESS WIRE)--The "Global Cell Therapies & Oncolytic Virus Market Analysis 2019" report has been added to ResearchAndMarkets.com's offering.

The Global Cell Therapies & Oncolytic Virus market is growing at a CAGR of 17.32% from 2018 to 2026.

Factors such as rise in technological advancements and increase in usage of combination therapies are driving the market growth. Though, occurrence of immune reaction to the virus-based treatments caused by repeated administration of therapeutics to treat cancer and Staggering expense related to oncolytic virus therapy are projected to inhibit the growth of the market. Moreover, stimulation of the intrinsic vascular cell may provide ample opportunities for the market growth.

By application, melanoma segment acquired significant growth in the market because most of the cancer therapies are developed for melanoma cancers. Increasing prostate cancer cases are also leading to the strong growth rate in prostate cancer therapies in recent years which is driving the market growth.

The key vendors mentioned are PsiOxus Therapeutics, Cold Genesys, TILT Biotherapeutics, SillaJen Biotherapeutics, Genelux Corporation, Vyriad, Lokon Pharma, TILT Biotherapeutics and Sorrento Therapeutics.

Key Questions Answered in this Report

Key Topics Covered

1 Market Synopsis

2 Research Outline

3 Market Dynamics

3.1 Drivers

3.2 Restraints

4 Market Environment

4.1 Bargaining power of suppliers

4.2 Bargaining power of buyers

4.3 Threat of substitutes

4.4 Threat of new entrants

4.5 Competitive rivalry

5 Global Cell Therapies & Oncolytic Virus Market, By Type

5.1 Introduction

5.2 Adenoviruses-Based Oncolytic viruses

5.3 HSV-Based Oncolytic Viruses

5.4 Newcastle Disease Virus-Based Oncolytic Viruses

5.5 Vaccinia Virus-Based Oncolytic Viruses

5.6 Vesicular Stomatitis Virus-Based Oncolytic Viruses

6 Global Cell Therapies & Oncolytic Virus Market, By Application

6.1 Introduction

6.2 Breast Cancer

6.3 Melanoma

6.4 Ovarian Cancer

6.5 Prostate cancer

7 Global Cell Therapies & Oncolytic Virus Market, By Geography

7.1 Introduction

7.2 North America

7.3 Europe

7.4 Asia-Pacific

7.5 South America

7.6 Middle East & Africa

8 Strategic Benchmarking

9 Vendors Landscape

9.1 PsiOxus Therapeutics

9.2 Cold Genesys

9.3 TILT Biotherapeutics

9.4 SillaJen Biotherapeutics

9.5 Genelux Corporation

9.6 Vyriad

9.7 Lokon Pharma

9.8 TILT Biotherapeutics

9.9 Sorrento Therapeutics

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

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Global Cell Therapies & Oncolytic Virus Markets, 2016-2019 & 2026 - Stimulation of the Intrinsic Vascular Cell to Provide Ample Industry...

Leading from the front in cell therapy – – pharmaphorum

In these videos, senior stakeholders from Kite, a Gilead Company, describe how a culture of innovation and partnership help it lead from the front in the team sport of cell therapy.

Dick Sundh, Anne Kerber, and Bethany Dudek reflect on the leadership focus of the company and outline how Kite aims to become the cell therapy leader of Europe.

Embodying leadership in cell therapy

On my very first day at Kite one of my colleagues said to me Dick, theres only one thing you need to remember; cell therapy is a team sport, says Dick Sundh, Head of Europe for Kite, a Gilead Company, as he describes the process of building the organisations cell therapy business in Europe.

Kite has been at the forefront of cell therapy development and, since the first European approvals of CAR T cell therapies in 2018, the company has been establishing the necessary European infrastructures to deliver cell therapies in the region, and manufacture at scale.

According to pharma veteran Sundh, the job has proven to be an interesting challenge and Kite has broken new ground in establishing the foundations required to enable access to cell therapies. We are learning every day along with our partners and medical institutions across Europe.

And its not only about the science, but about embodying leadership qualities by demonstrating the companys core values on a daily basis. When healthcare professionals and employees feel you truly care about patients you have a real opportunity to become a true leader.

Sundh explains that Kites situation is unique; operating as a separate business unit within one of the worlds largest pharma companies, Gilead Sciences.

Its about expanding the footprint for cell therapy in Europe. We have the best of both worlds because we have a big pharma company in the background with resources where we need it. Otherwise were independent and allowed to go as fast and as innovatively as we want to. Teamwork is at the heart of everything we do.

I feel very passionate and privileged about being part of redefining not only how we understand oncology, but how we treat it.

Delivering the potential of cell therapies

Anne Kerber, Vice President, Head of Clinical Development in Europe at Kite, a Gilead Company, describes the clinical process the company is undertaking to deliver on the potential of individualised cell therapies for patients with rare forms of blood cancer, who face ongoing unmet needs.

In her role, Kerber draws on her experience as a qualified haematologist/oncologist to inform her work with a diverse range of research partners across Europe, in the clinical development of cell therapies. We work closely with many healthcare professionals and see a high level of commitment from physicians to enrol in clinical trials; we understand that, for them, partnership, education and learning is key.

Having followed the development journey of cell therapies for several years, Kerber has gone on to work with healthcare professionals to build their knowledge of cell therapies for use in their own clinical research.

Kerber stresses the importance of teamwork in developing cell therapies, particularly in light of their complex manufacturing processes when compared to more conventional treatments.

Becoming closely involved with the experiences of patients on their treatment journey is another important part of Kerbers remit these are specialist therapies and as such the company has a much closer relationship with their stakeholders than would be the case with a conventional medicine.

In cell therapy, collaboration is very important and each person in the chain has a critical role to play in ensuring patients receive these therapies at the right time.

Translating innovation in quality systems into clinical delivery

Bethany Dudek, Executive Director, Head of Quality at Kite, a Gilead Company, describes the complex quality control processes that are required to deliver cell therapies to the exacting standards required.

Dudek and her team oversees everything from inspection of incoming apheresis white blood cells collected from patients using a specialised process to obtain vital T-cells to testing and releasing the final product once it has been manufactured.

Cell therapies are bespoke treatments derived from a persons own T-cells, genetically modified to recognise tumour cells, then returned to the individual patient to trigger their immune system to target certain cancers.

Once we receive the apheresis, its a continuous process until we get to the final product and its important that the quality organisation and manufacturing supply chain work closely as a team to get the product back to the hospital and to the patient.

The remit of the quality team also includes overseeing how the cell therapy product is delivered to patients, which requires the qualification of treating hospitals to undertake this process. This particular step is unique to cell therapies and ensures that treating hospitals understand how to receive and store the product at each stage, as well as maintaining the vital chain of custody throughout.

Its exciting because it allows us to collaborate more closely with our European colleagues at Gilead and Kite as well as healthcare professionals in the clinic. It helps us get the treatment to the patient, ensuring optimal quality.

According to Dudek, the leadership of Kites parent company Gilead has been central to allowing these highly specialised cancer therapies to reach patients in Europe.

The support from Gilead has been very important for our support to healthcare providers and to the centres that provide our product to patients.

We learn alongside colleagues, healthcare professionals and other stakeholders how to bring these innovative therapies to the people who need them.

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Leading from the front in cell therapy - - pharmaphorum

Neon Therapeutics Announces New Strategic Focus on Novel T Cell Programs – GlobeNewswire

Lead program, NEO-PTC-01, is a personalized neoantigen adoptive T cell therapy candidate to address refractory solid tumors

Corporate restructuring effected to focus resources while exploring strategic options

CAMBRIDGE, Mass., Nov. 20, 2019 (GLOBE NEWSWIRE) -- Neon Therapeutics, Inc. (Nasdaq: NTGN)today announced its new strategic focus on the development of its novel neoantigen-based T cell programs, in conjunction with a corporate restructuring. Neon will focus its efforts on the advancement of both personal and precision neoantigen-targeted T cell therapy candidates. Neons most advanced program is NEO-PTC-01, its personal neoantigen-targeted T cell therapy candidate consisting of multiple T cell populations targeting the most therapeutically relevant neoantigens from each patients tumor. Neon expects to file a Clinical Trial Application (CTA) in Europe by the end of 2019 to evaluate NEO-PTC-01 in patients with metastatic melanoma who are refractory to checkpoint inhibitors.

Prioritizing development of novel T cell therapies will leverage our years of expertise and learnings in pioneering neoantigen science, while positioning Neon to best deliver new therapies that could potentially improve patient outcomes and bring value to shareholders. The strategic restructuring will enable us to focus resources to execute on this vision. We acknowledge this decision impacts many talented employees who helped build Neon into a leader in neoantigen-based-therapies and we are grateful for their many contributions, said Hugh ODowd, Neons Chief Executive Officer.

NEO-PTC-01 leverages Neons neoantigen platforms, including RECON, its machine-learning bioinformatics platform, and NEO-STIM, its proprietary process to directly prime, activate and expand neoantigen-targeting T cells ex vivo. Neon believes that this approach will allow NEO-PTC-01, a non-engineered product that leverages peripheral blood mononuclear cells (PBMCs) as starting material, to specifically target each patient's individual tumor with T cells that can drive a robust and persistent anti-tumor response.

The initial clinical development of NEO-PTC-01 will be focused on demonstrating monotherapy activity targeting metastatic solid tumors that are refractory to checkpoint inhibitor therapy. Following the planned submission of a CTA by the end of 2019, the company plans to initiate a Phase 1 dose escalation clinical trial in second-line metastatic melanoma in collaboration with the Netherlands Cancer Institute. The second planned indication for NEO-PTC-01 is second-line metastatic ovarian cancer, with potential to expand to other solid tumor types and potential development in the United States.

NEO-PTC-01 has the potential to unlock the potency of cell therapy in solid tumors with several key advantages that overcome the challenges of other cell therapy approaches. In pre-clinical development and in several patient samples, we have demonstrated the ability to produce multiple enriched neoantigen-specific CD8+ and CD4+ T cell populations, including both memory and de novo T cell responses, that killed patient-specific tumors by targeting their tumor neoantigens. We believe that neoantigen targets will provide the tumor specificity required to develop safe, effective and durable T cell therapies for the treatment of solid tumors, said Richard Gaynor, M.D., Neons President of Research and Development.

Neon is also advancing a precision T cell therapy program targeting shared neoantigens in genetically defined patient populations. This process utilizes off-the-shelf targets with a patients own PBMCs to develop a novel cell-based immunotherapy enabling rapid deployment for each patient. The lead program from this approach, NEO-STC-01, is a T cell therapy candidate targeting shared RAS neoantigens initially in pancreatic cancer and is currently in preclinical development.

Corporate Restructuring

As part of this new strategic focus, Neon is reducing its workforce by approximately 24% of its current headcount. At this time, Neon will cease undertaking new additional spending commitments related to its cancer vaccine programs, NEO-PV-01 and NEO-SV-01. The company will continue to conduct follow-up from its NT-002 clinical trial of NEO-PV-01 in first-line patients with untreated advanced or metastatic non-small cell lung cancer, with plans to report clinical data from this trial in the third quarter of 2020. Neon also plans to cease future enrollment in its NT-003 trial in metastatic melanoma. Neon believes these actions will improve its potential to bring value to patients, employees and shareholders. As part of these cost reduction efforts, Neon intends to explore strategic options.

Neon expects that the restructuring and other cost-saving efforts will result in approximately $35 million in annualized cost savings. Neon estimates that it will incur approximately $1.5 million of pre-tax charges for severance and other costs related to the restructuring in 2019. With this restructuring, Neon now expects that its cash, cash equivalents and marketable securities will enable it to fund its operating expenses and capital expenditure requirements into the third quarter of 2020.

There can be no assurance that Neons restructuring and other cost-saving efforts will be sufficient to continue the development of NEO-PTC-01 or its other programs through completion of the planned or ongoing clinical trials, nor that Neons exploration of strategic alternatives will result in any transaction being entered into or consummated. Neon has not set a timetable for completion of this strategic review process and Neon does not intend to comment further unless or until its board of directors has approved a definitive course of action, the review process is concluded, or it determines that disclosure is required or appropriate.

About Neon Therapeutics

Neon Therapeuticsis a biotechnology company developing novel neoantigen-targeted T cell therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. Neon is using its neoantigen platform to develop both personal and precision neoantigen-targeted T cell therapy candidates. Neons most advanced program is NEO-PTC-01, its personal neoantigen-targeted T cell therapy candidate consisting of multiple T cell populations targeting the most therapeutically relevant neoantigens from each patients tumor. Neon expects to file a Clinical Trial Application (CTA) in Europe by the end of 2019 to evaluate NEO-PTC-01 in patients with metastatic melanoma who are refractory to checkpoint inhibitors.

For more information, please visitneontherapeutics.com.

Forward-Looking Statements

This press release contains forward-looking statements ofNeon Therapeutics, Inc.within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements may include, but may not be limited to, express or implied statements regarding our ability to obtain and maintain regulatory approval of our product candidates; the potential timing and advancement of our clinical trials; the potential timing and manner of data readouts from our ongoing and planned clinical trials; the design and potential efficacy of our therapeutic approaches; our plans to explore strategic alternatives; financial plans and projections, including its restructuring and other cost-saving efforts and impact on cash runway; and our ability to replicate results achieved in our preclinical studies or clinical trials in any future studies or trials. Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties related to the initiation, timing and conduct of studies and other development requirements for our product candidates; the risk that any one or more of our product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies and clinical trials may not be predictive of future results in connection with future studies or trials; the risk that Neons collaborations will not continue or will not be successful; risks related to our ability to protect and maintain our intellectual property position; risks related to our capital requirements, use of capital and unexpected expenditures, including our ability to manage operating expenses or obtain funding to support planned business activities or to explore and establish strategic alternative transactions; risks related to our ability to attract and retain personnel; and risks related to the ability of our licensors to protect and maintain their intellectual property position. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Neons actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Neons most recent Quarterly Report on Form 10-Q, as filed with theSecurities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Neons other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, and Neon undertakes no duty to update this information unless required by law.

Investor Contact:Will OConnor, Stern Investor Relationswill@sternir.com212-362-1200

Media Contact:Stephanie Simon,Ten Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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Neon Therapeutics Announces New Strategic Focus on Novel T Cell Programs - GlobeNewswire

New cell therapy player launches with $57M from marquee investors to go after ‘definitive’ tumor targets – Endpoints News

A crew of Amgen and Kite vets is publicly throwing their new startup into the cell therapy hat though they are saying little outside of the basics and $57 million in Series A cash.

The Column Group, Vida Ventures, Samsara BioCapital and Nextech Invest are backing A2 Biotherapeutics quest to find new ways of engaging immune cells in selectively attacking cancer. To do so, the biotech is working with two target classes: peptide MHC targets and targets that are irreversibly lost in tumor cells. The former builds on an increasingly popular strategy of locating neoantigens for T cells to home in on, while the latter is inspired by a mechanism used by natural killer cells.

A2 Biotherapeutics has potent, highly selective binders that we combine into molecular constructs to integrate multiple signals and potentially provide a large therapeutic window, said CSO Alexander Kamb, who co-founded the biotech after a stint as SVP of research at Amgen. They could be antibody and T-cell receptor fragments, according to its statement.

Scott Foraker, his former colleague at the Big Biotech, recently signed on as president and CEO.

Working out of Agoura Hills, California, their team of 40 expects to usher the first product candidate into the clinic next year. Its four programs in development span cancer testis antigen for solid tumors; targets lost antigens for solid tumors; neoantigens for head and neck cancer; and neoantigens for pancreatic, colorectal and lung cancer.

It also plans on constructing a manufacturing facility arguably the most important core competency that differentiates one cell therapy player from another in 2020 with the mindset of ultimately producing its own commercial supply. The site would support an autologous approach, though A2 Bio didnt specify what kind of cells it would use as the source.

Their lead in technical ops is Michelle Kreke, who was credited for leading chemistry, manufacturing and controls for the pioneering CAR-T Yescarta.

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New cell therapy player launches with $57M from marquee investors to go after 'definitive' tumor targets - Endpoints News

Zelluna Immunotherapy and Glycostem Therapeutics announce partnership to develop allogeneic TCR-NK therapies – BioSpace

Oslo and Oss, Norway and the Netherlands, November 19, 2019 - Zelluna Immunotherapy, a biopharma company developing T-cell receptor (TCR) guided adoptive cell therapy products for the treatment of multiple solid cancers, and Glycostem Therapeutics BV, a clinical stage and leading Natural Killer (NK) cell manufacturing company, today announce that they have entered into a development, license and supply agreement. This collaboration will focus on the development and manufacture of allogeneic TCR guided NK-cell therapies (TCR-NKs) for the treatment of patients with cancer.

This partnership between Zelluna and Glycostem represents a critical milestone in Zellunas aim to transform cell therapy treatments by enabling more patients to be treated effectively, rapidly and safely. It is the industrys first commercial development partnership of TCR-NK cells for treating cancer aiming at the rapid translation of our proprietary TCR-NK approach, said Miguel Forte, CEO, Zelluna. Zelluna selected Glycostem as a partner for the delivery of TCR-NK therapies for their expertise in NK-cell science, GMP manufacturing and clinical testing of NK-cell products. The development of TCR-NK programs will run in parallel to Zellunas autologous TCR-T development programs, including its lead autologous T-cell product entering the clinic next year. Zellunas vision of cell therapies continues to be autologous and allogeneic products delivering much needed patient benefit.

Currently, NK-cell based therapies such as CAR-NKs are receiving considerable interest from the wider cell therapy community. Through the partnership with Glycostem, Zelluna is enabling its proprietary TCR-NK approach with the potential to target a broader repertoire of tumor associated antigens by targeting NK-cells with TCRs for treatment of patients with solid cancers.

Zelluna will lead the development and commercialization of TCR-NK products with its TCRs and TCR development competence. Glycostem will contribute by manufacturing clinical grade umbilical cord derived NK-cells, NK-cell expertise, product process development, and clinical and commercial supply. Zelluna and Glycostem will be able to manufacture upfront a large number of patient doses to store and ship to clinical sites upon demand in an off-the-shelf manner.

We are very proud to be collaborating with Zelluna, a transformative TCR cell therapy company to jointly develop allogeneic TCR-NK products for the benefit of hard to treat patients. This partnership will enable Glycostems NK-cells to deliver increased efficacy against solid tumors in a TCR-NK product using Zellunas TCR targeting mechanism, said Troels Jordansen, CEO, Glycostem. This is further confirmation of the significant interest in NK-cell products and an external validation of Glycostems abilities which supports our commercial and scientific ambitions; taking cellular immunotherapy to the next level.

About Zelluna Immunotherapy

Zelluna Immunotherapy is a company developing transformative T cell receptor (TCR) based cellular immunotherapies for the treatment of solid cancers. The company is developing a unique portfolio of tumor specific TCRs that target the MHC class II pathway to broadly engage the host immune system to provide a safe, efficient and durable clinical response. The company is developing therapies based on the effector functions of autologous T cells (TCR-T) and allogeneic NK cells (TCR-NK). Both of these platforms have their unique advantages and provides the opportunity to attack cancers from multiple angles and improve access to these advanced therapies to a large patient population. For more information, please visit http://www.zelluna.com.

About Glycostem

Netherlands-based Glycostem Therapeutics BV, a clinical stage biotech company, develops allogeneic cellular immunotherapy to treat several types of cancer. By harnessing the power of stem cell-derived Natural Killer (NK) cells, Glycostems products are a safe alternative to CAR-T-cells. Glycostems lead product, oNKord(R), is manufactured from allogeneic raw material and is available off-the shelf. Thanks to its nine patent families, longstanding technical expertise and resources, as well as Orphan Drug Designation, Glycostem has secured a leadership position in the global NK-cell market. oNKord(R) is produced in a closed system in Glycostems state-of-the-art and GMP (Good Manufacturing Practice) licensed production facility in the Netherlands, from which it can be distributed globally. The production technology includes ex vivo generation of high numbers of NK-cells with a high degree of purity for clinical applications. oNKord(R) successfully passed phase I clinical trial (elderly and frail AML - Acute Myeloid Leukemia - patients), providing solid safety data and strong indication of clinical activity, including response on MRD (Minimal Residual Disease). Results indicate that oNKord(R) may be safely infused in AML patients. Glycostem is furthermore developing a range of CAR-NK products in-house and in cooperation with amongst other global partners MolMed SPA (BIT:MLM).

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Zelluna Immunotherapy and Glycostem Therapeutics announce partnership to develop allogeneic TCR-NK therapies - BioSpace