Category Archives: Cell Therapy

Cell therapy R&D is hot, and Charles River Labs $CRL knows it. The CRO is paying $380 million to acquire HemaCare, a company that provides material and services for companies developing off-the-shelf and personalized cell therapies. Charles River says its developing a turnkey service for cell therapy developers and manufacturers.

Cell and gene therapies are important new modalities, with an estimated 10 to 20 new product approvals per year within five years, notes Charles River CEO James Foster.

At the beginning of the year, Axsome Therapeutics rolled out positive data from a mid-stage study testing its lead CNS drug for patients with major depressive disorder (MDD), causing their stock $AXSM to leap. Today, the company is celebrating another win, as AXS-05 met the primary endpoint in their Phase III GEMINI study. The drug rapidly and significantly improved symptoms of depression compared to placebo at Week 6 meaning reductions from baseline of 16.6 points for AXS-05 and 11.9 for placebo, based on the Montgomery-sberg Depression Rating Scale (MADRS).

Immuneering, which has spent over a decade helping its partners (including Teva and J&J) understand how their existing drugs tend to work using gene expression data has moved into the arena of drug development with $17 million in backing from investors. The Cambridge, MA-based companys lead experimental drug is being developed for cancer cachexia.

The industry often looks at drugs as a bullet, something thats taking out one very precise target, noted co-founder and chief Ben Zeskind in an interview with Endpoints News. And we actually, through studying all these drugs, we have come to view the most successful medicine kind of differently not so much as bullets, but more as signal inducersand actually, we think the best metaphor is the noise-canceling headset.

Last October Alexion Pharmaceuticals, shopping for targets to fatten its pipeline, forked over $22 million upfront to co-develop two preclinical RNAi therapies owned by Dicerna Pharmaceuticals, the company is now dishing out an additional $20 million ($10 million each) to kick off discovery efforts against two more targets within the complement pathway.

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Charles River Labs forks over $380M cash for cell therapy player; Axsome soars on PhIII depression readout - Endpoints News

In a recent study published by Prophecy Market Insights, titled, Global Cell Therapy Manufacturing Market Research Report, analysts offers an in-depth analysis of global Cell Therapy Manufacturing market. The study analyses the various aspect of the market by studying its historic and forecast data. The research report provides Porters five force model, SWOT analysis, and PESTEL analysis of the Cell Therapy Manufacturing market. The different areas covered in the report are Cell Therapy Manufacturing market size, drivers and restrains, segment analysis, geographic outlook, major manufacturers in the market, and competitive landscape.

Key Players of Cell Therapy Manufacturing Market:

harmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

Download Sample Copy of This Report @ https://prophecymarketinsights.com/market_insight/Insight/request-sample/21

The research report, Cell Therapy Manufacturing Market presents an unbiased approach at understanding the market trends and dynamics. Analysts have studied the historical data pertaining to the market and compared it to the current market trends to paint an object picture of the markets trajectory. The report includes SWOT analysis and Porters five forces analysis to give the readers an in-depth assessment of the various factors likely to drive and restrain the overall market.

Market Segmentation:

Request PDF catalogue for this report @ https://prophecymarketinsights.com/market_insight/Insight/request-pdf/21

Table of Contents

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the global Cell Therapy Manufacturing market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the global Cell Therapy Manufacturing market is analyzed, taking into consideration price, revenue, sales, and market share by company, market concentration rate, competitive situations and trends, expansion, merger and acquisition, and market shares of top 5 and 10 companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the global Cell Therapy Manufacturing market as well as some useful information on their business. It talks about the gross margin, price, revenue, products and their specifications, applications, competitors, manufacturing base, and the main business of players operating in the global Cell Therapy Manufacturing market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Cell Therapy Manufacturing market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This part of the research study shows how different application segments contribute to the global Cell Therapy Manufacturing market.

Market Forecast: Here, the report offers complete forecast of the global Cell Therapy Manufacturing market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Upstream Raw Materials: The report provides analysis of key raw materials used in the global Cell Therapy Manufacturing market, manufacturing cost structure, and the industrial chain.

Marketing Strategy Analysis and Distributors: This section offers analysis of marketing channel development trends, indirect marketing, and direct marketing followed by a broad discussion on distributors and downstream customers in the global Cell Therapy Manufacturing market.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

Appendix: Here, we have provided a disclaimer, our data sources, data triangulation, market breakdown, research programs and design, and our research approach.

For More Info: https://prophecymarketinsights.com/market_insight/Global-Cell-Therapy-Manufacturing-Market-21

Contact Us:

Mr. Alex (Sales Manager)

Prophecy Market Insights

Phone: +1 860 531 2701

Email: [emailprotected]

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Cell Therapy Manufacturing Market Insights, New Project Investment and Potential Growth Scope - Info Street Wire

NANJING, China, Dec. 16, 2019 /PRNewswire/ -- GenScript Biotech Corp., one of the leadingbiotechnology companies inChina, today announcedits inaugural GenScript Biotech Global Forum on Jan. 14 in San Francisco, coinciding with the JP Morgan Healthcare Conference week. The Forum, exploring the theme "Cell and Gene Therapy and the Booming China Market," will feature gene and cell therapy leaders in industry, academia and the investment community and is expected to draw several hundred attendees.

"Advancements in cell and gene therapy have attracted global attention in recent years, as the promise of bringing life-changing treatments to cancer patients and others comes closer to reality," said Frank Zhang, PhD., founder and CEO of GenScript. "GenScript's Global Forum aims to foster closer collaborations among scientists, regulators, and industry, not just in the booming China market but around the globe. We hope that by working together we can advance the industry and accelerate drug development."

GenScript's Global Forum, will take place from 1:30 p.m. to 5:30 p.m. at the Grand Hyatt San Francisco. Highlights of the agenda include:

For more information about the Forum and to register for the event please visit hereor https://www.genscript.com/biotech-global-forum-2020.html.

About GenScript Biotechnology

GenScript Biotech Corporation (Stock Code: 1548.HK) is a global biotechnology group. GenScript's businesses encompass four major categories based on its leading gene synthesis technology, including operation as a Life Science CRO, enzyme and synthetic biology products, biologics development and manufacturing, as well as cell therapy.

Founded in 2002 and listed on the Hong Kong Stock Exchange in 2015, GenScript has an established global presence across Greater China, North America, the EU, and Asia Pacific. Today, over 300,000 customers from over 160 countries and regions around the world have used GenScript's premier, convenient, and reliable products and services.

GenScript currently has more than 2900 employees globally, 34% of whom hold master's and/or Ph.D. degrees. In addition, GenScript has a number of leading commercial technologies, including more than 100 patents and over 270 patent applications. As of June 2019, GenScript's products and services have been cited by 40,300 scientific papers worldwide.

GenScript is committed to striving towards its vision of being the most reliable biotech company in the world to make humans and nature healthier through biotechnology.

For more information, please visit https://www.genscript.com/

Contact:

Corporate:Fiona CheCorporate Communication Manager, GenScript+86 -025-58897288-6321Fiona.che@genscript.com

Media Susan ThomasPrincipal, Endpoint Communications(619) 540-9195susan@endpointcommunications.net

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SOURCE GenScript Biotech Corp.

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GenScript Biotech to Host Global Forum on Cell and Gene Therapy and the Booming China Market During JPM Week - P&T Community

Mike Berthiaume, 31, a Franklin firefighter, was transferring a patient at the hospital when he suddenly felt pain in his knee.

The injury would cause him to miss work, his one true passion, for the next year.

I basically just twisted my knee the wrong way, it was like a compression and twisting injury, and from there it just escalated, said Berthiaume.

He was injured in March 2018 and quickly learned he would need surgery, which would take him out of his beloved job as a firefighter for a year.

I enjoy working, so I think that was the hardest part, it was looking at the rehab being a year-plus, it was hard, said Berthiaume. I had one thought in my mind to get better.

Berthiaumes surgeon, Dr. Kai Mithoefer of Boston Sports and Shoulder, recommended an innovative treatment called MACI that uses a patients own cells to repair knee cartilage damage instead of using traditional metal or plastic devices.

With little hesitation, Berthiaume agreed and a sample of his cartilage cells was sent off to the MACI lab in Cambridge, the only lab in the country that manufactures and produces the MACI product by embedding cells on a special collagen membrane, which is implanted into the knee.

Mithoefer said Berthiaumes injury was large and severe, making him a good candidate for the technology. For larger defects that are not involving the bone underneath MACI, I think, is the perfect technology because it has really good outcomes.

Inside the ultra-controlled, sealed and pressurized MACI lab, workers in full white medical gowns complete the meticulous process of growing cells to produce the final product that is shipped back to surgeons.

The MACI lab is extremely clean, but not sterile, as it would kill the cells. The full process of creating MACI, whose full name is autologous cultured chondrocytes on porcine collagen membrane, takes about four to six weeks.

Chris Parrish, vice president of manufacturing at Vericel Corporation, which markets MACI, said, Its kind of like a two-part process receive the biopsy, grow your cells and freeze them down, receive your order, and the second part of the process becomes thaw some cells and now, performing intuitive process to produce MACI.

The final product is placed on a membrane so that the new, repaired cells will bind when transported into the patients knee.

Jon Hopper, chief medical officer at Vericel, said patients essentially have a natural cartilage surface over where the defect is and thats very helpful in terms of normal function for everyday people.

The MACI lab sees thousands of cases a year for its customized cell therapy that provides long-lasting pain relief and improved function for patients.

Berthiuame got MACI treatment in November of last year and returned to work just last month with little to no pain after rigorous rehabilitation and physical therapy.

Mithoefer said Berthiuame hit all the milestones perfectly, adding, He was motivated, I think he likes his job a lot, I think he was pretty motivated to go back and that kept him on track.

The rest is here:
Knee replacement made from own cells allows Franklin firefighter to get back to work - Boston Herald

It was a decade that began with the electrifying results of a clinical trial for a revolutionary new cancer therapy and ended with a Nobel Prize in Medicine for very different cancer-related research. In between those dramatic bookends, the 2010s were packed with progress, with discoveries leading to the FDAs 2017 approval of the first CAR T-cell therapy. Additional approvals would follow.

The 2010s started with clinical trial results centered on the use of checkpoint inhibitors, drugs that unleash a powerful immune system attack on cancer cells. The results founded on decades of research by scientists like Dana-Farbers Gordon Freeman, PhD helped usher in a new era of cancer immunotherapy.

Checkpoint blockersare transformational, Laurie H. Glimcher, MD, president and CEO of Dana-Farber and a prominent immunologist, said back in 2017, but they are only the tip of a proverbial immunotherapy iceberg.

On the other side of the last 10 years in cancer research was the Nobel Prize in Medicine, shared by Dana-Farbers William G. Kaelin, Jr., MD, for discoveries into the mechanism that enables cells to sense and adapt to changes in oxygen abundance research that has already led to exciting new treatments for cardiovascular disease and cancer.

As cancer research pioneer and Dana-Farber founder Sidney Farber, MD, said back in 1965, I have never accepted the incurability of cancer. And I have remained hopeful, not because of wishful thinking thats not progress but because of the factual evidence of progress. There is no such thing as a hopeless case.

Aside from these prominent discoveries, what were the most significant advances in cancer research and treatment? Heres what scientists and clinicians from around Dana-Farber said.

William Hahn, MD, PhD, Chief Research Strategy Officer

The sequencing of human cancer genomes over the past decade has demystified the genetics of cancer. We now have a blueprint of cancer genes in every type of cancer and information about the frequency and type of mutations that occur. This has revealed new genes and pathways important for cancer development and in some cases has already led to new approved cancer therapies.

In addition, geneticallysequencing tumor tissue samples guides the therapeutic agents selected for asubset of cancer patients. This tailored approach, termed precision medicine,selects patients most likely to respond and spares those that are unlikely torespond from untoward side effects. Recent discoveries that its possible tosequence DNA in the blood to detect cancers provide hope that this approach canbe used to identify cancers earlier and follow the response to therapy.

Through the study of rare cancers, we have identified mutations in genes that regulate the epigenome, the cells machinery for activating and deactivating genes. These studies have revealed that these same pathways are dysregulated in many common cancers and play key roles in cancer pathogenesis and resistance to therapy.

Sapna Syngal, MD, MPH, Director of Research, Center for Cancer Genetics and Prevention

The realization that upto 10% of many solid tumors have an inherited genetic basis provides us with agreat opportunity for precision prevention and early interception.

Scott Armstrong, MD, PhD, President, Dana-Farber/Boston Childrens Cancer and Blood Disorders Center

Were now able to identify several premalignant states that significantly increase peoples risk of developing certain hematologic cancers. Individuals with clonal hematopoiesis of indeterminate potential (CHIP), for example, have certain genetic mutations in their blood-forming stem cells that are associated with leukemia.

People with CHIP dont have symptoms of disease, but their risk of developing a blood cancer such as leukemia is 10 times higher than average and their risk of cardiovascular disease is elevated as well. Being able to identify high-risk individuals means we can begin to think about early-intervention strategies to prevent these cancers from developing an active area of research.

Ursula Matulonis, MD, Chief, Division of Gynecologic Oncology

The introduction of drugs known as PARP inhibitors has had a major impact on the treatment of ovarian cancer, and now they are showing effectiveness against other cancers including breast and pancreatic. PARP inhibitors work by blocking one of the key routes by which cells repair damaged DNA and are especially effective in cancers with existing DNA-repair deficiencies such as those harboring BRCA mutations.

Also, better understanding of the genomics of gynecologic cancers the set of genetic mutations within the cancer cells is transforming the way we approach treatment and prevention. Its now widely recognized that women with ovarian cancer, regardless of age, histology type, or the stage at which their cancer is diagnosed, should undergo genetic testing. A percentage of them will have a predisposing mutation in one of the BRCA genes. Women with newly diagnosed endometrial cancer should have their cancer tested for mismatch repair deficiencies, which interfere with the proper copying of DNA during cell division.

The presence of these genetic features not only influences the treatment patients receive, but, because they can be inherited, often enable us to identify family members who are also at risk and can benefit from more intensive monitoring or preventive treatment.

Richard Stone, MD, Program Director in Adult Leukemia

Morethan 10 drugs have been approved for acute leukemia in the past three years,whereas there had been very few new agents in the previous 25 years.

DNA sequencing of patients leukemia cells to identify mutations is being used to help guide treatment decisions.

Eric Winer, MD, Senior Vice President for Medical Affairs and Faculty Development; Chief, Division of Breast Oncology

In the treatment of breast cancer, we now know for a certainty that one size does not fit all. This allows us to personalize therapy to a much greater extent than ever before. In some patients, this means we can treat them with less-intensive therapy and still obtain excellent results. Others may require more extensive therapy or benefit from a different therapeutic approach. For all patients, this means better, more effective care, fewer side effects, and, for many, a longer life.

Kimberly Stegmaier, MD, Vice Chair of Pediatric Oncology Research

There have been multiple approvals of new targeted drugs in adult acute myeloid leukemia (AML) in the past two years, as well as TRK inhibitor approval for adult and pediatric patients with TRK fusion-positive cancers.

Bruce Johnson, MD, Chief Clinical Research Officer

Addingthe kinase inhibitor midostaurin to standard chemotherapy significantlyprolonged overall and event-free survival in patients with acute myeloidleukemia whose cancer cells have a FLT3 mutation.

Enzalutamide,an androgen receptor inhibitor, was associated with significantly longer progression-freeand overall survival than standard care in men with metastatic,hormone-sensitive prostate cancer receiving testosterone suppression.

Dana-Farberscientists reported on the feasibility, safety, and immunogenicity of apersonalized cancer vaccine that caused immune T cells to recognizecancer-related neoantigens on tumor cells. These results have promptedfurther development of a neoantigen vaccine approach.

Nadine Jackson McCleary, MD, MPH, Gastrointestinal Oncologist

Weve made strides in ensuring that evidence from cancer research studies actually makes its way into clinical practice. For too long, research findings often seemed to remain in academia without being translated to clinical medicine.

Professional and patient advocacy organizations have undertaken a variety of steps to not only implement these advances in the clinical setting but also to make sure theyre sustainable. For example, organizations such as the American Society of Clinical Oncology (ASCO) and cooperative research groups regularly inform the broader public about research results and work at the state and federal level on behalf of patients. The development of implementation science is having a sizable impact on clinical practice.

Were also making progress in improving equity in cancer care delivery. Where equity issues have traditionally involved issues such as race, gender, and socioeconomic status, were broadening the focus to include considerations of gender identity, patient location (where patients receive treatment may affect their outcome), and treatment of the very youngest and oldest patients. These efforts will help ensure that advances in cancer medicine reach all populations.

Toni Choueiri, MD, Director of the Lank Center for Genitourinary Oncology

An important ongoing approach is liquid biopsies obtaining tumor-related DNA in the blood as a means of early cancer detection. Liquid biopsies also have the potential to detect minimal residual disease in the body following surgery to predict the risk of relapse.

Rameen Beroukhim, MD, PhD, Physician-Scientist in Neuro-Oncology

This decade is the first in which targeting collateral vulnerabilities in cancer cells has become an important strategy. Most efforts at treating cancer focus treatment on the genetic changes within cells that cause them to become cancer. But along the way, many genes that have nothing to do with cancer are also affected, and scientists have found that targeting these genes on which the cancer cells depend can be an effective way of attacking cancer. Immunotherapy, for example, detects cancer cells based on this collateral damage.

I predict that targeting collateral vulnerabilities will become increasingly important in future decades. Another recent strategy is based on the emerging technology of protein degradation, which removes cancer-related proteins from cells rather than simply binding to these proteins to inhibit their activity.

Link:
The Most Significant Cancer Research Advances of the 2010s - Dana-Farber Cancer Institute

WASHINGTON, Dec. 20, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM), the international advocacy organization for the cell and gene therapy and broader regenerative medicine sector, today released the initial slate of presenting companies at the 2020 Cell & Gene Meeting on the Mediterranean. The event will be held April 15-17, 2020 in Barcelona, Spain.

The event, modeled after ARMs highly successful Cell & Gene Meeting on the Mesa, is expected to attract more than 500 attendees, including senior executives from leading cell therapy, gene therapy, and tissue engineering companies worldwide, large pharma and biotech representatives, institutional investors, academic research institutions, patient foundations, disease philanthropies, and members of the life science media community.

The second annual Cell & Gene Meeting on the Mediterranean will feature presentations by 50+ leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies.

The initial slate of 2020 presenting companies includes: Adaptimmune, AGTC, Ambys Medicines, American Gene Technologies, AskBio, Aspect Biosystems, Atara, Autolus Therapeutics, Avectas, AVROBIO, Axovant Gene Therapies, bluebird bio, Bone Therapeutics, Caribou Biosciences, Celavie Biosciences, Cellatoz Therapeutics, CEVEC, Cynata Therapeutics, Flexion Therapeutics, Fraunhofer IZI, GenSight Biologics, Healios, Iovance Biotherapeutics, Kiadis Pharma, Kytopen, LogicBio Therapeutics, MeiraGTx, Minerva Biotechnologies, MolMed, Novadip Biosciences, Orchard Therapeutics, Oxford Biomedica, PDC*line Pharma, Precision BioSciences, Promethera Biosciences, PTC Therapeutics, Recombinetics, REGENXBIO, ReNeuron, Rexgenero, Sangamo, SmartPharm Therapeutics, Standards Coordinating Body for Regenerative Medicine, Theradaptive, ThermoGenesis, Tmunity Therapeutics, Ultragenyx Pharmaceutical, VERIGRAFT, and Zelluna Immunotherapy.

Additional event details will be updated regularly on the conference website http://www.meetingonthemed.com.

Registration is complimentary for investors and credentialed members of the media. To learn more and to register, please visitwww.meetingonthemed.com. For members of the media interested in attending, please contact Kaitlyn Donaldson Dupont at kdonaldson@alliancerm.org.

For interested organizations looking to increase exposure to this fields top decision-makers via sponsorship, please contact Kelly McWhinney at kmcwhinney@alliancerm.org for additional information.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

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The Alliance for Regenerative Medicine Releases Initial Slate of Presenting Companies at the 2020 Cell & Gene Meeting on the Mediterranean -...

The global Stem Cell Therapy market is forecasted to reach a market value of ~US$ XX Mn/Bn by the end of 2029 registering a CAGR growth of around XX% during the forecast period (2019-2029). The recent market report provides a detailed analysis of the current structure of the Stem Cell Therapy market along with the estimated trajectory of the market over the course of the stipulated timeframe.

The report provides an in-depth assessment of the numerous factors that are anticipated to impact the market dynamics with utmost precision and accuracy. The SWOT and Porters Five Forces Analysis provides a clear picture about the current operations of the various market players operating in the global Stem Cell Therapy market.

Enticing discounts for new Stem Cell Therapy market players! Offer expires soon!

Request For Discount On This Report @ https://www.transparencymarketresearch.co/sample/sample.php?flag=D&rep_id=28262

Segmentation analysis of Stem Cell Therapy Market

Market segments and sub-segments

The regional analysis covers:

The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points in the industrys value chain.

A separate analysis of prevailing trends in the parent market, macro- and micro-economic indicators, and regulations and mandates is included under the purview of the study. By doing so, the report projects the attractiveness of each major segment over the forecast period.

Highlights of the report:

Note:Although care has been taken to maintain the highest levels of accuracy in TMRs reports, recent market/vendor-specific changes may take time to reflect in the analysis.

The report ponders over the market scenario in various geographies and highlights the major opportunities, trends, and challenges faced by market players in each region. An in-depth country wise analysis of each major region provides readers a deep understanding of the regional aspects of the market including, the market share, pricing analysis, revenue growth, and more.

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The market report on the Stem Cell Therapy market addresses some important questions such as:

Crucial data enclosed in the Stem Cell Therapy Market report:

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This post was originally published on Market Reports Observer

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Stem Cell Therapy Market Revenue and Value Chain 2017 2025 - Market Reports Observer

ZURICH (Reuters) - Swiss drugmaker Novartiss (NOVN.S) new $90 million cell and gene therapy factory in northern Switzerland is on track to begin commercial production of its cell therapy Kymriah for cancer in 2020.

The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann

The new factory, expected to employ 450 people, will allow the drugmaker to make its Kymriah treatment for European patients without first having to fly their immune cells across the Atlantic Ocean.

The Swiss factory, as well as a separate French site also being expanded, are central to Novartiss plans to transform Kymriah from a modest $250 million-per-year seller into a $1 billion blockbuster as European demand rises.

We have already started to produce clinical batches at both sites and are on track to begin producing commercial product in 2020, Novartis said.

Basel-based Novartis sees cell and gene therapies eventually contributing about 15% of its revenue.[nL8N2874FU][nL5N25V4JJ]

T cells currently harvested from Europeans getting the $400,000-per-patient Kymriah blood cancer therapy must now be sent to a U.S. laboratory for re-engineering. Analysts have called the lack of manufacturing capacity a major bottleneck.

Novartis is investing some $500 million in new facilities around the world to address production bottlenecks and drug companies including Novartis, Pfizer (PFE.N) and others, have plans to spend $2 billion building out gene and cell therapy manufacturing after rapid expansion into these treatments.

Kymriahs global rollout in 20 countries, plus Novartiss efforts to expand the therapys indications, make adding commercial production a priority, including in China and Japan. [nL8N1WD64W]

Kymriahs 2018 approval was hailed as a breakthrough as a last-ditch treatment for acute lymphoblastic leukemia and diffuse large B-cell lymphoma. T-cells are extracted from patients, modified to attack their cancer, then re-infused in the hope they work where other treatments failed.

But its complex production, tailor-made for each patient, poses logistical challenges compared to off-the-shelf treatments, especially in Europe where Novartis lacked commercial manufacturing.

The key factor in bringing local manufacturing to each region is to make Kymriah available as quickly as possible, Novartis said.

(This story removes reference to Zolgensma, which will not be made at new Swiss factory in 2020.)

Reporting by John Miller; Editing by Elaine Hardcastle

Continued here:
Novartis's $90 million Swiss factory to help solve cell therapy bottleneck - Reuters

A first-of-kind multi-antigen targeted off-the-shelf chimeric antigen receptor (CAR)-natural killer (NK) cell with engineered persistence has the potential to be a readily available treatment option for patients, Robert A. Brodsky, MD, said in a preview of the 61st American Society of Hematology (ASH) Annual Meeting & Exposition.

As most of you are well aware, CAR T-cell (therapy has) captured the imagination of physician scientists and patients alike, mainly for their incredible efficacy in treating B-cell malignancies like acute lymphocytic leukemia and non-Hodgkin lymphomas, said Brodsky, who serves as secretary of ASH and is also the director of the division of hematology at Johns Hopkins Medicine.

However, he added, this treatment option does not come without its drawbacks: namely, time, expenses, toxicity.

Only about two-thirds of patients enrolled in CAR T-cell trials will actually see infusion because often the disease will progress during the time it takes to make a successful product, Brodsky said.

Therefore, there is a need to develop a more timely infusion that can be associated with lower costs, and hopefully, less toxicity.

At the upcoming ASH Annual Meeting & Exposition, being held from December 7-10 in Orlando, Florida, Jode P. Goodridge, PhD, will present on his teams proof-of-concept study of induced pluripotent stem cell (iPSC)-derived effector cells.

iPSC-derived effector cells offer distinct advantages for immune therapy over existing patient- or donor- derived platforms, both in terms of scalable manufacturing from a renewable starting cellular material and precision genetic engineering that is performed at the single-cell level, the researchers wrote in their abstract. iPSC derived natural killer (iNK) cells offer the further advantage of innate reactivity to stress ligands and MHC downregulation and the potential to recruit downstream adaptive responses.

The candidate, called FT596, is consistently manufactured from a master iPSC line engineered to uniformly express an NK cell-calibrated CD19-targeting CAR, an enhanced functioning high-affinity, non-cleavable CD16, and a recombinant fusion of IL-15 and IL-15 receptor alpha for cytokine-autonomous persistence, according to the abstract.

What the authors here did is take advantage of the use of induced pluripotent stem cells and differentiated them to natural killer cells. Natural killer cells are not T cells but they are another form of lymphocytes that can be very effective in killing cancer cells. What they did is they engineered these pluripotent stem cells to target B cells, and they are specifically targeting the CD19 antigen on B cells and showing that these are very effective in cell line models and animal models, explained Brodsky.

However, of note, this product has not been tested in humans yet.

The big advance here is that this offers the potential of having a readily available source of basically CAR-NK cells that wouldnt need time to grow them up before they would be infused, Brodsky concluded.

Goodridge JP, Mahmood S, Zhu H, et al. FT596: Translation of First-of-Kind Multi-Antigen Targeted Off-the-Shelf CAR-NK Cell with Engineered Persistence for the Treatment of B Cell Malignancies. Presented at: 61st ASH Annual Meeting & Exposition Meeting preview; to be presented December 7, 2019; Orlando, Fla. Abstract 301.

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Proof-of-Concept Study of CAR-NK Cell Therapy with Engineered Persistence Shows Potential - Cancer Network

Vertex Pharmaceuticals is preparing to grow even biggeranother 256,000 square feet bigger, to be exact.

The drugmaker is in advanced talks to lease a building in Innovation Square, a research campus in Bostons Raymond Flynn Marine Industrial Park, The Boston Globe reported.

The target is the entire second phase of the new R&D hub that developer Related Beal is building on the South Boston waterfront. Its close to Vertexs existing 1.1 million-square-feet Fan Pier headquarters and would serve as a research and manufacturing facility for gene and cell therapies, according to the newspaper.

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Vertex scoured the greater Boston region for a new foothold, including sites in Cambridge, Waltham and Watertown, but picked the Innovation Square because its one of the most advanced projects in the neighborhood, on track to open in 2021, Vertex CEO Jeffrey Leiden reportedly said.

Expansion at the Innovation Square comes as the biotech giant diversifies beyond its fundamental cystic fibrosis business and into the burgeoning gene and cell therapy arena.

The question became how are we going to grow those programs if were running out of space at Fan Pier? said Leiden, as quoted by the Globe. The answer is a new building.

Leiden is transitioning to executive chairman, handing the baton to Chief Medical Officer Reshma Kewalramani. But before he moves up, a blueprint for Vertexs future growth has been laid out.

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In June, Vertex put down $245 million upfront to acquire Exonics and its gene editing technology, which uses CRISPR to repair dystrophin, the protein missing in patients with Duchenne muscular dystrophy (DMD). At the same time, it shelled out $175 million upfront to deepen its ties with CRISPR Therapeutics, also for using CRISPR-Cas9 to develop DMD and myotonic dystrophy Type 1 therapies.

The first project coming out of the CRISPR-Vertex partnership has just shown promise. CTX001, a CRISPR-based therapy for severe blood disorders marked by abnormal hemoglobin, helped a beta thalassemia patient live without transfusions for nine months, and a sickle cell patient was free of the painful vaso-occlusive crises after four months, the pair unveiled last week.

Vertex also agreed to pay $950 million to snatch up Semma Therapeutics and its stem cell treatment for Type 1 diabetes.

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The new building Vertex plans to lease will house 300 to 400 people, including employees from Exonics and Semma, as well as new hires, Leiden said, according to the Globe. Besides the lab and office space at its Fan Pier HQ, Vertex also has a lease for about 100,000 square feet of space in the Marine Industrial Park for certain logistical and laboratory operations and manufacturing equipment, the companys annual securities filing shows.

On the companys third-quarter earnings call in October, Leiden said the company will continue to do deals on early-stage assets, especially bolt-on deals to furtherits gene editing strategy.

Meanwhile, the cystic fibrosis franchise will continue to provideVertexs revenue backbone for some time. Last month, the company wonFDA approval for Trikafta, a triple combo designed to treat cystic fibrosis patients with a mutated delF508 gene, which is found in 90% of the U.S. cystic fibrosis population.

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Vertex plans major Boston expansion to support gene, cell therapy ambitions - FiercePharma