Category Archives: Cell Therapy

As a regulatory lawyer long-steeped in the world of stem cells and regenerative medicine, Im bound to latch onto the title of a recent Nature piece with particular interest. Cells or drugs? Its a great question from a regulatory point of view.

You cant place any medicine on the market, whether its a small molecule, a gene, or the fanciest of engineered cells, without a marketing authorisation. However, getting an MA can be pretty demanding, and especially when it comes to an advanced therapy product (ATMP) such as a population of cells. Avoiding cells has some obvious attractions.

The article reminds us that cell therapy is less about cells than their fate. Of course, manufacturers of such cells are obsessively concerned about their fate from a product point of view.

However, the bigger question is about what happens to the cells once theyre implanted. At least as important, what happens to the cells that were already at the implant site? The more we learn about how damaged tissues behave and what implanted cells do in their new home, the more it comes down to an understanding of the conversation between cells that determines their phenotype. If we assume that the conversation is mediated by molecules, then we only need cells because we dont yet know what those molecules are or because cells happen to be the best delivery device for them. If the cellular conversation is about growing new cells to replace those lost to ischaemia, for example, the proliferation of any cardiomyocytes surviving implantation may be of less importance than getting the wound site to grow new cardiomyocytes.

An alternative approach would be to mimic that molecular environment to administer well-characterised molecules at known doses. They wouldnt be ATMPs at all, so the extra ATMP burdens that fall on such products under regulations such as the ATMP Regulation would not apply; just those for a conventional biological medicinal products.

Regulatory reflection

Anyone familiar with the manufacturing requirements for somatic cell or tissue engineered products will know that the logistical and quality burdens on those seeking an MA for an ATMP far surpass even those for non-biological medicinal products. It is perfectly true that, under the EUs 2007 Advanced Therapy Medicinal Products (ATMP) Regulation there are only three varieties of ATMP: medicinal products comprising somatic cells or genes, and cells which have been substantially manipulated or used to fulfil a different function in the recipient than in the donor.

But why shouldnt an advanced therapy be delivered the old-fashioned way, as a drug?

The European Commission is reviewing EU blood, cell and tissue (BCT) quality and safety regulation (more on this soon), but has excluded the ATMP Regulation from its review. The Nature article does not touch upon regulation, but to me it suggests another reason for revisiting the ATMP Regulation in the light of changes in scientific understanding. The problem is that, although the ATMP Regulation shoe-horned cell and gene medicinal products into the well-worn boot of European medicines regulation, the foot doesnt entirely fit.

If you recall the heady days of the early 2000s in which the Regulation was developed, the future was one in which bags of embryonically-derived cells for fixing hearts and spines were distributed around the world like blister packs of ibuprofen. The future unfolded rather differently. Within months of the Regulation coming into effect, an invention for inducing pluripotent stem cell induction was published, reducing the necessity for embryonic cells (with the development of stem-cell based embryo models making even embryos unnecessary). More fundamentally, the future turned out to be dominated, not by packages of cells derived from other peoples or embryos, but by the use of the patients own cells; autologous cells.

However, the word autologous doesnt appear in the ATMP Regulation at all. One perfectly logical reason for their non-appearance is that its questionable as to whether a product intended solely for one person can be truly said to be placed on a market at all. If so, its at least odd to talk about authorising such a product to be placed on one. Sure, we legal our way around this so that autologous products do come bearing MAs, but this regulatory chafing of the cellular heel implies that the paradigm is wonky. The EUs BCT regulations already address quality and safety framework for cells, so perhaps the autologous cell therapy market is better regulated as a service than on the basis of a marketable product?

You get another idea about how awkwardly cell products fit into the regime for marketing medicines when you ask questions such as, what is the mechanism of action? and what is the dose? Again, we can get around irritating questions of this sort, but we should certainly pause for thought, and the Nature piece rather sharpens our appreciation.

Javaria Tehzeeb, author of a 2019 literature review on stem cell heart treatments thats cited in the Nature piece, wonders how things are likely to pan out. Perhaps stem-cell therapies will be approved first, but then be overtaken by drugs once the science has caught up? When we get to the end of the line with molecules, then maybe we can say stem cells are a thing of the past, says Tehzeeb. The manufacturing logistics and regulatory path to market would certainly be more straightforward, and there would be real control over the nature and amount of substances applied, in contrast to the informed-wing-and-prayer approach of cell injection. But were not in that future yet. As Tehzeeb says, until then, we should continue to pursue their potential.

As in life science, so in life science regulation its a work in progress

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Cell therapies without cells? - Lexology

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Global Immuno-Cell Therapy market: Application segmentsOncology Cardiovascular Disease (CVD) Orthopedic Wound Healing Others

Global Immuno-Cell Therapy market: Type segmentsPassive Immune (NK LAK etc) Active Immune

Table of Content1 Report Overview1.1 Product Definition and Scope1.2 PEST (Political, Economic, Social and Technological) Analysis of Immuno-Cell Therapy Market2 Market Trends and Competitive Landscape3 Segmentation of Immuno-Cell Therapy Market by Types4 Segmentation of Immuno-Cell Therapy Market by End-Users5 Market Analysis by Major Regions6 Product Commodity of Immuno-Cell Therapy Market in Major Countries7 North America Immuno-Cell Therapy Landscape Analysis8 Europe Immuno-Cell Therapy Landscape Analysis9 Asia Pacific Immuno-Cell Therapy Landscape Analysis10 Latin America, Middle East & Africa Immuno-Cell Therapy Landscape Analysis 11 Major Players Profile

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Immuno-Cell Therapy Market Exhibits a Stunning Growth by 2027 with Covid-19 Impact The Courier - The Courier

The frequency of moderate to severe cytokine release syndrome was reduced in patients with relapsed or refractory multiple myeloma who received anakinra prophylaxis with orvacabtagene autoleucel, a BCMA-targeted CAR T-cell therapy, according to findings presented at the European Hematology Association 2021 Virtual Congress.

The frequency of moderate to severe cytokine release syndrome (CRS) was reduced in patients with relapsed or refractory multiple myeloma (R/R MM) who received anakinra (Kineret) prophylaxis with orvacabtagene autoleucel (orva-cel), a B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T-cell therapy, according to findings presented at the European Hematology Association (EHA) 2021 Virtual Congress.

The findings showed that the use of anakinra prophylaxis had no adverse effect on the incidence of neurological events (NEs), infection, macrophage activation syndrome/hemophagocytic lymphohistiocytosis (MAS/HLH), orva-cel expansion or disease response.

Thirty-three patients with R/R MM who had previously received at least 3 prior lines of therapy were sequentially enrolled onto the phase 1/2 EVOLVE study (NCT03430011). Anakinra 100 mg was administered subcutaneously the night prior to orva-cel infusion, 3 hours before the infusion on day 1 and every 24 hours on days 2 through 5. If patients developed CRS, the dosing was increased to every 12 hours.

The median follow-up was 3 months for the anakinra prophylaxis arm (n = 14) and 8.8 months in the non-anakinra prophylaxis arm (n = 19). In the anakinra prophylaxis and non-anakinra prophylaxis arms, the median number of prior regimens was 6 and 5; bridging therapy was used in 57% and 68% of patients, respectively. The total frequency of CRS was similar in the two groups, however there were less grade 2 events in patients receiving anakinra prophylaxis (HR, 0.54; 95% CI, 0.21-1.38). Tocilizumab (Actemra) and corticosteroid use was numerically lower in patients treated with anakinra prophylaxis.

Most baseline demographics and disease characteristics were similar between groups, said Luciano J. Costa, MD, PhD, associate director for clinical research, ONeal Comprehensive Cancer Center at University of Alabama at Birmingham School of Medicine, during a presentation of the findings. But potentially important differences were noted in measurable serum M-protein, serum free light chain only, extramedullary plasmacytoma and lactate dehydrogenase above the upper limit of normal.

After a 2-month efficacy assessment, the objective response rate (ORR) was 100% in the anakinra prophylaxis arm and 95% in the non-anakinra prophylaxis arm.

At month six after orva-cel treatment, 87.5% of anakinra prophylaxis patients and 62% of non-anakinra prophylaxis patients retained detectable CAR transgene copy, Costa concluded.

The authors noted that these results warrant further study of anakinra prophylaxis in combination with CAR T-cell therapy in these patients.

Reference

Costa L, Mailankody S, Shaughnessy P, et al. Anakinra prophylaxis in patients with relapsed/refractory multiple myeloma receiving orvacabtagene autoleucel. Presented at: 2021 European Hematology Association Congress; June 9-17, 2021; Virtual. Abstract EP747.

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CAR T-Cell Therapy Prophylaxis With Anakinra May Reduce CRS and Toxicities in Multiple Myeloma - Targeted Oncology

California headquartered, Caribou, isa clinical-stage CRISPR genome editing biotechnology company.

The collaboration leverages Caribous next-generation CRISPR genome editing technology platform and AbbVies antigen-specific binders. AbbVie will utilize Caribous next-generation Cas12a CRISPR hybrid RNA-DNA (chRDNA) genome editing and cell therapy technologies to research and develop two new CAR-T cell therapies.

Under the terms of deal, Caribou will receive US$40m in an upfront cash payment and equity investment and up to US$300m in future development, regulatory, and launch milestones.

Although allogeneic CAR-T cell therapies have shown early promise in some cancer patients, the need for overcoming the rejection of allogeneic CAR-T cells by the host immune system remains a key challenge to their broader development, said the companies.

Employing Caribous CRISPR genome editing platform to engineer CAR-T cells to withstand host immune attack would enable the development of the next-generation cellular therapies to benefit a broader patient population, claim the partners.

Caribou will conduct certain pre-clinical research, development, and manufacturing activities for the collaboration programs, and AbbVie will reimburse Caribou for all such activities pursuant to the collaboration, while AbbVie will be responsible for all clinical development, commercialization, and manufacturing efforts.

AbbVie also has the option to pay a fee to expand the collaboration to include up to an additional two CAR-T cell therapies.

We are excited to partner with AbbVie on the development of new CAR-T cell therapies. This collaboration validates Caribous differentiated next-generation CRISPR genome editing technologies that provide best-in-class efficiency and specificity, said Rachel Haurwitz, CEO of Caribou.

CAR-T therapies have shown to be a promising breakthrough in cancer treatment, Collaborating with Caribou and their cutting-edge CRISPR platform will help AbbVie advance our efforts to deliver new hope for patients, said Steve Davidsen, VP, oncology discovery, AbbVie.

Outside of this collaboration, Caribou is advancing an internal pipeline of allogeneic cell therapies for oncology. CB-010, its lead allogeneic CAR-T cell program, targets CD19 and is being evaluated in a Phase 1 clinical trial for patients with relapsed/refractory B cell non-Hodgkin lymphoma.

CB-011, Caribous second allogeneic CAR-T cell therapy, targets BCMA for multiple myeloma, while CB-012, Caribous third allogeneic CAR-T cell therapy, targets CD371 for acute myeloid leukemia.

CB-011 and CB-012 are in preclinical development.

Additionally, Caribou is developing iPSC-derived allogeneic natural killer (NK) cell therapies for solid tumors.

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AbbVie and Caribou partner on CAR-T cell therapy development - BioPharma-Reporter.com

Dublin, Feb. 09, 2021 (GLOBE NEWSWIRE) -- The "Cell Therapy Market by Cell Type, Therapy Type, Therapeutic Area, and End User: Global Opportunity Analysis and Industry Forecast, 2020-2027" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market accounted for $7,754. 89 million in 2019, and is expected to reach $48,115. 40 million by 2027, registering a CAGR of 25. 6% from 2020 to 2027.

Cell therapy involves administration of somatic cell preparations for treatment of diseases or traumatic damages. Cell therapy aims to introduce new, healthy cells into a patient's body to replace diseased or missing ones.

This is attributed to the fact that specialized cells, such as brain cells, are difficult to obtain from human body. In addition, specialized cells typically have a limited ability to multiply, making it difficult to produce sufficient number of cells required for certain cell therapies. Some of these issues can be overcome through the use of stem cells. In addition, cells such as blood and bone marrow cells, mature, immature & solid tissue cells, adult stem cells, and embryonic stem cells are widely used in cell therapy procedures.

Moreover, transplanted cells including induced pluripotent stem cells (iPSCs), embryonic stem cells (ESCs), neural stem cells (NSCs), and mesenchymal stem cells (MSCs) are divided broadly into two main groups including autologous cells and non-autologous cells. Development of precision medicine and advancements in Advanced Therapies Medicinal Products (ATMPS) in context to their efficiency and manufacturing are expected to be the major drivers for the market. Furthermore, automation in adult stem cells and cord blood processing and storage are the key technological advancements that fuel growth of the market for cell therapy.

In addition, growth in aging patient population, The rise in cell therapy transplantations globally, and surge in disease awareness drive growth of the global cell therapy market. Furthermore, The rise in adoption of human cells over animal cells for cell therapeutics research, technological advancements in field of cell therapy, and increase in incidences of diseases such as cancer, cardiac abnormalities, and organ failure are the key factors that drive growth of the global market.

Moreover, implementation of stringent government regulations regarding the use of cell therapy is anticipated to restrict growth of the market. On the contrary, surge in number of regulations to promote stem cell therapy and increase in funds for research in developing countries are expected to offer lucrative opportunities to the market in the future.

The global cell therapy market is categorized on the basis of therapy type, therapeutic area, cell type, end user, and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutics, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others.

The global cell therapy market is categorized on the basis of therapy type, therapeutic, cell type, end user and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutic area, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others. On the basis of cell type, it is segregated into stem cell therapy and non-stem cell type. On the basis of end user, it is segregated into hospital & clinics and academic & research institutes. On the basis of region, the market is studied across North America, Europe, Asia-Pacific, and LAMEA.

Key Benefits

Story continues

The study provides an in-depth analysis of the global cell therapy market along with the current trends and future estimations to elucidate the imminent investment pockets.

Comprehensive analysis of factors that drive and restrict the market growth is provided in the report.

Comprehensive quantitative analysis of the industry from 2019 to 2027 is provided to enable the stakeholders to capitalize on the prevailing market opportunities.

Extensive analysis of the key segments of the industry helps in understanding the forms and types of cell therapy used across the globe.

Key market players and their strategies have been analyzed to understand the competitive outlook of the market.

Key Topics Covered:

Chapter 1: Introduction1.1. Report Description1.2. Key Benefits for Stakeholders1.3. Key Market Segments1.4. Research Methodology1.4.1. Secondary Research1.4.2. Primary Research1.4.3. Analyst Tools & Models

Chapter 2: Executive Summary2.1. Key Findings of the Study2.2. Cxo Perspective

Chapter 3: Market Overview3.1. Market Definition and Scope3.2. Key Findings3.2.1. Top Player Positioning3.2.2. Top Investment Pockets3.2.3. Top Winning Strategies3.3. Porter'S Five Forces Analysis3.4. Impact Analysis3.4.1. Drivers3.4.1.1. Technological Advancements in the Field of Cell Therapy3.4.1.2. The Rise in Number of Cell Therapy Clinical Studies3.4.1.3. The Rise in Adoption of Regenerative Medicine3.4.2. Restraint3.4.2.1. Developing Stage and Pricing3.4.3. Opportunity3.4.3.1. High Growth Potential in Emerging Markets3.5. Impact of Covid-19 on Cell Therapy Market

Chapter 4: Cell Therapy Market, by Cell Type4.1. Overview4.1.1. Market Size and Forecast4.2. Stem Cell4.2.1. Key Market Trends and Opportunities4.2.2. Market Size and Forecast, by Region4.2.3. Market Size and Forecast, by Type4.2.3.1. Bone Marrow, Market Size and Forecast4.2.3.2. Blood, Market Size and Forecast4.2.3.3. Umbilical Cord-Derived, Market Size and Forecast4.2.3.4. Adipose-Derived Stem Cell, Market Size and Forecast4.2.3.5. Others (Placenta, and Nonspecific Cells), Market Size and Forecast4.3. Non-Stem Cell4.3.1. Key Market Trends and Opportunities4.3.2. Market Size and Forecast, by Region

Chapter 5: Cell Therapy Market, by Therapy Type5.1. Overview5.1.1. Market Size and Forecast5.2. Autologous5.2.1. Key Market Trends and Opportunities5.2.2. Market Size and Forecast, by Region5.2.3. Market Analysis, by Country5.3. Allogeneic5.3.1. Key Market Trends and Opportunities5.3.2. Market Size and Forecast, by Region5.3.3. Market Analysis, by Country

Chapter 6: Cell Therapy Market, by Therapeutic Area6.1. Overview6.1.1. Market Size and Forecast6.2. Malignancies6.2.1. Market Size and Forecast, by Region6.2.2. Market Analysis, by Country6.3. Musculoskeletal Disorders6.3.1. Market Size and Forecast, by Region6.3.2. Market Analysis, by Country6.4. Autoimmune Disorders6.4.1. Market Size and Forecast, by Region6.4.2. Market Analysis, by Country6.5. Dermatology6.5.1. Market Size and Forecast, by Region6.5.2. Market Analysis, by Country6.6. Others6.6.1. Market Size and Forecast, by Region6.6.2. Market Analysis, by Country

Chapter 7: Cell Therapy Market, by End-user7.1. Overview7.1.1. Market Size and Forecast7.2. Hospitals & Clinics7.2.1. Key Market Trends and Opportunities7.2.2. Market Size and Forecast, by Region7.2.3. Market Analysis, by Country7.3. Academic & Research Institutes7.3.1. Key Market Trends and Opportunities7.3.2. Market Size and Forecast, by Region7.3.3. Market Analysis, by Country

Chapter 8: Cell Therapy Market, by Region8.1. Overview8.2. North America8.3. Europe8.4. Asia-Pacific8.5. LAMEA

Chapter 9: Company Profiles9.1. Allosource9.1.1. Company Overview9.1.2. Company Snapshot9.1.3. Operating Business Segments9.1.4. Product Portfolio9.1.5. Key Strategic Moves and Developments9.2. Cells for Cells9.2.1. Company Overview9.2.2. Company Snapshot9.2.3. Operating Business Segments9.2.4. Product Portfolio9.3. Holostem Terapie Avanzate Srl9.3.1. Company Overview9.3.2. Company Snapshot9.3.3. Operating Business Segments9.3.4. Product Portfolio9.4. Jcr Pharmaceuticals Co. Ltd.9.4.1. Company Overview9.4.2. Company Snapshot9.4.3. Operating Business Segments9.4.4. Product Portfolio9.4.5. Business Performance9.4.6. Key Strategic Moves and Developments9.5. Kolon Tissuegene, Inc.9.5.1. Company Overview9.5.2. Company Snapshot9.5.3. Operating Business Segments9.5.4. Product Portfolio9.5.5. Key Strategic Moves and Developments9.6. Medipost Co. Ltd.9.6.1. Company Overview9.6.2. Company Snapshot9.6.3. Operating Business Segments9.6.4. Product Portfolio9.6.5. Business Performance9.7. Mesoblast Ltd9.7.1. Company Overview9.7.2. Company Snapshot9.7.3. Operating Business Segments9.7.4. Product Portfolio9.7.5. Business Performance9.8. Nuvasive, Inc.9.8.1. Company Overview9.8.2. Company Snapshot9.8.3. Operating Business Segments9.8.4. Product Portfolio9.8.5. Business Performance9.9. Osiris Therapeutics, Inc.9.9.1. Company Overview9.9.2. Company Snapshot9.9.3. Operating Business Segments9.9.4. Product Portfolio9.10. Stemedica Cell Technologies, Inc.9.10.1. Company Overview9.10.2. Company Snapshot9.10.3. Operating Business Segments9.10.4. Product Portfolio

For more information about this report visit https://www.researchandmarkets.com/r/bja7iz

Link:
Outlook on the Cell Therapy Global Market to 2027 - Opportunity Analysis and Industry Forecasts - Yahoo Finance

Gamida Cell Ltd., a maker of cell and immune therapy technologies, on Wednesday published the results of a key late-stage clinical trial of its flagship drug, which aims to increase the success of bone marrow transplants in blood cancer patients triggering a surge in its Nasdaq-traded shares.

Gamidas shares jumped almost 26% on the US exchange Wednesday. The shares have advanced 163% in the past 12 months, leading to a company valuation of $733 million. The firm held its initial public offering of shares on the Nasdaq in 2019 to help finance the final stage trial of the drug.

The phase three study tested the safety and efficacy of its omidubicel drug, previously called NiCord, in patients with blood-based cancers.

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The data showed that omidubicel resulted in faster blood count recovery, fewer bacterial and viral infections, and fewer days in the hospital.

Stem cells in bag in Gamida Cells Jerusalem lab, July 2017 (Shoshanna Solomon/TimesofIsrael)

All of these are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant, said Mitchell Horwitz, principal investigator and professor of medicine at the Duke Cancer Institute, in a statement released by the Jerusalem-based firm.

The data was from the international, multi-center, randomized Phase 3 study of the drug, designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared with a group of patients who received a standard umbilical cord blood transplant.

In May, the firm said that the study yielded positive results and met a major target showing that omidubicel led to a statistically significant reduction in the time needed to for patients to begin making healthy new cells after receiving stem cells, which is a key milestone in a patients recovery from a bone marrow transplant.

Omidubicel, which would be the first drug developed by Gamida to hit the market, is believed to increase the chances of a successful bone marrow transplantation process for patients who do not have a rapidly available, fully matched bone marrow donor.

Julian Adams, chief executive officer of Gamida Cell (Courtesy)

Despite the curative potential of bone marrow transplant, it is estimated that more than 40 percent of eligible patients in the United States do not receive a transplant for various reasons, including the lack of a matched donor.

Today some high-risk blood cancers cannot be cured unless the patient undergoes a bone marrow graft. For that purpose, a perfect match needs to be found, a process that in the US takes an average of three to four months, if the patient is lucky. Sometimes, no match is found.

Umbilical cord blood collected from newborn babies contains stem cells, which can be used to treat diseases. Today cord-blood banks around the world store the cord blood. Its great advantage is that because it is so young, there is no need for a full tissue matching and a partial match is enough. Most patients generally find at least one unit of cord blood that partially matches them.

The problem is that the quantity of cells in each unit is not huge, and it is the number of stem cells in the cord blood that is critical to the success of transplantation.

Gamida overcomes this limitation by expanding the number of stem cells within one unit of umbilical cord blood and enhancing their performance.

The firm said Wednesday that it expects to submit a Biologics License Application to the US Food and Drug Administration for omidubicel in the second half of this year.

We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor, said Julian Adams, chief executive officer of Gamida Cell, which was founded in 1998.

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Hope grows for blood cancer patients, as cell therapy firm's key trial succeeds - The Times of Israel

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AlloVir (Nasdaq: ALVR), a late clinical-stage cell therapy company, today announced results of a subgroup analysis from a Phase 2, proof-of-concept study (CHARMS) evaluating the companys lead product candidate, Viralym-M (ALVR105), an allogeneic, off-the-shelf, multi-virus specific investigational T-cell therapy (VST), in allogeneic hematopoietic stem cell transplant (allo-HSCT) recipients with virus-associated hemorrhagic cystitis (V-HC). These data are being presented in an oral presentation during the Transplantation & Cellular Therapy (TCT) Meeting of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR). Additionally, two separate oral presentations characterize the high economic and clinical burden of V-HC and double-stranded (ds) DNA viral infections in allo-HSCT recipients. Preclinical data was also presented in a poster presentation on ALVR109, AlloVirs virus-specific T-cell therapy targeting SARS-CoV-2, the virus responsible for COVID-19.

The data from the Phase 2 CHARMS study highlight Viralym-M's potential to treat and possibly prevent multiple viral infections and viral diseases. The findings presented at TCT show that this novel virus-specific T cell therapy has the potential to rapidly and effectively resolve macroscopic hematuria in allo-HSCT recipients with virus-associated hemorrhagic cystitis a disease that currently has no effective treatment options and causes significant morbidity and increased risk of mortality, said Agustin Melian, MD, Chief Medical Officer and Head of Global Medical Sciences of AlloVir. We have recently initiated our Phase 3, pivotal study of Viralym-M for the treatment of virus-associated hemorrhagic cystitis and look forward to advancing this therapy through development for patients in need.

Data of Viralym-M in fifty-eight allo-HSCT recipients with at least one treatment-refractory viral infection caused by BK virus (BKV), cytomegalovirus (CMV), adenovirus (AdV), Epstein Barr virus (EBV), human herpesvirus 6 (HHV-6), and/or JC virus (JCV) were evaluated in the CHARMS Phase 2 study. The subgroup analysis presented at TCT included 26 patients who received intravenous VST infusions for the treatment of V-HC due to infection with BKV (n=23), AdV (n=2) and BKV and AdV (n=1). Infusions were well tolerated with mild, grade 1, de novo skin rash from graft-versus-host disease (GVHD) occurring in 15% of patients (n=4). In the 20 patients with available V-HC grading, resolution of macroscopic hematuria was observed in 60% and 80% of patients at two- and six-weeks post-infusion, respectively. In comparison, resolution of macroscopic hematuria was observed in <10% and 30% of patients at weeks two and six, respectively, in a contemporary cohort of allo-HSCT recipients (n=33) with V-HC who were not treated with Viralym-M.

Health economic outcomes data was also presented in two separate oral presentations at the conference. The two presentations analyzed U.S. claims data to compare health care reimbursement, health resource utilization, and clinical outcomes in pediatric and adult allo-HSCT recipients with V-HC and those without V-HC, and allo-HSCT recipients with or without dsDNA infections, respectively. Both studies found that allo-HSCT recipients with V-HC and those with any dsDNA infection had higher reimbursement costs, increased hospital and ICU length of stay, and increased hospital readmission rates. The presence of V-HC or any dsDNA viral infection was associated with a higher risk of mortality.

In addition, a poster presentation at the conference demonstrated the in vitro effector and safety profile of ALVR109, an allogeneic, off-the-shelf investigational VST therapy designed to target SARS-CoV-2, the virus that causes the severe and life-threatening viral disease, COVID-19. These data suggest the potential for using these VSTs to treat COVID-19 in hospitalized high-risk patients to prevent the development of severe disease. A clinical trial evaluating these banked, off-the-shelf SARS-CoV-2 specific T cells has been initiated at the Center for Cell and Gene Therapy, Baylor College of Medicine (BCM), Texas Children's Hospital, and Houston Methodist Hospital.

Viral Infections in Immunocompromised Patients

In healthy individuals, virus-specific T cells (VSTs) from the bodys natural defense system provide protection against numerous disease-causing viruses. However, in patients with a weakened immune system these viruses may be uncontrolled. Viral diseases are common and can cause potentially devastating and life-threatening consequences in immunocompromised patients. For example, up to 90% of patients will reactivate at least one virus following an allogeneic stem cell transplant and two-thirds of these patients reactivate more than one virus, resulting in significant and prolonged morbidity, hospitalization, and premature death. Typically, when viruses infect immunocompromised patients, standard antiviral treatment does not address the underlying problem of a weakened immune system and therefore many patients suffer with life-threatening outcomes such as multi-organ damage and failure, and even death.

Viralym-M

Viralym-M (ALVR105) is an allogeneic, off-the-shelf, multi-virus specific investigational T-cell therapy targeting five devastating viral pathogens: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. Viralym-M has the potential to transform care for transplant recipients as well as individuals who are at high risk for opportunistic viral infections by reducing or preventing disease morbidity and dramatically improving patient outcomes. Three pivotal and proof-of-concept clinical (POC) trials are ongoing and actively recruiting patients in indications such as treatment of virus-associated hemorrhagic cystitis and multi-virus prevention following allo-HSCT, and preemptive treatment of BK viremia in adult kidney transplant recipients. Additional pivotal and POC trials are expected to initiate for the treatment of CMV and the treatment of AdV in allo-HSCT recipients and in CMV for solid organ transplant recipients, respectively. For more information on the ongoing clinical trials visit clinicaltrials.gov.

Viralym-M has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA), as well as PRIority MEdicines (PRIME) and Orphan Drug Designations (ODD) from the European Medicines Agency.

About AlloVir

AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in pediatric and adult patients with weakened immune systems. The companys innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, multi-virus specific T-cells targeting devastating viruses for patients with T-cell deficiencies who are at risk from the life-threatening consequences of viral diseases. AlloVirs technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio. For more information visit http://www.allovir.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding AlloVirs development and regulatory status of our product candidates, the planned conduct of its preclinical studies and clinical trials and its prospects for success in those studies and trials, and its strategy, business plans and focus. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to AlloVirs financial results, the timing for the initiation and successful completion of AlloVirs clinical trials of its product candidates, whether and when, if at all, AlloVirs product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, the impact of the COVID-19 pandemic on AlloVirs product development plans, supply chain, and business operations and other risks identified in AlloVirs SEC filings. AlloVir cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. AlloVir disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent AlloVirs views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

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AlloVir Research Presented at the 2021 Transplantation & Cellular Therapy Meeting Digital Experience - Business Wire

BEDMINSTER, N.J. and SINGAPORE, Feb. 10, 2021 /PRNewswire/ -- Tessa Therapeutics Ltd. (Tessa), a clinical-stage cell therapy company developing next-generation cancer treatments for hematological malignancies and solid tumors, today announced the successful completion of dosing of the first patient cohort (n=3) in a Phase I dose escalation study, evaluating the safety and efficacy of Tessa's TT11X Allogeneic CD30-CAR Epstein Barr Virus Specific T-cell (EBVST) therapy.

The Phase 1 study being conducted at Baylor College of Medicine aims to enroll up to 18 patients with CD30+ lymphoma across three dose levels. Study objectives are to evaluate safety and efficacy and establish dosing for the next phase. "TT11X has been administered to three patients so far at Houston Methodist Hospital with a favorable safety profile. The therapy has been well tolerated with no evidence of GVHD or any severe adverse events associated with allogeneic therapies," said Dr. Carlos Ramos, Lead Principal Investigator on the study and Professor at the Center for Cell and Gene Therapy and member of the Dan L Duncan Comprehensive Cancer Center at Baylor College of Medicine. For more information, visitwww.clinicaltrials.gov(Study Identifier NCT04288726).

Off-the-shelf, allogeneic cell therapy has significant advantages and is the next frontier in cancer treatment. Tessa is developing a unique and potentially transformational allogeneic cell therapy platform based on decades-long research and development on Virus Specific T-cells (VSTs) by Tessa's Scientific Co-Founder Dr. Malcolm Brenner and his team at Baylor College of Medicine.

"VSTs are highly specialized T cells with the ability to recognize and kill infected cells while activating other parts of the immune system for a coordinated response. Allogeneic VSTs without any form of genetic modification have demonstrated a strong safety profile and efficacy in early trials with minimal risk of graft versus host disease and graft rejection," said Malcolm Brenner, M.D., Ph.D., Founding Director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston Methodist Hospital and Texas Children's Hospital. "The fundamental qualities of VSTs therefore make them a strong candidate for allogeneic application, and we are working closely with Tessa to advance this potential new platform therapy."

Tessa's allogeneic platform enhances inherent non-alloreactive properties of VSTs with CD30- CAR targeting. Preclinical studies have demonstrated that CD30 targeting potentially helps eliminate alloreactive T-cells and may improve allogeneic cell expansion and persistence. Tessa and Baylor College of Medicine are jointly developing this platform.

"We are quite excited about the therapeutic potential and broad applicability of our allogeneic CD30-CAR EBVST platform. The clinical progress on the ongoing study has been very encouraging and represents a significant milestone for Tessa," said Ivan D. Horak, M.D., Chief Medical Officer and Chief Scientific Officer of Tessa Therapeutics."Longer term, we aim to develop this platform to tackle solid tumors where there is significant patient need."

About Tessa Therapeutics

Tessa Therapeutics is a clinical-stage biotechnology company developing a portfolio of next-generation cell therapies for cancer. It has a pipeline of therapies in clinical development for the treatment of hematological malignancies and solid tumors.

Tessa's lead autologous therapy is in late-stage clinical development for treatment of lymphomas. It has shown strong clinical responses in patients with relapsed/refractory classical Hodgkin lymphoma, based on which it was granted the RMAT designation by the U.S. FDA and the PRIME designation by EMA.

Tessa is also developing a novel, differentiated, allogeneic "off-the shelf" cell therapy platform to create more efficacious, reliable, and scalable therapies capable of targeting a broad range of cancers. A therapy using this platform is being evaluated in an ongoing clinical trial in United States.

For more information on Tessa, please visit http://www.tessacell.com.

Cautionary Note on Forward Looking Statements

This press release contains forward-looking statements (within the meaning of the Private Securities Litigation Reform Act of 1995, to the fullest extent applicable) including, without limitation, with respect to various regulatory filings or clinical study developments of the Company. You can identify these statements by the fact that they use words such as "anticipate", "estimate", "expect", "project", "intend", "plan", "believe", "target", "may", "assume" or similar expressions. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the Company's financial results, the ability to raise capital, dependence on strategic partnerships and licensees, the applicability of patents and proprietary technology, the timing for completion of the clinical trials of its product candidates, whether and when, if at all, the Company's product candidates will receive marketing approval, and competition from other biopharmaceutical companies. The Company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made, and disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the Company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The Company's products are expressly for investigational use pursuant to a relevant investigational device exemption granted by the U.S. Food & Drug Administration, or equivalent competent body.

SOURCE Tessa Therapeutics

http://www.tessacell.com

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Tessa Therapeutics Announces Successful Dosing of First Patient Cohort in Phase I Allogeneic Cell Therapy Trial - PRNewswire

A new clinical trial at the University of Colorado Anschutz Medical Campus, using cells genetically engineered by medical faculty to fight stubborn cancers, is showing encouraging results.

Created at the Gates Biomanufacturing Facility a few steps from CU Anschutz, the chimeric antigen receptor T cells, or CAR-T cells, are being infused into patients with difficult to treat or frequently recurring cancers at UCHealth University of Colorado Hospital.

Heather Pidcoke was among

the first patients to receive

the locally produced

UCD19 CAR-T cells. She

now has no cancer symptoms.

Its the first cellular immunotherapy project where the developmental science, regulatory filing and approvals, manufacturing process and infusion of patients in clinical trials have all been done at CU Anschutz.

Heather Pidcoke, 56, a physician, medical researcher and now a patient, was among the first to receive the locally produced `UCD19 CAR-T cells.

She was diagnosed in 2014 with follicular lymphoma, a slow growing but often recurring cancer. Since then, Pidcoke has endured multiple chemotherapy cycles, most recently going into remission in 2018 before her lymphoma returned again in 2020.

She and her oncologist, Manali Kamdar, MD, an associate professor of medicine at the University of Colorado School of Medicine, decided to watch and wait.

At this point, Kamdar told her of the clinical trial and she quickly enrolled.

It takes courage and it takes a lot of faith in science to do this, Kamdar said. And I think her medical background helped.

Pidcoke knew of CAR-T cell therapy. She also knew it was reserved for the most difficult cases.

As a resident I treated patients, as a researcher I worked on things that affected patients but it is different being a patient, she said. Still, I have dedicated my life to research and I think it is important to take an active part in it.

Physicians at the CU School of Medicine took samples of Pidcokes own blood lymphocytes. The Gates Biomanufacturing Facility then separated out a certain subset of cells that were cultured and genetically altered to recognize and kill cancer cells.

CAR-T cells, often used in otherwise untreatable leukemias and lymphomas, can trigger serious side-effects.

They can create an environment of inflammation. Patients can get a fever, shortness of breath, and sometimes neurological side effects that can affect speech and cognition, Kamdar said. We see side-effects in about 15-30% of patients. Thankfully, we now know of strategies to mitigate them. However, our goal is to develop new ways of administering this therapy with excellent efficacy and less toxicity.

Pidcoke earns a living with her mind so potential neurological damage weighed heavily on her.

You meet those trepidations with prayer, trust in science, and resolve, she said. I prayed for my brain to be protected.

She checked into UCHealth in late September for a two week stay. She was given Benadryl to tamp down side-effects, then infused with the Gates-engineered CAR-T cells.

Nothing happened.

Kamdar feared the lack of side-effects meant the treatment wasnt working. Then Pidcokes next scan revealed something extraordinary. Every tumor in her abdomen had melted away. After 90 days, she was in complete remission with no side-effects.

The other patients in the trial have also seen encouraging results.

The UCD19 trial is a huge milestone. It shows we can deliver,

offering real results and hope for patients and their families.

Diane Gates Wallach, co-chair of the Gates Center Advisory Board

This is the realization of years of innovative research and meticulous preparation, said Michael Verneris, MD, professor of pediatrics-hematology/oncology and director of Bone Marrow Transplantation and Cellular Therapy at Childrens Hospital Colorado. New capabilities at the Gates Biomanufacturing Facility make CU Anschutz one of the few academic medical campuses in the nation where bench science can move to manufacturing and on to patient treatments all within walking distance.

Verneris, who co-leads the Tumor Host Interactions program in the CU Cancer Center, said the new trial closes the loop for patient-centered research in Colorado.

This approach, enrolling patients who have no other viable therapeutic options, will expand to pediatric patients at Childrens Hospital Colorado in early 2021.

Past trials employing other CAR-T cells, saw remission in about 60% of previously untreatable adult lymphoma cases and in approximately 80% of pediatric patients with resistant leukemia. Scientists hope this trial will prompt the development of new CAR-T therapies and lead to the eventual treatment of other forms of cancer.

We believe our ability to support on-campus clinical trials will lead to multiple similar efforts in the near future by other Gates Center researchers in other disciplines, and continue to bolster the reputation of the Gates Biomanufacturing Facility and CU Anschutz as a leader in the manufacture and administration of academic and commercial cellular therapies, said CU School of Medicine Dean John J. Reilly, Jr., MD.

The goal was to develop and produce clinical grade biological products and cellular therapies from bench research through trials to patient care, said Diane Gates Wallach, co-chair of the Gates Center Advisory Board. The UCD19 trial is a huge milestone.It shows we can deliver, offering real results and hope for patients and their families.Congratulations to the entire team.

Pidcoke is now back at work as chief medical research officer at Colorado State University. She looks forward to kayaking again and roaming the foothills with her husband and dog near her home outside Fort Collins.

She has no symptoms and feels `wonderful.

I have had just fantastic doctors who have been warriors with me in meeting this challenge and I know they will be there again if necessary, she said. I have learned to live in the moment and not let the good moments of today be overshadowed by what could happen in the future.

And those cells?

The cells worked beautifully, she said.

Link:
Cancer Patient Sees Total Remission With CU Anschutz-Created Cell Therapy - CU Anschutz Today

Dr. Patrick Hwu became CEO of the Moffitt Cancer Center in November. He took over at a crucial time for the Tampa, Fla., organization, which saw several leaders resign in late 2019 due to concerns over conflicts of interest regarding research being conducted in China. He wants to make Moffitt a leader in cell therapy. And in late January, Pasco County officials approved a $25 million incentive and financing deal for Moffitt to build on a 775-acre site. It includes infrastructure improvements, as well as waiving nearly $800,000 in permit fees. Hwu spoke with Modern Healthcare Managing Editor Matthew Weinstock about his plans for Moffitt, as well as the impact of COVID-19 on cancer care. The following is an edited transcript.

MH: Coming into an organization thats gone through a bit of turmoil, are there things that you had to address right away or since it was almost a year after the turnover, had some of that taken care of itself?

Hwu: A lot of it had settled down already, but the bottom line is we need to help cancer patients. We still have 45,000 deaths a year in Florida, 600,000 in America and over 10 million globally, although thats probably an underestimate. If we focus everyone on trying to cut down the number of deaths from cancer, everything aligns. Because that, in the end, is what we wantto prevent cancer and cure all these patients, and I think its possible.

MH: What are some of your priorities?

Hwu: Its very straightforward actually. We have to implement in 2021 all of things we know are effective to prevent, treat and cure cancer patients. So we need to have widespread implementation, which includes building additional centers where we can take care of more patients. The second thing we have to do is come up with better therapies and better ways to prevent cancer. In doing both of those things, I think we can really decrease these deaths over the years and so thats really our only goal.

People used to tell me, Isnt it depressing taking care of advanced melanoma patients, because my own clinic would turn over every six months. But now over half of my patients are doing well for many, many years because of the advances that weve made in targeted therapy and immunotherapy.

MH: One of the things youve talked about growing at Moffitt is cell therapy. Can you talk a little bit about what that might mean both for Moffitt and for cancer care broadly?

Hwu: Our immune systems are incredible. In fact, thats whats going to turn us around from COVIDstimulating the bodys immune system.

Weve evolved cells called lymphocytes that go around the body and try to protect against viruses, but weve also learned to trigger them against cancer. So when we get these immune cells, lymphocytes, touching the cancer cell they can do what I call the kiss of death, where they secrete proteins that cause the holes in the cancer membrane itself, and the cancer itself blows up. Then the immune cell goes to another cancer cell, does the kiss of death, and does the same thing.

Weve learned to grow these immune cells out of the body, grow them to large numbers, give them back to patients, (which) causes the tumors to regress.

Weve also learned to grow cells and put in genes, like CAR T-cell genes, chimeric antigen receptor genes, to help them recognize those tumor cells to cause that kiss of death. Thats a technology that we started years ago, in the early 90s when I was at the National Cancer Institute. And its really been fun for me to see this now approved by the Food and Drug Administration for some lymphomas.

MH: Moffitt is also expanding its physical presence. You recently got approval from Pasco County, which is putting in about $25 million in incentives, for a new project. What are you looking to do there?

Hwu: Its a 775-acre plot of land, which were very excited about. Its a growing area and a growing community, and more and more we have to take our care out to patients. So this is going to be an important facility. Were going to start with an outpatient facility to provide care in the clinic, and infusions, radiology, radiation. But we also hope to do some very novel kinds of treatment as well, such as carbon ion therapy, which is even the next level above proton therapy where you take a carbon ion, which is six protons, and hit it against the cancer. It should be much more effective and less toxic. Were really excited by that. Pasco County is now helping us by putting in the interstate exchanges so its very easy to get to and from the (Tampa) airport.

MH: Weve seen in other cases, including the Mayo Clinic, backlash when these large taxpayer financial incentives come into play. Have you heard any of that from people in Pasco County?

Hwu: (Theres been) nothing but positive feedback from everybody. Theyre very excited that weve chosen to go in there and expand. We think itll ultimately result in over 14,000 jobs in the area, so its going to build all sorts of industries. And it will also help with the biotech entrepreneurship community.

MH: Switching to the COVID-19 pandemic, weve been seeing reports that patients are skipping some of their screenings, including for some cancers. What level of concern do you have on that front?

Hwu: Initially we were seeing thatpeople did not want to come in. But then we really got on top of this and we explained to everybody that Moffitt Cancer Center, were probably the safest environment on the planet. Everyone gets screened, were all wearing masks, and weve had very low numbers of infections here at Moffitt. And I think cancer patients, in general, are more careful to begin with. So weve been able to get our activity back up to pre-COVID numbers.

Were also working with our communications team to make sure patients know that they need to get their mammograms, they need to get their colonoscopies, because the earlier we can catch their cancers, the more curable they are. Just because theres COVID, cancer does not stop, so its really important that we get people to follow through on their appointments and their screenings.

MH: And for a lot of cancer care, you cant rely on telehealth as heavily as other disciplines have, right?

Hwu: Absolutely. At the same time, were using a lot of telemedicine. What telemedicine is really helpful with is in between visits. They might have to come in to get an infusion or to be seen, or examined or for surgery, but in between, we could do some things on telemedicine and weve just scratched the surface of this.

The other area it can help is when the patient first calls us, we can get a much better idea (about their concerns), so the first appointment is much more efficient. We have a lot of the paperwork done and we have a lot of the screening done, we know exactly what we need to do for them initially. Weve had some positive learnings.

MH: Regarding the vaccine, what are you seeing in terms of patients getting vaccinated and do you feel like we know enough about the impact those vaccines might have on cancer patients?

Hwu: First, I want to point out that the COVID vaccine was developed through cancer research. It was first a cancer vaccine. (BioNTech CEO Dr.) Ugur Sahin was a cancer researcher. He was designing vaccines against cancer and then quickly flipped that to COVID when this became an issue, so its not a new platform. People say, Oh, I dont know about a new platform. Well, weve been giving this kind of RNA vaccine to cancer patients in clinical trials for years. We know its safe. I know that its going to be safe in cancer patients. Weve immunized over 5,000 cancer patients here.

The question is whether it is effective. Now thats an important question and hasnt been totally answered. At Moffitt, weve drawn blood samples on over 600 patients to try to answer this question. Weve taken their blood so we can understand their development of antibodies, which is what neutralizes the COVID virus. Were going to take it before (their vaccination); four weeks after their first vaccine were going to take another sample. Four weeks after the second vaccine were going to take another sample. And were going to look at six months, 12 months, 24 months, just to make sure that its still working. Well be able to define those populations.

What I predict is that some patients, especially if theyve had their cancer a number of years ago, will be just like the non-cancer population. But others, especially the ones who are having disorders like leukemia or lymphomadisease of the immune system or they got Rituxan or CD19 CAR-T where theyre knocking out the B cells that make the antibodiesthey may have lesser of a response and may need a third, or a fourth, vaccine to boost that up to levels that will protect them against COVID.

MH: When do you expect to start seeing some of that data roll out?

Hwu: Two to three months. I want to get that information as quickly as possible because its possible well need to pull back some of those patients and say, Hey, you need vaccine number three or vaccine number four. So thats why Ive urged the teams to try to get that data almost in real time. And well try to get this published just as quickly as possible, so everybody knows how to deal with this in their cancer patients.

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Moffitt Cancer Center CEO on COVID vaccines, expanding cell therapy - Modern Healthcare