Intellia Therapeutics Reports Progress on CRISPR/Cas9 AML Cancer Therapy Using Proprietary Cell Engineering Process at the 23rd Annual Meeting of the…

CAMBRIDGE, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology bothin vivoandex vivo,is presenting three oral presentations and two poster presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place virtually from May 12-15, 2020. Intellia researchers are presenting new data in support of NTLA-5001, the companys engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML). Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE).

At Intellia, we are applying our CRISPR/Cas9 technology to develop new processes that can produce enhanced engineered cell therapies to treat severe cancers, such as AML, that traditional approaches cannot address. Our proprietary platform provides a powerful tool to generate more potent TCR-directed cells, that can treat blood cancers initially and potentially solid tumors. The data being presented today validate Intellias approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year, said Intellia President and CEO John Leonard, M.D. We are also pleased to present data that support our recently announced HAE development candidate, NTLA-2002, Intellias second systemic therapy employing our in vivo knockout approach and modular delivery platform.

Data Presentations on Intellias First Engineered Cell Therapy Development Candidate, NTLA-5001 for the Treatment of AML, and Proprietary Cell Engineering Process

NTLA-5001 is Intellias first engineered T cell receptor (TCR) T cell therapy development candidate, which targets the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001 is being developed in collaboration with Chiara Boninis team at IRCCS Ospedale San Raffaele to treat AML patients regardless of the genetic subtype of a patients leukemia. AML is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment and is the most common type of acute leukemia in adults(Source:NIH SEER Cancer Stat Facts: Leukemia AML).

Intellias proprietary process is a significant improvement over standard engineering processes commonly used to introduce nucleic acids into cells. Intellias process enabled multiple gene edits using CRISPR/Cas9, while maintaining cell products with high expansion potential and minimal undesirable chromosomal translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR in locus, while eliminating the expression of the endogenous TCRs, with the goal of producing homogeneous T cell therapies like NTLA-5001.

Intellias novel approach with NTLA-5001 can overcome the challenges of standard T cell therapy, including risks of reduced specificity associated with mixed expression and mispairing of endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease (GvHD) risks, which could lead to an attack on the patients healthy cells; and reduced efficacy tied to lower tgTCR expression per T cell. Intellias unprecedented process is expected to streamline cell engineering and manufacturing, yielding a homogenous product comprising WT1-targeted T cells with high anti-tumor activity. Data highlights from todays presentation include the following:

Intellias cell engineering efforts are focused on its initial clinical investigation of NLTA-5001 on AML, while continuing preclinical studies exploring the potential for targeting WT1 in solid tumors. The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021.

The presentation titled, Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering, will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell Therapy, and can be found here, on the Scientific Publications & Presentations page of Intellias website. These data were a follow-on to the study presented at Keystone Symposias Engineering the Genome Conference from this past February.

In Vivo Data Supports Intellias Novel TCR Candidate

A second presentation on engineered cell therapy progress, in collaboration with IRCCS Ospedale San Raffaele, showed in vivo data demonstrating the potential of TCR-edited T cells to effectively target WT1 tumor cells in AML. In addition to the previously disclosed results of effective in vitro recognition of primary AML tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells), new data indicate that the selected TCR also enables T helper cells (CD4 T cells) to react to WT1-expressing tumor cells, providing cytokine support. This distinguishes Intellias TCR from other therapeutic TCR candidates, which either exclusively activate toxic CD8 T cells or require the co-transfection of CD8 into CD4 T cells to render them functional.

Using a mouse model carrying disseminated human primary AML, researchers observed a significant therapeutic effect, including decreased AML tumor burden. In addition, no signs of GvHD were observed in mice treated with the WT1-specific T cells. The data show that tgTCR-engineered cells have targeted anti-cancer activity in a challenging model of systemic AML, demonstrating the therapeutic potential of Intellias engineered TCR T cell approach.

The presentation titled, Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia, will be given today by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy. Notably, ASGCT meeting organizers selected this presentation as one of six to receive the ASGCT Excellence in Research Award this year.

Continued Progress on Intellias Second In Vivo Development Candidate, NTLA-2002 for the Treatment of HAE

Intellia is presenting development data updates on its potential HAE therapy, NTLA-2002, which utilizes the companys systemic in vivo knockout approach, including its proprietary lipid nanoparticle (LNP) system. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. NTLA-2002 aims to prevent unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.

The KLKB1 gene knockout in an ongoing non-human primate (NHP) study resulted in a sustained 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates(Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least six months, demonstrating the same high level of efficacy and durability seen in earlier rodent studies.

The short talk titled, CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema, will be given by Jessica Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when it will be made available here, on the Scientific Publications & Presentations page of Intellias website. The presented data include results from ongoing collaborations with researchers at Regeneron, and the program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent to initiate a Phase 1 trial for NTLA-2002 in the second half of 2021.

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its: planned submission of an investigational new drug (IND) application or similar clinical trial application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020 and its planned dosing of first patients in the second half of 2020; plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; plans to submit an IND or similar clinical trial application for its hereditary angioedema (HAE) program in the second half of 2021; plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program and HAE programs, and other animal studies supporting other in vivo and ex vivo programs, including its AML program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion; further development of its proprietary cell engineering process for multiple sequential editing; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML, and HAE programs, in any future studies, including human clinical trials; ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis or Regeneron Pharmaceuticals, Inc., and Regenerons ability to enter into a co-development and co-promotion agreement for the HAE program; statements regarding the timing of regulatory filings regarding its development programs.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Novartis or Regeneron or its other ex vivo collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917 lynnea.olivarez@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Reports Progress on CRISPR/Cas9 AML Cancer Therapy Using Proprietary Cell Engineering Process at the 23rd Annual Meeting of the...

Orgenesis sees 1Q revenue rocket driven by its Cell and Gene Therapy Biotech platform – Proactive Investors USA & Canada

For its first quarter ended March 31, 2020, the Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million a year earlier

Inc (), a global biotech company focused on accelerating and transforming the delivery of cell and gene therapies, posted first-quarter results on Monday that saw its revenue soar 348% year-over-year driven by its Cell and Gene Therapy (CGT) Biotech platform.

For its first quarter ended March 31, 2020, the Germantown, Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million in the first quarter of 2019.

Orgenesis achieved net income of $75.6 million, or $4.23 per share, reflecting the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO).

READ:Orgenesis boss Vered Caplan makes top 20 list of inspirational leaders in advanced medicine

On February 11, Orgenesis completed the successful sale of its CDMO business to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

As a result, Orgenesis reported cash and equivalents of $107.1 million as of March 31, 2020.

In a statement accompanying the numbers, Orgenesis CEO Vered Caplan said: Step by step, our CGT Biotech Platform is gaining traction within the market, as illustrated by the year-over-year growth.

In the first quarter of 2020, revenue increased to $1.9 million, or nearly an $8 million revenue run rate compared to $3.1 million for all of 2019. We believe our CGT Biotech Platform is poised for growth this year through industry partnerships that are currently underway with leading research institutes and hospitals around the world, she added.

The companys CGT Biotech platform consists of three core elements:point-of care Therapeutics, point-of care Technologies, and point-of care Network.

Caplan also noted that earlier this year, the company struck collaboration agreements with two leading healthcare research institutes in the US.

We plan to utilize our point-of-care Network to support their growing development and processing needs in order to advance and accelerate cell and gene-based clinical therapeutic research, said Caplan.

Orgenesis is using the Masthercell sale proceeds to expand the companys point-of-care cell therapy business. The biotech is currently focused on therapies which span a wide range of treatments.

In addition to our POCare Network, we are building our pipeline of POCare Therapeutics and Technologies, with an ultimate goal of providing life-changing treatments to large numbers of patients at reduced costs within the point-of-care setting, said Caplan.

Specifically, we are focusing on immune-oncology, metabolic and autoimmune diseases, as well as anti-viral therapies.

Orgenesis also recently completed the acquisition of Tamir Biotechnology and its broad-spectrum antiviral platform, ranpirnase in a cash and stock deal for roughly $21 million. The company will use ranpirnase to target human papillomavirus (HPV), which causes genital warts.

Ranpirnase has demonstrated clinical efficacy against HPV and other hard to target viruses based on its unique mechanism of action of killing the virus and modulating the immune system, said Caplan.

Going forward, Orgenesis plans to move the program through a Phase 2b trial in the US.

Meanwhile, the Orgenesis boss said the company has received a nod from regulators to keep research alive at its labs during the coronavirus (COVID-19) pandemic.

We are leveraging all our knowledge and expertise in the field of cell and gene therapy, including anti-viral technologies, in an attempt to find potential COVID-19 cures and therapies, said Caplan.

Importantly, we have a strong balance sheet and are strategically positioned to bring a variety of therapies to market in a cost-effective, high-quality and scalable manner.

At the start of April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous CGT Biotech platform to advance clinical trials.

Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and various other cellular products.

Our plan is to combine RevaTis patented technique to obtain mdMSCs through a minimally invasive muscle micro-biopsy with our own automated/closed-systems, 3D printing, and bioreactor technologies, said Caplan.

The goal of this JV is to lower the costs and accelerate the timeline of bringing these innovative therapies through the clinic and into commercialization.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Orgenesis sees 1Q revenue rocket driven by its Cell and Gene Therapy Biotech platform - Proactive Investors USA & Canada

Vasomune Therapeutics Announces Clinical and Scientific Advisory Board – BioSpace

TORONTO--(BUSINESS WIRE)-- Vasomune Therapeutics, Inc., a therapeutic biopharmaceutical company developing a novel first-in-class medicine for the treatment of COVID-19 pneumonia and pathogen-induced ARDS, today announced the formation of a Clinical and Scientific Advisory Board. The Board is composed of key opinion leaders in their respective fields of medicine and translational therapeutic development.

We are very excited to be working with this esteemed group of medical and scientific advisors as we transition to the clinic with our novel therapeutic approach to COVID-19 that involves targeting the vasculature, said Douglas A. Hamilton, President and Chief Executive Officer, Vasomune.

Members of the Vasomune Clinical and Scientific Advisory Board are:

David Andrews, PhD Dr. Andrews is Director of and Senior Scientist in Biological Sciences at Sunnybrook Research Institute, Professor of Biochemistry and Medical Biophysics at University of Toronto and a Tier 1 Canada Research Chair in Membrane Biogenesis. His research areas include regulation of apoptosis at mitochondrial level, high throughput and image-based high-content screening using automated imaging and analysis of cells in monolayer and 3D cultures, gene knockdown and screening of libraries of small molecules. Dr. Andrews participated in the start-up of MBI Fermentas and Isogenica, and his group performs collaborative and contract research for a variety of biotech companies. He holds licensed patents in regulation of translation and in vitro evolution.

Carolyn S. Calfee, MD, MAS Dr. Calfee, MD, MAS is Professor of Medicine and Anesthesia at the University of California, San Francisco (UCSF), where she attends intensive care units. She completed her undergraduate studies at Yale University and medical school at the University of Pennsylvania before moving to UCSF for her residency, chief residency and fellowship training, as well as her Masters degree in Clinical Research. Her primary academic focus is the pathogenesis and treatment of acute respiratory distress syndrome (ARDS). Her main research interests include molecular subphenotypes of ARDS and precision medicine in critical care; the role of environmental exposure, including smoking, air pollution and novel tobacco products in susceptibility to lung injury; and novel treatments for ARDS.

Eddie Fan, PhD, MD Dr. Fan is an Associate Professor in the Interdepartmental Division of Critical Care Medicine and the Institute of Health Policy, Management and Evaluation at the University of Toronto and a Staff Intensivist at the University Health Network/Mount Sinai Hospital. He is also currently the Medical Director of the Extracorporeal Life Support Program at Toronto General Hospital. Dr. Fans research has focused on advanced life support for acute respiratory failure and patient outcomes from critical illness. Dr. Fan obtained his undergraduate degree from the University of Toronto, his medical degree from the University of Western Ontario and a PhD in Clinical Investigation from Johns Hopkins University.

Michael Julius, PhD Dr. Julius is a professor emeritus in the Department of Immunology at the University of Toronto. His four decades of research experience at Stanford University, the Basel Institute for Immunology, the Institute Pasteur, McGill University and the University of Toronto, where he chaired the Department of Immunology, is dedicated to understanding the biochemistry and genetics of lymphocyte activation. Dr Julius has in-depth knowledge of multiple therapeutic areas, including neuroscience, cancer, cardiovascular and immune system; and expertise across multiple platforms, including high-content cellular analyses, artificial intelligence, health informatics, and imaging-guided interventions and therapeutics. Most recently, Dr. Julius was recruited to the position of Vice President, Research, at Sunnybrook Health Sciences Centre where he created an international hub for life sciences dedicated to both discovery and commercialization with an annual budget of $125M. This initiative achieved a functional integration of researchers, clinicians, business and patients towards moving discoveries into the clinic; and has spun-off 15 startup companies. Dr. Julius received his BSc at McGill University and his PhD at Stanford University, and has authored 230 publications.

Dana McClintock, MD Dr. McClintock is the Chief Medical Officer of Alladapt Immunotherapeutics, Inc., a role she has held since 2018. Prior to joining Alladapt, Dr. McClintock was Global Head of Innovation for Immunology, Infectious Disease and Ophthalmology, at Genentech/Roche. Prior to this position, Dr. McClintock held roles of increasing responsibility at Genentech/Roche, including Global Head of Pipeline and Portfolio Planning for Immunology, Infectious Disease and Ophthalmology, as well as Interim Global Co-Head of Ophthalmology. Earlier in Dr. McClintocks pharmaceutical career, she was deeply involved in respiratory, immunology and ophthalmology clinical development activities across a range of phases, from IND-enabling work and early clinical trials through post-marketing commitments and medical affairs activities. Dr. McClintock was involved in key clinical development activities for omalizumab (Xolair) and ranibizumab (Lucentis) as along with other pipeline molecules. Prior to joining the pharmaceutical industry, Dr. McClintocks academic research focus was in ARDS, with publications evaluating ventilator parameters as well plasma biomarkers of epithelial and endothelial cell injury. Dr. McClintock received a BA in Chemistry from Duke University and an MD from the University of Virginia. Dr. McClintock completed her training in Internal Medicine and Pulmonary and Critical Care Medicine, both at the UCSF.

Renato Skerlj, PhD Dr. Skerlj has 25 years of experience leading the discovery and development of small molecule drugs to treat rare diseases, cancer, infection and neurodegenerative diseases, and deep scientific expertise in the research and development of innovative, genetically-targeted treatments. Currently, he is Senior Vice President of Research and Development at X4 Pharmaceuticals in Cambridge, MA, and previously held drug discovery and development leadership roles at Cambridge-based Lysosomal Therapeutics, Inc. Prior to that, he was interim Head of Small Molecule Discovery at Genzyme, and was part of the executive team at AnorMED, a publicly-traded company that was acquired by Genzyme in 2006. Dr. Skerlj is an inventor of both plerixafor, a stem cell mobilizer approved by the U.S. Food and Drug Administration (FDA) in 2008, and ertapenem, an anti-bacterial approved by the FDA in 2001, and has been responsible for delivering multiple drug candidates into early clinical research. He has authored 65 publications and holds 50 patents. Dr. Skerlj received his PhD in Synthetic Organic Chemistry from the University of British Columbia and completed postdoctoral fellowships at the University of Oxford and Ohio State University.

About AV-001

AV-001 is an investigational medicine designed to activate the Tie2 receptor and restore normal barrier defense in the vasculature. Following trauma or infection, the bodys host vascular response can become unchecked, leading to vascular leak and ultimately organ failure and death. Vasomune is developing AV-001 for the treatment of COVID-19 pneumonia and pathogen-induced moderate-to-severe ARDS. ARDS is a life-threatening condition that can develop after pneumonia, trauma, shock and sepsis, and is also the leading cause of death for patients infected with COVID-19. Prior to the onset of the coronavirus pandemic, the combined annual incidence of ARDS was approximately 370,000 patients per year in the US and EU with an average mortality rate of 40%. At present, there are no effective therapeutics to treat ARDS.

About Vasomune Therapeutics

Vasomune Therapeutics is a private early stage biopharmaceutical company developing the next generation of medicines to harness the bodys ability to defend against illness. The company is transitioning in the near term to the clinic with a novel therapeutic approach to ARDS that involves targeting a signaling molecule in the vasculature responsible for regulating barrier defense. Vascular dysfunction is associated with the pathology of several disease states, including COVID-19 pneumonia, acute respiratory distress syndrome (ARDS), acute lung injury (ALI), acute kidney injury (AKI), hemorrhagic shock, sepsis and stroke. Vasomunes head office and laboratory is located in Toronto, Canada with US offices in San Mateo, CA. For more information about the company and its product candidates, please visit http://www.vasomune.com or email the President and CEO of Vasomune at dhamilton@vasomune.com.

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Vasomune Therapeutics Announces Clinical and Scientific Advisory Board - BioSpace

Orgenesis First Quarter 2020 Revenue Increases 348% to $1.9 Million Reflecting Success of CGT Biotech Platform – GlobeNewswire

GERMANTOWN, Md., May 11, 2020 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS) (Orgenesis or the Company), a pioneering, global biotech company committed to accelerating commercialization and transforming the delivery of cell and gene therapies (CGTs) while lowering costs, today reported financial results for the first quarter ended March 31, 2020.

First Quarter 2020 Financial Highlights:

Vered Caplan, CEO of Orgenesis, commented, Step by step, our CGT Biotech Platform is gaining traction within the market, as illustrated by the year-over-year growth. In the first quarter of 2020, revenue increased to $1.9 million, or nearly an $8 million revenue run rate compared to $3.1 million for all of 2019. We believe our CGT Biotech Platform is poised for growth this year through industry partnerships that are currently underway with leading research institutes and hospitals around the world.

Earlier this year, we entered into a collaboration agreement with two of the leading healthcare research institutes in the U.S., whereby we plan to utilize our POCare Network to support their growing development and processing needs in order to advance and accelerate cell and gene-based clinical therapeutic research. We believe these collaborations with such high-profile institutions in the field of cell and gene therapy further validate the significant value proposition of our platform.

In addition to our POCare Network, we are building our pipeline of POCare Therapeutics and Technologies, with an ultimate goal of providing life-changing treatments to large numbers of patients at reduced costs within the point-of-care setting. Specifically, we are focusing on immuno-oncology, metabolic and autoimmune diseases, as well as anti-viral therapies. Most recently, we completed the acquisition of Tamir Biotechnology, Inc., including ranpirnase, a broad spectrum anti-viral platform. Ranpirnase has demonstrated clinical efficacy against HPV and other hard to target viruses based on its unique mechanism of action of killing the virus and modulating the immune system. Having demonstrated clinical activity against human papillomavirus, as well as preclinical activity against some of the worlds most persistent viral threats, we plan to aggressively pursue a number of complementary approaches with a goal to maximize the potential of ranpirnase.

We have received approval from regulators to continue working in our research and development labs during the current COVID-19 pandemic, and we are leveraging all our knowledge and expertise in the field of cell and gene therapy, including anti-viral technologies, in an attempt to find potential COVID-19 cures and therapies. Importantly, we have a strong balance sheet and are strategically positioned to bring a variety of therapies to market in a cost-effective, high-quality and scalable manner.

We also announced a joint venture agreement with RevaTis S.A. to advance the development of autologous therapies utilizing and banking muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and other cellular products. Our plan is to combine RevaTis patented technique to obtain mdMSCs through a minimally invasive muscle micro-biopsy with our own automated/closed-systems, 3D printing, and bioreactor technologies. The goal of this JV is to lower the costs and accelerate the timeline of bringing these innovative therapies through the clinic and into commercialization.

The Companys complete financial results are available in the Companys Form 10-Q filed with the Securities and Exchange Commission on May 8, 2020 which is available at http://www.sec.gov and on the Companys website.

About Orgenesis

Orgenesis is a pioneering global biotech company which is unlocking the full potential of personalized therapies and closed processing systems through its Cell & Gene Therapy Biotech Platform, with the ultimate aim of providing life changing treatments at the Point of Care to large numbers of patients at low cost. The Platform consists of: (a) POCare Therapeutics, a pipeline of licensed cell and gene therapies (CGTs), and proprietary scientific knowhow; (b) POCare Technologies, a suite of proprietary and in-licensed technologies which are engineered to create customized processing systems for affordable point of care therapies; and (c) POCare Network, a collaborative, international ecosystem of leading research institutes and hospitals committed to clinical development and supply of CGTs at the point of care. By combining science, technologies and a collaborative network, Orgenesis is able to identify the most promising new therapies and provide a pathway for them to reach patients more quickly, more efficiently and at scale, thereby unlocking the power of cell and gene therapy for all. Additional information is available at: http://www.orgenesis.com.

Notice Regarding Forward-Looking Statements

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, the risk that the acquisition of Tamirs assets will not be successfully integrated with our technologies or that the potential benefits of the acquisition will not be realized, our ability to further develop ranpirnase following the acquisition, our reliance on, and our ability to grow, our point-of-care cell therapy platform, our ability to effectively use the net proceeds from the sale of Masthercell, our ability to achieve and maintain overall profitability, the development of our POCare strategy, the sufficiency of working capital to realize our business plans, the development of our trans-differentiation technology as therapeutic treatment for diabetes which could, if successful, be a cure for Type 1 Diabetes; our technology not functioning as expected; our ability to retain key employees; our ability to satisfy the rigorous regulatory requirements for new procedures; our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31 2019, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

Contact for Orgenesis:David WaldmanCrescendo Communications, LLCTel: 212-671-1021ORGS@crescendo-ir.com

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Orgenesis First Quarter 2020 Revenue Increases 348% to $1.9 Million Reflecting Success of CGT Biotech Platform - GlobeNewswire

Research Antibodies Market – Insights into how contours of market will change in coming years – BioSpace

The global research antibodies market is predicted to be a progressive one over the forecast period. The exceptional physiological properties of antibodies make them sought-after for cell research. Antibodies have the ability to bind to specific molecules, which allows molecules of interest to be isolated for cell research. This is a key factor for continual research to study the anatomy and physiology of antibodies.

The report serves readers to gauge prevailing trends and future growth opportunities in the global research antibodies market. It comprises the most relevant data pertaining to market dynamics. A detailed analysis of the competitive landscape is a key feature of the report.

Global Research Antibodies Market: Key Trends

At present, the number of R&D activities carried out by pharmaceutical and biopharmaceutical companies to introduce novel products is on the rise. These pursuits involve the use of antibodies that exhibit exceptional physiological properties. This, in turn is boosting research on antibodies for their use for secondary cell research.

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Hefty investments from pharmaceutical giants for advancement of antibodies research is also boosting the research antibodies market.

Furthermore, increasing incidence of cancer and other chronic diseases is encouraging research organizations and pharmaceutical and biotechnology companies to develop advanced therapeutics and personalized medicine. The use of antibodies for early detection of cancer is also increasingly recognized. These factors collectively are stimulating the research antibodies market.

Besides this, increasing public and private initiatives and adequate funds for stem cell research are aiding the growth of research antibodies market.

Global Research Antibodies Market: Market Potential

In a new development, the FDA has approved the clinical use of DNA-encoded monoclonal antibody (DMAb) therapy to prevent Zika virus infection. Unlike conventional therapeutic antibodies manufactured in industrial units, DMAb employed for the therapy is made inside the body. Patients are administered DNA instructions for the body to equip itself with necessary tools, and make its own highly specific antibodies to fight infection. The antibodies produced fight pathogenic targets such as virus-infected cells, bacteria, and cancer cells.

DMAb therapy is bringing about a clinical change in antibody therapy. Over the recent past, detailed preclinical studies using DMAb have been conducted, resulting in successful in vivo production of DMAb. The new approach displays extensive potential for major advancement over traditional monoclonal antibody therapy. Further, DMAb therapy could broaden the spectrum of antibody therapeutics, thereby opening new patient markets for antibody-based therapies for disease prevention or treatment.

Global Research Antibodies Market: Regional Outlook

North America holds supremacy among the key regions for research antibodies globally. Presence of a large number of biopharmaceutical and biotechnology companies coupled with immense government support for research are key factors behind the growth of North America research antibodies market. In addition, manufacturers of antibodies need to adhere to stringent guidelines for high quality products. This provides enhance reliability for researchers and clinicians involved in antibodies research and secondary cell research.

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Asia Pacific is expected to display remarkable growth in the research antibodies market over the forecast period. Increasing R&D and adoption of novel techniques for antibodies production is serving to boost this region. Furthermore, improving healthcare infrastructure and implementation of stringent quality adherence policies by public organizations in emerging economies is spurring growth of Asia Pacific research antibodies market.

Global Research Antibodies Market: Competitive Landscape

Some key companies operating in the global research antibodies market are Abcam PLC, Thermo Fisher Scientific Inc., and Agilent Technologies. Top companies in the market are adopting novel growth strategies to sustain the highly competitive environment.

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Research Antibodies Market - Insights into how contours of market will change in coming years - BioSpace

Atara Biotherapeutics Announces Appointment of Cell & Gene Therapy Expert Maria Grazia Roncarolo, MD to Board of Directors – Business Wire

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune diseases, today announced the appointment of immunology and cell & gene therapy expert Maria Grazia Roncarolo, MD, to the Board of Directors.

Dr. Roncarolo is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and Medicine, Director of the Center for Definitive and Curative Medicine, and Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine at Stanford University.

In 2014, Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine. The center, which is dedicated to the development of innovative stem cell and gene therapies for patients with currently incurable diseases, spans a wide range of bench and clinical research activities from basic biology through translational research and features its own GMP cell processing and Phase 1 study units.

I am thrilled to have one of the worlds leading experts in immunology and T cells, Dr. Roncarolo, bringing to our Board her experience and strategic vision in cell therapy and gene editing as well as her passion for transformative immunotherapies, said Pascal Touchon, President and Chief Executive Officer of Atara. She has dedicated her life to caring for patients with severe immunological and hematological diseases and has an impressive record in translating scientific discoveries in cell and gene therapy into novel treatments which aligns very well with Ataras mission.

Dr. Roncarolo has served as the primary investigator in several landmark trials involving the development of innovative stem cell- and gene-based therapies. She worked at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto for several years, where she contributed to the discovery of novel cytokines, cell-signaling molecules that are part of the immune response. She studied the role of these cytokines in inducing immunological tolerance and in promoting stem cell growth and differentiation. As Director of the Telethon Institute for Cell and Gene Therapy and the San Raffaele Scientific Institute in Milan, Dr. Roncarolo was the principal investigator leading the successful gene therapy trial in SCID, a severe life threatening disorder in which patients lack an enzyme critical to DNA synthesis.

Beyond studying new therapies, Dr. Roncarolo has also helped elucidate drivers of disease at the molecular and cellular level, as she has investigated the mechanisms of immune-mediated diseases throughout her career and helped advance the understanding of immunological tolerance. Dr. Roncarolo was the recipient of the outstanding achievement award from the European Society of Gene and Cell Therapy (ESGCT) in 2010 and from the American Society of Gene and Cell Therapy (ASGCT) in 2017. She is currently the president of the Federation of Clinical Immunology Societies.

About Atara Biotherapeutics

Atara Biotherapeutics, Inc. (@Atarabio) is a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune disease. With our lead program in Phase 3 clinical development, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other severe diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform to create a robust pipeline including: tab-cel (tabelecleucel) in Phase 3 development for Epstein-Barr virus-driven post-transplant lymphoproliferative disease (EBV+ PTLD); ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell (CAR T) immunotherapies for both solid tumors and hematologic malignancies. Improving patients lives is our mission and we will never stop working to bring transformative therapies to those in need. Atara is headquartered in South San Francisco and our leading-edge research, development and manufacturing facility is based in Thousand Oaks, California. For additional information about the company, please visit atarabio.com and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Because such statements deal with future events and are based on Atara Biotherapeutics' current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara Biotherapeutics could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including those discussed in Atara Biotherapeutics' filings with the Securities and Exchange Commission (SEC), including in the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of the Companys most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara Biotherapeutics disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

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Atara Biotherapeutics Announces Appointment of Cell & Gene Therapy Expert Maria Grazia Roncarolo, MD to Board of Directors - Business Wire

CBG Called the ‘Stem Cell’ Of Cannabinoids and Could be More Profitable Than CBD – PRNewswire

PALM BEACH, Florida, May 11, 2020 /PRNewswire/ --The CBD market surge in the last few years has been well documented and all reports seem to project continued growth of revenues over the coming years.CNNsaid,"These hemp farmers are getting rich with CBD."For farmers, growing hemp is a booming business. And it's much more profitable than growing corn or soybeans. In the U.S., sales could surge from $591 million last year . . . to $22 billion within just three years." An articlefrom Wyatt Research added: "Today, CBD is getting most of the attention. For growers, the next generation of hemp will be even more profitable. It's called CBG. It's the precursor cannabinoid to both CBD and THC. And it's currently being researched for a wide range of uses including psoriasis, eczema and as a cosmetic additive. CBG is essentially the next type of cannabinoid that will be researched, developed and put into thousands of mainstream products. The article continued projecting that hemp growers will be getting into CBG: "Today, production of CBG is very low. And that means prices for CBG are five times higher than CBD! Consider these numbers: One acre of farmland planted for CBD will generate $61,380 in revenue. Yet that same acre planted with hemp for CBG will earn $306,900!". Active cannabis companies in the markets this week include: SponsorsOne Inc. (OTCPK: SPONF) (CSE: SPO), The Supreme Cannabis Company, Inc. (OTCQX: SPRWF) (TSX: FIRE), Cresco Labs Inc. (OTCQB: CRLBF) (CSE: CL), Aurora Cannabis Inc. (NYSE: ACB) (TSX: ACB), Aphria Inc. (NYSE: APHA) (TSX: APHA).

An articlein The Green Fund echoed those reports saying: "CBD or Cannabidiol, has taken the world by storm over the last few years, thanks to the introduction of the US Farm Bill in 2018. CBD has appeared in every product from coffee to cocktails, to even pet treats and beauty products. However, CBD isn't the only cannabinoid in town it's time to learn about CBG." The Green Fund commentary continued: "Although CBG is just hitting the markets, it was actually first discovered by researchers in the 1960's. CBG is the main element that the other cannabinoids are created from, hence being commonly referred to as the "stem cell."

SponsorsOne Inc. (OTCPK: SPONF) (CSE: SPO) BREAKING NEWS:SponsorsOne Introduces Oral Dissolving CBG Tablet - Company Adds Another CBG Product To Its GO Nutraceutical Branded Line Of "Cannabis 2.0" Products - SponsorsOne, acompany that utilizes its proprietary platform that combines digital marketing, wholesale and retail distribution, branding and operational & funding capital, giving it a competitive first mover advantage in rapidly building, launching and selling proprietary brands focused in the craft Alcohol, Functional Beverage and Cannabis Sectors, today announced that It has introduced another innovative CBG product, under the marketing management agreement with GO Nutraceutical's branded line of "Cannabis 2.0" products.

The company, which recently launched a line of flavored, chewable tablets delivering a 20mg dose of CBG, has now added a CBG sublingual dissolving tablet that delivers 10mg in a very small 6mm size also produced by GO Nutraceuticals. Sublingual dissolving tablets are placed under the tongue triggering much higher bioavailabilitywhich results in faster absorption into the body.

As with the chewable product, this will be sold direct to consumer through GO Nutraceuticals' online store and through SPO's wholesale and retail distribution channel and the company's rapidly expanding influencer network.

Myles Bartholomew, CEO of SponsorsOne, said: "Oral dissolving CBG tablets are one of the more unique offerings in our solid oral dose line. At 10mg and only 6mm round, the tiny size tablet melts perfectly in the mouth without the need for digestion. Oral dissolving tablets are new to the cannabis industry, but they are showing great potential. The product is easy and enjoyable to consume, the deliveries are small, and it tastes great. But it is not only our delivery system that is the key here, it is fact that we are utilizing CBG, which as we have said before, has been shown to possess enormous therapeutic promise, and has been called theholy grail of cannabinoids.We have taken the knowledge of the pharmaceutical industry and applied it to natural extracts to create the next innovative wave of cannabis deliveries. We believe this will help us separate the company from others in the market and will increase revenues and bring value to our shareholders." Read this and more news for SponsorsOne at: https://www.financialnewsmedia.com/news-spo/

Other recent developments in the cannabis/cbd/hemp industries:

The Supreme Cannabis Company, Inc. (OTCQX: SPRWF) (TSX: FIRE) recently announced the completion of its first international cannabis shipment fromCanadaintoIsrael. Supreme Cannabis partnered with Breath of Life International Ltd.("BOL Pharma"),Israel'slargest and leading producer of medical cannabis and cannabis products, to offer Truverra-branded premium medical cannabis to patients inIsrael.

"This transaction represents a new international revenue opportunity for Supreme Cannabis and builds Truverra's global medical brand in one of the most sophisticated medical cannabis markets in the world," saidColin Moore, Interim President and CEO of Supreme Cannabis. "With the support of BOL Pharma, we navigated bothCanadaandIsrael'scomplex regulatory landscapes to achieve compliance with regulatory authorities in both countries, including Health Canada, the Canadian Food Inspection Agency andIsrael'sMinistry of Health and Ministry of Agriculture and Rural Development. We benefitted from BOL Pharma's skilled team, international experience and solidified position as a leading medical cannabis company inIsrael. As we pursue capital-light international opportunities, we will continue to look toJeff Adams, Truverra's CEO, and his exceptional team to build Truverra's medical brand globally."

Cresco Labs Inc. (OTCQB: CRLBF) (CSE: CL) one of the largest vertically integrated multistate cannabis operators in the United States, recently announced that it has completed the expansion project for its cultivation and manufacturing facility located in Brookville, PA. The expansion project provides an additional 66,000 square feet of indoor and greenhouse cultivation area, bringing the total cultivation space1in the facility to 88,000 square feet. The Brookville site supplies Cresco's house of brands to 100% of the licensed dispensaries in Pennsylvania and the Company currently maintains one of the largest wholesale market shares.

The Company also concluded a significant expansion of its manufacturing capabilities with new and advanced extraction booths and integrated safety systems. The expansion project provides a 300% increase in capacity for butane extraction and a 90% increase in ethanol extraction which enables Cresco to efficiently process its increased biomass to manufacture its full portfolio of brands, including Cresco, Remedi and Reserve, with new brands launching soon. As a result of tripling the site's capacity in a phased approach, the Company expects products from the third, 22,000-square-foot phase to enter the market this month, with production from the fourth, 22,000-square-foot phase starting in July 2020 and gradually increasing throughout the remainder of the year.

Aurora Cannabis Inc. (NYSE: ACB) (TSX: ACB) the Canadian company defining the future of cannabis worldwide, recently provided an update related to its balance sheet flexibility, business transformation initiatives and COVID-19 operational response plans.

As ofMarch 31, 2020, the Company had approximately$205 millionof cash. This includes all amounts raised under the existing, and now completed,US$400 millionAt-the-Market Offering program ("ATM"), initially announced inMay 2019. To support the strength of the Company's balance sheet and provide continued access to equity capital, the Company today stated that it intends to file a new prospectus supplement for a renewed ATM program, to enable Aurora to raise additional equity capital pursuant to its outstanding base shelf prospectus datedMay 14, 2019under which approximatelyUS$350 millionremains available. The Company intends to use a portion of this available capacity to provide further balance sheet strength and preserve flexibility given macroeconomic uncertainty caused by COVID-19.

Aphria Inc. (NYSE: APHA) (TSX: APHA) recently announced that its subsidiary, ASG Pharma Ltd. ("ASG"), has received its European Union Good Manufacturing Practices ("EU GMP") certification from the Malta Medicines Authority("MMA") in respect of production of cannabis for medicinal and research purposes. Following receipt of its first medical cannabis import license for analytical testing and research in 2018 and the subsequent ASG facility upgrade, the certification provides Aphria with the ability to ship finished dried flower and finished oil for medicinal and research use in permitted jurisdictions throughout the European Union.

"We are pleased to receive EU GMP certification for ASG inMalta, our third facility to achieve this milestone, which really speaks to the Company's commitment to quality," saidIrwin D. Simon, Chief Executive Officer. "We remain excited about growth opportunities as this increases our ability to serve, and further strengthens Aphria's leadership, in the European Union."

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates FinancialNewsMedia.com and MarketNewsUpdates.com, is a third party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM has been compensated forty six hundred dollars for news coverage of the current press releases issued by SponsorsOne, Inc. by a non-affiliated. FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

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CBG Called the 'Stem Cell' Of Cannabinoids and Could be More Profitable Than CBD - PRNewswire

Were All Casualties of Trumps War on Coronavirus Science – The New York Times

In 2004, 60 Minutes aired a segment on what it called virus hunters, scientists searching for bugs that can leap from animals to humans and cause pandemics. What worries me the most is that we are going to miss the next emerging disease, said a scientist named Peter Daszak, describing his fear of a coronavirus that moves from one part of the planet to another, wiping out people as it moves along.

In the intervening years, Daszak became president of the EcoHealth Alliance, a nonprofit research organization focused on emerging pandemics. EcoHealth worked with Chinas Wuhan Institute of Virology to study coronaviruses in bats that could infect humans, and, as Science magazine put it, to develop tools that could help researchers create diagnostics, treatments and vaccines for human outbreaks. Since 2014, the EcoHealth Alliance has received a grant from the National Institutes of Health, until its funding was abruptly cut two weeks ago.

The reason, as 60 Minutes reported on Sunday evening, was a conspiracy theory spread by Representative Matt Gaetz, the Florida Republican who in March wore a gas mask on the House floor to mock concern about the new coronavirus. On April 14, Gaetz appeared on Tucker Carlsons Fox News show and claimed that the N.I.H. grant went to the Wuhan Institute, which Gaetz intimated might have been the source of the virus the institute may have birthed a monster, in his words.

The first of Gaetzs claims was flatly false, and the second unlikely; the C.I.A. has reportedly found no evidence of a link between the virus and the Wuhan lab. But at a White House briefing a few days later, a reporter from the right-wing website Newsmax told President Trump that under Barack Obama, the N.I.H. gave the Wuhan lab a $3.7 million grant. Why would the U.S. give a grant like that to China? she asked.

In fact, Trumps administration had recently renewed EcoHealths grant, but Trump didnt appear to know that. The Obama administration gave them a grant of $3.7 million? he asked. Then he said, We will end that grant very quickly.

And they did. But ending the grant dealt a blow to efforts to find treatments and a vaccine for the coronavirus. Remdesivir, the antiviral drug thats shown some promise in Covid-19 patients, was earlier tested against bat viruses EcoHealth discovered. Now the nonprofit is facing layoffs.

This political hit on Daszaks work is far from the only way that the Trump administrations contempt for science has undermined Americas coronavirus response. Conservative antipathy to science is nothing new; Republicans have long denied and denigrated the scientific consensus on issues from evolution to stem cell research to climate change. This hostility has several causes, including populist distrust of experts, religious rejection of information that undermines biblical literalism and efforts by giant corporations to evade regulation.

But its grown worse under Trump, with his authoritarian impulse to quash any facts, from inauguration crowd sizes to hurricane paths, that might reflect poorly on him.

Until recently, it seemed as if Trumps sabotage of efforts to combat climate change would be the most destructive legacy of his disregard for science. But the coronavirus has presented the country with an emergency that only sound science can solve. That means that the Trump administrations disdain for expertise, its elevation of slavish loyalty over technical competence, has become a more immediate threat.

Months before this pandemic began, Reuters reported, the Trump administration axed the job of an epidemiologist working for the Centers for Disease Control and Prevention in China to help detect emerging disease outbreaks. As the pandemic raged, the administration removed Rick Bright, one of Americas premier experts on vaccine development, from an agency overseeing efforts to develop a coronavirus vaccine. Last week Bright filed a whistle-blower complaint claiming hed suffered retaliation because he resisted funding potentially dangerous drugs promoted by those with political connections and by the administration itself. (A federal watchdog agency has called for him to be reinstated pending its investigation.)

Another whistle-blower complaint, filed by a former volunteer on the coronavirus team assembled by Trumps son-in-law, Jared Kushner, claims the effort has been beset by inexperience and incompetence. The Associated Press reported on how the White House buried guidance from the C.D.C. on how communities could safely reopen. Now the president is urging Americans to return to work even as the White House itself has proved unable to keep the coronavirus at bay.

According to Axios, Trump has even privately started expressing skepticism of the coronaviruss death toll, suggesting its lower than official statistics say. (Most experts believe the opposite.) A senior administration official said he expects the president to begin publicly questioning the death toll as it closes in on his predictions for the final death count and damages him politically, reported Axios. The Trump administrations approach to the coronavirus began with denialism, and thats likely how it will end.

Any progress America makes in fighting Covid-19 will be in spite of its federal government, not because of it. I am speaking out because to combat this deadly virus, science not politics or cronyism has to lead the way, Dr. Bright said when he went public with his complaint in April. Trump wont let that happen. Hed rather essentially give up on combating it at all.

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Were All Casualties of Trumps War on Coronavirus Science - The New York Times

Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd Annual Meeting of the American Society of Gene and…

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, today presents new clinical data supporting longer-term efficacy and durability of gene therapy for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held virtually May 12-15, 2020. Two oral presentations highlight updates from the companys Phase 1/2 study of RP-L201 for the treatment of severe LAD-I and the Phase 1/2 study of RP-L102 Process A for the treatment of FA.

The latest additional data from both our LAD-I and FA programs demonstrate sustained engraftment and durable clinical impact, said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. These results further support the viability of gene therapy in LAD-I and FA, disorders in which bone marrow transplant is the primary curative option and is associated with high rates of toxicity.

Patients with severe LAD-I have neutrophil CD18 expression of less than 2% of normal, with extremely high mortality in early childhood, said Dr. Schwartz. In this first patient treated with RP-L201 using Process B, an increase from less than 1% to 47% CD18 expression sustained over six months demonstrates that RP-L201 has the potential to correct the neutrophil deficiency that is the hallmark of LAD-I. We are also pleased with the continued visible improvement of multiple disease-related skin lesions. These results lend further support to the applicability of Process B across the lentiviral portfolio. The second patient has also recently been treated, and we look forward to completing the Phase 1 portion of the registrational trial for this program.

Dr. Schwartz continued, In our FA program, patients followed for a year or more after treatment with RP-L102 Process A continue to demonstrate durable engraftment and hematologic correction, without the use of pre-treatment conditioning regimens. All six patients who received minimally adequate drug product and were followed for more than one year display sustained and progressive engraftment. Notably, hemoglobin levels have normalized to baseline in two patients treated. Todays update not only gives us confidence as we transition to our improved Process B drug product, but also supports the potential of gene therapy in the absence of any conditioning regimen as a definitive hematologic treatment for FA. The ability to treat patients without the side effects associated with allogeneic transplant or the use of genotoxic conditioning, and to restore blood cell counts is a major milestone for the FA scientific community.

Details on Rockets oral presentations at ASGCT:

Title: A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Initial Results from the First Treated Patient Session: HSPC Gene Therapies for Blood and Immune DisordersPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and principal investigator of the Phase 1 trialDate: Tuesday May 12, 2020Time: 4:30 p.m. - 4:45 p.m. EDT

Additional results from the first patient treated with RP-L201 for LAD-I continue to demonstrate evidence of safety and potential efficacy. Analyses of peripheral vector copy number (VCN) and CD18-expressing neutrophils were performed six months post treatment with RP-L201 to evaluate engraftment and phenotypic correction. The patient demonstrated peripheral blood VCN levels of 1.3 and CD18-expression of 47%, which is sustained from the 45% expression observed three months post treatment; pretreatment CD18 expression was <1%. The drug product VCN was 3.8. Additionally, the patient continues to display visible improvement of skin lesions. No safety or tolerability issues related to RP-L201 administration have been identified to date.

RP-L201 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria Fundacin Jimnez Daz (IIS-FJD). The lentiviral vector was developed in a collaboration between The University College of London (UCL) and CIEMAT.

Title: Updated Results of a European Gene Therapy Trial in Fanconi Anemia Patients, Subtype ASession: HSPC Gene Therapies for Blood and Immune DisordersPresenter: Juan A. Bueren, Ph.D., Scientific Director of the FA gene therapy program and Head of the Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBERER / IIS-FJD Date: Tuesday May 12, 2020Time: 4:45 p.m. - 5:00 p.m. EDT

Nine pediatric patients have been enrolled and treated in the Phase 1/2 clinical trial of RP-L102 Process A for the treatment of Fanconi Anemia, seven of whom are evaluable at or beyond the one year mark following treatment. The first four patients (02002, 02004, 02005 and 02006) exhibit robust and durable engraftment, continued hematologic correction and blood count stabilization. Importantly, hemoglobin levels for patients 02002 and 02006 have increased to a healthy, normal range; these patients received more optimal product consistent with the minimal dose criteria established for the Process B registrational program. Two additional patients (02008 and 02013) who have been followed for a year or more after treatment display early evidence of engraftment, as measured by increases in peripheral blood VCNs. Patient 02007 received a lower than optimal dose and is beginning to demonstrate preliminary signs of engraftment. Blood counts are not yet available in these patients. Two patients, patients 01003 and 02009, have not been included in this analysis. Patient 02009 is only six months post treatment and will continue to be followed. Patient 01003 received a drug product that did not meet full release criteria due to a technical issue this was a one-time lab-specific issue that was addressed. To date, no patients in this trial have undergone allogeneic bone marrow transplant.

RP-L102 is being developed in conjunction with CIEMAT, CIBERER and IIS-FJD.

The presentations will be made available on Rockets website at http://www.rocketpharma.com/asgct-presentations/ following presentation at the conference.

About Leukocyte Adhesion Deficiency-ISevere Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Fanconi AnemiaFanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, filed May 8, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd Annual Meeting of the American Society of Gene and...

Cell Expansion Market : Facts, Figures and Analytical Insights 2020-2026 – Cole of Duty

The report provides a valuable source of insightful data for business strategists and competitive analysis of Cell Expansion Market. It provides the Cell Expansion industry overview with growth analysis and futuristic cost, revenue and many other aspects. The research analysts provide an elaborate description of the value chain and its distributor analysis. This Tire Cell Expansion study provides comprehensive data which enhances the understanding, scope and application of this report.

the cell expansion market is projected to display a robust growth represented by a CAGR of 17.33% during 2019 2024.

A comprehensive research report created through extensive primary research (inputs from industry experts, companies, stakeholders) and secondary research, the report aims to present the analysis of cell expansion market. The report analyses the Global Cell Expansion Market: Analysis By Product Type (Instruments, Consumables, Disposables), By Cell Type (Human Cell and Animal Cell), By Application (Regenerative Medicine & Stem Cell Research and Cancer & Cell Based Research), By Region (North America, Europe, Asia Pacific, South America, and Middle East & Africa), (U.S, Canada, Germany, France, U.K., Japan, China, India): Opportunities and Forecast (2019 Edition): Forecast to 2024, for the historical period of 2018-2019 and the forecast period of 2019-2024.

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https://bit.ly/35chcJP

Prominent Players in the global Cell Expansion market are

Merck Millipore, Eppendorf, ThermoFisher Scientific, Becton Dickinson, Danaher Corporation, Corning Inc., Terumo Medical Corporation, CellGenix Technologie Transfer GmbH, Synthecon Inc., Stem Cell Technologies Inc.

SAN ANTONIO Oct. 8, 2019 Southwest Research Institute has received new funding from the Medical Technology Enterprise Consortium (MTEC) and the Food and Drug Administration (FDA) to use an SwRI-developed bioreactor to propagate cells for personalized regenerative medicine applications. A $3 million MTEC program will demonstrate stem cell manufacturing, and a $2.3 FDA contract is funding CAR T-cell production. Cell and gene therapies hold the promise of revolutionizing treating diseases using a patients own immune or stem cells. But existing technologies dont meet clinical needs for high-quality and cost-effective cell manufacturing. They typically require an expensive, labor-intensive cell culture process in high-cost clean room conditions.

Jun 13, 2013: Merck Millipore has announced the availability of its PluriSTEM Human ES/iPS Medium for the routine expansion of human embryonic and induced pluripotent stem cells in feeder- and serum-free conditions with less frequent feeding and cell culture time.

It is the first human ES/iPS media to combine small molecule inhibitors with pluripotent specific growth factors and supplements to enable a less rigorous cell culture regiment. These features save researchers time and lead to lower costs.

Scope of the Report

-Cell Expansion Market Size, Share & Forecast-Segmental Analysis Standalone and Chained Chained/Organized Cell Expansion Sizing, Growth, Forecast-Market Entry Strategies for Domestic/Foreign Players-Policy & Regulatory Landscape-Changing Market Trends & Emerging Opportunities-Competitive Landscape & Strategic Recommendations

The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.

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What are the market factors that are explained in the report?

Key Strategic Developments:The study also includes the key strategic developments of the market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

Analytical Tools:The Global Cell Expansion Market Report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, SWOT analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

Key Market Features:The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

We Offer Customization on report based on specific client Requirement:

Free country Level analysis for any 5 countries of your choice. Free Competitive analysis of any 5 key market players. Free 40 analyst hours to cover any other data point.

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https://bit.ly/2yPtGLk

How we have factored the effect of Covid-19 in our report:

All the reports that we list have been tracking the impact of COVID-19. Both upstream and downstream of the entire supplychain has been accounted for while doing this. Also, where possible, we will provide an additional COVID-19 update supplement/report to the report in Q3, please check for with the sales team.

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Cell Expansion Market : Facts, Figures and Analytical Insights 2020-2026 - Cole of Duty