Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

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The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

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Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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COVID 19 to Lead the Sales of Myelofibrosis Treatment to Register Stellar Growth in the Next 10 Years - Cole of Duty

Prophecy Market Insights Cell Therapy Manufacturing market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges. The research report study offers keen competitive landscape analysis including key development trends, accurate quantitative and in-depth commentary insights, market dynamics, and key regional development status forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.

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An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Cell Therapy Manufacturing market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.

Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Segmentation Overview:

Cell Therapy ManufacturingMarket Key Companies:

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

The Cell Therapy Manufacturing research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Cell Therapy Manufacturing market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. This report is a must-read for investors, entrepreneurs, consultants, researchers, business strategists, and all those who have any kind of stake or are planning to foray into the Cell Therapy Manufacturing industry in any manner.

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Market Analysis and Technological Opportunities of Cell Therapy Manufacturing Market till 2030 - Medic Insider

BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc.(Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53, today announced the oral presentation of updated data from its French Phase 1b/2 clinical trial at the 25th European Hematology Association Annual Meeting (EHA). The trial is evaluating the safety and efficacy of APR-246 (eprenetapopt) in combination with azacitidine (AZA) for the treatment of TP53 mutant myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). The clinical trial is sponsored by the Groupe Francophone des Mylodysplasies (GFM).

As of the April 1, 2020 data cutoff, the overall response rate (ORR) in 28 evaluable MDS patients was 75%, with a 57% complete remission (CR) rate, by International Working Group (IWG) criteria. With a median duration of follow-up of 9.7 months, the median overall survival (OS) for all enrolled patients (n=52) was 12.1 months and in MDS patients (n=34) was 12.1 months. For patients who remained on treatment for 3 or more cycles of treatment the median OS was higher at 13.7 months versus 2.8 months for patients who were on treatment for fewer than 3 cycles. Relative to baseline, mutant TP53 variant allele frequency (VAF) was decreased in responding patients by 3 cycles of treatment, including 20 (51%) patients who achieved mutant TP53 negativity by next-generation sequencing (NGS).

The data from this ongoing trial of eprenetapopt with azacitidine continue to be very encouraging in these most difficult-to-treat TP53 mutant MDS and AML patients, who not only have at least one TP53 mutation but the majority of whom also have high risk cytogenetic abnormalities, said Thomas Cluzeau, M.D., co-lead investigator for the GFM trial. We continue to observe ORR and CR rates in these patients that are substantially higher than the GFMs experience with azacitidine monotherapy. Furthermore, with increased duration of follow-up, we now also see the emergence of highly encouraging overall survival that appears to be better than azacitidine alone or in combination with others agents in this very high-risk molecular group of patients with a TP53 mutation.

Details of the on-demand oral presentation are as follows:

Title: APR-246 Combined with Azacitidine in TP53 Mutated Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia. A Phase 2 Study by the Groupe Francophone des Mylodysplasies (GFM)

Oral Abstract Session: Novel treatments for MDS I

Abstract: S181

About the Clinical Trial

Eligible patients in the Phase Ib/II clinical trial include hypomethylating agent (HMA) nave, TP53 mutated MDS and AML. All enrolled patients were to receive APR-246 as a 4,500 mg fixed dose IV daily for 4 days and AZA over 7 days in 28-day cycles. The primary endpoint of the trial is CR rate.

AboutAprea Therapeutics, Inc.

Aprea Therapeutics, Inc.is a biopharmaceutical company headquartered inBoston, Massachusettswith research facilities inStockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivatemutant tumor suppressor protein, p53. The Companys lead product candidate is APR-246 (eprenetapopt), a small molecule in clinical development for hematologic malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). APR-246 has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. For more information, please visit the company website atwww.aprea.com.

The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis.

About p53 and APR-246 (eprenetapopt)

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 (eprenetapopt) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein by restoring wild-type p53 conformation and function and thereby induce programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with APR-246 in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with APR-246 has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

Forward-Looking StatementCertain information contained in this press release includes forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as predicts, believes, potential, continue, anticipates, estimates, expects, plans, intends, targeting, confidence, may, could, might, likely, will, should or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with theU.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

Source:Aprea Therapeutics, Inc.

Excerpt from:
Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 (Eprenetapopt) and Azacitidine in Patients with TP53 Mutant...

Bone marrow aspirationand trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific.

Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.

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Bone Marrow Processing Systems Market : Industry Trends and Developments 2018 2025 - Cole of Duty

THE HAGUE, Netherlands, June 12, 2020 /PRNewswire/ -- Treatment of childhood cancer is a success story, particularly for acute lymphoblastic leukemia (ALL). More than 90% of ALL patients below 18 years of age are rescued with contemporary chemotherapy. However, the remaining 10% have resistant or reoccurring leukemia and require alternative treatment regimens. One of the most powerful leukemia therapies is hematopoietic stem cell transplantation from a donor (allogeneic HSCT). Approximately 50-80% of pediatric ALL patients that receive allogeneic HSCT are cured, 20% experience leukemic reoccurrence (relapse), and 10% die from complications.

Allogeneic HSCT is a multistep procedure:

For high-risk leukemia, the gold standard conditioning procedure is a combination of total body irradiation (TBI) and high dose chemotherapy. This approach is very effective in controlling leukemia in the conditioning step, but patients may experience highly negative consequences of this procedure later in life: sterility, growth retardation, lung problems, and secondary cancer.

Therefore, a large consortium of pediatric transplant experts initiated a global study to investigate whether chemotherapy-based conditioning could substitute TBI. The study is called FORUM (For Omitting Radiation Under Majority Age) and had to be stopped because chemotherapy-based conditioning had significantly poorer outcomes (i.e., lower overall survival rates) than the combination of TBI and chemotherapy. The researchers will now perform prospective monitoring to better define the advantages and limitations of various conditioning approaches.

Presenter:Dr Christina PetersAffiliation:Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, AustriaAbstract:#S102 TBI OR CHEMOTHERAPY BASED CONDITIONING FOR CHILDREN AND ADOLESCENTS WITH ALL: A PROSPECTIVE RANDOMIZED MULTICENTER-STUDY "FORUM" ON BEHALF OF THE AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG AND EBMT-PD-WP

Embargo: Please note that our embargo policy applies to all selected abstracts in the Press Briefings. For more information click here.

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SOURCE European Hematology Association (EHA)

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EHA25Virtual: Combined Irradiation and Chemotherapy Better Prepares Children for Stem Cell Transplantation than Chemotherapy Alone - PR Newswire UK

Management to Host One-on-One Investment Meetings

NEW YORK, June 11, 2020 /PRNewswire/ --BrainStorm Cell Therapeutics Inc.(NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced Chaim Lebovits, CEO and Ralph Kern, MD, MHSc, President and Chief Medical Officer, will present a corporate overview on Thursday, June 18 at 9:00 am EST, during theRaymond James Human Health Innovations Conference, a virtual event connecting institutional investors with company management teams that will be held June 15-18, 2020.

Mr. Lebovits and Dr. Kern will update conference participants on the Company's investigational therapeutic, NurOwn, that is currently in a fully enrolled phase 3 study for the treatment of ALS and a phase 2 study for the treatment of progressive multiple sclerosis. Additionally, they will present an overview of the Company's financial position and pipeline. After the presentation, the management team will participate in a question and answer session with institutional investors.

Mr. Lebovits and Dr. Kern will be joined by David Setboun, PhD, MBA, Chief Operating Officer, Stacy Lindborg, PhD, Head of Global Clinical Research, and Preetam Shah, PhD, MBA, Chief Financial Officer, for a series of one-on-one meetings, with select institutional investors arranged by Raymond James.

Participants can view the presentation via the event link and those unable to join will have access to an archived link on the Company's Events and Presentation webpage after the conclusion of the conference.

EVENT: Raymond James Human Health Innovations Conference

PRESENTATION: Thursday, June 18th at 9:00 am EST

LINK: https://bit.ly/2YmZf8u

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently receivedU.S.FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began inMarch 2019.

AboutBrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at sixU.S.sites supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a filing forU.S.FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently receivedU.S.FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment inMarch 2019.

Story continues

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: +1-862-397-1860pshah@brainstorm-cell.com

Media:

Sean LeousWestwicke/ICR PRPhone: +1-646-677-1839sean.leous@icrinc.com

View original content:http://www.prnewswire.com/news-releases/brainstorm-to-present-at-the-raymond-james-human-health-innovations-conference-301074370.html

SOURCE Brainstorm Cell Therapeutics Inc

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BrainStorm to Present at the Raymond James Human Health Innovations Conference - Yahoo Finance

The company said the move was aimed at the development of next generation cellular immunotherapy FLASH-CAR technology

(), a clinical-stage developer of cell-based technologies and therapeutics, announced Thursday that it has struck a three-way material transfer agreement (MTA) with Weill Cornell Medicine in New York City and the companys strategic partner, Arbele Limited.

With this agreement, Avalon GloboCare and Arbele Limited intend to collaborate with Weill Cornell Medicine and co-develop the standardized laboratory steps necessary to generate clinical-grade CAR-T and CAR-natural killer (NK) cells for use in future human clinical trials with Avalons first FLASH-CAR platform candidate, AVA-011.Similar to T-cells, NK cells are a type of white blood cell, also able to attack cancer cells, but utilize different mechanisms.

The company said this process development step will provide the bridge between Avalons benchtop research and the bio-manufacturing processes to potentially deliver the clinical-grade cellular immunotherapy product to patients.

READ:Avalon GloboCare advancing immune cell therapy to treat blood cancers using FLASH-CAR technology

We are excited about this agreement to translate our cellular therapy candidates into standardized, clinical-grade cell products that could be used in future clinical trials, Avalon GloboCare CEO David Jin said in a statement.

This step reflects our dedication to establishing an infrastructure to develop our cellular immunotherapy candidates and to maintain the highest possible standards for generating clinical-grade cells for human cancer trials, he added.

AVA-011 is a next generation cellular immunotherapy candidate using Avalons FLASH-CAR technology that targets both CD19 and CD22 tumor antigens on cancer cells. Avalon has already successfully completed pre-clinical research on AVA-011, including tumor cytotoxicity studies.

Avalon expects to begin a first-in-human clinical trial with AVA-011 for the treatment of relapsed or refractory B-cell lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma in the first quarter of 2021. The goal is to use AVA-011 as a bridge to bone marrow stem cell transplant therapy, currently the only curative approach for patients with these blood cancers.

Avalons next generation immune cell therapy using FLASH-CAR technology is being co-developed with the companys strategic partner Arbele Limited. The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor, expanding the reach of cancer patients that can be treated.

Avalon, based in Freehold, New Jersey, specializes in developing cell-based technologies and is involved in the management of stem-cell banks and clinical laboratories.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Avalon GloboCare strikes three-way material transfer agreement with Weill Cornell Medicine and Arbele Limited - Proactive Investors USA & Canada

-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia-

-Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-

-Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted-

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany.

CLIMB-111 Trial in Transfusion-Dependent Beta Thalassemia Updated ResultsData presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRISPR Therapeutics and Vertex announced initial data for the first TDT patient in November of 2019.

Patient 1 with TDT has the 0/IVS-I-110 genotype, which is associated with a severe phenotype similar to 0/0, and had a transfusion requirement of 34 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented today show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 100.0%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year.

Patient 2 with TDT has the 0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.4%.

Hemoglobin data over time are presented for Patient 1 and Patient 2 below.

Figure 1accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/35581299-d683-44b0-a75e-7a1a9b9fe9eb

CLIMB-121 Trial in Severe Sickle Cell Disease Updated Results Data presented today at EHA reflect longer-duration follow-up data for the first patient with SCD treated with CTX001. CRISPR Therapeutics and Vertex announced initial data for this first SCD patient in November of 2019.

Patient 1 with SCD experienced seven vaso-occlusive crises (VOCs) and five packed red blood cell transfusions per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. After CTX001 infusion, three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain; all subsequently resolved. New data presented today show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. Bone marrow allelic editing was 81.4% at 6 months. Figure 2 presents the hemoglobin data over time for this patient.

Figure 2 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7610c5bd-25c8-4f5b-be86-8bc16ed57eb1

With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as consistency of the therapeutic effect across patients, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. These highly encouraging early data represent one more step toward delivering on the promise and potential of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases.

The data announced today are remarkable, including the demonstration of clinical proof-of-concept in TDT, said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. While these are still early days, these data mark another important milestone for this program and for the field of gene editing. The results presented at this medical conference add to results previously shared demonstrating that CRISPR/Cas9 gene editing has the potential to be a curative therapy for severe genetic diseases like sickle cell and beta thalassemia.

In my 25 years of caring for children and young adults facing both sickle cell disease and beta thalassemia, I have seen how these diseases can adversely affect patients lives in very significant ways, said Dr. Haydar Frangoul, Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center and senior author of the abstract presented at the EHA virtual congress. I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease.

Recent Progress in the Phase 1/2 Clinical TrialsCLIMB-111 for TDT has dosed a total of 5 patients, and all patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of the first two patients. Additionally, CLIMB-111 has been expanded to allow enrollment of 0/0 patients and is in the process of being expanded to allow enrollment of pediatric patients ages 12 years or older.

CLIMB-121 for SCD has dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients.

The initial safety profile in these trials appears to be consistent with myeloablative busulfan conditioning and an autologous hematopoietic stem cell transplant.

In March 2020, clinical trial sites in the U.S. and Europe temporarily paused their elective hematopoietic stem cell transplant programs due to the COVID-19 pandemic, and as a result, CRISPR and Vertex temporarily paused conditioning and dosing in these trials. Enrollment, mobilization and drug product manufacturing in each trial remains ongoing. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites. The CLIMB-111 and CLIMB-121 clinical trials are ongoing, and patients will be followed for 2 years following CTX001 infusion. The companies expect to provide additional data in the second half of 2020.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT) from the U.S. FDA, Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), and Fast Track Designation from the FDA for both SCD and TDT.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex Collaboration CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases and activities at clinical trial sites) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is being presented at the European Hematology Associations virtual congress; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, and statements regarding our plans and expectations for our clinical trials and clinical trial sites, and our expectations regarding future data announcements. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies at the 25th...

On Friday, June 5, a few days after World MS Day on May 30, there was a day of online conferences and workshops to learn more about multiple sclerosis. It was an opportunity to shed light on autologous bone marrow transplantation, a little known treatment that could cure multiple sclerosis.

Neuron

Multiple sclerosis (MS) is a neurodegenerative autoimmune disease that causes stiffness, pain, and fatigue. It is the main cause of disability, exclusion from the labor market, and social exclusion among young people, as it occurs mainly among people between 25 and 35 years old. According to the National MS Society, approximately 1 million people in the United States suffer from MS.

Currently, there is no treatment to cure MS, but there is hope: Autologous bone marrow transplantation or autologous hematopoietic stem cell transplantation. This treatment allows patients to go from the more common forms of multiple sclerosis into remission. If carried out early enough, it enables at least partial recovery from the disability.

Read Also: Combo of Diabetes and Hypertension Drugs Causes Cancer Cell Death, Researchers Find

The aim of this treatment is to rebuild a new immune system in patients. This includes intensive chemotherapy followed by reinjection of the patients hematopoietic stem cells. Several studies conducted between 2015 and 2019 on this technique have shown that 83.3 of patients with the relapsing-remitting form had no attack in the four years following auto-transplantation and three years after transplantation 78% of patients with secondary progressive multiple sclerosis and 66% of patients with primary progressive multiple sclerosis experienced no worsening of their disability, Mediapart continues.

One of the main obstacles to this treatment remains the difficulty of access. Many patients testify that their neurologist often finds this method too experimental and too risky. Another factor that discourages the use of autologous bone marrow transplantation is the risk-benefit ratio, which is considered unbalanced. Transplant-related mortality is between 5 and 10%, which justifies doctors preference for a treatment that is considered safer.

Read Also: Diabetes: Metformin Transfers Blood Sugar From the Blood to the Intestines

Another treatment has shown encouraging results in multiple sclerosis. This is a drug for diabetes, metformin, which rejuvenates stem cells to convert them into myelin-producing cells and thus help combat multiple sclerosis. These results have been published in the journal Cell Stem Cell, and it is expected that the tests, which are currently only carried out on mice, will also be carried out on humans within a year. I am very optimistic, study author Professor Robin Franklin told The Guardian newspaper.

References

Metformin Restores CNS Remyelination Capacity by Rejuvenating Aged Stem Cells

https://blogs.mediapart.fr/noelle-tassy/blog/300520/journee-mondiale-de-la-sep-et-si-parlait-du-traitement-dont-ne-parle-pas

Autologous Hematopoietic Cell Transplantation in Multiple Sclerosis: Changing Paradigms in the Era of Novel Agents

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Autologous Bone Marrow Transplantation and Metformin, a Hope for the Cure of Multiple Sclerosis - Gilmore Health News

The Hematopoietic Stem Cell Transplantation (HSCT) Market research report enhanced worldwide Coronavirus COVID19 impact analysis on the market size (Value, Production and Consumption), splits the breakdown (Data Status 2014-2020 and 6 Year Forecast From 2020 to 2026), by region, manufacturers, type and End User/application. This Hematopoietic Stem Cell Transplantation (HSCT) market report covers the worldwide top manufacturers like (Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc) which including information such as: Capacity, Production, Price, Sales, Revenue, Shipment, Gross, Gross Profit, Import, Export, Interview Record, Business Distribution etc., these data help the consumer know about the Hematopoietic Stem Cell Transplantation (HSCT) market competitors better. It covers Regional Segment Analysis, Type, Application, Major Manufactures, Hematopoietic Stem Cell Transplantation (HSCT) Industry Chain Analysis, Competitive Insights and Macroeconomic Analysis.

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Scope of Hematopoietic Stem Cell Transplantation (HSCT) Market:In 2019, the market size of Hematopoietic Stem Cell Transplantation (HSCT) is million US$ and it will reach million US$ in 2025, growing at a CAGR of from 2019; while in China, the market size is valued at xx million US$ and will increase to xx million US$ in 2025, with a CAGR of xx% during forecast period.

In this report, 2018 has been considered as the base year and 2019 to 2025 as the forecast period to estimate the market size for Hematopoietic Stem Cell Transplantation (HSCT).

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, shipments, revenue (Million USD), price, and market share and growth rate foreach application.

Peripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)

On the basis of product type, this report displays the shipments, revenue (Million USD), price, and market share and growth rate of each type.

Allogeneic Autologous

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Hematopoietic Stem Cell Transplantation (HSCT) Market Trends 2020: In-Depth Analysis of Industry Growth & Forecast Up To 2026 - Cole of Duty

Stephanie Matto suffers from a rare and life-threatening disease. Pic credit: @stepankamatto/Instagram.

Stephanie Matto has one of the most interesting storylines on this season of 90 Day Fiance: Before the 90 Days.

Matto and Erika Owens made headlines as the first same-sex couple on the TLC show, but its Stephanies personal battle with a rare illness that caught viewers by surprise.

The 29-year-old New Yorker was diagnosed with the disease only two years ago.

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Throughout the show, Stephanie has shared information about her battle and how it has affected her.

She has spoken about the limitations it has put on her life, which we saw during an episode of the show, as she prepared to make the long journey to Australia to meet Erika.

Clad in a surgical mask while equipped with hand sanitizers and a bag full of medication viewers got a glimpse of the seriousness of Stephanies illness.

Matto is battling this rare blood disorder that occurs when the bone marrow does not make enough new blood cells for the body to work in a normal way.

It means that the stem cells inside the bone marrow are damaged, thus restricting the ability to make enough white blood cells, red blood cells, and platelets.

The bone marrow can become damaged due to a variety of different diseases and conditions, but the most common cause is when the immune system attacks and destroys the stem cells in the bone marrow.

The symptoms can range from mild to severe and cause the sufferer to bruise and bleed easier than the average person. It can also cause infections to last longer than normal. When the blood cell levels are low it can increase the risks for leukemia, blood disorders, and other complications.

Furthermore, when left untreated, it can lead to very serious issues such as arrhythmia and even heart failure.

The disease can be treated via blood and bone marrow transplants and blood transfusions.

As for Matto, she needs a bone marrow transplant, which has been known to cure the disease in some people.

So far, her search has been futile and she recently shared an update on Instagram about how rare it is for patients like her to find a match.

Did you know that less than 30% of patients seeking a bone marrow transplant have a full match? I remember meeting my transplant doctor last year and hearing from him that I had absolutely no matches in the registry.

She added, I am lucky, however. My immunosuppressive therapy has bought me time and so the urgency for transplant has faded away.

The TLC alum also shared in the post that the relapse rate is high for people who battle aplastic anemia, I have already suffered one complete relapse since my diagnosis.

Matto urged her followers to join the Be The Match Registry in order to get tested and become a donor for people battling the deadly disease.

90 Day Fiance: Before the 90 Days airs Sundaya at 8/7c on TLC.

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Aplastic anemia: Heres what to know about Stephanie Mattos life-threatening illness - Monsters and Critics

By Dr Edward Nazareth

Jun 9: During our school days, most of us had learnt in biology classes that we have a cardiovascular system, a respiratory system, a digestive system, a reproductive system and a nervous system but had not learnt about the immune system. With COVID 19 infection spreading everywhere, a lot of discussion about the immune system is going on. People with a good immune system are unlikely to get the disease and those with a poor immune system may be unlucky to get a fatal version of the disease from the same virus. Meanwhile there are a lot of promotions for immune boosters, which are supposed to make our immune system strong. It is worthwhile to understand the basics of the immune system and learn how to make it strong naturally.

Military of the body

The immune system is a defense system of body. It can be compared to the military of any nation. Each nations military system is unique, it has its own mechanisms to identify the enemy, remember who its enemy is and, fight to protect the nation from any attack. Every individuals immune system is unique and works similarly.

In general, the immune system is made up of special organs, cells and chemicals that fight infection (microbes) and the toxins that may be produced by them. The main parts of the immune system are: white blood cells, antibodies, the complement system, the lymphatic system, the spleen, the thymus, and the bone marrow. These are the parts of the immune system that actively fight infection. These different systems and cell types work in perfect synchrony (most of the time) throughout the body to fight off pathogens and clear up dead cells. However the front line soldiers of this system are lymphocytes, a type of white blood cells.

Identify, destroy and remember

Our immune system has different wings similar to a military system - a surveillance wing to identify the enemy, a destroyer (fighter) wing to eliminate the enemy and an intelligence wing to remember the enemy. All these different wings are managed by lymphocytes.

Whenever a foreign material - such as bacteria, virus, fungi or any other matter with protein or, the toxins produced by these organisms enter the body, the immune system identifies it as foreign. The immune system is able to identify self from non-self. This is done by detecting the proteins that are found on the surface of the cells or by the chemicals produced by the organisms. Like the defense personnel, the immune system learns to ignore its own or self-proteins and identify the intruder. The intruder is now known as antigen. An antigen is any substance that can spark an immune response.

In many cases, an antigen is a bacterium, fungus, virus, toxin, or foreign body. But it can also be one of our own cells that are faulty or dead (like our own people turning as terrorists).

In the immune system we have two important types of white blood cells- B lymphocytes and T lymphocytes.

The B lymphocytes spot the antigen and they begin to secrete antibodies. The antibodies are special proteins (called immunoglobulins) that lock on to specific antigens. Antibodies are the ammunition to eliminate a particular antigen. The specialty of the immune system is that it produces specific ammunition to kill a particular enemy. For example to eliminate COVID 19 viruses the antibodies produced can eliminate only COVID 19 viruses and cannot act against the viruses that produce common cold, even though both the viruses belong to the same family of corona viruses.Antibodies lock onto the antigen, but they do not kill it, only mark it for death. (B lymphocytes arrest the enemy). There are three main types of T lymphocytes: Helper T cells, killer T cells and memory T cells. Helper T cells they coordinate the immune response and stimulate B cells to produce more antibodies. Killer T cells (cytotoxic T lymphocytes) as the name suggests, these T cells attack the antigen. They are particularly useful for fighting viruses. They work by recognizing small parts of the virus on the outside of infected cells and destroy the infected cells. The memory T cells are produced following an infection; they are antigen-specific and live long. Memory T cells are important because they can quickly respond to re-exposure to the antigen. They provide the immune system with memory against previously encountered antigens. Once an antibody has been produced, a copy remains in the body system, and should the same antigen invade again, it can be dealt with more swiftly. That is why with some diseases, such as measles or chickenpox we only get infected once as the body has the measles or the chickenpox antibodies stored, ready and waiting to destroy them next time when they attack. This is called immunity.

In COVID 19 infections it takes about two to four weeks for the human body to eliminate all the viruses by producing specific antibodies against them. Then these antibodies remain in the system and will not allow the virus to multiply in the body. But here two issues are to be understood. We are not yet sure if the antibodies will remain forever or disappear after a few months or years. Another point is the virus changes its protein component called genome and can attack in a new form. This is exactly like terrorists who keep on changing the names of their organization, but have the same motto.

Immunization, infection and immunity

Before the advent of immunization many people used to die from infective diseases as it happens in COVID 19 infection now. Thousands of people have died due to small pox. Babies used to die due to measles, whooping cough, tetanus and similar other illnesses. The babies are now immunized using vaccines. A particular vaccine against an infective organism is produced using the same organism. The disease causing pathogens are attenuated (weakened) or part of their protein is extracted and introduced into the healthy individual. This material is termed as vaccine. Once the vaccine enters the system, it produces antibodies and when the real organism, invades the system it is eliminated. By immunization, deadly diseases like small pox have been totally eliminated and poliomyelitis is now almost removed completely.

As we are exposed to certain other organisms, we get infected and then our system develops antibodies and we remain immune thereafter. For example most of us were infected by chickenpox when we were children. As we had been infected by the chickenpox virus once, we will not get it again. In this way we build up a store house of antibodies to different pathogens. This protection from pathogens develops as we go through life. This is also referred to as immunological memory because our immune system remembers its previous enemies.

Elderly and immunity

As humans age, the immune response becomes weak, which leads to more infections. This is again comparable to a nation with a weak military system which can be attacked and defeated easily. It is a known fact that compared with younger people, the elderly are more likely to contract infectious diseases and, more likely to die from them. Respiratory infections, influenza and the COVID 19 causes pneumonia which are a leading cause of death in people over the age of 65 worldwide. It is now known that this increased risk correlates with a decrease in T cells, because as we age fewer T cells are produced to fight off infection. It is believed that the bone marrow becomes less efficient at producing stem cells that give rise to the cells of the immune system.

Immune deficiency

In some individuals the immune system may be weak. The most common causes for deficiency of immunity worldwide include malnutrition, poor sanitary conditions and human immune deficiency virus (HIV) infection. Other causes of temporary or permanent damage to the immune system include old age, medications (e.g. cortisone, cytostatic drugs used to treat malignancies), radiotherapy, stress after surgery and malignant tumors of the bone marrow and the lymph nodes. Innate deficiencies of the immune system are comparatively rare.

This is the reason people with these diseases, probably with deficient immune system are required to be cautious of contracting the COVID 19 infection. If they are infected, their immune system may not be able to eliminate the virus and they might get severe illness.

Herd immunity

When most of the members of a community are immune to an infectious disease, it is known as herd immunity (also called herd protection). For example, if 80% of people in a community are immune to COVID 19 virus, eight out of every ten people who get infected will not become sick from the disease. In this way, the spread of infectious diseases is kept under control. Depending on how contagious an infection is, usually 70% to 90% of a population needs immunity to achieve herd immunity.

Small pox, measles, mumps and polio are examples of infectious diseases that were once very common but are now rare in our region because vaccines helped to establish herd immunity. For infections without a vaccine, even if many adults have developed immunity because of prior infection, the disease can still circulate among children and can still infect those with weakened immune systems. Chickenpox is an example for this.

Can we achieve herd immunity against COVID 19 infection? As with any other infection, there are two ways to achieve herd immunity: A large proportion of the people either gets infected or gets a protective vaccine. Based on early estimates of this viruss infectiousness, we will likely need at least 70% of the population to be immune to have herd protection. As we do not have vaccine against COVID 19 infection now, it may take years for us to develop herd immunity for it.

Can medicines boost the immune system?

As the COVID 19 infection is spreading widely and people with good immunity are reported as unaffected, a lot of information about boosting immunity is circulated in the media.

Many products claim to boost or support immunity. But the concept of boosting immunity makes little sense scientifically. Boosting immunity actually means boosting the lymphocytes (the soldiers of immune system, as explained above). In fact, boosting the number of cells in the body immune cells or others is impossible. What is known is that the body is continually generating immune cells. In fact, it produces many more lymphocytes than it can possibly use. The extra cells remove themselves through a natural process of cell death called apoptosis.

As already stated above, efficacy of herbs or any substance enhancing the immunity is a highly complicated matter. There is no research supporting that any herb or substance can increase the levels of antibodies in the body.

Healthy ways to strengthen the immune system

The best way to improve the immune system is to choose a healthy life style. A healthy life style has to be followed from a young age and attempts should be made to maintain good health naturally. Immunity depends on general health and general health depends on good immunity. Every part of the body, including the immune system, functions better when protected and improved by healthy-living strategies such as these:

Smoking has to be stopped. Smoking is bad for overall health. Many elderly who have succumbed to COVID 19 infection were chronic smokers. A diet high in fruits and vegetables is good for health. The deficiencies of zinc, selenium, iron, copper, folic acid, and vitamins A, B6, C, and E alter immune responses in experimental animals. These micronutrients are commonly present in fruits and vegetables. If not possible to consume the fruits or vegetables, dietary supplements may be used. Regular exercise is a boost to health, it is one of the pillars of healthy living. It improves cardiovascular health, helps to control body weight, and protects against a variety of diseases. Just like a healthy diet, exercise can contribute to general good health and therefore to a healthy immune system. It may contribute even more directly by promoting good circulation, which allows the cells and substances of the immune system to move through the body freely and do their job efficiently. Adequate sleep is also a natural immune booster. The human body prepares and releases cytokines, a type of protein that targets infection effectively creating an immune response. It is proved that chronic deficiency of sleep reduces bodys ability to respond to infection. Try to reduce stress. When a person is stressed, the immune system's ability to fight off antigens is reduced. That is why humans are more susceptible to infections during stressful situations. The stress hormone corticosteroid can suppress the effectiveness of the immune system (e.g. lowers the number of lymphocytes).

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Immune system, COVID 19 infection, and building up immunity - By Dr Edward Nazareth - Daijiworld.com

Five years ago, we had only one treatment for sickle cell disease, a disease that should not be taken lightly.

This disease can be a pain-generating disease that actually affects all organs of the body. This can start at the heart, blood vessels, brain, joints, bones and also the lungs.

Sickle cell is due to a mutation of a tiny gene that leads to an unstable hemoglobin. The sickles in the hemoglobin, when stressed, deprive tissue from oxygen that can lead to what we call crisis.

Crisis starts with pain, but it can also lead to stroke, heart attack and limb loss. Sickle cell crisis is when the abnormal cell gets stuck in the small blood vessels.

Sickle cell disease affects approximately 100,000 people in the United States. For years, the only therapeutic option was Hydroxyurea. This drug has been in existence since 1984. We know that this drug works, since it has proved to be effective in increasing hemoglobin, reducing pain and acute chest syndrome.

This drug has also decreased the number of blood transfusions in patients who suffer from sickle cell disease. Unfortunately, Hydroxyurea is chemotherapy and requires close monitoring. This therapy works over time with each patient; therefore, not all patients will respond equally. Since Hydroxyurea was introduced, there has been a need for new treatments. For the past several years, more therapies have started to emerge.

The first notable drug that has been FDA approved in 2017, since Hydroxyurea, is L-glutamine (Endari). This drug works on the inflammatory part of the disease. It has also proved to decrease the number of pain crisis and lessen acute chest syndrome.

The second drug is Voxelotor. This drug is a once-daily pill that stabilizes the oxygenated hemoglobin. Trials have proved to make patients less anemic, but events are not necessarily less painful. More long-term studies are looking at this issue. This drug is available, and FDA approved, through an accelerated program.

The third drug is Crizanlizumab. This drug helps with the stickiness of the red blood cells against the sticky vessel wall. This is one of the detrimental aspects of this disease. A randomized study called SUSTAIN proved that this intravenous drug decreases the number of painful crisis. This drug was FDA approved through a breakthrough therapy program.

Lastly, there is gene therapy. This type of treatment consists of an auto stem cell transplant of a viral infected, anti-sticking hemoglobin. This therapy still requires chemotherapy to wipe out the bone marrow so that space can be made for the transplant. The results of this treatment have been very successful.

Many promising therapies are seeing the light and are changing the care of this complex disease so that patients with sickle cell disease can lead a semi-normal lifestyle.

Dr. Ziad Skaff is board certified in hematology and oncology. He serves as chief of staff of MUSC Health-Florence Medical Center and Medical Director of Oncology Services. Dr. Skaff is associated with MUSC Health Hematology & Oncology, located at 805 Pamplico Highway, Medical Pavilion A, Suite 315. To schedule an appointment, call 843-674-6460.

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Sickle cell treatment then and now - SCNow

Myeloproliferative disorders are disease of blood and bone marrow which have unknown cause and there are wide range of symptoms. The treatment of myeloproliferative disorders generally depends on the type and presence of symptoms. Myeloproliferative disorders is generally considered as clonal disorder which begins with one or more change in the DNA of a single stem cells in the bone marrow. The changes to the hematopoietic stem cell cause the cell to reproduce repeatedly, creating more abnormal stem cells and these abnormal cells become one or more types of blood cells. Myeloproliferative disorders gets worst with time as the number of extra blood cells build up in the bone marrow and bloodstream.

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Emergence of new treatment for the myeloproliferative disorders and availability of novel drug drive the market for myeloproliferative disorders drugs market in the near future. Rising incidence of myeloproliferative disorders and presence of strong product pipeline spur the myeloproliferative disorders drugs market. Growing geriatric population, change in lifestyle and growing awareness among general population is expected to drive the market of myeloproliferative disorders in the forecast period.

Advancement in the treatment for oncology further expand the treatment option for myeloproliferative disorders. Various clinical trial undergoing for the treatment of myeloproliferative disorders which further drive the growth of the myeloproliferative disorders drugs market. However, high cost of drug and treatment along with the lack of awareness among the population in developing and under developed nations hinder the growth of myeloproliferative disorders drugs market.

The global myeloproliferative disorders drugs market is segmented on basis of Type, Drug Type, Distribution Channel, End User and Geography.

Improvement in the symptoms and reduction of in splenomegaly among patients receiving available therapy is expected to boost the market of myeloproliferative disorders. Development in new therapeutic drug and target therapy further drive the market growth of myeloproliferative disorders. Increased research and development and increased funding by the government towards the development of novel therapy spur the market growth. With the discovery of specific gene mutations in myeloproliferative disorders the market is expected to grow in the forecast period owing to increased adoption of new drugs and increased awareness along with the favorable reimbursement scenarios for the treatment of myeloproliferative disorders.

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The North America market holds the largest revenue share for myeloproliferative disorders drugs, due to presence of major pharmaceutical players undergoing various clinical innovation, government initiative and increase research and development funding for the Myeloproliferative disorders. Europe is expected to contribute for the second largest revenue share after North America in the global myeloproliferative disorders drugs market, owing to merging treatment option and development of oncology drug discovery and rising prevalence of myeloproliferative disorders.

Asia Pacific is expected to show rapid growth, due to increasing number of vascular surgeons and low cost of peripheral interventions. China is expected to register fast growth, due to significant increase in the number of innovative firm and research organization and increasing importance of pharmaceutical research & development activities and investments in research for developing new drugs. Latin America and Middle East & Africa are projected to exhibit sluggish growth in myeloproliferative disorders Drugs market, due to proper healthcare systems and adoption of new drug and therapy.

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Examples of some of the key manufacturer present in the global myeloproliferative disorders drugs market are,

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Myeloproliferative Disorders Drugs Market Industry Trends and Developments Through 2026 - Cole of Duty

Prophecy Market Insights Cell Therapy Manufacturing market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges. The research report study offers keen competitive landscape analysis including key development trends, accurate quantitative and in-depth commentary insights, market dynamics, and key regional development status forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.

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An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Cell Therapy Manufacturing market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.

Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Segmentation Overview:

Cell Therapy ManufacturingMarket Key Companies:

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

The Cell Therapy Manufacturing research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Cell Therapy Manufacturing market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. This report is a must-read for investors, entrepreneurs, consultants, researchers, business strategists, and all those who have any kind of stake or are planning to foray into the Cell Therapy Manufacturing industry in any manner.

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Prophecy Market Insights is specialized market research, analytics, marketing/business strategy, and solutions that offers strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high-value opportunities in the target business area. We also help our clients to address business challenges and provide the best possible solutions to overcome them and transform their business.

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Market Analysis and Technological Opportunities of Cell Therapy Manufacturing Market till 2030 - Cole of Duty

Researchers believe the link is so strong that they have called for baldness to be listed as a risk factor called the Gabrin sign This is after Dr Frank Gabrin, the first US physician to die of coronavirus, who was also bald. Lead author of the study, Professor Carlos Wambier of Brown University, told The Telegraph: "We really think that baldness is a perfect predictor of severity."

Recently scientists have hypothesised that coronavirus is more deadlier for men due than women because of testosterone.

Italian doctors have previously found patients given androgen deprivation therapy, which radically cuts testosterone levels, were four times less likely to die from coronavirus.

A protein, TMPRSS2, is driven up by testosterone and scientists think the virus could use this protein to help it unlock cells.

Researchers at Londons Institute for Cancer Research are examining the link further, whilst the University of California, Los Angeles is looking at testosterone-blocking therapy to help with coronavirus treatment.

Testosterone can produce dihydrotestosterone (DHT) which can lead to hair loss.

Though as per South China Morning Post, it is possible to have low levels of testosterone but high levels of DHT.

The further data on baldness further raises the possibility that treatments that cut testosterone could be used to slow the virus down.

Discussions have taken place about a trial using baldness drugs to treat coronavirus.

READ MORE:Chinas Xi Jinping vows to strengthen public health system

A study of 122 patients in three Madrid hospitals found 79 percent of the men were bald.

Howard Soule, executive vice president at the Prostate Cancer Foundation, told Science Magazine: "Everybody is chasing a link between androgens and the outcome of Covid-19.

Karen Stalbow, Head of Policy at Prostate Cancer UK, said: There have been several recent pieces of research which indicate there may be a link between male hormones and increased risk of Covid-19.

This has led some researchers to investigate whether hormone therapies commonly used to treat prostate cancer, such as enzalutamide, could reduce this risk.

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However, most of the research so far has been in the lab, and there is conflicting evidence over whether the hormone therapies have the same impact in the lungs as they would in prostate cells.

There are now several clinical studies starting which hope to address these issues, but much more evidence is needed before we can know whether these hormone therapies would be an effective treatment for Covid-19.

Professor Nick James of the Institute for Cancer Research has warned against using testosterone cutting treatment as a preventive measure due to their severe side effects.

He explained to the Mail on Sunday: Being on these drugs is the male equivalent of going through the menopause.

You would almost certainly cause more harm than good.

According to the NHS, the following people are considered clinically extremely vulnerable: people who have had an organ transplant, people undergoing chemotherapy or antibody treatment for cancer, intense radiotherapy, cancer treatments which affect the immune system, people who have blood or bone marrow cancer, those have had a bone marrow or stem cell transplant in the previous six months, or are still taking immunosuppressant medicine, those with a severe lung condition, those with a condition which puts them at high risk of infection or taking medicines which makes them more likely to do so and expecting mothers with heart conditions.

The list of people are considered at moderate risk includes: Those over 70, expecting mothers, those who have lung conditions which are not severe, those with heart disease, diabetics, those with chronic kidney disease, those with liver disease, those with a condition affecting the brain or nerves and those who are obese.

Previously, environmental factors were hypothesised as being behind the higher risk to men.

This includes the fact men are more likely to be smokers.

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Bald men at higher risk of having severe coronavirus, shocking new research suggests - Express

BALTIMORE, June 4, 2020 /PRNewswire/ -- Elixirgen Therapeutics, Inc., a Baltimore-based biotechnology company focused on the discovery, development, and commercialization of therapies for genetic diseases and vaccines, received confirmation from the U.S. Food and Drug Administration (FDA) that its Investigational New Drug (IND) application for its lead candidate, EXG34217, was approved on May 23, 2020. EXG34217 is an autologous cell therapy for telomere biology disorders with bone marrow failure.

The FDA's approval allows Elixirgen Therapeutics to proceed with its planned Phase I/II, open label, single center clinical trial to assess the safety and tolerability of EXG34217 at Cincinnati Children's Hospital Medical Center (ClinicalTrials.gov Identifier: NCT04211714). This program's treatment paradigm uses Elixirgen Therapeutics' proprietary ZSCAN4 technology to extend the telomeres of the hematopoietic stem cells of the patients.

About Elixirgen Therapeutics, Inc.Elixirgen Therapeutics, Inc. is a Baltimore-based biotechnology company co-founded by Akihiro Ko and Minoru Ko, MD, PhD, which is focused on curing humanity's ailments through innovations in stem cell biology.The company's experienced team of researchers has a wide variety of specialties, enabling it to use both basic and translational research approaches to developing therapies for genetic diseases and vaccines. For more information visit https://ElixirgenTherapeutics.com

Forward-Looking StatementsThis press release may contain "forward-looking" statements, including statements regarding the effectiveness of EXG34217 to treat telomere biology disorders with bone marrow failure and statements relating to the planned clinical trials of EXG34217. Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in drug research and development. In light of these and other uncertainties, the forward-looking statements included in this press release should not be regarded as a representation by Elixirgen Therapeutics that its plans and objectives regarding EXG34217 will be achieved. Any forward-looking statements in this press release speak only as of the date of this press release, and Elixirgen Therapeutics undertakes no obligation to update or revise the statementsin the future, even if new information becomes available.

Contact:Media RelationsElixirgen Therapeutics, Inc.(443) 869-5420Media@ElixirgenTherapeutics.com

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FDA Approves Elixirgen Therapeutics IND Application for Therapy for Telomere Biology Disorders with Bone Marrow Failure - Herald-Mail Media

The current coronavirus pandemic clearly illustrates how dangerous viral infections can become for us. Independent of the present situation, there are people whose bodies are defenseless against infections because their immune systems are unable to combat them - they suffer from immunodeficiency diseases such as ADA-SCID (adenosine deaminase severe combined immunodeficiency) or Wiskott-Aldrich syndrome. Prof. Dr. Alessandro Aiuti, a physician and research scientist based in Milan who works at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) and at the Vita Salute San Raffaele University, is now being honored with the Else Krner Fresenius Prize for Medical Research 2020 for his groundbreaking successes in the development of gene therapies. The award is coupled to 2.5 million euros in prize money.

In the case of the rare immune disorder ADA-SCID, which exclusively afflicts young children and occurs about 15 times a year in Europe, a defective ADA gene within the genome disrupts lymphocyte development, leaving the young patient's body defenseless against infections. "Without effective therapy, the children rarely survive for more than 2 years because any infection can become perilous for them," Aiuti explains. Standard for this therapy is a bone marrow transplantation from a fully matched sibling. However, a suitable donor is available only for a minority of patients. "Meanwhile children with such a condition benefit from the advances we have made in the field of gene therapy. So far we have treated 36 children from 19 countries using the therapy we developed. In more than 80 percent of the cases, the treatment has had such an impact that no enzyme replacement therapy or transplantation is needed. This achievement has been made possible by the extraordinary effort and dedication of SR-Tiget researchers and clinical team throughout 25 years," Aiuti adds. All of the patients are still alive.

For these successes and his other work in the field of gene therapy, Alessandro Aiuti has now been honored with the Else Krner Fresenius Prize for Medical Research 2020 awarded by the Else Krner-Fresenius-Stiftung (EKFS) foundation. At 2.5 million euros, this award is one of the highest endowed prizes for medical research in the world. "Still young by comparison, this year the prize is being awarded for the third time. It honors research scientists for pioneering contributions in the areas of biomedical science. A major percentage of the prize money flows into the prizewinner's research and is supposed to contribute toward achieving further groundbreaking findings and medical breakthroughs in the future as well," emphasizes Prof. Dr. Michael Madeja, scientific director and member of the management board at EKFS.

The decision regarding the prize recipient was made by a ten-member international jury composed of renowned research scientists in the fields of genome editing and gene therapy along with delegates from the Scientific Commission at EKFS. Prof. Dr. Hildegard Bning, chairwoman of the jury and president of the European Society for Gene and Cell Therapy (ESGCT), substantiates the jury's decision: "Alessandro Aiuti is a truly outstanding physician and scientist. His work has decisively contributed to the development and successful treatment of rare, genetically caused disorders such as SCID. Thanks not least of all to the contributions he has made, even patients with other inheritable illnesses can presumably be treated successfully in the future."

After successful clinical trials, the gene therapy developed for ADA-SCID patients was approved as a pharmaceutical remedy in Europe. It is considered to be one of the key findings in the development of gene therapies worldwide. With this treatment certain blood stem cells (CD34+) are taken from the patient, then the cell DNA is modified. The cells are treated outside the body using a viral vector to accomplish this. The correct version of the gene for the ADA enzyme is introduced into the genome of the cells that were collected. The genetically modified cells are returned to the patient's bloodstream via intravenous infusion. A portion of the modified cells subsequently establish themselves in bone marrow again. The patient now has blood stem cells that function properly and produce lymphocytes to defend against infections - presumably on a life-long basis.

Alessandro Aiuti wants to utilize the prize money from EKFS to set the success story forth, to optimize the therapies further and map out the healing mechanisms involved in a better fashion. The scientist sees another major challenge in conveying the acquired knowledge beyond the successful gene therapies from Milan to as many other genetic disorders as possible. Alongside the therapy for ADA-SCID, the San Raffaele Telethon Institute for Gene Therapy has also developed gene therapies for four more hereditary diseases, among them the Wiskott-Aldrich syndrome and metachromatic leukodystrophy (MLD). To this day a total of more than 100 patients from 35 different countries have been treated.

Biography of Alessandro Aiuti

Alessandro Aiuti was born in Rome in 1966 and studied medicine there at Sapienza University. Following a stay at Harvard Medical School in Boston, Massachusetts in the USA, he received his doctorate in Human Biology in 1996 from Sapienza University. Since 1997 he has been active at the San Raffaele Scientific Institute in Milan, where he meanwhile also teaches as a professor at the Vita Salute San Raffaele University. He is furthermore Deputy Director of Clinical Research at the San Raffaele Telethon Institute for Gene Therapy and Head of the Pediatric Immunohematology Unit at San Raffaele Hospital.

Aiuti is the author of numerous and highly acclaimed publications. Over the course of his career he has received a number of prizes from national and international institutions. Aiuti is a member of the board of the European Society of Gene and Cell Therapy, and a member of the EMA Committee for Advanced Therapies since 2019.

The Else Krner Fresenius Prize for Medical Research

The international Else Krner Fresenius Prize for Medical Research came into existence in 2013 on the occasion of the 25th anniversary of Else Krner's death and is awarded in alternating fields of biomedical science. Endowed with 2.5 million euros, the prize is one of the most highly endowed medical research awards in the world. It honors and supports research scientists who have made significant scientific contributions in their fields and whose work can be expected to yield groundbreaking findings and medical breakthroughs in the future as well.

The Else Krner-Fresenius-Stiftung (EKFS) foundation - Advancing research. Helping people.

The Else Krner-Fresenius-Stiftung, a non-profit foundation, is dedicated to the funding of medical research and supports medical/humanitarian projects. The foundation was established in 1983 by entrepreneur Else Krner and appointed as her sole heir. EKFS receives virtually all of its income in dividends from the Fresenius healthcare group, in which the foundation is the majority shareholder. To date, the foundation has funded around 2,000 projects. With a current annual funding volume around 60 million euros the EKFS is one of the largest foundations for medicine in Germany. More information:www.ekfs.de.

The San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than EUR 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit:www.telethon.it

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Groundbreaking Gene Therapies for Hereditary Diseases / Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Krner...

People usually volunteer to donate stem cells for an allogeneic transplant either because they have a loved one or friend who needs a match or because they want to help people. Some people give their stem cells so they can get them back later if they need an autologous transplant.

People who want to donate stem cells or join a volunteer registry can speak with a health care provider or contact the National Marrow Donor Program to find the nearest donor center. Potential donors are asked questions to make sure they are healthy enough to donate and dont pose a risk of infection to the recipient. For more information about donor eligibility guidelines, contact Be the Match or the donor center in your area.

Be the Match (formerly the National Marrow Donor Program)Toll-free number: 1-800-MARROW-2 (1-800-627-7692)Website: http://www.bethematch.org

A simple blood test is done to learn the potential donors HLA type. There may be a one-time, tax-deductible fee of about $75 to $100 for this test. People who join a volunteer donor registry will most likely have their tissue type kept on file until they reach age 60.

Pregnant women who want to donate their babys cord blood should make arrangements for it early in the pregnancy, at least before the third trimester. Donation is safe, free, and does not affect the birth process.

If a possible stem cell donor is found to be a good match for a recipient, steps are taken to teach the donor about the transplant process and make sure he or she is making an informed decision. If a person decides to donate, a consent form must be signed after the risks of donating are fully discussed. The donor is not pressured to take part. Its always a choice.

If a person decides to donate, a medical exam and blood tests will be done to make sure the donor is in good health.

Stem cells may be collected from these 3 different sources:

Each method of collection is explained here.

This process is often called bone marrow harvest. Its done in an operating room, while the donor is under general anesthesia (given medicine to put them into a deep sleep so they dont feel pain). The marrow cells are taken from the back of the pelvic (hip) bone. The donor lies face down, and a large needle is put through the skin and into the back of the hip bone. Its pushed through the bone to the center and the thick, liquid marrow is pulled out through the needle. This is repeated several times until enough marrow has been taken out (harvested). The amount taken depends on the donors weight. Often, about 10% of the donors marrow, or about 2 pints, are collected. This takes about 1 to 2 hours. The body will replace these cells within 4 to 6 weeks. If blood was taken from the donor before the marrow donation, its often given back to the donor at this time.

After the bone marrow is harvested, the donor is taken to the recovery room while the anesthesia wears off. The donor may then be taken to a hospital room and watched until fully alert and able to eat and drink. In most cases, the donor is able to leave the hospital within a few hours or by the next morning.

The donor may have soreness, bruising, and aching at the back of the hips and lower back for a few days. Over-the-counter pain medications or nonsteroidal anti-inflammatory drugs are helpful. Some people may feel tired or weak, and have trouble walking for a few days. The donor might be told to take iron supplements until the number of red blood cells returns to normal. Most donors get back to their usual activities in 2 to 3 days. But it could take 2 or 3 weeks before they feel completely back to normal.

There arent many risks for donors and serious complications are rare. But bone marrow donation is a surgical procedure. Rare complications could include anesthesia reactions, infection, nerve or muscle damage, transfusion reactions (if a blood transfusion of someone elses blood is needed this doesnt happen if you get your own blood), or injury at the needle insertion sites. Problems such as sore throat or nausea may be caused by anesthesia.

Allogeneic stem cell donors do not have to pay for the harvesting because the recipients insurance company usually covers the cost. Even so, be sure to ask about insurance coverage before you decide to have the bone marrow harvest done.

Once the cells are collected, they are filtered through fine mesh screens. This prevents bone or fat particles from being given to the recipient. For an allogeneic or syngeneic transplant, the cells may be given to the recipient through a vein soon after they are harvested. Sometimes theyre frozen, for example, if the donor lives far away from the recipient.

For several days before starting the donation process, the donor is given a daily injection (shot) of a drug that causes the bone marrow to make and release a lot of stem cells into the blood. Filgrastim can cause some side effects, the most common being bone pain and headaches. These may be helped by over-the-counter pain medications or nonsteroidal anti-inflammatory drugs. Nausea, sleeping problems, low-grade (mild) fevers, and tiredness are other possible effects. These go away once the injections are finished and collection is completed.

After the shots, blood is removed through a catheter (a thin, flexible plastic tube) thats put in a large vein in the arm. Its then cycled through a machine that separates the stem cells from the other blood cells. The stem cells are kept while the rest of the blood is returned to the donor, often through the same catheter. (In some cases, a catheter may be put in each arm one takes out blood and the other puts it back.) This process is called apheresis. It takes about 2 to 4 hours and is done as an outpatient procedure. Often the process needs to be repeated daily for a few days, until enough stem cells have been collected.

Possible side effects of the catheter can include trouble placing the catheter in the vein, blockage of the catheter, or infection of the catheter or at the area where it enters the vein. Blood clots are another possible side effect. During the apheresis procedure, donors may have problems caused by low calcium levels from the anti-coagulant drug used to keep the blood from clotting in the machine. These can include feeling lightheaded or tingly, and having chills or muscle cramps. These go away after donation is complete, but may be treated by giving the donor calcium supplements.

The process of donating cells for yourself (autologous stem cell donation) is pretty much the same as when someone donates them for someone else (allogeneic donation). Its just that in autologous stem cell donation the donor is also the recipient, giving stem cells for his or her own use later on. For some people, there are a few differences. For instance, sometimes chemotherapy (chemo) is given before the growth factor drug is used to tell the body to make stem cells. Also, sometimes it can be hard to get enough stem cells from a person with cancer. Even after several days of apheresis, there may not be enough for the transplant. This is more likely to be a problem if the patient has had certain kinds of chemo in the past, or if they have an illness that affects their bone marrow.

Cord blood is the blood thats left in the placenta and umbilical cord after a baby is born. Collecting it does not pose any health risk to the infant or the mother. Cord blood transplants use blood that would otherwise be thrown away. After the umbilical cord is clamped and cut, the placenta and umbilical cord are cleaned. The cord blood is put into a sterile container, mixed with a preservative, and frozen until needed.

Some parents choose to donate their infants cord blood to a public blood bank, so that it may be used by anyone who needs it. Many hospitals collect cord blood for donation, which makes it easier for parents to donate. Parents can donate their newborns cord blood to volunteer or public cord blood banks at no cost. For more about donating your newborns cord blood, call 1-800-MARROW2 (1-800-627-7692) or visit Be the Match.

Other parents store their newborns cord blood in private cord blood banks just in case the child or a close relative needs it someday. If you want to donate or bank (save) your childs cord blood, youll need to arrange it before the baby is born. Some banks require you to set it up before the 28th week of pregnancy, although others accept later setups. Among other things, youll be asked to answer health questions and sign a consent form.

Parents may want to bank their childs cord blood if the family has a history of diseases that may benefit from stem cell transplant. There are several private companies offer this service. But here are some things to think about:

More information on private family cord blood banking can be found at the Parents Guide to Cord Blood Foundation. You can visit their website at http://www.parentsguidecordblood.org.

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Donating Stem Cells and Bone Marrow

Mesenchymal Stem Cells Market Trends by Manufacturers, States, Type and Application, Forecast to 2019 2027

Global Mesenchymal Stem Cells Market: Snapshot

The increasing use of mesenchymal stem cells (MSCs) for the treatment of diseases and disabilities of the growing aging population is having a positive influence on the global mesenchymal stem cells market. Mesenchymal stem cells are adult stem cells that are of various types such as adipocytes, osteocytes, monocytes, and chondrocytes.

The main function of mesenchymal stem cells is to replace or repair damaged tissue.

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Mesenchymal stem cells are multipotent, i.e. they can produce more than one type of specialized cells.

These specialized cells have their own distinguishing shapes, structures, and functions, with each of them belonging to a particular tissue.

Mesenchymal stem cells are traditionally found in the bone marrow. However, these cells can also be separated from other tissues such as cord blood, fallopian tube, peripheral blood, and fetal liver and lung.

Mesenchymal stem cells have long thin cell bodies containing a large nucleus. MSCs have enormous capacity for renewal keeping multipotency.

Due to these virtues, mesenchymal stem cells have huge therapeutic capacity for tissue repair.

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Mesenchymal stem cells can differentiate into a number of cell types that belong to our skeletal tissues that include cartilage, bone, and fat. Research is underway to discover if mesenchymal stem cells can be used to treat bone and cartilage diseases.

Scientists are also exploring the possibility if mesenchymal stem cells differentiate into other type of cells apart from skeletal tissues. This includes nerve cells, liver cells, heart muscle cells, and endothelial cells.

This will lead to mesenchymal stem cells to be used to treat other diseases.

Stem cells are specialized cells which have the capability of renewing themselves through cell division and differentiate into multi-lineage cells. Mesenchymal stem cells (MSCs) are non- hematopoietic, multipotent adult stem cells which can be isolated from bone marrow, cord blood, fat tissue, peripheral blood, fallopian tube, and fetal liver and lung tissue.

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Mesenchymal stem cells have the capacity to differentiate into mesodermal lineages, such as chondrocytes, adipocytes, and osteocytes, and non-mesodermal lineages such as ectodermal (neurocytes) and endodermal lineages (hepatocytes). These stem cells have specific features such as multilineage potential, secretion of anti-inflammatory molecules, and immunomodulation.

These cells have emerged as promising therapeutic agents for regenerating skeletal tissues such as damaged bone and cartilage tissues and treatment of chronic diseases owing to their specific features.

The global mesenchymal stem cells market is expected to be driven by the increasing clinical application of mesenchymal stem cells for the treatment of chronic diseases, bone and cartilage diseases, and autoimmune diseases. Studies have shown that these stem cells enhance the angiogenesis in myocardium and allow the reduction of myocardial fibrotic area.

The pre-clinical studies for using mesenchymal stem cells in treatment of cardiovascular diseases, liver diseases, and cancer are projected to create new market opportunities for mesenchymal stem cells. Mesenchymal stem cells also produce anti-inflammatory molecules which modulate humoral and cellular immune responses.

Features of these stem cells such as ease of isolation, regenerative potential, and immunoregulatory, the mesenchymal stem cell therapy has emerged as a promising tool for the treatment of chronic diseases, degenerative, inflammatory, and autoimmune diseases.

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Clinical studies are exploring MSCs for various conditions such as orthopedic injuries, graft versus host disease following bone marrow transplantation, and genetic modification of MSCs to overexpress antitumor genes for use as anticancer therapy, which are exhibiting new opportunities in therapeutic area. However, the mesenchymal stem cell research studies are tedious, lengthy, and complex.

In some cases, due to some adverse effects transplanted mesenchymal stem cells rapidly removed from the body which limits use of stem cells in therapeutic treatments. The conflicting results and regulatory compliances for approvals may also hamper the growth of this market.

The global mesenchymal stem cells market is segmented on the basis of source of isolation, end-user, and region. Stem cells are isolated from the bone marrow, peripheral blood, lung tissue, umbilical cord blood, amniotic fluids, adipose tissues, and synovial tissues.

Traditionally the MSCs were isolated from bone marrow aspiration which is associated with risk of infection and painful for the patient. The MSCs from adipose tissues are usually isolated from the biological material generated during liposuction, lipectomy procedures by using collagenase enzymatic digestion followed by centrifugation and washing.

In terms of end-user, the market is segmented into clinical research organizations, biotechnological companies, medical research institutes, and hospitals.

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Geographically, the global mesenchymal stent cells market is distributed over North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America dominated the global market and is projected to continue its dominance in terms of market share during the forecast period owing to high R&D expenditure, availability of advanced research facilities and skilled professionals, and government initiatives.

Europe is the second largest market after North America. The Asia Pacific market is projected to expand at a high CAGR during the forecast period due to increased R&D budgets in Japan, China, and India.

Key global players operating in the mesenchymal stem cells market include R&D Systems, Inc., Cell Applications, Inc., Axol Bioscience Ltd., Cyagen Biosciences Inc., Cytori Therapeutics Inc., Stemcelltechnologies Inc., BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc., and Celprogen, Inc.

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Mesenchymal Stem Cells Market trends by manufacturers, states, type and application, forecast to 2019 2027 - WhaTech Technology and Markets News