KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that new data from its oncology program will be presented at the 2020 American Society of Clinical Oncology (ASCO20) Virtual Scientific Program from May 29-31. More than 80 abstracts in nearly 20 types of solid tumors and blood cancers have been accepted across Mercks broad cancer portfolio and investigational pipeline, including KEYTRUDA, Mercks anti-PD-1 therapy; LENVIMA (in collaboration with Eisai); LYNPARZA (in collaboration with AstraZeneca); and MK-6482 (formerly PT2977), an investigational, oral hypoxia-inducible factor-2 alpha (HIF-2) inhibitor.

Despite the challenges we all face due to the COVID-19 pandemic, Merck remains fully committed to supporting the cancer community and to advancing important scientific research from our clinical program, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. The data to be presented at this years ASCO demonstrate how our deep and diverse oncology portfolio continues to show meaningful outcomes for patients in new tumor types and stages of disease, while long-term survival data for KEYTRUDA in non-small cell lung cancer, renal cell carcinoma and melanoma further support its important role in these types of cancer.

Key abstracts including late-breakers, oral sessions, and select poster discussions and posters to be presented at ASCO include:

Merck Investor Event

Merck will hold a virtual investor event in conjunction with the ASCO20 Virtual Scientific Program on Tuesday, June 2 at 2 p.m. ET. Details will be provided at a date closer to the event at http://investors.merck.com/home/default.aspx.

Details on Abstracts Listed Above, Additional Presentations and Key Abstracts with Mercks Collaboration Partners

KEYTRUDA

Breast Cancer

Bladder Cancer

Classical Hodgkin Lymphoma

Colorectal Cancer

Lung Cancer

Renal Cell Carcinoma

Prostate Cancer

Melanoma

Ovarian Cancer

Head and Neck Cancer

KEYTRUDA plus LENVIMA (in collaboration with Eisai)

Hepatocellular Carcinoma

Renal Cell Carcinoma

Endometrial Cancer

LYNPARZA (in collaboration with AstraZeneca)

Ovarian Cancer

MK-6482

Renal Cell Carcinoma

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Endometrial Carcinoma

KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

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Merck to Present New Data from its Broad Oncology Portfolio and Pipeline at the ASCO20 Virtual Scientific Program - Business Wire

Cardiac Stem Cells Comments Off on Merck to Present New Data from its Broad Oncology Portfolio and Pipeline at the ASCO20 Virtual Scientific Program – Business Wire | May 7th, 2020

A recent market study on the global Hematopoietic Stem Cell Transplantation (HSCT) market reveals that the global Hematopoietic Stem Cell Transplantation (HSCT) market is expected to reach a value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% during the forecast period (2019-2029). The impact of the COVID-19 pandemic on the global Hematopoietic Stem Cell Transplantation (HSCT) market is discussed in the presented study.

The Hematopoietic Stem Cell Transplantation (HSCT) market study encloses a thorough analysis of the overall competitive landscape and the company profiles of leading market players involved in the global Hematopoietic Stem Cell Transplantation (HSCT) market. Further, the presented study offers accurate insights pertaining to the different segments of the global Hematopoietic Stem Cell Transplantation (HSCT) market such as the market share, value, revenue, and more.

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The following doubts are addressed in the market report:

Key Highlights of the Hematopoietic Stem Cell Transplantation (HSCT) Market Report

The presented report segregates the Hematopoietic Stem Cell Transplantation (HSCT) market into different segments to ensure the readers gain a complete understanding of the different aspects of the Hematopoietic Stem Cell Transplantation (HSCT) market.

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Segmentation of the Hematopoietic Stem Cell Transplantation (HSCT) market

Competitive Outlook

This section of the report throws light on the recent mergers, collaborations, partnerships, and research and development activities within the Hematopoietic Stem Cell Transplantation (HSCT) market on a global scale. Further, a detailed assessment of the pricing, marketing, and product development strategies adopted by leading market players is included in the Hematopoietic Stem Cell Transplantation (HSCT) market report.

Companies Mentioned in the Report

The report profiles key manufacturers in the hematopoietic stem cell transplantation (HSCT) Market based on various attributes such as company details, SWOT analysis, strategic overview, financials, and business overview. Major players profiled in this report include Regen Biopharma, Inc., Escape Therapeutics, Inc., Lonza Group Ltd., and Pluristem Therapeutics Inc.

The global hematopoietic stem cell transplantation (HSCT) Market has been segmented as follows:

By Transplant Type

By Disease Indication

By Application

By Region

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Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -...

Bone Marrow Stem Cells Comments Off on Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -… | May 6th, 2020

As the world continues to battle the coronavirus pandemic, scientists are looking towards gene therapy to find ways to develop vaccines for the Covid-19 virus. Gene therapy itself was developed based on how viruses work.

When a virus attacks a host, it introduces its genetic material into the host cell as part of its replication cycle. The genetic material serves as an instruction manual on how to produce more copies of the virus, hijacking the host body's normal production machinery to serve the needs of the virus. The host cells then produce additional copies of the virus, leading to more host cells being infected.

Like animals, humans have found a way to domesticate viruses as well, i.e., direct the virus's function to achieve favorable results, which is prominent in gene therapy. Such viruses which physically insert their genes into the host's genome could instead be used to carry "good" genes into a human cell. Scientists would first remove the genes in the virus that cause diseases, and replace those genes with genes encoding the desired effect.

All of this sounds quite sci-fi but it has been done numerous times in the past. Peter Kolchinsky, a virologist and a biotechnology investor, compiled how different viruses have been used for gene therapy in the past.

Kolchinsky tweeted, "SARS2 is a scary menace, but did you know that we've domesticated viruses? Like wolves vs dogs, we've tamed them, including some deadly ones, to perform many useful functions (and may help us stop SARS2)."

The human immunodeficiency virus (HIV) has killed millions of people. It works by disabling the host body's immune system until it can't defend the person against common, normally mild pathogens. Kolchinsky explained that HIV's special trick is to integrate its genome into that of the host body's cells.

This feature of HIV is used for gene therapy, as explained before, by replacing a chunk of the virus's genome with the hemoglobin gene to insert it into bone marrow stem cells of patients with sickle cell anemia, whose hemoglobin genes are malfunctioning.

Kolchinsky also tweeted, "Adenoviruses typically cause mild infections, including common colds. These, too, we are trying to use for gene therapies, particularly when we just want to temporarily make a protein in cells. One company is developing such an adenovirus gene therapy for heart disease to induce growth of new blood vessels when old ones are clogged. Another is using this virus to make oral vaccines that would otherwise require injection (eg flu vaccine pill). When we use a virus to deliver code for making something in cells, we call that a virus vector."

There is now a wealth of clinical experience with numerous vector types that include primarily vaccinia, measles, vesicular stomatitis virus (VSV), polio, reovirus, adenovirus, lentivirus, -retrovirus, adeno-associated virus (AAV) and herpes simplex virus (HSV).

However, as with all other procedures, viral vector-gene therapy has associated risks. Viruses can usually infect more than one type of cell, so, when viralvectorsare used to carrygenesinto the body, they might infect healthy cells as well as cancer cells.

Another danger is that the new gene might be inserted in the wrong location in the DNA, possibly causing harmful mutations to the DNA or even cancer. Moreover, when viruses are used to deliver DNA to cells inside the patient's body, there is a slight chance that this DNA could unintentionally be introduced into the patients reproductive cells. If this happens, it could produce changes that may be passed on if a patient has children after treatment.

One study to help find a vaccine for Covid-19 aims to use the principles behind gene therapy to get the vaccine ready. The researchers' method uses a harmless virus as a vector to bring DNA into the patient's cells. The DNA should then instruct the cells to make a coronavirus protein that would stimulate the immune system to fight off future infections.

While a mass-produced vaccine may still take a while, this study is one of at least 90 vaccine projects around the world trying to find a cure for Covid-19. However, some experts are worried that a vaccine may never be available. According to our previous report, Dr David Nabarro, a professor of global health at Imperial College London, who also serves as a special envoy to the WHO on Covid-19, said, "There are some viruses that we still do not have vaccines against."

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Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough - MEAWW

Bone Marrow Stem Cells Comments Off on Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough – MEAWW | May 6th, 2020

According to the World Health Organization (WHO), cardiovascular disease, specifically ischemic heart disease, is one of the leading causes of death worldwide. Cardiovascular diseases result in an estimated 17.9 million deaths each year. This is about 31% of all deaths worldwide (1). Medical researchers are continually working on ways to reduce those numbers, including the development of new technologies to combat premature deaths from cardiovascular diseases. This article will focus, in particular, on the value of induced pluripotent stem cells (iPSCs) in cardiac research.

iPSCs are a type of pluripotent stem cell. These are master cells that can differentiate into any cell or tissue the body needs. They are generated directly from somatic cells through ectopic expression of various transcription factors, such as

Theyve become key tools to model biological processes, particularly in cell types that are difficult to access from living donors. Many research laboratories are working to enhance reprogramming efficiency by testing different cocktails of transcription factors.

iPSCs have become essential in a number of different research fields, including cardiac research.

They are a valuable and advantageous technologic development for two main reasons:

Most people have heard of embryonic stem cells, which are one variation of pluripotent cells. Like iPSCs, they can be used to replace or restore tissues that have been damaged.

The problem is that embryonic stem cells are only found in preimplantation stage embryos (3). Whereas iPSCs are adult cells that have been genetically modified to work like embryonic stem cells. Thus, the term, inducedpluripotent stem cells.

The development of iPSCs was helpful because embryos are not needed. This reduces the controversy surrounding the creation and use of stem cells. Further, iPSCs from human donors are also more compatible with patients than animal iPSCs, making them even closer to their embryonic cousins.

The Japanese inventor of iPSCs, Professor Shinya Yamanaka earned a Nobel Prize in 2012 for the discovery that mature cells can be reprogrammed to become pluripotent. (4) The Prize was awarded to Dr. Yamanaka because of the significant medical and research implications this technology holds.

iPSCs hold a lot of promise for transplantation medicine. Further, they are highly useful in drug development and modeling of diseases.

iPSCs may become important in transplantation medicine because the tissues developed from them are a nearly identical match to the cell donors. This can potentially reduce the chances of rejection by the immune system (5).

In the future, and with enough research, it is highly possible that researchers may be able to perfect the iPSC technology so that it can efficiently reprogram cells and repair damaged tissues throughout the body.

iPSCs forgo the need for embryos and can be made to match specific patients. This makes them extremely useful in both research and medicine.

Every individual with damaged or diseased tissues could have their own pluripotent stem cells created to replace or repair them. Of course, more research is needed before that becomes a reality. To date, the use of iPSCs in therapeutic transplants has been very limited.

One of the most significant areas where iPSCs are currently being used is in cardiac research. With appropriate nutrients and inducers, iPSC can be programmed to differentiate into any cell type of the body, including cardiomyocyte. This heart-specific cell can then serve as a great model for therapeutic drug screening or assay development.

Another notable application of iPSCs in cardiac research is optical mapping technology. Optical mapping technology employs high-speed cameras and fluorescence microscopy to examines the etiology and therapy of cardiac arrhythmias in a patient-like environment. This is typically done by looking into electrical properties of multicellular cardiac preparations., e.g. action potential or calcium transient, at high spatiotemporal resolution (6).

Optical mapping technology can correctly record or acquire data from iPSCs. iPSCs are also useful in mimicking a patients cardiomyocytes with their specific behaviors, resulting in more reliable and quality data of cardiac diseases.

iPSCs are vital tools in cardiac research for the following reasons:

iPSCs are patient-specific because they are 100% genetically identical with their donors. This genomic make-up allows researchers to study patients pathology further and develop therapeutic agents for treating their cardiac diseases.

Induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs), help researchers predict the cardiotoxicity of drugs like with widely used chemotherapy reagents (10). Predictions like this were close to impossible before iPSC technology entered the research game.

iPSCs really come into play with their ability to model diseases. Because iPSCs are genetic matches to their living donors, they are uniquely useful for the study of genetic cardiac diseases like monogenic disorders. iPSCs help researchers understand how disease genotypes at the genetic level manifest as phenotypes at the cellular level (5).

Long QT syndrome, a condition that affects the repolarization of a patients heart after a heartbeat, is a notable example of iPSC-based disease modeling (7). This syndrome has been successfully modeled using iPSCs and is an excellent model for other promising target diseases (7).

Long QT syndrome is not the only disease that has been modeled by iPSCs. Other cardiac diseases like Barth syndrome-associated cardiomyopathy and drug-induced kidney glomerular injuries have been modeled as well (8).

The advent of iPSC technology has created a wealth of new opportunities and applications in cardiovascular research and treatments. In the near future, researchers hope that iPSC-derived therapies will be an option for thousands, if not millions of patients worldwide.

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What is the Value of iPSC Technology in Cardiac... - The Doctor Weighs In

Cardiac Stem Cells Comments Off on What is the Value of iPSC Technology in Cardiac… – The Doctor Weighs In | May 6th, 2020

MIKE TYSON revealed how he has started training again having not thrown a punch for a staggering 15 years - and is being aided by stem-cell research therapy.

The Baddest Man on the Planet, who hung up his gloves in 2005 following defeat by Kevin McBride, wouldn't elaborate on why he was having the treatment but added that it was 'really wild what scientists can do'.

4

Tyson was having an Instagram chat with basketball legend Shaquille O'Neal when he revealed that he had been training for the previous three days after 15 years away - and his new health regime.

Iron Mike said: "You know what I had done? I had stem-cell research therapy.

"I feel like a different person but I can't comprehend why I feel this way. It's really wild what scientists can do."

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition that usually takes the form of a bone marrow transplantation.

He did not reveal what the exact condition was that was being treated.

Despite letting the gloves gather dust in the corner, it didn't take long for Iron Mike to show off that lethal punching power.

4

The 53-year-old delighted fans in lockdown by uploading a viral video of him laying into a punchbag with his trademark speed and power.

Tyson's weight ballooned after retiring following battles with drug addiction and depression.

But he has since partnered with a new trainer, MMA coach Rafael Cordeiro, to kick-start his training after sensationally revealing his 15-year break.

During their chat, basketball legend O'Neal revealed how he ached for three days after playing with his sons.

Tyson responded: "That's just because you haven't done it for a while.

WHAT IS STEM CELL TREATMENT USED FOR?

Stem cell transplants are carried out when bone marrow is damaged or isnt able to produce healthy blood cells.

It can also be used to replace damaged blood cells as the result of intensive cancer treatment.

Here are conditions that stem cell transplants can be used to treat:

"If you continue to do it consistently you'll be back to normal.

"It's just like me, I haven't boxed or hit the bag for 15 years - it has been three days so far and I feel incredible."

Tyson, who has a 50-6 record, is reportedly gearing up for a sensational return amid plans to compete in exhibition bouts for charity later this year.

He told rapper T.I. last month: "I've been hitting the mitts for the last week.

"That's been tough, my body is really jacked up and really sore from hitting the mitts.

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"I've been working out, I've been trying to get in the ring, I think I'm going to box some exhibitions and get in shape.

"I want to go to the gym and get in shape to be able to box three or four-round exhibitions for some charities and stuff.

"Some charity exhibitions, make some money, help some homeless and drug-affected motherf****er like me."

4

4

Original post:
Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t - The Sun

Bone Marrow Stem Cells Comments Off on Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t – The Sun | May 5th, 2020

Veteran Bollywood actor Rishi Kapoor, who was battling leukemia for the last two years, died at Mumbais Sir HN Reliance Foundation Hospital on Thursday (April 30) morning. He was 67. Rishi Kapoor got treated for leukemia in the US for a year and had returned to India in September 2019.

Leukemia is a blood cancer caused by a rise in the number of white blood cells in your body. Those white blood cells crowd out the red blood cells and platelets that your body needs to be healthy. The extra white blood cells dont work right. A cancer of blood-forming tissues, hindering the body's ability to fight infection.

Dr Rahul Bhargava, Director, Haematology, Haemato Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram says:

Leukemia, often fatal, is a blood cancer. As with cancer, it is associated with an exponential rise in the number of white blood cells. Symptoms begin to manifest slowly and can often be mistaken for something else sweating uncontrollably, shaking, fever, vomiting. It is caused by huge was is reduced focus on ones own health.

Intake of processed food, soda, artificial and synthetic sweets and preservatives, pesticide use in farms, lack of physical exercise, excessive tobacco and drug consumption, excessive alcohol intake, caffeine consumption are the main culprits causing cancer. Family history also plays an important role in a leukemia diagnosis. Only through screening can it be identified and if caught early managed. One needs to be mindful and ensure that they are employing good lifestyle habits.

This means eating green leafy vegetables and fruits, going for a 30 minutes walk every day or engaging in any other form of exercise for 30 minutes a day, drinking adequate amount of water to keep flushing out the toxins, controlling or completely eliminating our use of tobacco and other unnatural substances. Processed food and sugars should be consumed in control, they are the single largest cause of mutation to cells.

Leukemia symptoms vary, depending on the type of leukemia. Common leukemia signs and symptoms include:

Leukemia can also cause symptoms in organs that have been infiltrated or affected by the cancer cells. For example, if the cancer spreads to the central nervous system, it can cause headaches, nausea and vomiting, confusion, loss of muscle control, and seizures.

Leukemia can also spread to other parts of your body, including:

The major types of leukemia are:

Blood has three types of cells: white blood cells that fight infection, red blood cells that carry oxygen, and platelets that help blood clot. Every day, your bone marrow makes billions of new blood cells, and most of them are red cells. When you have leukemia, your body makes more white cells than it needs. These leukemia cells cant fight infection the way normal white blood cells do. And because there are so many of them, they start to affect the way your organs work. Over time, you may not have enough red blood cells to supply oxygen, enough platelets to clot your blood, or enough normal white blood cells to fight infection.

Leukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer. The treatment depends on the type and stage of the cancer. Some forms of leukemia grow slowly and don't need immediate treatment. However, treatment for leukemia usually involves one or more of the following:

Source: Information has been taken from various health website

Read more| National Honesty Day 2020: Date, history, significance and quotes on the value of honesty

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What Is Leukemia? Here's all you need to know - India Today

Bone Marrow Stem Cells Comments Off on What Is Leukemia? Here’s all you need to know – India Today | May 5th, 2020

The first-in-human, universal chimeric receptor antigen (CAR) T-cell (CAR-T) therapy GC027 was tolerable and resulted in antileukemic responses among patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), according to results from a phase 1 trial presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020.1

Theuniversal CAR T cells target CD7, which, according to Xinxin Wang, PhD, ofGracell Biotechnologies Co, Ltd, in China, and lead author and presenter of thestudy, is a good target for T-ALL because it is expressed by more than 95% ofT-ALL patients.

GC027 isallogeneic, which may prevent the development of graft-versus-host disease. Theproduct is introduced using lentivirus for rapid elimination of T-ALL cells. Preclinicalstudies showed efficacy in a T-ALL xenograft model, and this prospective studyevaluated the safety and efficacy in humans.

Thesingle-arm, open-label study treated 5 adult patients with relapsed/refractoryCD7-positive T-ALL with a single infusion of 1 of 3 different dose levels ofG027: 0.6 x 107/kg, 3 x 107/kg, and 1.5 x 107/kg.Lymphodepletion therapy was administered prior to the G027 infusion. Theprimary endpoint was safety and the secondary endpoints included objectiveresponse rate (ORR) within 3 months after G027 infusion.

Patientswith extramedullary or central nervous system disease were excluded. Atbaseline, the median age was 24 (range, 19-38). Patients were heavilypretreated, with 5 median number of prior therapies (range, 1-9). Two patientshad high-risk disease and the median bone marrow tumor burden was a median of38.2% of blasts. None of the patients had undergone a prior allogeneic hematopoieticstem cell transplant.

Allpatients developed cytokine release syndrome (CRS), 4 of which were grade 3 and1 was grade 4. All cases were manageable and resolved with treatment andsupportive care. None of the patients developed neurotoxicity.

The completeremission (CR)/CR with incomplete hematologic recovery was 100%. By day 28, 4patients achieved a CR with negative for minimal residual disease (MRD) and 3of these patients remained MRD negative up to day 161. One patient achieved CRbut was MRD positive, and relapsed by day 29.

Peak CART-cell expansion in peripheral blood occurred between week 1 and 2.

As the first-in-human, universal CAR T-cell therapy for adult relapsed/refractory T-ALL, Dr Wang said, GC027 has demonstrated superior clinical efficacy and induced deep response in patients with acceptable safety profile. She added that trial enrollment is ongoing.

Reference

Wang X, Li S, Gao L, et al. Clinical safety and efficacy study of TruUCAR GC027: The first-in-human, universal CAR-T therapy for adult relapsed/refractory T-cell acute lymphoblastic leukemia (r/r T-ALL). Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I; April 27-28, 2020. Abstract CT052.

This article originally appeared on Cancer Therapy Advisor

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First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL - Oncology Nurse Advisor

Bone Marrow Stem Cells Comments Off on First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL – Oncology Nurse Advisor | May 5th, 2020

Most doctors are prisoners of their education and shackled by their profession.

- Richard Diaz

A month ago we offered up the analysis below on this 'watch item' biotech stock from across the pond exclusively to Biotech Forum members. The stock is up over 40% since then but the concern is still intriguing. Today, we update our original thesis to account for all company-specific news and analyst ratings on this name since our original research was posted.

Orchard Therapeutics (ORTX) is a London, United Kingdom-based biopharmaceutical company that IPOd in 2018. The companys mission is to help patients with rare conditions by leveraging its understanding of gene therapy. The companys approach is to take a patients own blood stem cells and insert into them a working copy of the missing or malfunctioning gene. The companys approach circumvents the need for a bone marrow transplant by taking advantage of blood stem cells' intrinsic capacity to self-renew in a patients bone marrow and produce new blood cells of all types. The ultimate goal is to permanently correct genetic disorders through the use of a single treatment. The companys pipeline is broken down into three overarching programs: neurometabolic disorders, primary immune deficiencies, and blood disorders. The companys lead product candidates are OTL-200, OTL-101 and OTL-103. The companys only commercial product is Strimvelis, which has been approved by the EMA but not by the FDA. Orchard Therapeutics has a market capitalization of just over $1 billion and trades for around $11 a share.

Pipeline:

Source: Company Presentation

OTL-200:

OTL-200 is an ex vivo autologous gene therapy in development to treat Metachromatic leukodystrophy. The drug uses a modified virus to insert an operational copy of the ARSA gene into a patients cells. OTL-200 has received rare pediatric disease designation from the FDA. MLD is a rare and deadly inherited disease. The disease is characterized by the accumulation of fats called sulfatides, which causes a breakdown in the protective fatty layer surrounding nerves in the central and peripheral nerve systems. It is estimated that 1 in 40,000 to 1 in 160,000 people have the disease worldwide. OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Source: Company Presentation

On December 2nd, 2019, under accelerated assessment status, The EMA accepted the review for a marketing application of OTL-200 for MLD. Under accelerated assessment, the review period is 150 days, compared to the normal 210 days. The action date should occur very shortly.

Source: Company Presentation

Looking ahead, the company expects to obtain approval and launch the drug in Europe in the second half of 2020. The company is currently preparing for a commercial launch. Furthermore, the company expects a U.S. regulatory filing, a BLA, in late 2020 to early 2021.

Source: Company Presentation

OTL-101:

OTL-101 is an ex vivo autologous gene therapy in development to treat adenosine deaminase severe combined immunodeficiency. The drug uses a modified virus to insert an operational copy of the ADA gene into a patients cells. OTL-101 has received both Breakthrough Therapy designation and Rare Pediatric Disease designation from the FDA. ADA-SCID is a rare, inherited, pediatric disorder that is commonly fatal when undiagnosed or left untreated. The disorder is the result of a deficiency in adenosine deaminase. The disease culminates in a complete lack of/minimal immune system development. It is estimated that the diseases annual incidence is between 1 in 200,000 and 1 in 1 million live births.

Looking ahead, Orchard Therapeutics will initiate a rolling BLA filing in the U.S. for OTL-101 in ADA-SCID in the first half of 2020 with an anticipated completion of the filing within 12 months.

Source: Company Presentation

OTL-103:

0TL-103 is an ex vivo autologous gene therapy in development to treat Wiskott Aldrich syndrome. The drug uses a modified virus to insert a working copy of the WAS gene into a patients cells. WAS is a rare, X-linked, recessive, inherited immune disorder, which is characterized by reoccurring severe infections, autoimmunity, eczema and severe bleeding episodes. The company has received Rare Pediatric Disease designation and Regenerative Medicine Advanced Therapy designation from the FDA. OTL-103 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Looking ahead, the company is preparing to file a BLA in the U.S. and an MAA in the EU for OTL-103 in WAS in 2021.

Source: Company Presentation

As of December 31st, 2019, the company had cash and cash equivalents of over $300 million including marketable securities. Research and development expenses for the fourth quarter were $30.8 million, compared to $17.4 million in Q4 of 2018. Selling, general and administrative expenses were $18.5 million in the quarter, compared to $11.9 million in the same quarter of 2018. Overall, the company had a net loss of $45.4 million in the quarter, compared to a net loss of $25 million in the same period of 2018. The company believes that its current balance sheet will allow it to fund all anticipated operations and capital expenditures into the second half of 2021.

Despite a sizable market cap, Orchard gets little in the way of attention from analysts. That is probably because it is headquartered overseas. The current median analyst price target on Wall Street is just over $25.00 a share.

Oppenheimer reissued its Buy rating and $28 price target on April 1st. In mid-March, Cowen & Co. did the same with an identical price target in Mid-March. This follows a similar call at Cowen in December with strongly bullish commentary:

Orchard Therapeutics has a differentiated and promising lentiviral, ex vivo pipeline with gene therapies for ADA-SCID, WAS and MLD likely to get approved in 2021-22. With a deep early stage pipeline focused on validated targets, we anticipate strong stock appreciation as drugs are approved and early stage programs yield positive clinical results and advance into pivotal studies in 2020/21."

On September 17th, 2019, Guggenheim initiated coverage with a buy rating and a $31 a share price target. The analyst at Guggenheim views the company's ex vivo, lentiviral gene therapy platform as differentiated and de-risked. Furthermore, three drug approvals within the next 24 months bode well for share appreciation. Finally, in early September of last year, Barclays (NYSE:BCS) initiated coverage with an overweight rating and a $21 price target. The analyst at Barclays sees a company that strategically targets genetic diseases that have validated approaches with hematopoietic stem cell transplant, a fairly de-risked pipeline and multiple catalysts over the next 24 months.

Orchard has multiple shots on goal and several definable milestones in the coming quarters. Given some of the recent turmoil in the credit markets, anything that will need to raise capital in the foreseeable future is taken out and shot when markets have deep down days.

Given that, I still have Orchard as a solid 'watch item' holding and added several hundred shares in my personal account in late March. The shares appear to have an attractive risk/reward profile on a longer term basis, but I would accumulate on dips given the shares run up in April. Once volatility ebbs and we have more confidence in the economy and markets, this is a name we will revisit and maybe upgrade to a larger recommended holding.

The securest place is a prison cell, but there is no liberty

- Ben Franklin

Bret Jensen is the Founder of and authors articles for the Biotech Forum, Busted IPO Forum, and Insiders Forum

Live Chat on The Biotech Forum continues to be very active with new trade and the lucrative covered call ideas available thanks to the spike in market volatility throughout the trading day. If you join the The Biotech Forum today by clicking HERE you will automatically get access to our model portfolio, Live Chat, investment archives and our next 'option play of the week.'

Disclosure: I am/we are long ORTX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Orchard Therapeutics: Updating The Investment Thesis - Seeking Alpha

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics: Updating The Investment Thesis – Seeking Alpha | May 5th, 2020

SAN CARLOS, Calif., May 05, 2020 (GLOBE NEWSWIRE) -- BioCardia, Inc.[Nasdaq: BCDA], a leader in the development of comprehensive solutions for cardiovascular regenerative therapies, today announced data from a recent animal study performed by the Company that demonstrate meaningful improvements in heart function for subjects treated with its allogenic (from another donor, or off the shelf) neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) program for heart failure, known as CardiALLO. In addition, the Company is planning further exploration and discussion with the U.S. Food and Drug Administration (FDA) on the use of its allogenic cells for COVID-19 related Acute Respiratory Distress Syndrome (ARDS).

In the 26 animals treated with both low dose and high dose NK1R+ MSC, echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow were meaningfully improved, with all three measures being statistically significant for both dosage levels over control animals.

The CardiALLO cell therapy is being developed initially to treat heart failure patients whose cells do not qualify for its lead autologous cell therapy, CardiAMP (BCDA-01).

BioCardia Chief Scientific Officer Ian McNiece, PhD, said, In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

COVID-19 Induced Acute Respiratory Distress Syndrome (ARDS) Exploration

The Company also intends to submit an IND for the use of its NK1R+ MSC delivered via intravenous (IV) infusion for Acute Respiratory Distress Syndrome (ARDS) caused by COVID-19.

Based on preliminary clinical reports on COVID-19, respiratory failure complicated by ARDs is the leading cause of death for COVID-19 patients.1 ARDS is a type of respiratory failure characterized by the rapid onset of widespread inflammation in the lungs.

The anti-inflammatory effects of MSC have been well-documented and MSC have been shown to reduce inflammation and injury in models of lung disease.2 The specific MSCs used in BioCardias allogenic cell therapy are expanded from cells selected for the presence of the NK1 receptor, which is known to bind to substance P, an important neuropeptide associated with inflammation throughout the body and a primary mediator of inflammation in the airways.3,4

Our NK1R+ allogenic MSC may have more potential than other MSC approaches being advanced today due to their interaction with Substance P, said BioCardia CEO Peter Altman, PhD. This COVID-19 related work will be the Companys first clinical investigation outside of the cardiac space and our first exploring therapy for the lung. A recent patent publication (US 2020/0101113 A1) shows that BioCardia has long intended for these remarkable reparative cells to be targeted for respiratory disorders, in addition to cardiovascular disease. Addressing inflammation in the lungs is an important contribution we may be able to make using our NK1R+ allogenic MSC therapy.

The Companys allogenic cells are expected to be manufactured at BioCardias clinical stage cell manufacturing facility in San Carlos, California.

About BioCardiaBioCardia, Inc., headquartered in San Carlos, California, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the Companys biotherapeutic product candidates in clinical development. The Company's approved products include the Helix transendocardial delivery system and its steerable guide and sheath catheter portfolio. BioCardia also partners with other biotherapeutic companies to provide its Helix System and clinical support to their programs studying therapies for the treatment of heart failure, chronic myocardial ischemia and acute myocardial infarction.

Forward Looking Statements This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include, among other things, references to the development of NK1R+ cells for the treatment of heart failure and ARDS secondary to COVID-19, potential FDA IND acceptances, and potential FDA filings, statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations. Such risks and uncertainties include, among others, the inherent uncertainties associated with developing new products or technologies. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.

We may use terms such as believes, estimates, anticipates, expects, plans, intends, may, could, might, will, should, approximately or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in BioCardias Form 10-K filed with the Securities and Exchange Commission on April 9, 2020, under the caption titled Risk Factors. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

Media Contact: Michelle McAdam, Chronic Communications, Inc.michelle@chronic-comm.com(310) 902-1274

Investor Contact: David McClung, Chief Financial Officerinvestors@BioCardia.com(650) 226-0120

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BioCardia Announces Positive Preclinical Results Supporting Investigational New Drug Application for Anti-Inflammatory Cell Therapy in Heart Failure -...

Cardiac Stem Cells Comments Off on BioCardia Announces Positive Preclinical Results Supporting Investigational New Drug Application for Anti-Inflammatory Cell Therapy in Heart Failure -… | May 5th, 2020

Too lazy to wear make-up while working from home? You're not alone - according to Bloomberg, a recent survey found about 90 per cent of women working remotely during the coronavirus pandemic are going barefaced.

Even celebrities who rarely show themselves without a face full of make-up have joined the fray.

View this post on Instagram

in return for this pic of me literally first thing in the morning, please donate to the intensive care support at https://platform.nationalfundingscheme.org/COVID.ICS?charity=COVID.ICS#.XoNf4y9lCfA thanks so much

A post shared by Helen Mirren (@helenmirren) on Mar 31, 2020 at 8:30am PDT

A-listers including Katy Perry, Julia Roberts, Julianne Moore, Salma Hayek, Halle Berry, Helen Mirren and Jessica Alba have all posted make-up-free selfies while stuck at home.

This does not bode well for beauty companies - data from market research firm NPD Group show total beauty sales in the United States declined 58 per cent year on year in the week ending March 28. It can be beneficial, though, if women begin to put their skincare needs first.

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Pre-quarantine ?? mid-quarantine ???? Hope your stay-at-home plans tonight include joining me for a beautiful and breezy episode of #AmericanIdol, taped in heavenly Hawaii back in January and February ?

A post shared by KATY PERRY (@katyperry) on Mar 29, 2020 at 2:19pm PDT

"Now that I am home more, I have no excuse to not be good to my skin. New habits include home facials and face rolling. I have also been using my Foreo face massager weekly and I've been thinking about adding in an LED face mask," says Los Angeles-based entrepreneur Tracy Fong.

Fong is not the only one spending more time on her skin. Beauty retailers such as Joyce Beauty may have noted a decline in lipstick sales, but customers are now stocking up on skincare items such as beauty tools and sheet masks.

"I've added steps to my usual, more basic routine and am trying new things," says fashion executive Jane Tong. "I'm enjoying the little bit of 'me time' that I didn't have pre-quarantine. My skin has definitely improved, but I think it can be attributed to giving it more attention, plus more sleep and a slower-paced lifestyle overall."

View this post on Instagram

My incredible friends @beloveapparel are donating $7 from every I Choose Love purchase to @together.rising who are helping children and families facing hunger due to mass school closures. Let’s help each other and small businesses! #ichooselove #beloveapparel #togetherrising

A post shared by Julia Roberts (@juliaroberts) on Mar 21, 2020 at 11:27am PDT

While staying at home has given women more time to pamper themselves, working from home can bring new skincare concerns. Being stuck indoors all day means that the skin is exposed to a range of new pollutants.

"We all know how damaging the sun's rays can be - but there are many other light wavelengths that we need to be careful of, including blue light. Wherever you live in the world, indoor pollution can be as bad as outdoor pollution. Additional factors such as mould, heating, air conditioning or poor ventilation can stress out the skin," says Nigma Talib, a Los Angeles-based naturopathic doctor and trained medical aesthetician who counts celebrities such as Gwyneth Paltrow and Penelope Cruz among her clients.

View this post on Instagram

I am grateful to my @theshalanyc teachers @mskleigh @barbaraverrochi @ashes76 for offering so many of us an online community during this difficult time. Those classes are something to look forward to everyday. ?? ???? Support small businesses - after my class I ordered a few things from @ondabeautynyc while wearing my cozy @suziekondi track suit. Love to u female entrepreneurs ?

A post shared by Julianne Moore (@juliannemoore) on Mar 20, 2020 at 1:15pm PDT

For this reason, Talib advocates that women simplify their routines and focus on high-quality products that help counteract environmental aggressors.

At the top of her list is a resurfacing cleanser, which contains exfoliating agents such as glycolic acid and salicylic acid, to help remove dead skin cells which can build up daily.

A nourishing moisturiser packed with ingredients such as collagen and hyaluronic acid can protect against moisture loss and oxidative stress, which occurs when there is an imbalance between free radicals (unstable molecules with the potential to damage cells) and antioxidants in your body.

Don't forget to apply sunscreen, either. "Layer it on top of a serum loaded with plant stem cells. Sunscreen alone won't prevent light damage to the skin, and the addition of plant stem cells makes your sunscreen 10 times more effective," she says.

Forgoing make-up entirely is not necessarily a good thing. Instead, Talib suggests women pare down their routine and choose products that are multipurpose. Invest in "clean" brands free of toxic chemicals and ingredients, and stick to brands that your skin is already familiar with.

"If it's good quality, make-up can sometimes act as a protective barrier to the skin. I like to suggest a no-make-up look that is simple and free from fussy techniques such as contouring." she says.

View this post on Instagram

Proudly wearing my @Bella_Freud jumper in support of the #ChangeAGirlsLife campaign in partnership with with Prince Charles’ @PrincesTrust ? Proceeds from all onlines sales throughout March from Bella’s #SheSaid collection will be donated to the The Prince’s Trust to support young women and girls. Bella’s inspiration behind the shirt is that “When a woman speaks it’s good to listen” ??

A post shared by Salma Hayek Pinault (@salmahayek) on Mar 10, 2020 at 7:59am PDT

"Start with a BB cream or tinted moisturiser with SPF that isn't heavy. Go for a natural colour on your lips - either a simple liner or lip gloss - and a coat of mascara to accentuate your eyes.

"Let your natural beauty shine. You might even come out of quarantine more confident in your skin."

For the latest updates on the coronavirus, visithere.

This article was first published in South China Morning Post.

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Make-up out, skincare in when working from home. Just ask the stars - AsiaOne

Skin Stem Cells Comments Off on Make-up out, skincare in when working from home. Just ask the stars – AsiaOne | May 5th, 2020

Marky Jaquez, 19, has recessive dystrophic epidermolysis bullosa, a condition found in fewer than one in one million newborns. (Picture: Melissa Jaquez/Metro.co.uk)

A teenager whose rare skin disorder means that even the slightest touch leaves him in extreme pain has defied doctors predictions to live to adulthood. Marky Jaquez, from Wichita, Kansas, cannot hug his parents, wear clothes or even walk because any friction causes layers of skin to peel away from his body.

Wheelchair-bound, Marky, 19, has never been able to walk because the ground would shred his feet like a cheese grater and must be covered head-to-toe in bandages to protect his fragile skin from tearing. He has recessive dystrophic epidermolysis bullosa, a condition found in an estimated one in one million newborns that often leads to life-threatening skin infections and even cancer.

The illness is caused by a faulty gene, passed onto children by parents. Tragically, Markys brother Carlos was born with it too, and died aged just 14. Doctors warned Markys parents he was unlikely to live beyond the age of 11, but he defied that grim prediction and will celebrate his 20th birthday later this year.

His mom, Melissa Jaquez, 40, said: Marky only has one layer of skin and because of his genetic makeup, the skin has no way of anchoring itself the body. Any form of touch or friction means his skins just tears away.

Its world shattering to think I will lose both of my boys to this and I dont want to think about what life will be like afterwards. I feel like my purpose in life is to give Mary the best life I can for the time that I have him.

I am not able to hug my son because any friction just causes his skin to come away and tear right off. It causes him excruciating pain and I feel so much guilt, but theres nothing I can do to help him.

But despite all of this he is a very happy and joyful boy and he amazes me every single day. Hes an inspiration and proves to everyone that you can keep going with whatever youre going through, no matter how bad it is.

Marky is the youngest of three brothers born to healthcare worker, Melissa. Eldest son Michael is 24, with Carlos dying of heart failure caused by the skin condition in 2015. Marky and Carlos, who would be 21 this year, were both born with the same condition after inheriting a faulty gene from their parents.

Melissa said doctors suspect both she and the boys father carry the faulty gene that causes the genetic mutation. Although both are unaffected themselves, they have an estimated 25% chance of having a child born with recessive dystrophic epidermolysis bullosa. Melissa has since split up with Marky and Carlos father neither of whom knew they carried the faulty gene until Carlos was born, she said.

Melissa said she became aware of the risks involved with having a child after Carlos was born, but claims she was told by a clinician that the chance of her having a second child with the same condition would be less than 1%.

Melissa said: I lost Carlos to the condition because his heart just gave up. His body was in a constant battle to repair his wounds and that took its toll eventually.

His condition was much more progressive than Markys. He got real bad, real quick and by the end could not even breathe on his own.

My pregnancy with Marky was unplanned, but when I got pregnant I was told the chance of me having another baby with the same condition is 1%. They said having two children with it is unheard of.

Marky was born with the most severe type of epidermolysis bullosa meaning he was delivered without skin on his hands, feet and chest because because of the friction of childbirth.

Ever since Marky was little, Melissa, who is now married to Marcos, 30, has had to spend up to three hours a day covering his skin in protective bandages and cleaning open wounds to prevent infection.

She has said every day of motherhood so far has felt like a battle and admits she has struggled to have a normal mother and son relationship with Marky, because of his condition.

Every day is such a massive challenge for us. I struggle with the grief of knowing that when I walk into my sons room in a morning, we cant have fun. I dont get to do the normal things that a mom and a son does.

Instead when I go into his room I know I have to cause him pain and spend two hours doing his bandages. That does give me a lot of anxiety and guilt.

Taking his bandages off does cause excruciating pain. We have to soak his skin every day to soothe his wounds and give him pain medication. Its incredibly upsetting for me.

Whilst there is no cure for epidermolysis bullosa and Marky is highly likely to sadly one day lose his life to the condition, Melissa is hopefully he will survive for several more years.

He has been selected to take part in experimental treatment to create genetically modified skin stem cells that can slow down the effects of the progressive disease. The family plan to travel to Stamford Hospital, Connecticut, later this year to undergo the treatment.

Melissa and Marcos work opposite shifts and have a carer who comes to help look after Marky for three hours each day. Melissa has paid tribute to her amazing son, who she believes can go on to live for many happy years, despite the predictions of doctors.

She said: The treatment at Stamford could add several years to his life. It will reduce the pain and make it easier for his body to cope.

We have tried for that treatment for years, so were thrilled.

Marky has always been an incredibly happy boy and he has just got on with it, without ever moaning or saying why me. Ive had messages from people all over the world to say Marky has inspired them.

Hes an amazing human being and he makes me so proud.

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Terminally-ill teen with agonizingly-sensitive skin defies doctors to live to 19 - Metro.co.uk

Skin Stem Cells Comments Off on Terminally-ill teen with agonizingly-sensitive skin defies doctors to live to 19 – Metro.co.uk | May 5th, 2020

A team of doctors and researchers at the Abu Dhabi Stem Cell Center (ADSCC) administered the treatment in the UAE to 73 COVID-19 patients, who were all successfully treated and cured, without any immediate side effects, according to a statement by the United Arab Emirates (UAE) health ministry from 1 May.

The process involved a minimally invasive method where the patients stem cells are extracted, activated and turned into fine mist to be inhaled into the lungs. This was done in addition to the conventional treatment and is expected to work by supporting the established protocol of management of symptoms.

The ministry said in the statement, It is hypothesised to have its therapeutic effect by regenerating lung cells and modulating the immune response to keep it from overreacting to the COVID-19 infection and causing further damage to healthy cells.

The treatment has successfully undergone the initial phase of clinical trials, demonstrating its safety, and further trials for its efficiency are ongoing; expected to be completed in a couple of weeks.

Dr Fatima al-Kaabi, head of haematology and oncology at the Sheikh Khalifa Medical City in the UAE, told CNBC, Its very early to say at this stage. If all went well, this could reach the market in three months, she added.

Going further back, a pilot study in China on seven COVID-19 patients found that intravenous infusions of donor mesenchymal stem cells (MSC) - multipotent stem cells - improved patient outcomes and helped all of them recover. An Israeli pharmaceutical company, Pluristem Therapeutics, also tested stem cells in seven critical hospitalized patients and found positive results.

Additionally, the US Food and Drug Administration (FDA) approved MSC use in extremely sick COVID-19 patients under expanded access compassionate use on 5 April according to a report in The Scientist, even though the experts seemed divided on the logic on which the investigative treatment may have worked.

A hospital in New York tried the therapy as an experiment on 12 patients, 10 of whom were able to come off ventilators, reports CBS news. The Australian regenerative medicine company Mesoblast has also announced a 300-person trial for its stem cell therapy remestemcel-L (which was used in the New York trial) to determine whether it will work on patients suffering with severe lung inflammation.

Currently, there are over 20 active stem cell trials for COVID-19, most focusing on the use of MSCs.

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Stem Cell Treatment for COVID-19; Doctors Divided on Its Scope - The Quint

Skin Stem Cells Comments Off on Stem Cell Treatment for COVID-19; Doctors Divided on Its Scope – The Quint | May 5th, 2020

The global Plant Stem Cell Market research report thoroughly explains each and every aspect related to the Plant Stem Cell Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business. The growth trend forecasted on account of thorough examination offers in-depth information regarding the global Plant Stem Cell Market. A pathway of development is offered by the market to the several connected networks of businesses under it, which include different firms, industries, organizations, vendors, distributors, and local manufacturers too. All the key Plant Stem Cell Market players compete with each other by offering better products and services at a reasonable price in order to grab significant share at the regional and global level market.

Plant stem cells are undifferentiated cells within the meristems of plants.Plantstem cellsserve as the origin of plant vitality as they maintain themselves and provide a steady supply ofprecursor cellsto form various tissues and organs in plants. Plant stem cells are characterized by two distinctive properties namely its ability to create differentiated celltypes and to self-renew such that the number of stem cells remain stable. The plant stem cell market focuses on this ability of plant cells and thus, processed extracts from the buds, roots and shoots are in high demand and are widely used for topical uses.ription

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The report incorporates an estimated impact of strict standards and regulations set by the government over the market in the upcoming years. The market report also comprises exhaustive research done using several analytical tools such as SWOT analysis to identify the market growth pattern.

Top Manufacturers in GlobalPlant Stem CellMarket Includes:Oriflame Holding AG, MyChelle Dermaceuticals, LLC, Natura Therapeutics Inc, Aidan Products LLC, Mibelle Biochemistry, Phyto Science SDN BHD, and Renature Skin Care Inc.

Regions & Countries Mentioned In The Plant Stem Cell Market Report:

North America ( United States)

Europe ( Germany, France, UK)

Asia-Pacific ( China, Japan, India)

Latin America ( Brazil)

The Middle East & Africa

Key Highlights of the Table of Contents:

Plant Stem Cell Market Study Coverage: It includes key manufacturers covered, key market segments, the scope of products offered in the global market, years considered, and study objectives. Furthermore, it tuches the segmentation study provided in the report on the basis of the type of product and applications.

Plant Stem Cell Market Executive Summary: This section emphasizes on the key studies, market growth rate,Competitive landscape, market drivers, trends, and issues.

Plant Stem Cell Market Production by Region: The report provides information related to import and export, production, revenue, and key players of all regional markets studied are covered in this section.

Plant Stem Cell Market Profile of Manufacturers: Analysis of each market player profiled is detailed in this section. This also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

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Table of Contents

Report Overview:It includes the Plant Stem Cell market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary:This section of the report gives information about Plant Stem Cell market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players:Here, key players of the Plant Stem Cell market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study:All of the regions and countries analyzed in the Plant Stem Cell market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

An Overview of the Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

National governments and international bodies are focused on adopting collaborative efforts to encourage financial institutions to meet the financial needs of customers and members affected by the coronavirus. However, there are some sectors that have remained unscathed from the impact of the pandemic and there are some that are hit the hardest.

We, at Coherent Market Insights, understand the economic impact on various sectors and markets. Using our holistic market research methodology, we are focused on aiding your business sustain and grow during COVID-19 pandemics. With deep expertise across various industries-no matter how large or small- and with a team of highly experienced and dedicated analysts, Coherent Market Insights will offer you an impact analysis of coronavirus outbreak across industries to help you prepare for the future.

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Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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Plant Stem Cell Market 2026 Growth Trends by Manufacturers, Regions, Type and Application, Forecast - Latest Herald

Skin Stem Cells Comments Off on Plant Stem Cell Market 2026 Growth Trends by Manufacturers, Regions, Type and Application, Forecast – Latest Herald | May 5th, 2020

You might feel pressured to follow a 10-step skincare routine to have the complexion of your dreams. But FYI, a fuss-free routine can be just as effectiveespecially if your goal is as simple as looking and feeling fresh and hydrated. After all, hydration is the key to having a soft, smooth, and clear face. And if you're worried about future fine lines and wrinkles, then you definitely need to use moisturizing products. If youre still not sure about how to build a simple routine, check out our 3-step one below.

Cleansing your skin in the morning can freshen you up for a new day, and at night can remove dirt and grime and prepare you for bed. When choosing a facial wash, look for one that doesnt strip your skin of its natural oils. That way, your skin stays hydrated and wont overproduce oils, which can lead to shine and breakouts.

In the morning, use a gentle cleanser like Cetaphils Gentle Foaming Cleanser. It can remove dirt and oil while moisturizing the skin with vitamins B5 and E. It also has a hypoallergenic, fragrance-free formula, making it great for those with sensitive skin.

At night, use an exfoliating cleanser to get rid of the grime, sweat, and makeup from the day. Try Cetaphils Daily Exfoliating Cleanser, which can gently slough off dead skin cells and clean dirt and oil away without stripping or irritating your skin. It contains bamboo stem extract exfoliant and moisturizing ingredients like Vitamin B5 and E to help your skin feel soft, smooth, and radiant.

While you may only be in your 20s, that doesnt mean you dont have to apply eye cream. Doing so will not only help prevent wrinkles from forming in the future, but will also help address problems you might be dealing with now (hello, dark circles!). Try Cetaphils Hydrating Eye Cream-Serum with Hyaluronic Acid, a cream-serum hybrid that absorbs quickly and moisturizes deeply. Its formulated with hyaluronic acid, a moisturizing agent that can help improve the skins natural elasticity, hold on to moisture in the skin, and improve the appearance of fine lines and fatigue. Those all-nighters youve been pulling dont stand a chance against this product.

Using a creamy moisturizer might sound counterintuitive to feeling fresh, but hear us out! Sometimes, lightweight moisturizers arent enough to keep your skin hydrated all day and all night. Using a cream can help retain your skins natural moisture and protect it from dryness (perfect if youre struggling with dry patches!).

For this, why not try Cetaphils Rich Night Cream? It can help rehydrate and soothe skin, plus, it contains vitamins C, A, and E, plant extracts, and hyaluronic acid to help protect your skin from dryness. BTW, you can even use it during the day if you want! Just lightly apply it on areas that need a little extra TLC.

Shop Cetaphils face range on Lazada and learn more by following Cetaphil on Facebook.

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This Low-Maintenance Skincare Routine Will Leave Your Skin Feeling Fresh And Hydrated - Cosmopolitan Philippines

Skin Stem Cells Comments Off on This Low-Maintenance Skincare Routine Will Leave Your Skin Feeling Fresh And Hydrated – Cosmopolitan Philippines | May 5th, 2020

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced a new development and commercialization agreement with Saladax Biomedical, Inc. (Saladax), a leading diagnostics provider focused on developing blood tests for personalized dosing, to develop and validate a fully automated nanoparticle immunoassay kit designed to simplify and streamline therapeutic drug monitoring (TDM) for patients treated with the conditioning agent busulfan.

At AVROBIO, we push ourselves to be at the forefront of technologies advancing lentiviral gene therapy, and its in this spirit that were funding the development of this kit, said Geoff MacKay, AVROBIOs president and CEO. Our personalized conditioning approach is already delivering results. We believe this new assay kit will, for the first time, provide convenient busulfan TDM close to the patient, potentially improving both the patient experience and long-term outcomes, as well as enabling many more hospitals and clinics to become TDM-capable sites.

AVROBIOs state-of-the-art plato gene therapy platform incorporates TDM protocols designed to optimize busulfan dosing over four days, with the goal of maximizing stem cell engraftment while minimizing side effects. TDM evaluates how quickly a patient metabolizes busulfan a rate that can vary significantly from patient to patient and even from one day to the next for the same patient. Current assays that inform that dose adjustment can take hours to return results and must be processed at specialized laboratories with trained staff that may not be geographically convenient to the gene therapy dosing site.

The technology used to deliver these rapid test results is based on an extensive intellectual property portfolio developed by Saladax in the field of TDM. The new assay kit under development by Saladax, which collects a small blood sample, is able to return results on patient metabolization of busulfan in minutes using hospitals standard analytical devices, greatly expanding access to personalized conditioning with busulfan.

Personalized Gene Therapy to Optimize Durable Protein Expression including in Brain, Muscle and Bone

AVROBIOs investigational gene therapies start with collecting the patients own hematopoietic stem cells. In the companys manufacturing process, a lentiviral vector is used to integrate a therapeutic gene designed to produce functional protein essential to cellular health into the patients chromosomes. Prior to dosing, treating clinicians use busulfan, an extensively validated conditioning agent generally considered to be the gold standard for ex vivo lentiviral gene therapy, to create space in the patients bone marrow. Finally, the patient receives the gene therapy and the therapeutic stem cells are expected to engraft in the bone marrow and produce generations of daughter cells, each containing the therapeutic gene. This approach is designed to drive durable production of the functional protein throughout the patients body, including hard-to-reach tissues such as the brain, muscle and bone. A distinguishing feature of this type of gene therapy with busulfan conditioning is that some of the corrected cells are expected to cross the blood-brain barrier and thereby potentially address central nervous system manifestations.

Earlier this year, AVROBIO reported initial clinical results for the first patient conditioned with busulfan using TDM prior to dosing in AVROBIOs Phase 2 clinical trial of its investigational gene therapy, AVR-RD-01, for Fabry disease. The early data from this patient showed increased endogenous enzyme activity at one month following dosing, as compared to other patients in the trial who received a different conditioning agent. Initial data suggest side effects, including nausea, mucositis, fever, rash and hair loss, which were consistent with those expected based on clinical experience of busulfan, developed eight to 10 days after dosing with busulfan and resolved quickly.

About AVROBIO

Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits of Saladaxs immunoassay kits, including the ability to improve, simplify and streamline therapeutics drug monitoring for patients treated with the conditioning agent busulfan and enable local commercialization of AVROBIOs proprietary platform worldwide, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that AVROBIO may not realize the intended benefit of Saladaxs immunoassay kits, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO to Collaborate with Saladax Biomedical on New High-Speed Diagnostic Assay Used with Busulfan Conditioning to Enable Widespread...

Bone Marrow Stem Cells Comments Off on AVROBIO to Collaborate with Saladax Biomedical on New High-Speed Diagnostic Assay Used with Busulfan Conditioning to Enable Widespread… | May 4th, 2020

People with cancer who contract the new coronavirus appear to have a greater risk for severe COVID-19 illness and death, but this may depend on their cancer stage and the type of treatment they are receiving, according to recent research. In fact, those with early-stage cancer may fare as well as people who have not had cancer.

Researchers from some of the earliest and hardest hit epicenters of the COVID-19 pandemic described outcomes among cancer patients with the coronavirus (officially known as SARS-CoV-2) during a special session the American Association for Cancer Research (AACR) virtual annual meeting last week. Soon after the conference, another group of researchers published an analysis of mortality among cancer patients in New York City.

Early reports from China, where the pandemic originated in late December, showed that older people, those with compromised immune systems and those with underlying health conditions are more susceptible to severe COVID-19. One study saw a death rate of 6% for people with cancermore than twice as high as the overall estimated COVID-19 mortality rate in China, but lower than the rates seen in people with diabetes (7%) or cardiovascular disease (11%).

Chemotherapy medications and some targeted therapies for cancer can cause neutropenia, a temporary depletion of immune system white blood cells that fight infection. People who receive bone marrow stem cell transplants or CAR-T therapy or for blood cancers typically receive strong chemotherapy to kill off existing blood cells and make room for the new ones. Conversely, immunotherapies such as checkpoint inhibitors and CAR-T therapy unleash natural or engineered T cells to fight cancer, which in some cases can trigger an excessive immune response that leads to harmful inflammation.

Two reports at the AACR meeting provided updates from China. Li Zhang, MD, PhD, of Tongji Medical College described outcomes among 28 cancer patients with COVID-19 in Wuhan, the initial epicenter of the pandemic.

Seven had lung cancer and the remainder had 13 other cancer types. Just over a third had Stage IV, or metastatic, cancer. Nearly 30% acquired the coronavirus at medical facilities. About half had severe disease, 10 patients required mechanical ventilators and eight diedmostly from acute respiratory distress syndromegiving a mortality rate of 29%.

Although three quarters had ever undergone surgery, radiation or chemotherapy, a majority had not received treatment recently. Only one person received radiation, three received chemotherapy, two received targeted therapy and one received immunotherapy within two weeks prior to their COVID-19 diagnosis. Recent cancer treatment was associated with a fourfold increased risk of severe outcomes. However, the single patient treated with a checkpoint inhibitor (for liver cancer) had mild COVID-19 and a short hospital stay.

Similarly, as part of his discussion of immunotherapy for cancer in the COVID-19 era, Paolo Ascierto, MD, of the National Tumor Institute in Naples, noted that just two out of 400 patients on immunotherapy at his institute tested positive for the coronavirus, they were asymptomatic and they recovered quickly, leading him to speculate that immunotherapy might somehow be protective against COVID-19.

Hongbing Cai, MD, of Zhongnan Hospital of Wuhan University, presented data on 105 cancer patients and 536 age-matched people without cancer at 14 hospitals in Hubei province who developed COVID-19. Results were also published in Cancer Discovery. Twenty-two had lung cancer, 13 had gastrointestinal cancers, 11 each had breast cancer and thyroid cancer, nine had blood cancers such as leukemia or lymphomawhich affect white blood cells that carry out immune responsesand six each had cervical and esophageal cancer.

In general, patients with cancer deteriorated more rapidly than those without cancer, Cais team reported. Cancer patients with COVID-19 were nearly three times more likely to have severe or critical illness (34%), be admitted to an intensive care unit ICU (19%) or be put on a ventilator (10%). Whats more, people with cancer were about twice as likely to die as COVID-19 patients without cancer (11% versus 5%, respectively).

People with blood cancers or lung cancer, as well as those with metastatic cancer, had a higher risk of severe events. Two thirds of the blood cancer patients and half of the lung cancer patients had such events. Among the lung cancer patients, 18% were put on ventilators and 18% died. In contrast, no one with breast, thyroid or cervical cancer required ventilators or died.

In particular, those with blood cancersmore than half of whom had severe immune suppressionhad about a 10-fold higher risk of severe events or death. Two thirds had severe symptoms, 22% were put on ventilators and 33% died. These patients all had a rapidly deteriorated clinical course once infected with COVID-19, the researchers wrote.

People with metastatic cancer had about a six-fold higher risk of severe events or death. But people whose cancer had not yet spread were not significantly more likely to have severe events or die than COVID-19 patients without cancer. People currently on cancer treatment and those with a history of cancer who had completed treatment were both at higher risk.

People who underwent surgery within the previous 40 days had higher rates of severe events, ICU admission, ventilator use and death, but this was not the case for those who received only radiation. In this study, unlike Zhangs and Asciertos, people treated with immunotherapy did not fare so well. Four of the six patients who recently received checkpoint inhibitors had critical symptoms and two died.

Based on our analysis, COVID-19 patients with cancer tend to have more severe outcomes when compared to the non-cancer population, the researchers wrote. Although COVID-19 is reported to have a relatively low death rate of 2% to 3% in the general population, patients with cancer and COVID-19 not only have a nearly three-fold increase in the death rate than that of COVID-19 patients without cancer, but also tend to have much higher severity of their illness.

In a related study, Marina Chiara Garassino, MD, of Fondazione IRCCS National Tumor Institute in Milan, presented the first data from the international TERAVOLT registry, which is collecting data about COVID-19 among people with lung cancer and other thoracic malignancies. She noted that TERAVOLT was registering around 70 new cases per week from around the world per week.

This population may be especially vulnerable to COVID-19 due to older age, lung damage, smoking and underlying health conditions, Garassino said. Whats more, the symptoms of COVID-19 overlap with lung cancer, making diagnosis very challenging.

Garassino described results from the first 200 cancer patients with COVID-19 in more than 20 countries. Non-small-cell lung cancer was the most common type, and nearly three quarters had metastatic disease. About 20% received only targeted therapy, 33% received chemotherapy alone and 23% received immunotherapy alone.

A majority (76%) were hospitalized, but most were not offered intensive care for COVID-19; just 9% were admitted to an ICU and 3% were put on ventilators. More than a third (35%) died, mostly due to COVID-19 rather than cancer. Specific types of cancer treatment were not significantly associated with an increased risk of death.

But not all studies have seen worse COVID-19 outcomes among people with cancer. Fabrice Barlesi, MD, PhD, and colleagues looked at 137 COVID-19 patients with cancer at Gustave Roussy, a cancer center near Paris. They had a variety of cancer types, with blood cancers and breast cancer being most common. Nearly 60% had active advanced disease while 40% were in remission or being treated with potentially curative therapy.

Within this group, 25% had worsening COVID-19 after admission, 11% were admitted to the intensive care unit (ICU) and 15% died. Again, people with blood cancers were more likely to have worse outcomes. Treatment with chemotherapy within the past three monthsbut not targeted therapy or immunotherapydoubled the likelihood of worsening disease. But this only applied to people with active or metastatic cancer, not those who had localized disease or were in remission.

The 15% death rate among people with cancer at Gustave Roussy was lower than the 18% rate for all COVID-19 patients in Paris and in France, Barlesi said. His team concluded that both incidence and outcomes of COVID-19 among cancer patients seem to be comparable to the population as a whole. However, people with blood cancers, those treated with chemotherapy and frail patients are at greater risk.

Discussing how to manage cancer patients during the COVID-19 pandemic, Cai recommended self-protective isolation, strict infection control in hospitals and shifting some medical services online.

With regard to cancer treatment, she said, clinicians need to develop individualized plans based on a patients tumor type and stage of disease. She added that postponing surgery, if appropriate, should be considered in areas with current outbreaks. Radiation therapy, she said, could go ahead according to existing treatment plans with intensive protection and surveillance. Whether people with early-stage cancer need to postpone their treatment remains an unanswered question, she said.

Click hereto read the abstracts from the AACR COVID-19 and cancer session.Learn about What People With Cancer Need to Know About the New Coronavirus.

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Cancer Patients With COVID-19 May Have Higher Risk of Severe Illness and Death - Cancer Health Treatment News

Bone Marrow Stem Cells Comments Off on Cancer Patients With COVID-19 May Have Higher Risk of Severe Illness and Death – Cancer Health Treatment News | May 4th, 2020

Researchers are learning more about how to diagnose and better treat blastic plasmacytoid dendritic cell neoplasm, a rare cancer that often presents with skin manifestations, according to a review published March 2020 in Current Opinion in Hematology.1

Blastic plasmacytoid dendritic neoplasm patients have suffered historically poor outcomes. Years ago, doctors were limited to treating these patients primarily with intensive chemotherapy regimens used to treat acute myeloid leukemia or acute lymphoblastic leukemia patients.

But in 2018, the U.S. Food and Drug Administration (FDA) approved tagraxofusp-erzs (Elzonris, Stemline).

Tagraxofusp-erz is the first approved drug indicated specifically for blastic plasmacytoid dendritic neoplasm, and its use is recommended in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology.Newer targeted agents to treat the hematologic malignancy are in the pipeline.

Notable changes in recent years

The World Health Organization (WHO) named blastic plasmacytoid dendritic cell neoplasm (BPDCN) and classified it under acute myeloid leukemia and related precursor neoplasms in 2008. Some eight years later, WHO established BPDCN as a distinct entity.

Just how many people have BPDCN isnt clear. But it is thought that there are about 0.04 cases of the cancer per 100,000 people. And about three in four patients are older men.

Derived from plasmacytoid dendritic cells, BPDCN generally is an aggressive disease. It presents clinically on the skin in about nine out of every 10 cases. Skin lesions tend to be asymptomatic, often appearing as bruise-like lesions, plaques or nodules, according to the paper.

While a small percentage of patients will present with skin disease only, most show signs of BPDCN in the bone marrow, lymph nodes or visceral organs. Rarely, patients will have no cutaneous evidence and instead present with the leukemic phase of the cancer. About 30% of patients also have central nervous system involvement.

Flow cytometry to determine the immunophenotype is an essential component of diagnosing [blastic plasmacytoid dendritic cell neoplasm], the author wrote.

CD123, an interleukin-3 receptor alpha, is over expressed in nearly all BPDCN cases. These cancer cells also may be positive for CD4, CD56, CD303 or TCL1, according to the paper.

Some authors have found a recurrent MYC gene rearrangement in these patients. That particular genetic aberration is associated with an older age at diagnosis and worse prognosis.

Treatment is evolving

Unfortunately, doctors have to rely largely on retrospective studies looking at BPDCN treatment options.Those studies suggest that BPDCN, generally, responds better to acute lymphoblastic leukemia regimens compared to acute myeloid leukemia treatment options. However, most responses to these regimens are transient, the author reported.

Retrospective studies suggest allogeneic stem cell transplant for eligible patients in their first remission offer the highest overall survival rates, including 3- and 4-year overall survival rates ranging from 74% to 82%.Tagraxofusp-erzs targets CD123. It consists of recombinant human interleukin-3 fused to a truncated diphtheria toxin, according to the paper.

Binding the drug to CD123 on the cell surface leads to cellular internalization of the diphtheria toxin, which ultimately leads to inhibition of protein synthesis and cell death, the author wrote.

In a phase I/II clinical trial of 44 untreated or relapsed/refractory BPDCN patients, 21 of 29 previously untreated patients achieved complete remission and 13 of those went on to have a stem cell transplant. Overall response rate of the 15 patients with relapsed/refractory BPDCN was 67% with tagraxofusp-erzs, with an average overall survival of 8.5 months.

Eighteen of the 44 patients studied developed the most critical treatment-related adverse event, capillary leak syndrome. Two patients died from capillary leak syndrome during the study.Researchers are studying investigational agents aimed at treating BPDCN. These include IMGN632, a humanized antibody-drug conjugate with an anti-CD123 monoclonal antibody conjugated to a DNA-alkylating payload, the author wrote.

Researchers are evaluating the safety and efficacy of treating CD123-positive malignancies including BPDCN with the monoclonal antibody targeting CD123 and CD3 XmAb14045.

Venetoclax, a BCL-2 inhibitor, is yet another agent in the pipeline for BPDCN patients.

as knowledge is gained on the molecular changes that occur in [blastic plasmacytoid dendritic cell neoplasm], this will ideally lead to more targeted and effective therapies in the years to come, the author wrote.

Disclosures:

Kendra Sweet, MD, has received honoraria from Stemline Therapeutics.

References:

1 Sweet K. Blastic plasmacytoid dendritic cell neoplasm: diagnosis, manifestations, and treatment. Curr Opin Hematol. 2020;27(2):103-107.

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Investigational agents to treat hematologic malignancy in pipeline - Dermatology Times

Bone Marrow Stem Cells Comments Off on Investigational agents to treat hematologic malignancy in pipeline – Dermatology Times | May 4th, 2020

A novel, bispecific CD19/CD22 chimeric antigen receptor T-cell (CAR-T) therapy was tolerable and resulted in responses among patients with acute lymphoblastic leukemia (ALL), according to results from a phase 1 trial presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020.

Thenovel CAR-T therapy was developed with the hypothesis that dual antigen-targetingstrategies may prevent antigen negative escape, Haneen Shalabi, DO, of theNational Cancer Institute and lead author and presenter of the study, said.

Thephase 1, dose-escalation study treated 13 young patients with ALL with theCD19/CD22 CAR-T therapy at 3 different dose levels, including 3 x 105,1 x 106, and 3 x 106. The bispecific construct containedFMC63 (CD19 scFv) linked with m971 (CD22 scFv) and a 4-1 BB costimulatorydomain.

Patientsunderwent lymphodepletion with fludarabine plus cyclophosphamide prior to theirCAR-T infusion. The primary endpoints were safety and toxicity, and thesecondary endpoints were efficacy, chimeric antigen receptor (CAR) expansion,and CAR persistence.

Atbaseline, the median age was 19.6 (range, 5.4-28.5). Patients had receivedprevious treatments, including hematopoietic stem cell transplant (54%),CD19-targeted therapy (69%), prior CD19 CAR T cell therapy (38.4%),blinatumomab (61.5%), CD22-targeted therapy (38.4%), inotuzumab (30.7%), andCD22 CAR-T therapy (15.4%). Extramedullary disease was present in 46.2% ofpatients.

CAR Tcells were well tolerated and toxicities were reversible in all patients, DrShalabi said.

Cytokinerelease syndrome (CRS) developed in 46% of patients, 15.4% of which was grade 3or higher. Both patients who developed grade 3 or higher CRS had received the 1x 106 dose level of the CD19/CD22 CAR-T product and both requiredtreatment with tocilizumab. One patient developed neurotoxicity, and hadreceived the 3 x 106 dose level.

Of the12 patients evaluable for efficacy, a complete response (CR) was achieved by42% (5) of patients, including all patients who received the 1 x 106 or 3 x 106 dose levelsof the CD19/CD22 CAR-T therapy. There were 2 nonresponders.

Two patientswho received 1 x 106 CAR-T and all patients who received the 3 x 106dose level were negative for minimal residual disease (MRD), with the remainingCRs demonstrating bone marrow clearance. Four of the 5 patients who were MRDnegative were also naive to CAR-T therapy.

Of the 5patients who achieved a CR, 2 relapsed with CD19-positive/CD22-positive diseaseand 3 remained in remission at a median 7 months after CAR T cell infusion.

Severalpatients, however, who were MRD negative in the bone marrow did not achieve CRin their extramedullary disease. Dr Shalabi said that these discrepant resultsbetween marrow and extramedullary disease suggests potentially limited CAR-Ttrafficking to sites of extramedullary disease. She suggested that treatmentat higher dose levels may be needed to overcome this limitation.

CAR T-cellexpansion occurred in all patents who responded, with a median peak inperipheral blood of 7%. At day 28, there were 1.3% CAR T cells in the bonemarrow. The persistence of the CAR T cells in peripheral blood was a median of45.6 days, as measured by flow cytometry.

Dr Shalabi concluded that this early experience with bispecific CD19/CD22 CAR T cells demonstrates clinical activity with reversible CRS and limited neurotoxicity. She noted that future studies will explore a 1 x 107 dose level, intensification of lymphodepletion prior to CAR-T infusion, and consideration of the potential role of immune checkpoint inhibitors to augment CAR-T in extramedullary disease.

References

Shalabi H, Yates B, Shahani S, et al. Safety and efficacy of CD19/CD22 CAR T cells in children and young adults with relapsed/refractory ALL. Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020; April 27-28, 2020. Abstract CT051.

This article originally appeared on Cancer Therapy Advisor

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Global Cell Therapy Technologies Market was valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

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The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

The report study has analyzed revenue impact of covid-19 pandemic on the sales revenue of market leaders, market followers and disrupters in the report and same is reflected in our analysis.

Global Cell Therapy Technologies Market: Overview

Cell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

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Global Cell Therapy Technologies Market: Drivers and Restraints

The growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation Analysis

On the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional Analysis

North America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company GE Healthcare Lonza Merck KGaA MiltenyiBiotec STEMCELL Technologies, Inc. Terumo BCT, Inc. Thermo Fisher Scientific, Inc. Sartorius AG

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

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Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2019-2026) - MR Invasion

Bone Marrow Stem Cells Comments Off on Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2019-2026) – MR Invasion | May 4th, 2020

Leukemia are a heterogeneous group of cancers affecting the bone marrow and White Blood Cells (WBC). Leukemia is characterized by the rapid increase of abnormal blood cells growth or blasts, resulting in a decrease in the numbers of healthy, normal fully modified blood cells, leading to the typical symptoms of bleeding, anemia, and high risk of infection. Leukemia can grow along either the myeloid or lymphoid stem cell lines, it depends on the effect of genetic and epigenetic mutations on the progression of pluripotent stem cells to the various lines of mature cells which then pass into the blood. The effected line, combined with the rate of action and growth of disease reflects the four types of leukemias- Acute Myeloid Leukemia (AML), chronic lymphoblastic leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia. AML: Acute Myeloid Leukemia, is a serious condition, its the most common leukemia suffered by adult people. According to a report from American Cancer Society, the average age for first diagnostic for AML is 64. With few days without treatment, AML develops fast, in duration of few weeks, the patient becomes severely ill. Due to its fast onset and acuteness in nature, there is no staging system for Acute Myeloid Leukemia (AML).The treatment for Acute Myeloid Leukemia (AML) has changed in last 4 decades.

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The drug approval process is difficult in AML, (many drugs have not been approved by USFDA, for instance Laromustine, Dacogen and Tipitarnib) efforts have been made to introduce new therapies in the AML market.

Primary drivers boosting the growth of acute myeloid leukemia (AML) therapeutics market are minimal but increased prevalence of acute myeloid leukemia (AML), increased drug approval rate for AML, classification of acute myeloid leukemia (AML) as an orphan disease. Over the forecast period, population of people over 65 year is anticipated to increase, which is another key driver for acute myeloid leukemia (AML) therapeutics market.

However, lack of targeted therapies in current acute myeloid leukemia (AML) therapeutics landscape, the drug difficult approval process in AML can hinder the growth of acute myeloid leukemia (AML) therapeutics market, but this restraint has opened an opportunity for key players to innovate acute myeloid leukemia (AML) therapeutics market.

How the Coronavirus Threat has Taken Global Business into Uncharted Waters

The global acute myeloid leukemia (AML) therapeutics market is segmented on the basic of disease subtype, treatment type, end user and region.

Based on the disease subtype, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on treatment type, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on end user, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

The global acute myeloid leukemia (AML) therapeutics market is anticipated to show lucrative growth owing to increased investment in innovative technologies by key players. Players in this market using various strategies to fuel their global footprint and to gain a competitive edge. Product pipelines, new product launches, agreements and collaborations, acquisitions, mergers and clinical trials are some key strategies applied from global players in recent years are anticipated to give a robust hike to the market in the forecast period.

Geographically, acute myeloid leukemia (AML) therapeutics market is segmented into regions viz. North America, Latin America, Europe, Asia Pacific and Japan, Middle East and Africa. North America is anticipated to be major contributor to this market accounting maximum percent of share in AML therapeutics market followed by Europe. Slow but constant growth in prevalence for AML in North America is anticipated to fuel the growth in acute myeloid leukemia (AML) therapeutics market. In Asia pacific region, China and India are anticipated to show high growth in acute myeloid leukemia (AML) therapeutics market due to new developments in healthcare infrastructure in the region.

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The players in acute myeloid leukemia (AML) therapeutics market include,

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