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Raleigh man delivers stem cells to patients around the world – WRAL.com

By daniellenierenberg

By Adam Owens, WRAL anchor/reporter

A 74-year-old Raleigh man spends an average of two weeks a month traveling around the world delivering stem cells or bone marrow to patients as part of Be The Match, a volunteer-based donor program.

According to Troy "Davis" Moore, Be The Match uses a database of 135 countries to find life-saving bone marrow or stem cells for patients with leukemia and other blood diseases. The stem cells are delivered from donors to patients around the world by volunteers like Moore.

Moore said, when he retired 17 years ago, gardening and working around the house just wasn't enough. His friend, who was already a volunteer courier with Be The Match, told him about the opportunity.

To date, Moore has logged more than 5,000 hours as a volunteer courier. He has traveled as far as London, Barcelona, Croatia, Portugal, Singapore and Taiwan.

On Thanksgiving week, Moore will travel to South America to pick up blood stem cells and deliver them to a patient in the United States. He had to get a rapid COVID-19 test before his trip.

"It's been a lot more challenging during COVID-19 because the rules have changed so much in these countries," said Moore, explaining he recently ran into some trouble in Croatia when he hadn't had a coronavirus test in 72 hours.

Moore said, although he doesn't get to meet the families he helps due to confidentiality, the job is incredibly special. In an interview with WRAL's Adam Owens, Moore described a trip he made on Christmas Day, 10 years ago, to deliver bone marrow from the United Kingdom to a hospital in Columbus, Ohio.

Moore said a nurse was walking him through the hospital hallways when she tapped on his shoulder and pointed to a patient room. Inside, he saw the parents of a child waiting for a transplant. "I have children and I could only imagine," Moore said. "They were just pacing down the room, waiting for it."

Moore said he plans to keep traveling until he can't anymore. Volunteering works well for him, he said, especially now that his children are grown.

Some people might get caught up in the adventure of travel, Moore said, but he's usually only in another country for one day.

"At some point, you realize that's not what it's all about," he said. "It's about getting [a cure] to someone."

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St. Louis Native Heather Lynn Discusses Motivation for Donating Stem Cells – Ladue News

By daniellenierenberg

Since losing her cousin to leukemia during childhood, Heather Lynn made it her mission to ensure others battling blood cancers get a second chance at life. Earlier this year, the St. Louis native fulfilled that life purpose: saving a stranger by donating her stem cells.

Five years ago, Lynn became the director of global special events for DKMS, the worlds largest bone marrow and blood stem cell donor center, and registered as a potential donor with the hope that someday she could give a blood cancer patient what her cousin didnt have a second chance at life and more time with the patients family.

Amid this years coronaviral pandemic, Lynn received the life-changing call from a colleague that she was a match for a 58-year-old man battling acute myeloid leukemia. I screamed with joy, Lynn recalls. I was a match for someone with blood cancer and was about to be the first employee at DKMS to donate and ultimately save someones life. After the call, Lynn realized she would be giving more than stem cells: I was giving something much bigger: hope.

Despite the uncertainty surrounding COVID-19, Lynn felt a strong sense of purpose to help this man and donated her stem cells to save his life. I have seen how much someones life can change with a blood cancer diagnosis, Lynn says. The fear, the pain, the loss it can be devastating. I have spent the past five years working to elevate the message about donating and how easy it is to sign up and give back it simply requires swabbing the inside of each cheek for 60 seconds.

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Actinium Highlights Foundational Patents Covering the Composition of Apamistamab Antibody and Iomab-B Antibody Radiation Conjugate for Targeted…

By daniellenierenberg

NEW YORK, Nov. 23, 2020 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today highlighted its intellectual property portfolio for apamistamab, a CD45 targeting antibody, and the Antibody Radiation Conjugate (ARC) comprised of apamistamab and the radioisotope iodine-131 used in the Company's lead Phase 3 candidate, Iomab-B, and its Iomab-ACT programs. Actinium owns issued or pending patents within the United States and globally covering composition of matter, formulation, methods of use, and methods of administration with potential coverage for 19 years or longer. Importantly, Actinium owns an issued patent in the US covering composition of matter, for which the Company expects validity until 2037.In addition, the Company owns a second issued US patent that further covers composition of matter, methods of use, and methods of administration for Iomab-B. The company has also received a notice of allowance in Europe for this second patent and expects it to be in force until 2036.

Iomab-B is currently being investigated in the ongoing pivotal Phase 3 SIERRA trial, which is over 75% enrolled, for targeted conditioning prior to potentially curative bone marrow transplant (BMT) for patients with relapsed or refractory Acute Myeloid Leukemia ("R/R AML"). In addition, Actinium is utilizing apamistamab with lower doses of iodine-131, known as Iomab-ACT, for targeted conditioning prior to gene therapy and adoptive cell therapy ("ACT"), namely CAR-T, including in its recently announced collaboration with Memorial Sloan Kettering Cancer Center that is supported by NIH STTR Fast Track grant funding.

"The continued protection of our lead asset Iomab-B, our Iomab-ACT program and apamistamab by a strong patent position is an important component of our development efforts, particularly as we approach the conclusion of our pivotal Phase 3 SIERRA trial for BMT conditioning in R/R AML The growth of BMT, ACT and Gene Therapy has highlighted the importance of conditioning and the need to move beyond non-targeted chemotherapy to increase the number of patients that could benefit from these potentially curative therapies.CD45 is an ideal target for conditioning applications given its unique expression on blood cancer cells and blood forming stem and immune cells and with no expression outside the hematopoietic or blood system," said Dr. Dale Ludwig, Actinium's Chief Scientific Officer. "Apamistamab is well characterized and its use in conditioning is supported by extensive clinical data across multiple clinical trials and indications. Our robust data shows that apamistamab has a favorable biodistribution profile that, together with our ARC technology, has significant advantages over other approaches such antibody drug conjugates that require payload internalization, making them impractical for targeting CD45.Further, our ARC approach allows us to use varying intensities of targeted radiation to achieve our desired conditioning outcome.With these important patents in place, and continued expansion of our patent portfolio in the US, EU and other select countries, we look forward to continuing to build out our targeted conditioning strategic business unit."

About Iomab-B

Iomab-B is Actinium's lead product candidate that is currently being studied in a 150-patient, multicenter pivotal Phase 3 clinical trial in patients with relapsed or refractory acute myeloid leukemia who are age 55 and above. Upon approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant, also referred to as a hematopoietic stem cell transplant, which is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B targets cells that express CD45, a pan-leukocytic antigen widely expressed on white blood cells with the monoclonal antibody, apamistamab (formerly BC8), labeled with the radioisotope, iodine-131. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid the side effects of radiation on most healthy tissues while effectively killing the patient's cancer and marrow cells. In a Phase 1/2 clinical study in 68 patients with advanced AML or high-risk myelodysplastic syndrome (MDS) age 50 and older, Iomab-B produced enabled 100% of patients to proceed to transplant with all patients achieving transplant engraftment by day 28. Iomab-B was developed at the Fred Hutchinson Cancer Research Center where it has been studied in almost 300 patients in a number of blood cancer indications, including acute myeloid leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia, chronic lymphocytic leukemia, Hodgkin's disease, Non-Hodgkin lymphomas and multiple myeloma. Actinium obtained the worldwide, exclusive rights to apamistamab (BC8) and Iomab-B from the Fred Hutchinson Cancer Research Center. Iomab-B has been granted Orphan Drug Designation for relapsed or refractory AML in patients 55 and above by the U.S. Food and Drug Administration and the European Medicines Agency.

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation.Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities.With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently.Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning.The SIERRA trial is over seventy-five percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings.More information on this Phase 3 clinical trial can be found at sierratrial.com. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1 study with a CD19 CAR T-cell Therapy and Phase 1/2 anti-HIV stem cell gene therapy with UC Davis. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform.This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes.Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Clayton Robertson Actinium Pharmaceuticals, Inc. [emailprotected]

Hans VitzthumLifeSci Advisors, LLC[emailprotected](617) 430-7578

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com

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Adaptive Biotechnologies and Collaborators to Present Data from More Than 35 Abstracts at ASH 2020 Highlighting Clinical Relevance of MRD Testing with…

By daniellenierenberg

SEATTLE, Nov. 23, 2020 (GLOBE NEWSWIRE) -- Adaptive Biotechnologies Corporation (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, together with its collaborators will present data from more than 35 abstracts studying the use of Adaptives clonoSEQ Assay for minimal residual disease (MRD) assessment at the American Society of Hematology (ASH) virtual 62nd Annual Meeting and Exposition, December 5-8. clonoSEQ is the first and only U.S. Food and Drug Administration (FDA)-cleared assay for MRD assessment in chronic lymphocytic leukemia (CLL), multiple myeloma and B-cell acute lymphoblastic leukemia (B-ALL) and is widely available to clinicians and patients across the U.S.

We are thrilled to see so many investigators presenting clonoSEQ data at ASH this year, among the more than 300 ASH studies highlighting MRD data, significantly growing the body of evidence validating this tool as a critical measure of patient outcomes, said Lance Baldo, MD, Chief Medical Officer of Adaptive Biotechnologies. As innovation continues for the treatment of blood cancers with novel and highly targeted therapies that create deep and durable responses for patients, we see clinicians increasingly utilizing clonoSEQ to help guide day-to-day patient care.

Assessment of MRD is a way to directly detect and quantify remaining disease, even in the absence of symptoms, across a spectrum of blood cancers. A patients MRD status gives clinicians timely information about how a patient may be responding to treatment, so patients and providers can be in control when it comes to managing their disease and treatment decisions.

clonoSEQ, the first clinical application of Adaptives immune medicine platform, will be featured in 14 oral presentations and 23 posters at ASH. Data on clinical and research utility from studies, as well as findings based on real-world experience, will be presented across a range of cancers including multiple myeloma, ALL, CLL and non-Hodgkins lymphoma (NHL). These new data show a correlation between clonoSEQ MRD results and improved blood cancer patient outcomes, enhanced clinical decision-making, and potential savings to the healthcare system.

Additional data at ASH this year will highlight Adaptives immune profiling research tool, immunoSEQ, to quantitatively assess the immune response to novel therapies in development.

Key presentations include:

AbouttheclonoSEQ AssayThe clonoSEQ Assay is the first and only FDA-cleared assay for MRD in chronic lymphocytic leukemia (CLL), multiple myeloma (MM) and B-cell acute lymphoblastic leukemia (ALL). Minimal residual disease (MRD) refers to the small number of cancer cells that can stay in the body during and after treatment. clonoSEQ was initially granted De Novo designation and marketing authorization by the FDA for the detection and monitoring of MRD in patients with MM and B-ALL using DNA from bone marrow samples.InAugust 2020, clonoSEQ received additional clearance from theFDA to detect and monitor MRD in blood or bone marrow from patients with CLL.

The clonoSEQ Assay leverages Adaptives proprietary immune medicine platform to identify and quantify specific DNA sequences found in malignant cells, allowing clinicians to assess and monitor MRD during and after treatment. The assay provides standardized, accurate and sensitive measurement of MRD that allows physicians to predict patient outcomes, assess response to therapy over time, monitor patients during remission and predict potential relapse. Clinical practice guidelines in hematological malignancies recognize that MRD status is a reliable indicator of clinical outcomes and response to therapy, and clinical outcomes have been shown to be strongly associated with MRD levels measured by the clonoSEQ Assay in patients diagnosed with CLL, MM and ALL.

The clonoSEQ Assay is a single-site test performed at Adaptive Biotechnologies.In addition to its FDA-cleared uses, clonoSEQ is also available as a CLIA-validated laboratory developed test (LDT) service for use in other lymphoid cancers and sample types. For important information about the FDA-cleared uses of clonoSEQ, including the full intended use, limitations, and detailed performance characteristics, please visitwww.clonoSEQ.com/technical-summary.

About immunoSEQ Assay Adaptives immunoSEQ Assay helps researchers make discoveries in areas such as oncology, autoimmune disorders, infectious diseases and basic immunology. The immunoSEQ Assay can identify millions of T- and B-cell receptors from a single sample in exquisite detail. Offered as a Service or Kit, the immunoSEQ Assay is used to ask and answer translational research questions and discover new prognostic and diagnostic signals in clinical trials. The immunoSEQ Assay provides quantitative, reproducible sequencing results along with access to powerful, easy-to-use analysis tools. The immunoSEQ Assay is for research use only and is not for use in diagnostic procedures.

About AdaptiveAdaptive Biotechnologies is a commercial-stage biotechnology company focused on harnessing the inherent biology of the adaptive immune system to transform the diagnosis and treatment of disease. We believe the adaptive immune system is natures most finely tuned diagnostic and therapeutic for most diseases, but the inability to decode it has prevented the medical community from fully leveraging its capabilities. Our proprietary immune medicine platform reveals and translates the massive genetics of the adaptive immune system with scale, precision and speed to develop products in life sciences research, clinical diagnostics and drug discovery. We have two commercial products and a robust clinical pipeline to diagnose, monitor and enable the treatment of diseases such as cancer, autoimmune conditions and infectious diseases. Our goal is to develop and commercialize immune-driven clinical products tailored to each individual patient.

For more information, please visit adaptivebiotech.com and follow us on http://www.twitter.com/adaptivebiotech.

Forward Looking Statements This press release contains forward-looking statements that are based on managements beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including statements regarding our ability to develop, commercialize and achieve market acceptance of our current and planned products and services, our research and development efforts, and other matters regarding our business strategies, use of capital, results of operations and financial position, and plans and objectives for future operations.

In some cases, you can identify forward-looking statements by the words may, will, could, would, should, expect, intend, plan, anticipate, believe, estimate, predict, project, potential, continue, ongoing or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors are described under "Risk Factors," "Management's Discussion and Analysis of Financial Condition and Results of Operations" and elsewhere in the documents we file with theSecurities and Exchange Commissionfrom time to time. We caution you that forward-looking statements are based on a combination of facts and factors currently known by us and our projections of the future, about which we cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent our views as of the date hereof. We undertake no obligation to update any forward-looking statements for any reason, except as required by law.

MEDIA CONTACT:Beth Keshishian917-912-7195media@adaptivebiotech.com

ADAPTIVE INVESTORS:Karina Calzadilla201-396-1687

Carrie Mendivil, Gilmartin Groupinvestors@adaptivebiotech.com

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Adaptive Biotechnologies and Collaborators to Present Data from More Than 35 Abstracts at ASH 2020 Highlighting Clinical Relevance of MRD Testing with...

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Novartis bags Mesoblast’s stem cell therapy for ARDS, including in Covid-19, in a deal worth up to $1.2B+ – Endpoints News

By daniellenierenberg

Novartis has licensed a new stem cell therapy from Mesoblast, just weeks after the FDA rejected the Australian biotechs pitch for an approval on a separate indication.

The Swiss pharma announced Thursday afternoon it is partnering with Mesoblast $MESO to develop remestemcel-L for the treatment of acute respiratory distress syndrome, including ARDS related to Covid-19. As part of the deal, Novartis will shell out $25 million in upfront cash and take a $25 million stake in the biotech, while offering up to $1.255 billion in potential milestone payments.

Mesoblast investors embraced the news, sending shares up 11% on the Australian stock exchange Friday. The companys stock was also up roughly 17% on the Nasdaq before Fridays opening bell.

The milestone payments are split as such, per Mesoblast: $505 million will be available pre-commercialization, with an additional $750 million set aside for hitting certain sales targets and double-digit royalties.

Remestemcel-L, or Ryoncil, acts as an anti-inflammatory and consists of culture-expanded mesenchymal stem cells derived from a bone marrow donor. Currently, the drug is being evaluated in a Phase III study for Covid-19-related ARDS with 300 patients, and the first cut of data is expected in early 2021.

Should that outcome prove successful, Novartis will launch a Phase III in non-Covid ARDS after the deal closes. The companies highlighted Novartis ability to rapidly scale up cell-based therapies from the clinic to the commercial phase as a motivator for the collaboration.

The drug had been examined in a small compassionate use program for Covid-19 ARDS back in March, which included 12 patients requiring ventilators. Remestemcel-L treatment demonstrated an 83% survival rate in that program and was the basis for the ongoing Phase III.

Thursdays deal comes less than two months after the FDA issued a CRL for remestemcel-L in Mesoblasts pediatric acute graft-versus-host disease program. The rejection, which denied the company an accelerated approval, came after an ODAC adcomm in August voted 9 to 1 in favor of approval as panel members struggled to envision what a pivotal trial might look like.

During both the adcomm and in their CRL, regulators took issue with Mesoblasts study design given that the company submitted its application on the basis of one, single-arm and open-label trial. In the study, Remestemcel-L demonstrated a statistically significant benefit in its primary endpoint against the historical control rate.

But because many parents and pediatricians are reluctant to risk putting children into the placebo arm of a randomized study, Mesoblast argued that key opinion leaders said an additional study was not feasible. The veto came despite the FDA approving a similar drug Incyte and Novartis Jakafi based on one single-arm trial, something for which ODAC members chastised the FDA.

Earlier this week, Mesoblast met with the agency for its Type A meeting, and the company reported in its third quarter earnings that it does not expect the FDA to reverse its decision for accelerated approval. Mesoblast is still waiting to receive final meeting minutes to know whether thats indeed the case. The CRL set back potential approval in GvHD from 2021 to 2024, per analysts.

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BioRestorative Therapies Emerges from Chapter 11 Reorganization – OrthoSpineNews

By daniellenierenberg

MELVILLE, N.Y., Nov. 20, 2020 (GLOBE NEWSWIRE) BioRestorative Therapies, Inc. (BioRestorative or the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, announced today that its amended joint plan of reorganization has become effective and it has emerged from Chapter 11 reorganization. Pursuant to the confirmed plan of reorganization, the Company has received $3,848,000 in financing. The confirmed plan of reorganization also provides for additional funding, subject to certain conditions, of $3,500,000 less the sum of the debtor-in-possession financing provided to the Company during the reorganization (approximately $1,227,000) and the costs incurred by the debtor-in-possession lender.

In connection with the reorganization, Lance Alstodt has been appointed the Companys President, Chief Executive Officer and Chairman of the Board. Mr. Alstodt said, This process has been a long and challenging journey for the Company. Im inspired by the great resolve and execution from our employees, professionals and investors. We are very pleased that all requirements have been met for us to emerge. Allowed creditor claims have been fully satisfied and, as importantly, our equity holders have retained their shares in this exciting new opportunity. We were able to preserve all of our intellectual property assets and look forward to initiating our Phase 2 clinical trial.

Based upon the Companys emergence from Chapter 11 reorganization, FINRA has removed the Q at the end of its trading symbol. Shareholders do not need to exchange their shares for new shares.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate,BRTX-100,is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders. TheBRTX-100production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure,BRTX-100is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial usingBRTX-100to treat persistent lower back pain due to painful degenerative discs.

Metabolic Program (ThermoStem): We are developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release containsforward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Companys latest Form 10-K filedwith the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

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Rescue Therapy with Bendamustine and Other Agents May Result in High Response Rates in Relapsed or Refractory NHL – Curetoday.com

By daniellenierenberg

Salvage chemotherapy, or treatment for cancer nonresponsive to other chemotherapy regimens, with bendamustine, ofatumumab, carboplatin and etoposide in patients with relapsed or refractory aggressive B-cell non-Hodgkin lymphoma (NHL) served as a safe and effective regimen to bridge patients to stem cell transplantation.

In the era of high value, cost-conscious medicine, delivering high-quality care in the outpatient setting is becoming crucial to allow delivery of cost-effective care, the study authors wrote. (Bendamustine, ofatumumab, carboplatin and etoposide) offers a safe and effective outpatient alternative to currently available inpatient salvage chemotherapy regimens and has the potential to minimize hospitalizations and thus lower health care costs.

Aggressive salvage treatments commonly used in these patients are often administered in an inpatient or outpatient setting. A more ideal regimen, according to the study authors, would be administered in an outpatient setting, provide a favorable toxicity profile and high disease response rate and offer an alternative to CD20-targeted therapy, which focuses on a protein on the surface of B cells that also be found in bone marrow.

In this phase 1/2 study, 35 patients (median age, 62 years; 15 women) with relapsed or refractory aggressive B-cell NHL were treated with a salvage chemotherapy regimen including bendamustine, ofatumumab, carboplatin and etoposide. In particular, 57% of patients had de novo large cell or grade 3B follicular lymphoma, 26% had transformed de novo large cell lymphoma, 9% had grade 3A follicular lymphoma and 3% had mantle cell lymphoma.

The first cycle of this treatment was administered in an inpatient setting to monitor for infusion-related reactions. If toxicities of grade 3 or higher did not occur, which included severe, life-threatening events, subsequent cycles were given in an outpatient setting.

The primary objective of this study was to assess the tolerability and safety of this regimen and to determine its overall response rate (ORR). In addition, secondary end points included overall survival (OS), progression-free survival (PFS), duration of response and the number of patients who advanced to stem cell transplantation. Follow-up was conducted for a median of 24.1 months.

The ORR in all patients was 69%, with 49% of patients having a complete response and 20% of patients having a partial response. When patients with de novo large cell lymphoma and grade 3B follicular lymphoma were assessed, the ORR was 70%, with 50% of patients having a complete response and 20% of patients having a partial response.

Patients in the study had a median PFS of 5.1 months and OS of 26.2 months. Twelve patients moved on to undergo stem cell transplantation.

The most common grade 3-4 nonhematologic toxicities, or those not related to cancer originating in the bone marrow or blood, included hypophosphatemia and neutropenic fever. Hypophosphatemia refers to an electrolyte disorder resulting from low phosphate levels in blood, whereas neutropenic fever occurs when a patient has a temperature of 100.4 F or greater while having low counts of a type of white blood cell called neutrophil.

While the protocol mandated inpatient monitoring after the first cycle for (infusion-related reactions), we found that (infusion-related reactions) were all grade 1-2, the study authors wrote. Therefore, (bendamustine, ofatumumab, carboplatin and etoposide) should be safe for outpatient administration, which is our current institutional practice when using it outside of a clinical trial setting.

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Global Stem Cell Banking Market 2020: Overview, Trends, Opportunities, Impact of Drivers, Key Vendors, Types, Applications, Forecast by Focusing…

By daniellenierenberg

Global stem cell banking market is set to witness a substantial CAGR of 11.03% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. The increased market growth can be identified by the increasing procedures of hematopoietic stem cell transplantation (HSCT), emerging technologies for stem cell processing, storage and preservation. Increasing birth rates, awareness of stem cell therapies and higher treatment done viva stem cell technology.

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Competitive Analysis:

Global stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of inflammatory disease drug delivery market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Competitors:

Few of the major competitors currently working in global inflammatory disease drug delivery market are: NSPERITE N.V, Caladrius, ViaCord, CBR Systems, Inc, SMART CELLS PLUS, LifeCell International, Global Cord Blood Corporation, Cryo-Cell International, Inc., StemCyte India Therapeutics Pvt. Ltd, Cordvida, ViaCord, Cryoviva India, Vita34 AG, CryoHoldco, PromoCell GmbH, Celgene Corporation, BIOTIME, Inc., BrainStorm Cell Therapeutics and others

Market Definition:Global Stem Cell Banking Market

Stem cells are cells which have self-renewing abilities and segregation into numerous cell lineages. Stem cells are found in all human beings from an early stage to the end stage. The stem cell banking process includes the storage of stem cells from different sources and they are being used for research and clinical purposes. The goal of stem cell banking is that if any persons tissue is badly damaged the stem cell therapy is the cure for that. Skin transplants, brain cell transplantations are some of the treatments which are cured by stem cell technique.

Cord Stem Cell Banking MarketDevelopment and Acquisitions in 2019

In September 2019, a notable acquisition was witnessed between CBR and Natera. This merger will develop the new chances of growth in the cord stem blood banking by empowering the Nateras Evercord branch for storing and preserving cord blood. The advancement will focus upon research and development of the therapeutic outcomes, biogenetics experiment, and their commercialization among the global pharma and health sector.

Cord Stem Cell Banking MarketScope

Cord Stem Cell Banking Marketis segmented on the basis of countries into U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

All country based analysis of the cord stem cell banking marketis further analyzed based on maximum granularity into further segmentation. On the basis of storage type, the market is segmented into private banking, public banking. On the basis of product type, the market is bifurcated into cord blood, cord blood & cord tissue. On the basis of services type, the market is segmented into collection & transportation, processing, analysis, storage. On the basis of source, market is bifurcated into umbilical cord blood, bone marrow, peripheral blood stem, menstrual blood. On the basis of indication, the market is fragmented into cerebral palsy, thalassemia, leukemia, diabetes, autism.

Cord stem cell trading is nothing but the banking of the vinculum plasma cell enclosed in the placenta and umbilical muscle of an infant. This ligament plasma comprises the stem blocks which can be employed in the forthcoming time to tackle illnesses such as autoimmune diseases, leukemia, inherited metabolic disorders, and thalassemia and many others.

Market Drivers

Increasing rate of diseases such as cancers, skin diseases and othersPublic awareness associated to the therapeutic prospective of stem cellsGrowing number of hematopoietic stem cell transplantations (HSCTs)Increasing birth rate worldwide

Market Restraint

High operating cost for the therapy is one reason which hinders the marketIntense competition among the stem cell companiesSometimes the changes are made from government such as legal regulations

Key Pointers Covered in the Cord Stem CellBanking MarketIndustry Trends and Forecast to 2026

Market SizeMarket New Sales VolumesMarket Replacement Sales VolumesMarket Installed BaseMarket By BrandsMarket Procedure VolumesMarket Product Price AnalysisMarket Healthcare OutcomesMarket Cost of Care AnalysisMarket Regulatory Framework and ChangesMarket Prices and Reimbursement AnalysisMarket Shares in Different RegionsRecent Developments for Market CompetitorsMarket Upcoming ApplicationsMarket Innovators Study

Key Developments in the Market:

In August, 2019, Bayer bought BlueRock for USD 600 million to become the leader in stem cell therapies. Bayer is paying USD 600 million for getting full control of cell therapy developer BlueRock Therapeutics, promising new medical area to revive its drug development pipeline and evolving engineered cell therapies in the fields of immunology, cardiology and neurology, using a registered induced pluripotent stem cell (iPSC) platform.In August 2018, LifeCell acquired Fetomed Laboratories, a provider of clinical diagnostics services. The acquisition is for enhancement in mother & baby diagnostic services that strongly complements stem cell banking business. This acquisition was funded by the internal accruals which is aimed to be the Indias largest mother & baby preventive healthcare organization.

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Research objectives

To perceive the most influencing pivoting and hindering forces in Cord Stem Cell Banking Market and its footprint in the international market.Learn about the market policies that are being endorsed by ruling respective organizations.To gain a perceptive survey of the market and have an extensive interpretation of the Cord Stem Cell Banking Market and its materialistic landscape.To understand the structure of Cord Stem Cell Banking Market by identifying its various sub segments.Focuses on the key global Cord Stem Cell Banking Market players, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).To project the consumption of Cord Stem Cell Banking Market submarkets, with respect to key regions (along with their respective key countries).To strategically profile the key players and comprehensively analyze their growth strategiesTo analyze the Cord Stem Cell Banking Market with respect to individual growth trends, future prospects, and their contribution to the total market.

Customization of the Report:

All segmentation provided above in this report is represented at country levelAll products covered in the market, product volume and average selling prices will be included as customizable options which may incur no or minimal additional cost (depends on customization)

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Global Stem Cell Banking Market 2020: Overview, Trends, Opportunities, Impact of Drivers, Key Vendors, Types, Applications, Forecast by Focusing...

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St. Louis Native Heather Lynn Discusses Motivation for Donating Her Stem Cells – Ladue News

By daniellenierenberg

Since losing her cousin to leukemia during childhood, Heather Lynn made it her mission to ensure others battling blood cancers get a second chance at life. Earlier this year, the St. Louis native fulfilled that life purpose: saving a stranger by donating her stem cells.

Five years ago, Lynn became the director of global special events for DKMS, the worlds largest bone marrow and blood stem cell donor center, and registered as a potential donor with the hope that someday she could give a blood cancer patient what her cousin didnt have a second chance at life and more time with the patients family.

Amid this years coronaviral pandemic, Lynn received the life-changing call from a colleague that she was a match for a 58-year-old man battling acute myeloid leukemia. I screamed with joy, Lynn recalls. I was a match for someone with blood cancer and was about to be the first employee at DKMS to donate and ultimately save someones life. After the call, Lynn realized she would be giving more than stem cells: I was giving something much bigger: hope.

Despite the uncertainty surrounding COVID-19, Lynn felt a strong sense of purpose to help this man and donated her stem cells to save his life. I have seen how much someones life can change with a blood cancer diagnosis, Lynn says. The fear, the pain, the loss it can be devastating. I have spent the past five years working to elevate the message about donating and how easy it is to sign up and give back it simply requires swabbing the inside of each cheek for 60 seconds.

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Jakafi and Dacogen May Improve Overall Survival in Patients with MPN – Curetoday.com

By daniellenierenberg

Results from a phase 2 study demonstrated that treatment with Jakafi (ruxolitinib) and Dacogen (decitabine) was well tolerated and contributed to favorable overall survival (OS) in patients with myeloproliferative neoplasm (MPN) in the accelerated or blast phase.

MPN is a blood cancer that develops when a stem cell mutation in the bone marrow leads to an overproduction of white cells, red cells and/or platelets. The accelerated phase of MPN refers to when 10% to 19% of blasts, or immature blood cells, are in the blood circulating through the body or in the bone marrow, whereas the blast phase refers to 20% or greater blasts in the circulating blood or bone marrow, according to the study published in Blood Advances.

This study was important, as patients with an antecedent (pre-existing) myeloproliferative neoplasm that evolves into an acute myeloid leukemia have a dismal prognosis of several months, and induction chemotherapy alone does not improve outcome unless followed by consolidation hematopoietic stem cell transplantation, Dr. John O. Mascarenhas, director of the adult leukemia program and leader of the myeloproliferative neoplasm clinical research program at Tisch Cancer Institute at Icahn School of Medicine at Mount Sinai, said in an interview with CURE.

The study authors previously assessed this therapy in a multicenter, phase 1 trial.

We had previously shown that the epigenetic modifying agent decitabine can be administered (on) an outpatient (basis) and improve outcome with a median survival of nine to 10 months, Mascarenhas said. This prospective, phase 2, multicenter, investigated-initiated trial built on the phase 1 trial of combination decitabine and ruxolitinib based on supportive preclinical data from the laboratory of our collaborator, (Dr.) Ross Levine.

In this current trial, 25 patients (median age, 71 years; 56% women) with MPN either in the accelerated phase (10 patients; median age, 70.1 years; 70% women) or blast phase (15 patients; median age, 71.6 years; 46.7% women) were treated with Jakafi and Dacogen. A 25 mg dose of Jakafi was administered orally twice per day for 28 days in addition to a 20 mg/m2 dose of Dacogen intravenously during days 8 through 12. After that first cycle, the dose of Jakafi was reduced to 10 mg.

The prespecified primary endpoint, or goal, was best response by six months, and the predetermined secondary endpoint focused on the safety and tolerability of Jakafi and Dacogen. Study authors defined OS as the time from the first dose of Jakafi to death from any cause.

During follow-up, 19 patients died from causes including respiratory failure, disease progression, sepsis and pneumonia. Patients in this study had a median OS of 9.5 months. Overall response rate, which included complete remission, incomplete platelet recovery and partial remission, occurred in 44% of patients. Response to this treatment was not linked with improved survival.

This combination is well tolerated and can provide spleen symptom benefit and survival advantage compared to cytotoxic chemotherapy, Mascarenhas said. This study supports the use of this approach to maintain ambulatory care of these very advanced patients with a limited lifespan. This is one therapeutic approach that is now included in the (National Comprehensive Cancer Network) guidelines.

Mascarenhas added that more research is needed in this area. Ultimately, we need to identify active agents that can fully eliminate the malignant hematopoietic stem cell and attain molecular remissions that afford patients long-term survival, he said. This is an ongoing area of active translational research of our group.

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Community Cord Blood Banking saves life of child with aplastic anaemia – The Hindu

By daniellenierenberg

Community Cord Blood Banking, a stem cell banking initiative introduced by LifeCell in 2017, has helped save the life of a seven-year-old girl from Nashik in Maharashtra who was suffering from aplastic anaemia, a rare and serious blood disorder.

In a major breakthrough, a team of senior doctors from LOTUS Institute of Haematology, Oncology and Bone Marrow Transplantation, recently conducted Indias first dual cord blood transplant through an unrelated donor using Community Stem Cell Banking. People familiar with the development said the transplant was challenging because no apt bone marrow donors were available and the cost of retrieval of matching units from public cord blood banks would have been high.

The girls parents, as members of LifeCell Community Bank, placed a request for two matching cord blood units after the childs sibling was found to be only a 50% (4/8) match. Two high-quality matches (7/8) were found in the registry, which fulfilled the requirement for umbilical cord blood transplantation. The parents could withdraw the matched units at no extra expense, which would have cost around 45 lakh per unit.

Mayur Abhaya, MD, LifeCell International said, The purpose of Community Cord Blood Banking is to ensure easy and rapid access to stem cells for every Indian without the hurdles of public and private banking models. While stem cells from the umbilical cord blood can be procured from global public banks, the probability of finding a match for a patient of Indian origin is less than 10% because of the low inventory of available units plus the big issue of donor dropouts.

Mr. Abhaya said, Luckily, since the family was a part of LifeCells community banking programme, they could gain quick, free access to the huge inventory of over 50,000 qualified and consented units available at LifeCell, which provides greater than 97% probability of finding a match.

In majority of blood-related disorders treatable by transplants, patients own stem cells are not suitable. Hence, the best donor is a close family member, usually a sibling. However, in this case, there was only a 50% match with the sibling, thus needing a match from an unrelated donor, LifeCell said in a statement. It said while a regular stem cell transplant requires a dose of 25 million cells per kg of the patients weight, for aplastic anaemia, the recommended minimum dose is 40 million cells per kg, which is not easy to find.

The community banking model made it possible and the child received a timely transplant with an encouraging prognosis. Just 18 days after the transplant, white blood cells were completely engrafted, and platelets and red blood cell production also increased drastically, the statement added.

Dr. Pritesh Junagade, director, LOTUS Hospital, expressed surprise that the retrieval process for two cord blood units was smooth and at no additional cost to the patient as compared to other banking models.

Tasneem Bohari, the childs father, said: It was two years ago that my daughter was diagnosed with aplastic anaemia. At the time of diagnosis, the doctors had suggested she would need a stem cell transplant eventually and it would be the best possible treatment in the future.

The family did research and made enquiries about which stem cell bank to opt for as they were expecting their second child. It was during this time that their friends and relatives suggested LifeCell to them. Their doctor also suggested that they could go ahead and preserve their babys stem cell with LifeCell.

At the time of preservation, we didnt have much idea about Community Stem Cell Banking and its benefits, but today we are happy to associate with LifeCell, who have helped my daughter lead a quality life through cord blood transplant, Mr. Bohari said.

Community Cord Blood Banking allows sharing of preserved umbilical cord stem cells from a common pool amongst the members of the community. The model offers greater and easier access to donor stem cells, unlimited retrievals at the cost of one enrolment, and a higher probability for finding a close match for potentially life-saving treatments.

This facility ensures complete protection to the child, siblings, parents, and maternal and paternal grandparents from more than 80 disorders treatable by stem cells.

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Gene-edited monkey embryos give researchers new way to study HIV cure – University of Wisconsin-Madison

By daniellenierenberg

Egg cells harvested from Mauritian cynomolgus macaques (top left) were fertilized (top right) and injected with CRISPR gene editing materials to insert a genetic mutation that cured two men of HIV in the last decade. The growing embryos (developing in the bottom images), if carried to maturity by surrogates, will help researchers study the mutation as a potential treatment for HIV. Courtesy of Golos and Slukvin labs

A gene that cured a man of HIV a decade ago has been successfully added to developing monkey embryos in an effort to study more potential treatments for the disease.

Timothy Brown, known for years as the Berlin Patient, received a transplant of bone marrow stem cells in 2007 to treat leukemia. The cells came from a donor with a rare genetic mutation that left the surfaces of their white blood cells without a protein called CCR5. When Browns immune system was wiped out and replaced by the donated cells, his new immune systems cells carried the altered gene.

This mutation cuts a chunk out of the genome so that it loses a functional gene, CCR5, that is a co-receptor for HIV, says Ted Golos, a University of WisconsinMadison reproductive scientist and professor of comparative biosciences and obstetrics and gynecology. Without CCR5, the virus cant attach to and enter cells to make more HIV. So, in Timothy Browns case, his infection was eliminated.

In 2019, a second cancer patient Adam Castillejo, initially identified as the London patient was cleared of his HIV by a stem cell transplant conferring the same mutation.

Thats very exciting, and there have been some follow up studies. But its been complicated, to say the least, Golos says.

Between the two transplants came a more infamous application of the mutation, when in 2018 Chinese biophysicist He Jiankui announced he had used the DNA-editing tool CRISPR to write the mutation into the DNA of a pair of human embryos. His work drew criticism from scientists concerned with the ethics of altering genes that can be passed down to human offspring, and he was jailed by the Chinese government for fraud.

The promise of the CCR5 mutation remains, but not without further study. The mutation occurs naturally in fewer than 1 percent of people, suggesting that it may not be associated only with positive health outcomes. An animal model for research can help answer open questions.

Given interest in moving forward gene-editing technologies for correcting genetic diseases, preclinical studies of embryo editing in nonhuman primates are very critical, says stem cell researcher Igor Slukvin, a UWMadison professor of pathology and laboratory medicine.

Golos, Slukvin and colleagues at UWMadisons Wisconsin National Primate Research Center and schools of Veterinary Medicine and Medicine and Public Health employed CRISPR to edit the DNA in newly fertilized embryos of cynomolgus macaque monkeys. They published their work recently in the journal Scientific Reports.

Slukvins lab had already established a method for slicing the CCR5-producing gene out of the DNA in human pluripotent stem cells, which can be used to generate immune cells resistant to HIV.

We used that same targeting construct that we already knew worked in cells, and delivered it to one-cell fertilized embryos, says Jenna Kropp Schmidt, a Wisconsin National Primate Research Center scientist. The thought is that if you make the genetic edit in the early embryo that it should propagate through all the cells as the embryo grows.

Primate Center scientist Nick Strelchenko found that as much as one-third of the time the gene edits successfully deleted the sections of DNA in CRISPRs crosshairs base pairs in both of the two copies of the CCR5 gene on a chromosome and were carried on into new cells as the embryos grew.

The goal now is to transfer these embryos into surrogates to produce live offspring who carry the mutation, Schmidt says.

Cynomolgus macaques are native to Southeast Asia, but a group of the monkeys has lived in isolation on the Indian Ocean island of Mauritius for about 500 years. Because the entire Mauritian monkey line descends from a small handful of founders, they have just seven variations of the major histocompatibility complex, the group of genes that must be matched between donor and recipient for a successful bone marrow transplant. There are hundreds of MHC variations in humans.

With MHC-matched monkeys carrying the CCR5 mutation, the researchers would have a reliable way to study how successful the transplants are against the simian immunodeficiency virus, which works in monkeys just like HIV does in humans.

Anti-retroviral drugs have really positively changed the expectation for HIV infection, but in some patients, they may not be as effective. And theyre certainly not without long-term consequences, says Golos, whose work is funded by the National Institutes of Health. So, this is potentially an alternative approach, which also allows us to expand our understanding of the immune system and how it might protect people from HIV infection.

The animal model could lead to the development of gene-edited human hematopoietic stem cells the type that work in bone marrow to produce many kinds of blood cells that Slukvin and Golos say could be used as an off-the-shelf treatment for HIV infection.

This research was supported by grants from the National Institutes of Health (R24OD021322, P51OD011106, K99 HD099154-01, RR15459-01 and RR020141-01).

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Ruxolitinib for Acute Graft-vs-Host Disease Improves Outcomes in the Second Line – Targeted Oncology

By daniellenierenberg

During a Targeted Oncology Case Based Peer Perspective event, Usama Gergis, MD, MBA, professor of Oncology, director, Bone Marrow Transplant and Immune Cellular Therapy at Sidney Kimmel Cancer Center, at the Thomas Jefferson University Hospital, discussed the case of a 48-year-old male patient with acute graft-versus-host-disease (GVHD).

Targeted Oncology: For a patient such as this, what is the risk of developing acute (GVHD) following transplant?

GERGIS: The list of risk factors for GVHD is huge: donor HLA [human leukocyte antigen] disparities that are major/minor, sex matching, donor parity, donor age, blood typewhile its controversialdonor CMV seropositivity, gene polymorphism, and stemcell graft source. Obviously, peripheral mobilized stem cells have more T cells than bone marrow. Other factors include graft composition, the higher CD34 the higher lymphocytes, and conditioning intensities. There are a bunch of factors here, at least for our patient, who had a MAC. The fact that his donor is a multirisk, 50-year-old woman and a MUD [infers] similar higher risk.

How quickly do you determine whether a patient is steroid refractory or steroid dependent?

Basically, steroid refractoriness or resistance versus dependence versus intolerance [can be determined in] as early as 3 days. If there is progression of the grade of GVHD, this is considered steroid refractory. If by 4 weeks it does not go to grade 0, its considered refractory. However, I disagree with the 28-day [timeline]. I think its a long time to wait. Usually, I look at 2 weeks.

This has been the consensus among many of us. I think the trials REACH-1 [NCT02953678] and REACH-2 [NCT02435433] looked at 28 days; but if you go to the 2 papers, most responses took place in 7 to 14 days. Again, this is in the second-line setting.

Ive been doing this for 15 years. Ive been through most of this; so many lines [of therapy and] so many heartbreaks. I teach my fellows to look at any [research] paper in second-line GVHDthe overall response rate is 30%. Ive done it with MMF [mycophenolate mofetil], sirolimus [Rapamune], infliximab [Remicade], ATG [antithymocyte globulin], cyclophosphamide, Ontak [denileukin diftitox], and mesenchymal stem cells and overall response rate is 30%.

Which data support the use of ruxolitinib (Jakafi) in patients who are steroid refractory?

Based on a small trial of 49 patients, a phase 2 trial [INCB 18424-271; NCT02953678], ruxolitinib was approved as a second-line therapy for patients with acute steroid-refractory GVHD.1

Review the details of the REACH-2 trial.

The REACH-2 trial data were just published in 2020. Ruxolitinib was approved based on a small trial of 49 patients with steroid-refractory acute GVHD. They enrolled 70 but only 49 were available for efficacy. Obviously, it was a pilot phase 2 with no comparative arm, and the response rates across the board were [somewhere in the range of] 50% or so by day 28.

Then they were asked to run a phase 3 trial comparing ruxolitinib at 10 mg twice daily versus best available care. The best available care goes through the list that I just mentioned, including photopheresis, ATG, and others. After 4 weeks, patients who were not responders on the best available care were allowed to cross over to ruxolitinib.

By day 28, the responders on the ruxolitinib composed 60% of the cohort versus 40% on the control arm with a P value of less than .001. The durability of response at 8 weeks was 40% versus 20% [odds ratio, 2.38; 95% CI, 1.43 to 3.94; P < .001].

This tell us that ruxolitinib works in this group, that it does not work that great in one-third of patients who lose their response at 8 weeks, and that its just better than anything else [thats available]. A good thing about the ruxolitinib arm is that the response duration was much longer than best available treatment, as illustrated by the Kaplan-Meier curve for response duration.

Failure-free survival [in this trial] was 5.0 months versus 1.0 month [HR, 0.46; 95% CI, 0.35-0.60].

Do you apply this therapy to any of your patients?

We have a patient [at my institution] who has steroid-refractory GVHD. Last Friday in our weekly meeting, we were discussing his [case]. He has lower GI [gastrointestinal] GVHDgrade IV, steroid-refractory, as bad as it getsand I said lets start him on ruxolitinib. One of my colleagues said it doesnt work well in the lower GI. I asked the organizers to pull [data] from the supplemental figures on the New England Journal of Medicine paper of REACH-2 [indicating that these patients did have a benefit with ruxolitinib].2

REFERENCES:

1. FDA approves ruxolitinib for acute graft-versus-host disease. FDA. May 24, 2019. Accessed October 10, 2020. https://bit.ly/2SMU7I8

2. Zeiser R, von Bubnoff N, Butler J, et al; REACH2 Trial Group. Ruxolitinib for glucocorticoid- refractory acute graft-versus-host disease. N Engl J Med. 2020;382(19):1800-1810. doi: 10.1056/NEJMoa1917635

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Hematopoietic Stem Cell Transplantation (HSCT) Market by Product Type, End User and by Region-Trends and Forecast to 2026|China Cord Blood Corp, CBR…

By daniellenierenberg

The global Hematopoietic Stem Cell Transplantation (HSCT) market is broadly analyzed in this report that sheds light on critical aspects such as the vendor landscape, competitive strategies, market dynamics, and regional analysis. The report helps readers to clearly understand the current and future status of the global Hematopoietic Stem Cell Transplantation (HSCT) market. The research study comes out as a compilation of useful guidelines for players to secure a position of strength in the global Hematopoietic Stem Cell Transplantation (HSCT) market. The authors of the report profile leading companies of the global Hematopoietic Stem Cell Transplantation (HSCT) market, such as Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc They provide details about important activities of leading players in the competitive landscape.

The report predicts the size of the global Hematopoietic Stem Cell Transplantation (HSCT) market in terms of value and volume for the forecast period 2019-2026. As per the analysis provided in the report, the global Hematopoietic Stem Cell Transplantation (HSCT) market is expected to rise at a CAGR of XX % between 2019 and 2026 to reach a valuation of US$ XX million/billion by the end of 2026. In 2018, the global Hematopoietic Stem Cell Transplantation (HSCT) market attained a valuation of US$_ million/billion. The market researchers deeply analyze the global Hematopoietic Stem Cell Transplantation (HSCT) industry landscape and the future prospects it is anticipated to create.

This publication includes key segmentations of the global Hematopoietic Stem Cell Transplantation (HSCT) market on the basis of product, application, and geography (country/region). Each segment included in the report is studied in relation to different factors such as consumption, market share, value, growth rate, and production.

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The comparative results provided in the report allow readers to understand the difference between players and how they are competing against each other. The research study gives a detailed view of current and future trends and opportunities of the global Hematopoietic Stem Cell Transplantation (HSCT) market. Market dynamics such as drivers and restraints are explained in the most detailed and easiest manner possible with the use of tables and graphs. Interested parties are expected to find important recommendations to improve their business in the global Hematopoietic Stem Cell Transplantation (HSCT) market.

Readers can understand the overall profitability margin and sales volume of various products studied in the report. The report also provides the forecasted as well as historical annual growth rate and market share of the products offered in the global Hematopoietic Stem Cell Transplantation (HSCT) market. The study on end-use application of products helps to understand the market growth of the products in terms of sales.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market by Product: , Allogeneic, Autologous

Global Hematopoietic Stem Cell Transplantation (HSCT) Market by Application: Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)

The report also focuses on the geographical analysis of the global Hematopoietic Stem Cell Transplantation (HSCT) market, where important regions and countries are studied in great detail.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market by Geography:

Methodology

Our analysts have created the report with the use of advanced primary and secondary research methodologies.

As part of primary research, they have conducted interviews with important industry leaders and focused on market understanding and competitive analysis by reviewing relevant documents, press releases, annual reports, and key products.

For secondary research, they have taken into account the statistical data from agencies, trade associations, and government websites, internet sources, technical writings, and recent trade information.

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Table Of Contents:

1 Market Overview of Hematopoietic Stem Cell Transplantation (HSCT)1.1 Hematopoietic Stem Cell Transplantation (HSCT) Market Overview1.1.1 Hematopoietic Stem Cell Transplantation (HSCT) Product Scope1.1.2 Market Status and Outlook1.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size Overview by Region 2015 VS 2020 VS 20261.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Region (2015-2026)1.4 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Region (2015-2020)1.5 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size Forecast by Region (2021-2026)1.6 Key Regions, Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.1 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.2 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.3 Asia-Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.4 Latin America Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026)1.6.5 Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Market Size YoY Growth (2015-2026) 2 Hematopoietic Stem Cell Transplantation (HSCT) Market Overview by Type2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type: 2015 VS 2020 VS 20262.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Type (2015-2020)2.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Type (2021-2026)2.4 Allogeneic2.5 Autologous 3 Hematopoietic Stem Cell Transplantation (HSCT) Market Overview by Application3.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application: 2015 VS 2020 VS 20263.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Application (2015-2020)3.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Application (2021-2026)3.4 Peripheral Blood Stem Cells Transplant (PBSCT)3.5 Bone Marrow Transplant (BMT)3.6 Cord Blood Transplant (CBT) 4 Global Hematopoietic Stem Cell Transplantation (HSCT) Competition Analysis by Players4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Players (2015-2020)4.2 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in Hematopoietic Stem Cell Transplantation (HSCT) as of 2019)4.3 Date of Key Manufacturers Enter into Hematopoietic Stem Cell Transplantation (HSCT) Market4.4 Global Top Players Hematopoietic Stem Cell Transplantation (HSCT) Headquarters and Area Served4.5 Key Players Hematopoietic Stem Cell Transplantation (HSCT) Product Solution and Service4.6 Competitive Status4.6.1 Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Rate4.6.2 Mergers & Acquisitions, Expansion Plans 5 Company (Top Players) Profiles and Key Data5.1 Regen Biopharma Inc5.1.1 Regen Biopharma Inc Profile5.1.2 Regen Biopharma Inc Main Business5.1.3 Regen Biopharma Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.1.4 Regen Biopharma Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.1.5 Regen Biopharma Inc Recent Developments5.2 China Cord Blood Corp5.2.1 China Cord Blood Corp Profile5.2.2 China Cord Blood Corp Main Business5.2.3 China Cord Blood Corp Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.2.4 China Cord Blood Corp Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.2.5 China Cord Blood Corp Recent Developments5.3 CBR Systems Inc5.5.1 CBR Systems Inc Profile5.3.2 CBR Systems Inc Main Business5.3.3 CBR Systems Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.3.4 CBR Systems Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.3.5 Escape Therapeutics Inc Recent Developments5.4 Escape Therapeutics Inc5.4.1 Escape Therapeutics Inc Profile5.4.2 Escape Therapeutics Inc Main Business5.4.3 Escape Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.4.4 Escape Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.4.5 Escape Therapeutics Inc Recent Developments5.5 Cryo-Save AG5.5.1 Cryo-Save AG Profile5.5.2 Cryo-Save AG Main Business5.5.3 Cryo-Save AG Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.5.4 Cryo-Save AG Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.5.5 Cryo-Save AG Recent Developments5.6 Lonza Group Ltd5.6.1 Lonza Group Ltd Profile5.6.2 Lonza Group Ltd Main Business5.6.3 Lonza Group Ltd Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.6.4 Lonza Group Ltd Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.6.5 Lonza Group Ltd Recent Developments5.7 Pluristem Therapeutics Inc5.7.1 Pluristem Therapeutics Inc Profile5.7.2 Pluristem Therapeutics Inc Main Business5.7.3 Pluristem Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.7.4 Pluristem Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.7.5 Pluristem Therapeutics Inc Recent Developments5.8 ViaCord Inc5.8.1 ViaCord Inc Profile5.8.2 ViaCord Inc Main Business5.8.3 ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT) Products, Services and Solutions5.8.4 ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT) Revenue (US$ Million) & (2015-2020)5.8.5 ViaCord Inc Recent Developments 6 North America6.1 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country6.2 United States6.3 Canada 7 Europe7.1 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country7.2 Germany7.3 France7.4 U.K.7.5 Italy7.6 Russia7.7 Nordic7.8 Rest of Europe 8 Asia-Pacific8.1 Asia-Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Region8.2 China8.3 Japan8.4 South Korea8.5 Southeast Asia8.6 India8.7 Australia8.8 Rest of Asia-Pacific 9 Latin America9.1 Latin America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country9.2 Mexico9.3 Brazil9.4 Rest of Latin America 10 Middle East & Africa10.1 Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Country10.2 Turkey10.3 Saudi Arabia10.4 UAE10.5 Rest of Middle East & Africa 11 Hematopoietic Stem Cell Transplantation (HSCT) Market Dynamics11.1 Industry Trends11.2 Market Drivers11.3 Market Challenges11.4 Market Restraints 12 Research Finding /Conclusion 13 Methodology and Data Source 13.1 Methodology/Research Approach13.1.1 Research Programs/Design13.1.2 Market Size Estimation13.1.3 Market Breakdown and Data Triangulation13.2 Data Source13.2.1 Secondary Sources13.2.2 Primary Sources13.3 Disclaimer13.4 Author List

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Hematopoietic Stem Cell Transplantation (HSCT) Market by Product Type, End User and by Region-Trends and Forecast to 2026|China Cord Blood Corp, CBR...

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Cord Stem Cell Banking Market 2020-2026 is Growing Globally With Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva…

By daniellenierenberg

Global Cord Stem Cell Banking Market report brings into light key market dynamics of the sector. It provides guidelines about planning of advertising and sales promotion efforts. The market report is a professional yet exhaustive study on the current state for the market. This world class market research report is a vital document in planning business objectives or goals. The credible market report is a window to the industry which talks about what market definition, classifications, applications, engagements and market trends are. While formulating this Cord Stem Cell Banking Market analysis report, client business competence is understood adeptly to identify tangible growth opportunities.

The wide ranging Cord Stem Cell Banking Market report evaluates CAGR value fluctuation during the forecast period of 2021-2028 for the market. All the data of research and analysis are mapped in an actionable model, with strategic recommendations from the experts. Base year for calculation in the report is considered as 2020 while the historic year is 2019 which will tell how the Cord Stem Cell Banking Market is going to act upon in the forecast years by giving information about the several market insights. Lastly, the large scale report makes some important proposals for a new project of the market before evaluating its feasibility.

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Global Cord stem cell banking market is estimated to reach USD 13.8 billion by 2026 registering a healthy CAGR of 22.4%. The increasing number of parents storing their childs cord blood, acceptance of stem cell therapeutics, high applicability of stem cells are key driver to the market.

Few of the major market competitors currently working in the globalcord stem cell banking marketareCBR Systems, Inc., Cordlife, Cells4Life Group LLP, Cryo-Cell International, Inc., Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva India, Global Cord Blood Corporation, National Cord Blood Program, Vita 34, ReeLabs Pvt. Ltd., Regrow Biosciences Pvt. Ltd. , ACROBiosystems., Americord Registry LLC., New York Blood Center, Maze Cord Blood, GoodCell., AABB, Stem Cell Cryobank, New England Cryogenic Center, Inc. among others

Browse Detailed TOC, Tables, Figures, Charts and Companies @https://www.databridgemarketresearch.com/toc?dbmr=global-cord-stem-cell-banking-market

Market Definition: Global Cord Stem Cell Banking Market

Cord stem cells banking is nothing but the storing of the cord blood cell contained in the umbilical cord and placenta of a newborn child. This cord blood contains the stem cells which can be used in future to treat disease such as leukemia, thalassemia, autoimmune diseases, and inherited metabolic disorders, and few others.

Segmentation: Global Cord Stem Cell Banking Market

Cord Stem Cell banking Market : By Storage Type

Cord Stem Cell banking Market : By Product Type

Cord Stem Cell banking Market : By Service Type

Cord Stem Cell banking Market : By Indication

Cord Stem Cell banking Market : By Source

Cord Stem Cell banking Market : By Geography

Key Developments in the Cord Stem Cell banking Market:

Cord Stem Cell banking Market : Drivers

Cord Stem Cell banking Market : Restraint

Scope of the Cord Stem Cell banking Market Report :

The report shields the development activities in the Cord Stem Cell banking Market which includes the status of marketing channels available, and an analysis of the regional export and import. It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments. This will benefit the reports users, that evaluates their position in Cord Stem Cell banking market as well as create effective strategies in the near future.

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Cord Stem Cell Banking Market 2020-2026 is Growing Globally With Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva...

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Stem Cell Banking Market is Projected to Reach $6,956 million by 2023 | Leading key players are Cord Blood Registry, ViaCord, Cryo-Cell, China Cord…

By daniellenierenberg

The global stem cell banking market was valued at $1,986 million in 2016, and is estimated to reach $6,956 million by 2023, registering a CAGR of 19.5% from 2017 to 2023. Stem cell banking is a process where the stem cell care isolated from different sources such as umbilical cord and bone marrow that is stored and preserved for future use. These cells can be cryo-frozen and stored for decades. Private and public banks are different types of banks available to store stem cells.

Top Companies Covered in this Report: Cord Blood Registry, ViaCord, Cryo-Cell, China Cord Blood Corporation, Cryo-Save, New York Cord Blood Program, CordVida, Americord, CryoHoldco, Vita34

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Increase in R&D activities in regards with applications of stem cells and increase in prevalence of fatal chronic diseases majorly drive the growth of the global stem cell banking market. Moreover, the large number of births occurring globally and growth in GDP & disposable income help increase the number of stem cell units stored, which would help fuel the market growth. However, legal and ethical issues related to stem cell collections and high processing & storage cost are projected to hamper the market growth. The initiative taken by organizations and companies to spread awareness in regards with the benefits of stem cells and untapped market in the developing regions help to open new avenues for the growth of stem cell banking market in the near future.

The global stem cell banking market is segmented based on cell type, bank type, service type, utilization, and region. Based on cell type, the market is classified into umbilical cord stem cells, adult stem cells, and embryonic stem cells. Depending on bank type, it is bifurcated into public and private. By service type, it is categorized into collection & transportation, processing, analysis, and storage. By utilization, it is classified into used and unused. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

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Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: STEM CELL BANKING MARKET, BY CELL TYPE

CHAPTER 5: STEM CELL BANKING MARKET, BY BANK TYPE

CHAPTER 6: STEM CELL BANKING MARKET, BY SERVICE TYPE

CHAPTER 7: STEM CELL BANKING MARKET, BY UTILIZATION

CHAPTER 8: STEM CELL BANKING MARKET, BY REGION

CHAPTER 9: COMPANY PROFILES

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BRTX-100; the Story of BioRestorative Therapies Inc (OTCMKTS: BRTX) – MicroCap Daily

By daniellenierenberg

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BioRestorative Therapies Inc (OTCMKTS: BRTXQ) (BRTX) is soaring up the charts after it was revealed at 12.04 pm Wednesday afternoon the Company was emerging from bankruptcy. BRTXQ came to the attention of many penny stock speculators after the Company partnered on a new bankruptcy reorganization plan with one of its creditors Auctus Capital in which it would emerge from bankruptcy with the commons intact, ready to begin phase 2 trials and get BioRestorative back on a national stock exchange.

BioRestorative Therapies has received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs. BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. It is the Companys intend that the product be used for the non-surgical treatment of painful lumbosacral disc disorders.

BioRestorative Therapies Inc (OTCMKTS: BRTX) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders: Disc/Spine Program (brtxDISC): Its lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders.

The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. The Company has received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs.

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Metabolic Program (ThermoStem): the Company is developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

BioRestorative owns a valuable intelectual property portfolio including unique international Stem Cell patents as well as 8 patents issued, in the United States and other countries, for the Companys brown fat technology related to BioRestoratives metabolic program (ThermoStem Program).

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BioRestorative Therapies is making a powerful move up the charts after it was it was revealed at 12.04 pm Wednesday afternoon the Company was emerging from bankruptcy. This comes after the Company successfully entered into a reorganization plan with one of its creditors Auctus Capital after its March Bankruptcy filing in which the Company would emerge from bankruptcy with the commons intact, ready to begin their phase 2 trials and get BioRestorative back on a national stock exchange. BioRestorative Therapies has received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs. BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. It is the Companys intend that the product be used for the non-surgical treatment of painful lumbosacral disc disorders. We will be updating on BioRestorative when more details emerge so make sure you are subscribed to Microcapdaily so you know whats going on with BioRestorative.

Disclosure: we hold no position in BRTX either long or short and we have not been compensated for this article.

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Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency – Health Europa

By daniellenierenberg

For the first time, researchers at the Center for Cell-Based Therapy (CTC) in Ribeiro Preto, Brazil, have identified a non-hereditary mutation in blood cells from a patient with GATA2 deficiency.

GATA2 deficiency is a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2), which regulates the expression of genes that play a role in developmental processes and cell renewal.

An article on the study is publishedin the journalBlood.

The non-hereditary mutation may have acted as a natural gene therapy which prevented the disease from damaging the process of blood cell renewal. This meant that the patient did not develop such typical clinical manifestations as bone marrow failure, hearing loss, and lymphedema.

The researchers say that the findings pave the way for the use of gene therapy and changes to the process of checking family medical history and medical records for families with the hereditary disorder.

Luiz Fernando Bazzo Catto, first author of the article, said: When a germline [inherited] mutation in GATA2 is detected, the patients family has to be investigated because there may be silent cases.

The discovery was made when two sons were receiving medical treatment at the blood centre of the hospital run by FMRP-USP, both of which, in post-mortem DNA sequencing, showed germline mutations and GATA2 deficiency diagnosis. The researchers used next generation sequencing to estimate the proportion of normal blood cells in the fathers bone marrow, preventing clinical manifestations of GATA2 deficiency, and of cells similar to his childrens showing that 93% of his leukocytes had the mutation that protects from the clinical manifestations of GATA2 deficiency.

Following the sequencing of the fathers T-lymphocytes, the researchers found that the mutation occurred early in their lives and in the development of hematopoietic stem cells, which have the potential to form blood.

They also measured the activity of the blood cells, to see if they could maintain the activity of inducing normal cell production for a long time, by measuring the telomeres of his peripheral blood leukocytes. Telomeres are repetitive sequences of non-coding DNA at the tip of chromosomes that protect them from damage. Each time cells divide, their telomeres become shorter. They eventually become so short that division is no longer possible, and the cells die or become senescent.

The telomeres analysed by the researchers were long, indicating that the cells can remain active for a long time.

The researchers hypothesised that the existence of the somatic mutation in the fathers blood cells, and its restoration of the blood cell renewal process, may have contributed to the non-manifestation of extra-haematological symptoms of GATA2 deficiency such as deafness, lymphedema, and thrombosis.

Professor Rodrigo Calado, a corresponding author of the article, said: A sort of natural gene therapy occurred in this patient. Its as if he embodied an experiment and a medium-term prospect of analogous gene therapy treatment in patients with GATA2 deficiency.

The findings help us understand better how stem cells can recover by repairing an initial genetic defect.

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Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency - Health Europa

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gift of life marrow registry announces new center for cell and gene therapy – The Boca Raton Tribune

By daniellenierenberg

Chamber Member Update

Boca Raton, FL, November 17 Every three minutes a person in the United States is diagnosed with a blood disease such as leukemia, immunodeficiencies and sickle cell. To deliver cutting-edge, lifesaving therapy services for stem cell transplantations and improve patient outcomes, the Gift of Life Marrow Registry has announced the newly opened the Gift of Life Center for Cell and Gene Therapy.

Located at Gift of Lifesheadquarters in Boca Raton, Fla., theCenter will provide transplant physicians, researchers, and cell and gene therapy developers with the donor cell products they need to help more patients than ever before. One of the Centers signature services will be a biobank of off-the-shelf, on-demand cell therapy products available for transplantation, as well as those for engineering and ethical research. The inventory will be comprised of products collected from super donors with high frequency genetic characteristics sourced from the Gift of Life Marrow Registry. These cells will be tested, processed and cryopreserved by the Centers cellular therapy laboratory.

Another specialty service is the Centers innovation program, which will collaborate with world-renowned South Florida research institutions to improve transplant outcomes, develop processes for cell expansion and much more.

While many advancements have been made in stem cell treatments for those with blood diseases, there are still critical barriers that can impeded success, said Gift of Life CEO and Founder Jay Feinberg.

The only registry in the world started and run by a transplant survivor, Gift of Life has pioneered key innovations in the stem cell transplantation and donation process. Last April, the organization become the first registry to operate an in-house stem cell collection center that has not only helped to bring stem cell donors to collection faster, but also greatly improves the experience for donors. Gift of life is also the first registry to use buccal cheek swabs at recruitment drives instead of blood draws and the first to do mobile registration, including the creation of a smart phone app to register individuals quickly and effectively.

Gift of Lifes overarching mission isto democratize celltherapy and ensure that every patient has an equal opportunity to receive the treatment that can save their lives, said Feinberg. Doing so requires innovation, passion and an entrepreneurial approach to the challenges at hand and we are excited to draw upon our 30 years of expertise as we embark on this next phase of our work.

About Gift of Life Marrow RegistryGift of Life Marrow Registry is a 501(c)(3) nonprofit organization headquartered in Boca Raton, Fla. The organization, established in 1991, is dedicated to saving lives by facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, and other blood-related diseases. To learn more about Gift of Life Marrow Registry, visitwww.giftoflife.org.

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gift of life marrow registry announces new center for cell and gene therapy - The Boca Raton Tribune

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Stem Cells Market Detailed Analysis by On-going Trends, Prominent Size, Share, Sales and Forecast to 2025 – PRnews Leader

By daniellenierenberg

A succinct analysis of market size, regional growth and revenue projections for the coming years is presented in GlobalStem Cells Marketreport. The study further sheds light on major issues and the new growth strategies implemented by manufacturers that are part ofcompetitive landscape of the studied market.Thereport offers key trends, investment opportunities and drivers in Global Stem Cells Marketwith the latest market intelligence by adopting primary and secondary research methods. It also includes strategies adopted in the context of acquisitions and mergers, and business footprint extensions.

In order to provide more exactmarket forecast, the report comprises a complete research study and analysis ofCOVID-19 impact on the Global Stem Cells market. It also considers the strategies that can be adopted to deal with the situation.

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Competitive Landscape and Stem Cells Market Share Analysis:

The competitive landscape of the Stem Cells market provides data about the players operating in the studied market. The report includes a detailed analysis and statistics onprice, revenue and market share of the playersfor the period 2020-2025. The major players covered are as follows:

Thermo Fisher Scientific Inc.

Cellular Engineering Technologies Inc

Qiagen N.V

Sigma Aldrich Corporation

Becton, Dickinson and Company

Miltenyi Biotec

International Stem Cell Corporation

Stem Cell Technologies Inc.

Pluristem Therapeutics Inc

Medtronic, Inc

Zimmer Holdings, Inc.

Bio Time Inc

Zimmer Holdings, Inc

Orthofix, Inc.

Osiris Therapeutics Inc

Others Prominent Players

Key segments covered:

By Product

Adult Stem Cells

Neural Stem Cells

Hematopoietic Stem Cells

Mesenchymal Stem Cells

Umbilical Cord Stem Cells

Epithelial Stem cells and Skin Stem Cells

Others

Human Embryonic Stem Cells

Induced Pluripotent Stem Cells

Others

By Application

Regenerative Medicine

Neurology Regenerative Medicine

Oncology Regenerative Medicine

Myocardial Infraction Regenerative Medicine

Diabetes Regenerative Medicine

Hematology & Immunology Regenerative Medicine

Orthopedics Regenerative Medicine

Other Regenerative Medicine

Drug Discovery and Development

Other Applications

By Technology

Cell Acquisition

Bone Marrow Harvest

Umbilical Blood Cord

Apheresis

Others

Cell Production

Therapeutic Cloning

In-vitro Fertilization

Cell Culture

Isolation

Cryopreservation

Expansion and Sub-Culture

By End-User

Biopharmaceutical

Biotechnology Industry

Research Institutes

By Treatment Type

Allogeneic Stem Cell Therapy

Auto logic Stem Cell Therapy

Syngeneic Stem Cell Therapy

By Banking Type

Public

Private

By Region:

North America (U.S. & Canada)

Europe (Germany, United Kingdom, France, Italy, Spain, Russia, and Rest of Europe)

Asia Pacific (China, India, Japan, South Korea, Indonesia, Taiwan, Australia, New Zealand, and Rest of Asia Pacific)

Latin America (Brazil, Mexico, and Rest of Latin America)

Middle East & Africa (GCC, North Africa, South Africa, and Rest of Middle East & Africa)

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Some Important Highlights from the Report include:

Market CAGR during the 2020-2025 forecast period.

Comprehensive analysis on factors that will speed up the growth of Stem Cells marketover the next five years.

Precise estimates about the market size of global Stem Cells market and itscontribution to the parent market.

Precise forecasts for future developments in the Stem Cells industry and shifts in consumer behavior.

The growth of the Stem Cells Market across the Americas, APAC, Europe and MEA.

A detailed analysis of the industry competition and quantitative data on various vendors.

Comprehensive information on variables that will impede the growth of Stem Cells companies.

The Following are the Key Features of Global Stem Cells Market Report:

Market Overview, Industry Development, Market Maturity, PESTLE Analysis, Value Chain Analysis

Growth Drivers and Barriers, Market Trends & Market Opportunities

Porters Five Forces Analysis & Trade Analysis

Market Forecast Analysis for 2020-2025

Market Segments by Geographies and Countries

Market Segment Trend and Forecast

Market Analysis and Recommendations

Price Analysis

Key Market Driving Factors

Stem Cells Market Company Analysis: Company Market Share & Market Positioning, Company Profiling, Recent Industry Developments etc.

Why Choose KD Market Insights?

Provides accurate data and best-in class solutions to our clients.

Offers optimal market predictions and analysis of the business.

Ensures that clients gain unmatched competitive advantage, builds more competent organizations, and ensures enduring outcomes.

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Stem Cells Market Detailed Analysis by On-going Trends, Prominent Size, Share, Sales and Forecast to 2025 - PRnews Leader

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