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Critically ill Indonesian woman thanks Taiwan for saving life – Taiwan News

By daniellenierenberg

Taipei, Nov. 5 (CNA) An Indonesian migrant worker who received a stem cell transplant in Taiwan in June thanked the nation on Thursday for expediting her treatment by lifting travel restrictions for her family amid COVID-19 thereby facilitating the operation that saved her life.

At a press conference that day to celebrate being discharged from the hospital, 23-year-old Nina Herlina thanked Taiwan for giving her a new lease of life and said her treatment was a testament to Taiwan's healthcare capabilities. In November last year, Nina began suffering from bouts of menorrhagia that lasted for about 20 days and came with symptoms that included dizziness, tiredness, and fever.

In February, she turned to the Taiwan International Workers' Association (TIWA), a local NGO that promotes migrant workers' rights when she was fired, shortly after a doctor diagnosed her as suffering from aplastic anemia, an autoimmune disease in which the bone marrow stops making new blood cells. With the help of the TIWA, the young woman was allowed to remain in Taiwan, where she had worked as a caregiver since October 2018.

In March, she was confirmed as having severe aplastic anemia, requiring an allogeneic stem cell transplant to treat the disease, according to the TIWA. However, at that time the COVID-19 pandemic was worsening and Nina's family were in rural Indonesia and local medical institutions lacked the technology and techniques to identify a donor in time for a bone marrow transplant.

At that time she was being kept alive in Taiwan by weekly blood transfusions. However, frequent blood transfusions can have a detrimental effect on the success of a transplant.

In addition, she also had leukopenia, a condition when a person has a reduced number of white blood cells, which increases the risk of infection. As a result, doctors at Taipei Veterans General Hospital (TVGH) determined the patient was in urgent need of a transplant, according to TIWA.

With the assistance of TIWA, a TVGH medical team explained the condition to Herlina and her family members in Indonesia via video calls. Doctors said the healthy cells for the transplant should ideally come from a family member, making her two younger sisters, aged 5 and 14, the best candidates for the operation, TIWA said.

Based on humanitarian considerations, the Central Epidemic Command Center decided in June to lift travel restrictions for her mother and sisters to visit Taiwan.

After undergoing special blood tests arranged by TVGH, the 5-year-old sister was identified as a suitable donor for a transplant. The operation was carried out after the three family members completed their 21-day quarantine in Taiwan and provided two consecutive negative COVID-19 test results.

After having received medical treatment in Taiwan for nine months, Nina was discharged from the hospital Thursday, after doctors confirmed she had recovered from the life-threatening illness.

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Novel Targeted Drugs are Changing the Treatment of Diffuse Large B-Cell Lymphoma – Curetoday.com

By daniellenierenberg

Several drugs that work by targeting genetic alterations in cancer cells have won recent approval from the Food and Drug Administration as treatments for patients with diffuse large B-cell lymphoma (DLBCL).

Dr. Germame Ajebo, assistant professor of medicine at Georgia Cancer Center at Augusta University, shared information on novel treatments for the disease during the recent virtual CURE Educated Patient Leukemia & Lymphoma Summit.

In his talk, Ajebo focused on drugs meant for use in disease that has recurred or become resistant to previous treatments.

DLBCL is a usually aggressive form of the blood cancer known as a B-cell non-Hodgkin lymphoma, which affects the immune system. The disease causes rapid growth of tumors in the lymph nodes, spleen, liver, bone marrow or other organs.

Approved by the FDA within the last two years to treat aggressive DLBCL are oral Xpovio (selinexor), Polivy (polatuzumab vedotin-piiq) and Monjuvi (tafasitamab-cxix). In addition, an immunotherapy, the chimeric antigen receptor (CAR)-T cell therapy Yescarta (axi-cel), was approved to treat the disease in 2017, Ajebo reported.

Read more: Monjuvi-Revlimid Combination Approval Fills Unmet Need for Certain Patients with DLBCL.

Xpovio is a nuclear export inhibitor, which prevents cancerous cells from pushing tumor-suppressing proteins out of their nuclei. This results in tumor suppressors accumulating in the nucleus, where they can work to kill the cell.

In the phase 2b clinical trial that led to its approval which administered Xpovio by itself to 134 previously treated older adult patients the partial response rate (including those with tumor shrinkage) was 16%, the complete response rate (including those with no sign of cancer remaining) was 13% and the rate of stable disease (including patients with no progression of cancer) was 8.2%, Ajebo reported. Looking at all patients who had partial or complete responses, 38% responded for at least six months and 15% for at least 12 months.

The most common side effects that were serious or worse were low blood counts, Ajebo summarized. Other serious side effects included nausea, vomiting, diarrhea, weight loss, dizziness and infections.

Polivy is an antibody-drug conjugate that uses a targeted drug to deliver a potent chemotherapy directly to cancer cells.

It was approved based on the results of a phase 2 study of 80 previously treated patients who were divided into equally sized groups to receive the chemotherapy Treanda (bendamustine) and the targeted drug Rituxan (rituximab) with or without Polivy every 21 days for six cycles. At the end of treatment, 40% of those receiving the triplet combination had experienced a complete response, compared with 18% of those receiving Treanda and Rituxan alone. In the 63% of patients who achieved a best overall response at any point in the study while receiving the drug triplet, 48% had a response that lasted at least 12 months and 64% responded for at least six months, Ajebo noted.

Major side effects, he said, included tingling or weakness in the extremities, low blood counts, liver toxicity and tumor lysis syndrome, a condition that can damage organs due to blood chemistry issues arising from the quick destruction of tumor cells. The most common side effects of any severity included low blood counts, fatigue, diarrhea and fever.

Monjuvi, a targeted drug that inhibits the activity of the DLBCL-fueling protein CD19, was approved based on results of the phase 2 L-MIND study that demonstrated a 43% complete response rate and an 18% partial response rate in 80 previously treated adult patients who were prescribed the drug along with the targeted medication Revlimid (lenalidomide), with a median duration of response of 21.7 months. The drug is approved in combination with Revlimid for patients with recurrent or resistant DLBCL who are not eligible for, or did not agree to undergo, bone marrow transplant using their own stem cells, Ajebo said.

Serious side effects occurred in 52% of patients, he said, and included low blood counts and infections. The drug caused fatal reactions in 5% of patients, including stroke, respiratory failure, progressive multifocal leukoencephalopathy (a virus that infects the brain) and sudden death.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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Stem Cell Therapy Market to Surge at a Robust Pace in Terms of Revenue Over2017 2025 – Royal Sutton News

By daniellenierenberg

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -…

By daniellenierenberg

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov 5, 2020--

bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from its gene and cell therapy programs for sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT) and multiple myeloma (MM) will be presented, including seven oral presentations, at the 62 nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.

Updated results from patients in Group C of the companys Phase 1/2 HGB-206 study of LentiGlobin for SCD gene therapy (bb1111) will be presented.

bluebird bio will also present updated long-term efficacy and safety results from the LTF-303 follow-up study; outcomes across genotypes; and outcomes in pediatric patients from Phase 3 studies HGB-207 and HGB-212 of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for -thalassemia) in TDT.

Data from across the companys multiple myeloma program will be presented. Presentations will include updated safety and efficacy results from the Phase 1 CRB-401 clinical study of idecabtagene vicleucel (ide-cel, bb2121) and preliminary data from the ongoing Phase 1 CRB-402 clinical study of bb21217, as well as subgroup analyses of the pivotal Phase 2 KarMMa study of ide-cel. Ide-cel and bb21217 are investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immune therapies being studied, in partnership with Bristol-Myers Squibb, for the treatment of adult patients with MM.

Sickle Cell Disease Data at ASH

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Presenting Author: Julie Kanter, MD, University of Alabama at Birmingham, Birmingham, AL

Date/Time: Oral #365, Sunday, December 6, 2020, 9:45 am PST

Resolution of Serious Vaso-occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Presenting Author: Alexis A. Thompson, MD, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital, Chicago, IL

Date/Time: Oral #677, Monday, December 7, 2020, 1:30 pm PST

The GRNDaD Registry: Contemporary Natural History data and an analysis of real-world patterns of use and limitations of Disease Modifying Therapy in adults with SCD

Presenting Author: Alexandra Boye-Doe, MD, University of North Carolina School of Medicine, Chapel Hill, NC

Date/Time: Poster #1730, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Transfusion-Dependent -Thalassemia Data at ASH

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent -Thalassemia: Results in Patients with up to 6 Years of Follow-up

Presenting Author: Janet L. Kwiatkowski, MD, MSCE, Director, Thalassemia Center at Children's Hospital of Philadelphia, Philadelphia, PA

Date/Time: Oral #153, Saturday, December 5, 2020, 12:00 pm PST

Favorable Outcomes in Pediatric Patients in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) Studies of betibeglogene autotemcel Gene Therapy for the Treatment of Transfusion-dependent -thalassemia

Presenting Author: Alexis A. Thompson, MD, MPH, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, IL

Date/Time: Oral #154, Saturday, December 5, 2020, 12:15 pm PST

Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent -Thalassemia in the Phase 3 HGB-207 Study

Presenting Author: John B. Porter, MA, MD, FRCP, FRCPath, Head of Red Cell Unit, University College London Hospital, London, UK

Date/Time: Poster #776, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Response of patients with transfusion-dependent -thalassemia (TDT) to betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy based on HBB genotype and disease genetic modifiers

Presenting Author: Mark C. Walters MD, Medical Director, Jordan Family Center for BMT & Cellular Therapies Research, UCSF Benioff Childrens Hospital Oakland, Oakland, CA

Date/Time: Poster #1699, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Multiple Myeloma Data at ASH

Updated results from the Phase I CRB-402 study of anti-BCMA CAR-T cell therapy bb21217 in patients with relapsed and refractory myeloma: correlation of expansion and duration of response with T cell phenotypes

Presenting Author: Melissa Alsina, MD, Department of Blood and Marrow Transplantation and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL

Date/Time: Oral #130, Saturday, December 5, 2020, 9:45 am PST

Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study

Presenting Author: Yi Lin, MD, PhD, Division of Hematology, Mayo Clinic, Rochester, MN

Date/Time: Oral #131, Saturday, December 5, 2020, 10:00 am PST

Secondary Quality-of-Life Domains in Patients With Relapsed and Refractory Multiple Myeloma Treated With the BCMA-Directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel; bb2121): Results from the KarMMa Clinical Trial

Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA

Date/Time: Oral #437, Sunday, December 6, 2020, 12:15 pm PST

Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients with Relapsed/Refractory Multiple Myeloma: KarMMa Subgroup Analysis

Presenting Author: Jess Berdeja, MD, Sarah Cannon Research Institute and Tennessee Oncology, Nashville, TN

Date/Time: Poster #1367, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Characterization of Cytokine Release Syndrome in the KarMMa Study of Idecabtagene Vicleucel (ide-cel, bb2121) For Relapsed and Refractory Multiple Myeloma

Presenting Author: Ankit Kansagra, MD, Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, TX

Date/Time: Poster #1378, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Molecular and Phenotypic Profiling of Drug Product and Post-infusion Samples from CRB-402, an Ongoing: Phase I Clinical Study of bb21217 a BCMA-directed CAR T Cell Therapy

Presenting Author: Olivia Finney, PhD, Associate Director, Immunotherapy, bluebird bio

Date/Time: Poster #1401, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Effects of Prior Alkylating Therapies on Preinfusion Patient Characteristics and Starting Material for CAR T Cell Product Manufacturing in Late-Line Multiple Myeloma

Presenting Author: Julie Rytlewski, PhD, Bristol Myers Squibb, Princeton, NJ

Date/Time: Poster #1405, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

KarMMa-4: Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy, in High-Risk Newly Diagnosed Multiple Myeloma

Presenting Author: Saad Z. Usmani, MD, Director, Clinical Research in Hematologic Malignancies, Levine Cancer Institute/Atrium Health, Charlotte, NC

Date/Time: Poster #1418, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Healthcare Resource Utilization and Cost of Cytokine Release Syndrome and Neurologic Events in Patients with Relapsed and Refractory Multiple Myeloma Receiving the BCMA-directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel, bb2121) in the KarMMa Trial

Presenting Author: Parmeswaran Hari, MD, Medical College of Wisconsin, Milwaukee, WI

Date/Time: Poster #1598, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

A Matching-Adjusted Indirect Comparison of Efficacy Outcomes for Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T Cell Therapy Versus Conventional Care in Triple-Class Exposed Relapsed and Refractory Multiple Myeloma

Presenting Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA

Date/Time: Poster #1653, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) Responses Are Characterized by Early and Temporally Consistent Activation and Expansion of CAR T Cells With a T Effector Phenotype

Presenting Author: Nathan Martin, PhD, Bristol Myers Squibb, Princeton, NJ

Date/Time: Poster #2315, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

KarMMa-3: A Phase 3 Study of Idecabtagene Vicleucel (ide-cel,bb2121), a BCMA-Targeted CAR T Cell Therapy Versus Standard Regimens in Relapsed and Refractory Multiple Myeloma

Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium

Date/Time: Poster #2323, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) in Relapsed and Refractory Multiple Myeloma: Analyses of High-Risk Subgroups in the KarMMa Study

Presenting Author: Noopur S. Raje, MD, Massachusetts General Hospital, Boston, MA

Date/Time: Poster #3234, Monday, December 7, 2020, 7:00 am 3:00 pm PST

Health State Utility Valuation in Patients with Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Treated with the BCMAdirected CAR T Cell Therapy, Idecabtagene Vicleucel (idecel, bb2121): Results from the KarMMa Trial

Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium

Date/Time: Poster #3465, Monday, December 7, 2020, 7:00 am 3:00pm PST

Abstracts outlining bluebird bios accepted data at ASH are available on the ASH conference website.

About LentiGlobin for SCD (bb1111)

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD (bb1111) is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

LentiGlobin for SCD was designed to add functional copies of a modified form of the -globin gene ( A-T87Q -globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once patients have the A-T87Q -globin gene, their red blood cells can produce anti-sickling hemoglobin (Hb A-T87Q ) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As of March 3, 2020, a total of 37 patients have been treated with LentiGlobin for SCD to-date in the HGB-205 (n=3) and HGB-206 (n=34) clinical studies. The HGB-206 total includes: Group A (n=7), B (n=2) and C (n=25).

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

The U.S. Food and Drug Administration (FDA) granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD. LentiGlobin for SCD continues to be evaluated in the ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About betibeglogene autotemcel

Transfusion dependent beta-thalassemia (TDT) is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT require chronic blood transfusions to maintain adequate Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel (beti-cel) adds functional copies of a modified form of the -globin gene ( A-T87Q -globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q -globin gene, they have the potential to produce HbA -T87Q, which is gene therapy-derived adult hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0 / 0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

As of March 3, 2020, a total of 60 pediatric, adolescent and adult patients, including 11 patients with at least 5 years of follow-up, across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. In studies of beti-cel, patients were assessed for transfusion independence, defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. FDA granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the United States. Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of beti-cel.

About idecabtagene vicleucel (ide-cel, bb2121)

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio. Ide-cel was granted accelerated assessment by the European Medicines Agency (EMA) on March 26, 2020, and the Marketing Authorization Application (MAA) was validated by the EMA on May 20, 2020. The FDA accepted the ide-cel Biologics License Application (BLA) for priority review on September 22, 2020.

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with RRMM in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other secondary endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 10 6 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

CRB-401 (NCT02658929) is an open-label Phase 1 study evaluating the preliminary safety and efficacy of ide-cel in patients with relapsed and refractory multiple myeloma (RRMM). The primary endpoint of the study is safety. CRB-401 was designed as a two-part (dose escalation and dose expansion) study to determine the maximum tolerated dose and further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose; these findings established the recommended dose of the Phase 2 KarMMa trial. All patients have been treated in the study and follow-up is ongoing.

In addition to the pivotal KarMMa and CRB-401 trials, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) exploring ide-cel combinations and activity in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel is not approved for any indication in any geography.

About bb21217

bb21217 is an investigational BCMA-targeted CAR T cell therapy that uses the ide-cel CAR molecule and is cultured with the PI3 kinase inhibitor (bb007) to enrich for T cells displaying a memory-like phenotype with the intention to increase the in vivo persistence of CAR T cells. bb21217 is being studied for patients with multiple myeloma in partnership with Bristol Myers Squibb.

bluebird bios clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with relapsed and refractory multiple myeloma (RRMM), designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM. For more information visit: clinicaltrials.gov using identifier NCT03274219.

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bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -...

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Generation of normal induced pluripotent stem cell line KUMCi002-A from bone marrow CD34+ cells of patient with multiple myeloma disease having 13q…

By daniellenierenberg

This article was originally published here

Stem Cell Res. 2020 Oct 15;49:102030. doi: 10.1016/j.scr.2020.102030. Online ahead of print.

ABSTRACT

Multiple myeloma (MM) is a hematological cancer characterized by an uncontrolled proliferation of antibody-secreting plasma cells within the bone marrow. Currently, cell therapy such as chimeric antigen receptor T-cell (CAR-T) based on induced pluripotent stem cells (iPSCs) has received attention for treating MM. However, the generation of iPSCs from MM patients appears to be very rarely reported. Here we generated an iPSC line from CD34+ bone marrow cells of a patient with MM using human placenta-derived cell conditioned medium (hPCCM), offering a relatively high efficiency in humanized conditions. This iPSC line might be a useful model for research on MM.

PMID:33142253 | DOI:10.1016/j.scr.2020.102030

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Survival Benefit of GVHD in MDS Is Limited to Specific Patient Subgroup – Cancer Therapy Advisor

By daniellenierenberg

Development of limited, chronic graft-vs-host disease (GVHD) following allogeneic hematopoietic stem cell transplantation (HSCT) was associated with a survival benefit for patients with high-risk myelodysplastic syndrome (MDS), according to results of a retrospective study published in Clinical Cancer Research.1

MDS is a heterogeneous group of clonal hematopoietic stem cell disorders characterized by an inadequate production of normal, mature blood cells in bone marrow.

At the present time, the only curative treatment for patients with MDS is allogeneic HSCT. Although risks associated with this approach include posttransplant relapse, as well as the development of acute and/or chronic GVHD, previous findings from studies of patients with acute leukemia treated with allogeneic HSCT have shown potent graft-vs-tumor effects manifesting as lower mortality in those patients who subsequently experienced chronic GVHD.

The clinical data used in this study were collected from more than 300 HSCT centers throughout Japan by the Japanese Data Center for Hematopoietic Cell Transplantation.

MDS was characterized as low- or high-risk disease according to the

French-American-British or World Health Organization classification schemes, depending on the year of diagnosis.2,3 Consensus criteria were used to assign GVHD severity and distinguish acute and chronic forms of the condition.

Study inclusion criteria dictated patients should be aged 16 through 70 years, had received first allogeneic HSCT between 2001 and 2017, had achieved neutrophil engraftment, and had a follow-up period of over 60 days.

Of the 3119 patients included in this study, 1193 and 1926 were classified as having low- and high-risk disease, respectively. In the overall group, the median patient age was 54 years, more than 90% of patients had an Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1, and 85% were diagnosed with de novo disease.

At a median follow-up of 55 months, rates of 5-year overall survival (OS) were 63% and 48% for those with low- and high-risk disease, respectively (P <.001). The cumulative incidence of posttransplantation relapse at 5 years was approximately twice as high for those with high-risk disease (29%) compared with those with low-risk disease (15%; P <.001), although the cumulative incidence of nonrelapse mortality was similar in the 2 groups: 25% (low-risk disease) and 27% (high-risk disease; P =.338).

Multivariate analyses accounting for all confounding variables performed to evaluate these transplant outcomes in patient subgroups defined according to disease risk, as well the severity of GVHD and whether it was classified as acute or chronic, revealed the following:

In summarizing the results of this study, the study authors emphasized that these data demonstrated a survival benet of the graft-versus-MDS effect is present only in high-risk MDS patients with limited chronic GVHD.1

Authors of an accompanying editorial noted that in recent years, the mutational spectrum in MDS has become more associated with clinical phenotype and prognosis, and selection of a patient with MDS for HSCT has begun to incorporate the mutational landscape of the patients disease. Thus, one will have to be cautious in extrapolating the current data to ongoing and future trials, which have begun to incorporate molecular information.4

References

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Actinium Announces Two Oral Presentations Featuring Data and Findings from the Phase 3 SIERRA Trial of Iomab-B at the 62nd American Society of…

By daniellenierenberg

NEW YORK, Nov. 4, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc.,(NYSE AMERICAN: ATNM) ("Actinium") today announced that two abstracts on the Company's Antibody Radiation Conjugate (ARC) Iomab-B were accepted for oral presentations at the 2020 American Society of Hematology (ASH) annual meeting that is being held virtually December 5-8, 2020.

"This is an exciting fourth quarter for the company and we are honored to have multiple oral presentations at this year's ASH conference. Our 3 oral presentations and one poster presentation demonstrate the clinical progress we have seen not only with Iomab-B, but our other programs including Actimab-A in combination with novel and approved therapeutic agents," stated Sandesh Seth, Actinium's Chairman and CEO. "We look forward to presenting the Iomab-B data in further detail during the two oral presentations on the Iomab-B SIERRA study at ASH in December. The company remains on track to report safety and feasibility data from 75% of the patients to be enrolled in SIERRA, as well as to complete the ad hoc interim analysis in the fourth quarter."

Mark Berger, Actinium's Chief Medical officer, said, "We are encouraged that we continue to see positive ongoing results from our Phase 3 pivotal SIERRA trial in relapsed or refractory Acute Myeloid Leukemia (R/R AML) patients. In the Iomab-B Phase 3 trial we continue to see 100% engraftment in patients receiving a therapeutic dose of Iomab-B in the treatment arm whereas 83% of control arm patients failed salvage therapy, which includes the recently approved targeting agents such as venetoclax. This high failure rate demonstrates the significant need that exists in R/R AML and represents the paradigm shift we are looking to initiate with Iomab-B. The strong safety and feasibility data we have seen thus far gives us confidence that these older patients with active AML may benefit by undergoing a potentially curative bone marrow transplant which they could not receive otherwise."

Details & Highlights for Oral Presentations

Note: The two abstractsincludeSIERRA Phase 3 trial data available to the company from its CRO prior to August 10, 2020, the ASH submission cutoff date. Per ASH rules, updated data sets are permitted to be included in the live presentations.

OralPresentationTitle:

Personalized Targeted Radioimmunotherapy with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] in Patients with Active Relapsed or Refractory Acute Myeloid Leukemia Results in Successful Donor Hematopoietic Cells Engraftment with the Timing of Engraftment Not Related to the Radiation Dose Delivered

Publication Number:

193

Session Name:

721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities

Session Date:

Saturday, December 5, 2020

Presentation Time:

1:00 PM PT / 4:00 PM ET

Phase 3 SIERRA Preliminary Results

Baseline Characteristics

Randomized to Iomab-B(N=53)

Randomized to Conventional Care (CC)(N=53)

Age (yrs, median, range)

64 (55-77)

65 (55-77)

Molecular & Cytogenetic Risk

Favorable: 2%Intermediate: 33%

Adverse: 65%

Favorable: 4%

Intermediate: 30%

Adverse: 66%

% TransplantedIntent-to-Treat Group

87% (46/53)

17% (9/53)

59% (26/44)

Results

Received Therapeutic dose of Iomab-B & Transplanted (N=46)****

Eligible to Receive Std. of Care Transplant Post-Salvage (N=9)

Evaluated for Crossover (N=44)*****

Cross-over Rate

n/a

n/a

Received Therapeutic Dose of Iomab-B (N=26)

Transplanted (N=26)

59% (26/44)

% Transplanted

100% (46/46)

17% (9/53)

100% (26/26)

BM Blast % @ baseline (median, range)

26 (4-95)

14 (5-97)

30 (6-87)

BM Blast % pre-HCT (median, range)

26 (4-95)

1 (0-3)*

32.5 (2-75)

Days to ANC Engraftment

14 (9-22)***

17 (13-83)#

14 (10-37)**

Days to Platelet Engraftment

17 (4-39)***

22 (8-35)#

19 (1-38)**

Days to HCT (Post Randomization)

30 (23-60)

66 (51-86)

65 (36-161)^

Myeloablative Dose Delivered to Bone Marrow

14.7 (4.6-32) Gv

n/a

14.4 (6.3-30) Gv

540 (313-1008) mCi

632 (354-1027) mCi

Chimerism>/=95% by D100

91% (39/43^ Evaluable)

67% (4/6^^ Evaluable)

87% (20/23^^^ Evaluable)

100-day non-Relapse Transplant-Related Mortality

5% (2/40 Evaluable)

25% (2/8 Evaluable)

8% (2/24 Evaluable)

*1 pt with 8% BM blasts on D42 with CRp on D50, ** ANC engraftment data not available (N=3), platelet engraftment data not available (N=4); *** ANC engraftment data not available (N=4), platelet engraftment data not available (N=9), ^ 1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab & transplant when stable, # ANC and platelet engraftment data not available (N=2)

**** No Therapy Dose (7) due to: Declining KPS (4), Infusion Reaction (1), Unfavorable Biodistribution (1), Post-Randomization Eligibility (1); 1 Pending Treatment.

*****Ineligible for Iomab-B HCT after crossover evaluation - 13: due to Hospice Care/Progression (4), Declined/Ineligible for HCT (5), Died Pre-Crossover (4). 4 Received Dosimetry but No Therapy Dose due to Declining KPS; 2 Pending Evaluation for Crossover.

^ Did not achieve 95% chimerism (4); Data pending (2); Died (1); ^^ Did not achieve 95% chimerism (4); Data pending (1); ^^^Did not achieve 95% chimerism (4); Data pending (2);

Oral PresentationTitle:

High Doses of Targeted Radiation with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Do Not Correlate with Incidence of Mucositis, Febrile Neutropenia or Sepsis in the Prospective, Randomized Phase 3 Sierra Trial for Patients with Relapsed or Refractory Acute Myeloid Leukemia

Publication Number:

135

Session Name:

721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities

Session Date:

Saturday, December 5, 2020

Presentation Time:

9:30 AM PT / 12:30 PM ET

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Stem Cell Therapy Market Size, Key Development Trends, and Growth Projection to 2027 – Stock Market Vista

By daniellenierenberg

Stem Cell Therapy Market Size And Forecast

A comprehensive overview of the Stem Cell Therapy Market is recently added by Verified Market Research to its humongous database. Furthermore, the Stem Cell Therapy Market report has been aggregated by collecting informative data of various dynamics such as market drivers, restraints, and opportunities. Furthermore, this innovative report makes use of SWOT, PESTLE, and Porters Five Forces analyses to get a closer outlook on the Stem Cell Therapy Market. Furthermore, the Stem Cell Therapy Market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth. Furthermore, this statistical market research repository examines and estimates the Stem Cell Therapy Market at the global and regional levels. The study covers the impact of various drivers and manacles on the Stem Cell Therapy Market growth opportunities over the forecast period.

Impact of Covid-19 :

During the first quarter of 2020, different global economies were badly impacted by a viral outbreak of COVID-19. This viral outbreak of the Covid-19 was later recognized as a global pandemic by the World Health Organization (WHO). COVID-19 spread in different global countries, affecting a large number of people in a short timeframe. The outburst of COVID-19 adversely hit different global economies in the world. The stringent regulations imposed by several governments, including complete lockdown and quarantine methodologies to fight against COVID-19, resulted in a massive impact on various business sectors. We at Verified Market Research offer an informative report on the Stem Cell Therapy Market which helps in making strategic decisions over the forecast period.

Competitive Landscape:

The degree of competition among leading global companies has been elaborated by examining various leading key players operating across the global regions An expert team of research analysts sheds light on various attributes such as -global market competition, market share, latest industry developments, innovative product launches, partnerships, mergers or acquisitions by leading companies in the Stem Cell Therapy Market. The leading manufacturers have been analyzed by using research methodologies for getting insight views on global competition.

Following key players have been profiled with the help of proven research methodologies:

The Stem Cell Therapy Market has been examined into different global market segments such as type, applications and global geographies. Each and every global market segment has been studied to get informative insights into various global regions.

Global Stem Cell Therapy Market Segmentation:

1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

Global Stem Cell Therapy Market Segment by Global Presence:

North America Latin America Middle East Asia-Pacific Africa Europe

The report has been aggregated by using a couple of research methodologies such as primary and secondary research techniques. It helps in collecting informative pieces of professional information for deriving effective insights into the market. This informative report helps in making well informed and strategic decisions throughout the forecast period.

Key questions answered through this analytical market research report include:

What are the latest trends, new patterns and technological advancements in the Stem Cell Therapy Market? Which factors are influencing the Stem Cell Therapy Market over the forecast period? What are the global challenges, threats and risks in the Stem Cell Therapy Market? Which factors are propelling and restraining the Stem Cell Therapy Market? What are the demanding global regions of the Stem Cell Therapy Market? What will be the global market size over the coming future? What are the different effective business strategies followed by global companies?

If you have any custom requirements, please let us know and we will offer you the customized report as per your requirements.

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Verified Market Research is a leading Global Research and Consulting firm servicing over 5000+ customers. Verified Market Research provides advanced analytical research solutions while offering information enriched research studies. We offer insight into strategic and growth analyses, Data necessary to achieve corporate goals, and critical revenue decisions.

Our 250 Analysts and SMEs offer a high level of expertise in data collection and governance use industrial techniques to collect and analyze data on more than 15,000 high impact and niche markets. Our analysts are trained to combine modern data collection techniques, superior research methodology, expertise, and years of collective experience to produce informative and accurate research.

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Stem Cell Therapy Market Size, Key Development Trends, and Growth Projection to 2027 - Stock Market Vista

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Aruvant Announces Updated Data to be Presented in Oral Presentation of ARU-1801 Data at the 62nd American Society of Hematology (ASH) Annual Meeting -…

By daniellenierenberg

NEW YORK, Nov. 4, 2020 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, announced that an abstract demonstrating clinical benefit of the company's lead product candidate ARU-1801 has been published online and will be the subject of an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place virtually from December 5 to 8. Michael S. Grimley, M.D., Professor of Clinical Pediatrics, Medical Director of the Division of Bone Marrow Transplantation and Immune Deficiency at the Cincinnati Children's Hospital Medical Center, will present the data at 2:15 PM ET on December 7, 2020. Today the abstract was published online and will be published in the upcoming supplemental issue of Blood.

Data presented at ASH is from the MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD). The MOMENTUM study, which continues to enroll patients, examines ARU-1801, an autologous cell therapy leveraging a modified gamma globin lentivirus vector, in individuals with severe SCD. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy. ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of Graft-versus-Host Disease (GvHD) seen with allogeneic stem cell transplants. The data to be presented at ASH highlights participants dosed with product manufactured with both the original academic manufacturing process and the enhanced Process II.

"The clinical results thus far demonstrate that ARU-1801 holds significant promise for achieving durable responses with a reduced intensity conditioning approach to gene therapy," said Dr. Grimley, a principle investigator in the MOMENTUM study. "Given the impact chemotherapy toxicity has on physician and patient decision making around treatment options, ARU-1801 has the potential to be a unique option for SCD patients seeking gene therapy."

Abstract and Oral Presentation Information

Title: Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning TransplantPublication Number: 680Session Name: 114. Hemoglobinopathies, Excluding ThalassemiaClinical: Novel Treatments for Sickle Cell DiseaseDate:Monday, December 7, 2020Session Time:1:30 PM - 3:00 PMPresentation Time:2:15 PM

About ARU-1801ARU-1801 is a one-time potentially curativeinvestigational gene therapy for individuals living with sickle cell disease. This product candidate was designed to address the limitations of current treatment options including chemotherapy toxicity, donor availability, andchronic administration and replace it with a curative therapy. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial in patients with sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM StudyAruvant is currently conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time only potentially curative gene therapy for patients with SCD. This Phase 1/2 study currently is enrolling individuals with SCD, and information may be found at http://www.momentumtrials.com.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromsickle cell disease(SCD). ARU-1801 is an investigational lentiviral gene therapy currently in aclinical trial,the MOMENTUM study, as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated engraftment of ARU-1801 and amelioration of SCD is possible with one dose of low intensity chemotherapy. For more information on the clinical study, please visit http://www.momentumtrials.com and for more on the company, please visitwww.aruvant.com.

SOURCE Aruvant Sciences

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Aruvant Announces Updated Data to be Presented in Oral Presentation of ARU-1801 Data at the 62nd American Society of Hematology (ASH) Annual Meeting -...

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Putting it all together – The Statesman

By daniellenierenberg

The ability to grow and reproduce is a fundamental property of living organisms. Whether an organism is composed of a single cell or trillions of cells, individual cells must be able to grow and divide in an appropriately regulated fashion.

Cell growth is accomplished through the synthesis of new molecules of proteins, nucleic acids, carbohydrates and lipids. As the accumulation of these molecules causes the volume of a cell to increase, the plasma membrane grows to prevent the cell from burst-ing.

But cells cannot continue to enlarge indefinitely; as a cell grows larger, there is an accompanying decrease in its surface area/volume ratio and hence in its capacity for ef-fective exchange with the environment.

Therefore, cell growth is generally accompanied by cell division, whereby one cell gives rise to two new daughter cells (The term daughter is used by convention and does not indicate that cells have gender).

For single-celled organisms, cell division increases the total number of individuals in a population. In multicellular organisms, cell division either increases the number of cells, leading to growth of the or-ganism, or replaces cells that have died. In an adult human, for example, about two million stem cells in bone marrow divide every second to maintain a constant num-ber of red blood cells in the body. When cells grow and divide, the newly formed daugh-ter cells are usually genetic duplicates of the parent cell, containing the same (or virtually the same) DNA sequences.

Therefore, all the genetic information in the nucleus of the parent cell must be duplicated and carefully distributed to the daughter cells during the division process.

In accomplishing this task, a cell passes through a series of discrete stages, collectively known as the cell cycle. The cell cycle begins when two new cells are formed by the division of a single parental cell and ends when one of those cells divides again into two cells.

To early cell biologists studying eukaryotic cells with the mi-croscope, the most dramatic events in the life of a cell were those associated with the point in the cycle when the cell actually divides. This division process, called the M phase, involves two overlapping events in which the nucleus divides first and the cytoplasm second. Nuclear division is called mitosis, and the division of the cytoplasm to pro-duce two daughter cells is termed cytokinesis.

The stars of the mitotic drama are the chromosomes.

The beginning of mitosis is marked by condensation (coiling and folding) of the cells chromatin, which generates chromosomes that are thick enough to be individually discernible under the micro-scope.

Because DNA replication has already taken place, each chromosome actually consists of two chromosome copies that remain attached to each other until the cell divides. As long as they remain attached, the two new chromosomes are referred to as sister chromatids.

As the chromatids become visible, the nuclear envelope breaks into fragments. Then, in a stately ballet guided by the mi-crotubules of the mitotic spindle, the sister chromatids separate and each now a full-fledged chromosome move to opposite ends of the cell.

By this time, cytokinesis has usually begun, and new nuclear membranes envelop the two groups of daughter chromosomes as cell division is completed. While visually striking, the events of the mitotic phase account for a relatively small portion of the total cell cycle; for a typical mammalian cell, the mitotic phase usually lasts less than an hour.

Cells spend the majority of their time in the growth phase between divisions, called interphase. Most cellular contents are synthesised continuously during interphase, so cell mass gradually increases as the cell approaches division.

During interphase the amount of nuclear DNA doubles, and experiments using radioactive DNA precursors have shown that the new DNA is synthesised during a particular portion of interphase named the S phase (S for synthesis). A time gap called G1 phase separates the S phase from the preceding M phase, and a second gap, the G2 phase, separates the end of S phase from the beginning of the next M phase.

Although the cells of a multicellular organism divide at varying rates, most studies of the cell cycle involve cells growing in culture, where the length of the cycle tends to be similar for different cell types. We can easily determine the overall length of the cell cycle the generation time for cultured cells by counting the cells under a microscope and determining how long it takes for the population to double.

In cultured mammalian cells, for example, the total cycle usually takes about 18-24 hours. Once we know the total length of the cycle, it is possible to determine the length of specific phases. To determine the length of the S phase, we can expose cells to a radioactively labelled DNA precursor for a short period of time and then examine the cells by autoradiography.

The fraction of cells with silver grains over their nuclei represents the fraction of cells that were somewhere in S phase when the radioactive compound was available. When we mul-tiply this fraction by the total length of the cell cycle, the result is an estimate of the average length of the S phase.

For mammalian cells in culture, this fraction is often around 0.33, which indicates that S phase is about six to eight hours in length. Similarly, we can estimate the length of the M phase by multiplying the generation time by the percentage of the cells that are actually in mitosis at any given time.

This percentage is called the mitotic index.

The mitotic index for cultured mammalian cells is often about three to five per cent, which means that M phase lasts less than an hour (usually 30-45 minutes). In contrast to the S and M phases, whose lengths tend to be quite similar for different mammalian cells, the length of G1 is quite variable, depending on the cell type. Although a typical G1 phase lasts 8-10 hours, some cells spend only a few minutes or hours in Gl, whereas others are delayed for long periods of time. During Gl, a major decision is made as to whether and when the cell is to divide again. Cells that become arrested in Gl, awaiting for a signal that will trigger re-entry into the cell cycle and a commitment to divide, are said to be in G0 (G zero).

Other cells exit from the cell cycle entirely and undergo terminal differentiation, which means that they are destined never to divide again; most of the nerve cells in our body are in this state. In some cells, a transient arrest of the cell cycle can also occur in G2. In general, however, G2 is shorter than Gl and is more uniform in duration among different cell types, usually lasting 4-6 hours.

The writer is associate professor and head, department of botany, Ananda Mohan College, Kolkata.

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Putting it all together - The Statesman

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Bragar Eagel & Squire, PC Reminds Investors That Class Action Lawsuits Have Been Filed Against Precigen, Royal Caribbean, Mesoblast, and Loop…

By daniellenierenberg

NEW YORK, Nov. 04, 2020 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized shareholder rights law firm, reminds investors that class actions have been commenced on behalf of stockholders of Precigen, Inc. f/k/a Intrexon Corporation (NASDAQ: PGEN; XON), Royal Caribbean Group (NYSE: RCL), Mesoblast Limited (NASDAQ: MESO), and Loop Industries, Inc. (NASDAQ: LOOP). Stockholders have until the deadlines below to petition the court to serve as lead plaintiff. Additional information about each case can be found at the link

Precigen, Inc. f/k/a Intrexon Corporation (NASDAQ: PGEN; XON)

Class Period: May 10, 2017 to September 25, 2020

Lead Plaintiff Deadline: December 4, 2020

On September 25, 2020, the U.S. Securities and Exchange Commission (SEC) issued a cease and desist order against Precigen. The cease and desist order involved inaccurate reports concerning the companys purported success converting relatively inexpensive natural gas into more expensive industrial chemicals using a proprietary methane bioconversion (MBC) program. The order noted that the Company was primarily using significantly more expensive pure methane for the relevant laboratory experiments but was indicating that the results had been achieved using natural gas. The cease-and-desist order further stated that although the Company pitched the MBC program privately to numerous potential business partners over the course of 2017 and 2018 and [a] number of these potential partners performed due diligence on the MBC program including reviewing lab results and plans for commercialization. [The Company] has not yet found a partner for the MBC program.

The complaint, filed on October 5, 2020, alleges that throughout the Class Period defendants made false and/or misleading statements and/or failed to disclose to investors that: (1) the Company was using pure methane as feedstock for its announced yields for its methanotroph bioconversion platform instead of natural gas; (2) yields from natural gas as a feedstock were substantially lower than the aforementioned pure methane yields; (3) due to the substantial price difference between pure methane and natural gas, pure methane was not a commercially viable feedstock; (4) the Companys financial statements for the quarter ended March 31, 2018 were false and could not be relied upon; (5) the Company had material weaknesses in its internal controls over financial reporting; (6) the Company was under investigation by the SEC since October 2018; and (7) as a result of the foregoing, defendants public statements were materially false and misleading at all relevant times.

For more information on the Precigen class action go to: https://bespc.com/cases/PGEN

Royal Caribbean Group (NYSE: RCL)

Class Period: February 4, 2020 to March 17, 2020

Lead Plaintiff Deadline: December 7, 2020

The complaint, filed on October 7, 2020, alleges that throughout the Class Period defendants failed to disclose material facts about the Companys decrease in bookings outside China, instead maintaining that it was only experiencing a slowdown in bookings from China. The Action further alleges that defendants failed to disclose material facts about the Companys inadequate policies and procedures to prevent the spread of COVID-19 on its ships. The truth about the scope of the impact that COVID-19 had on the Companys overall bookings and the inability of Royal Caribbean to prevent the virus spread on its ships was revealed through a series of disclosures.

First, on February 13, 2020, Royal Caribbean issued a press release stating that it had canceled 18 voyages in Southeast Asia due to recent travel restrictions and further warning that recent bookings had been softer for its broader business.

On this news, Royal Caribbean shares fell over 3 percent.

Second, on February 25, 2020, Royal Caribbean filed its 2019 Form 10-K, indicating that COVID-19 concerns were negatively impacting its overall business.

On this news, Royal Caribbean shares fell over 14 percent.

Third, on March 10, 2020, Royal Caribbean withdrew its 2020 financial guidance, increased its revolving credit facility by $550 million, and announced that it would take cost-cutting actions due to the proliferation of COVID-19, further revealing that COVID-19 was severely impacting Royal Caribbeans 2020 customer booking and that its safety measures were inadequate to prevent the spread of the virus on its ships.

On this news, Royal Caribbean shares fell over 14 percent.

Fourth, on March 11, 2020, Royal Caribbeans largest competitor, Carnival, announced a 60-day suspension of all operations, prompting concern that Royal Caribbean would follow suit. At the same time, Royal Caribbean also cancelled two cruises, beginning a series of cancellations and suspensions to follow.

On this news, Royal Caribbean shares fell almost 32 percent.

Fifth, on March 14, 2020, Royal Caribbean announced a suspension of all global cruises for 30 days.

On this news, Royal Caribbean stock fell over 7 percent.

Sixth, on March 16, 2020, the Company revealed that global operations could be suspended longer than anticipated, announcing the cancellations of two additional cruises throughout April and into May.

On this news, Royal Caribbean shares fell over 7 percent.

Finally, on March 18, 2020, analysts downgraded Royal Caribbeans stock and slashed their price targets.

On this news, Royal Caribbean shares fell more than 19 percent.

For more information on the Royal Caribbean class action go to: https://bespc.com/cases/RCL

Mesoblast Limited (NASDAQ: MESO)

Class Period: April 16, 2019 to October 1, 2020

Lead Plaintiff Deadline: December 7, 2020

Mesoblast develops allogeneic cellular medicines using its proprietary mesenchymal lineage cell therapy platform. Its lead product candidate, RYONCIL (remestemcel-L), is an investigational therapy comprising mesenchymal stem cells derived from bone marrow. In February 2018, the Company announced that remestemcel-L met its primary endpoint in a Phase 3 trial to treat children with steroid refractory acute graft versus host disease (aGVHD).

In early 2020, Mesoblast completed its rolling submission of its Biologics License Application (BLA) with the FDA to secure marketing authorization to commercialize remestemcel-L for children with steroid refractory aGVHD.

On August 11, 2020, the FDA released briefing materials for its Oncologic Drugs Advisory Committee (ODAC) meeting to be held on August 13, 2020. Therein, the FDA stated that Mesoblast provided post hoc analyses of other studies to further establish the appropriateness of 45% as the null Day-28 ORR for its primary endpoint. The briefing materials stated that, due to design differences between these historical studies and Mesoblasts submitted study, it is unclear that these study results are relevant to the proposed indication.

On this news, the Companys share price fell $6.09, or approximately 35%, to close at $11.33 per share on August 11, 2020.

On October 1, 2020, Mesoblast disclosed that it had received a Complete Response Letter (CRL) from the FDA regarding its marketing application for remestemcel-L for treatment of SR-aGVHD in pediatric patients. According to the CRL, the FDA recommended that the Company conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD. The CRL also identified a need for further scientific rationale to demonstrate the relationship of potency measurements to the products biologic activity.

On this news, the Companys share price fell $6.56, or 35%, to close at $12.03 per share on October 2, 2020.

The complaint, filed on October 8, 2020, alleges that throughout the Class Period defendants made materially false and/or misleading statements, as well as failed to disclose material adverse facts about the Companys business, operations, and prospects. Specifically, defendants failed to disclose to investors: (1) that comparative analyses between Mesoblasts Phase 3 trial and three historical studies did not support the effectiveness of remestemcel-L for steroid refractory aGVHD due to design differences between the four studies; (2) that, as a result, the FDA was reasonably likely to require further clinical studies; (3) that, as a result, the commercialization of remestemcel-L in the U.S. was likely to be delayed; and (4) that, as a result of the foregoing, defendants positive statements about the Companys business, operations, and prospects were materially misleading and/or lacked a reasonable basis.

For more information on the Mesoblast class action go to: https://bespc.com/cases/MESO

Loop Industries, Inc. (NASDAQ: LOOP)

Class Period: September 24, 2018 to October 12, 2020

Lead Plaintiff Deadline: December 14, 2020

On October 13, 2020, Hindenburg Research published a report alleging, among other things, that Loops scientists, under pressure from CEO Daniel Solomita, were tacitly encouraged to lie about the results of the companys process internally. The report also stated that Loops previous claims of breaking PET down to its base chemicals at a recovery rate of 100% were technically and industrially impossible, according to a former employee. Moreover, the report alleged that Executives from a division of key partner Thyssenkrupp, who Loop entered into a global alliance agreement with in December 2018, told us their partnership is on indefinite hold and that Loop underestimated both costs and complexities of its process.

On this news, the Companys share price fell $3.78, or over 32%, to close at $7.83 per share on October 13, 2020.

The complaint, filed on October 13, 2020, alleges that throughout the Class Period defendants made materially false and/or misleading statements, as well as failed to disclose material adverse facts about the Companys business, operations, and prospects. Specifically, defendants failed to disclose to investors: (1) that Loop scientists were encouraged to misrepresent the results of Loops purportedly proprietary process; (2) that Loop did not have the technology to break PET down to its base chemicals at a recovery rate of 100%; (3) that, as a result, the Company was unlikely to realize the purported benefits of Loops announced partnerships with Indorama and Thyssenkrupp; and (4) that, as a result of the foregoing, defendants positive statements about the Companys business, operations, and prospects were materially misleading and/or lacked a reasonable basis.

For more information on the Loop class action go to: https://bespc.com/cases/Loop

About Bragar Eagel & Squire, P.C.:Bragar Eagel & Squire, P.C. is a nationally recognized law firm with offices in New York and California. The firm represents individual and institutional investors in commercial, securities, derivative, and other complex litigation in state and federal courts across the country. For more information about the firm, please visit http://www.bespc.com. Attorney advertising. Prior results do not guarantee similar outcomes.

Contact Information:Bragar Eagel & Squire, P.C.Brandon Walker, Esq. Melissa Fortunato, Esq.Marion Passmore, Esq.(212) 355-4648investigations@bespc.comwww.bespc.com

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Stem Cell Therapy Market is estimated to be worth USD 8.5 Billion by 2030 – PRnews Leader

By daniellenierenberg

The success of approved stem cell therapies has caused a surge in interest of biopharma developers in this field; many innovator companies are currently progressing proprietary leads across different phases of clinical development, with cautious optimism

Roots Analysis has announced the addition of Global Stem Cells Market: Focus on Clinical Therapies, 20202030 (Based on Source (Allogeneic, Autologous); Origin (Adult, Embryonic); Type (Hematopoietic, Mesenchymal, Progenitor); Lineage (Amniotic Fluid, Adipose Tissue, Bone Marrow, Cardiosphere, Chondrocytes, Corneal Tissue, Cord Blood, Dental Pulp, Neural Tissue Placenta, Peripheral Blood, Stromal Cells); and Potency (Multipotent, Pluripotent)) report to its list of offerings.

There is a growing body of evidence supporting the vast applicability and superiority of treatment outcomes of stem cell therapies, compared to conventional treatment options. In fact, the unmet needs within this domain have spurred the establishment of many start-ups in recent years.

To order this 500+ page report, which features 185+ figures and 220+ tables, please visit this link

Key Market Insights

Over 280 stem cell therapies are under development, most of which are allogeneic productsMore than 50% of the pipeline candidates are in the mid to late phase trials (phase II and above), and allogenic therapies (majority of which are derived from the bone marrow) make up 65% of the pipeline.

70% of pipeline candidates are based on mesenchymal stem cellsIt is worth highlighting that the abovementioned therapies are designed to treat musculoskeletal (22%), neurological (21%) and cardiovascular (15%) disorders. On the other hand, hematopoietic stem cell-based products are mostly being evaluated for the treatment of oncological disorders, primarily hematological malignancies.

Close to 85% stem cell therapy developers are based in North America and Asia-Pacific regionsWithin these regions, the US, China, South Korea and Japan, have emerged as key R&D hubs for stem cell therapies. It is worth noting that majority of the initiatives in this domain are driven by small / mid-sized companies

Over 1,500 grants were awarded for stem cell research, since 2015More than 45% of the total amount was awarded under the R01 mechanism (which supports research projects). The NCI, NHLBI, NICHD, NIDDK, NIGMS and OD emerged as key organizations that have offered financial support for time periods exceeding 25 years as well.

Outsourcing has become indispensable to R&D and manufacturing activity in this domainPresently, more than 80 industry / non-industry players, based in different regions across the globe, claim to provide contract development and manufacturing services to cater to the unmet needs of therapy developers. Examples include (in alphabetical order) Bio Elpida, Cell and Gene Therapy Catapult, Cell Tech Pharmed, GenCure, KBI Biopharma, Lonza, MEDINET, Nikon CeLL innovation, Roslin Cell Therapies, WuXi Advanced Therapies and YposKesi.

North America and Asia-Pacific markets are anticipated to capture over 80% share by 2030The stem cell therapies market is anticipated to witness an annualized growth rate of over 30% during the next decade. Interestingly, the market in China / broader Asia-Pacific region is anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 8.5 billion (by 2030) financial opportunity within the stem cell therapies market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom stem cell therapies are currently considered to be a promising alternatives for the treatment of a myriad of disease indications, with the potential to overcome challenges associated with conventional treatment options. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles of several companies (including those listed below); each profile features an overview of the company, financial information (if available), stem cell therapy portfolio and an informed future outlook.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/stem-cells-market/296.html

or email [emailprotected]

You may also be interested in the following titles:

Contact:

Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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ADL live: Matters of the Heart Monash University – Monash Lens

By daniellenierenberg

With all the attention still focused on the devastating COVID-19 pandemic despite Australia slowly moving towards a new normal heart disease remains the countrys leading cause of death. It kills one person every 12 minutes, and causes one in four deaths in Australia and beyond.

The heart beats without our active thought, which is why, perhaps, we take for granted the health of this rhythmic, ticking pump; our most vital organ.

Watchthe 'A Different Lens' live event viaFacebookorLinkedInat 7.30pm on Thursday, 5 November

With the launch now of Monash Universitys Victorian Heart Institute (VHI), and the Victorian Heart Hospital (VHH) also opening on campus in 2022, the focus from the experts involved will be on this one-organ system, although from an expansive range of medical and other disciplines. When the under-construction hospital opens, the two new heart hubs will be housed in the same building, making it the centre of heart research, teaching and cardiac care in Australia. It will be the first and only dedicated heart hospital in the country.

Every year, 50,000 Australians will suffer a heart attack, killing 20 people a day, with one person dying every 12 minutes.

Professor Steve Nicholls, a world-renowned cardiologist originally from Adelaide, heads both. The work of the new institute is already underway, and is expected to break down barriers between research, teaching and clinical care.

There is something incredibly exciting about the fact that biomedical research will be happening in the same building that we will treat patients, Professor Nicholls says.

He's outlined what he calls the "five grand challenges"for the next decade of heart research, with the ambition of shifting the status quo in heart disease.

They are:

Living well is understanding that we need to take a more holistic approach to treating heart health, Professor Nicholls says.

We need to consider diet, sleep, mindfulness and wellbeing, but also survivorship. People who survive other diseases like cancer now find themselves at a great risk of heart disease. How do we improve health, and the patient experience for them?

Dr Emily Kotschet, a Monash cardiologist and electrophysiologist, is a specialist in the technology and "mechanical intervention"(pacemaker) side of the equation.

Technology is best when you are advancing in terms of treatment, not just prevention, she says.

The advances right now are miniaturisation and digital technology for managing patients remotely implantables that are remote-accessed, self-sufficient and patient driven. With the patients in more control than ever, their experience is continually improving.

Technologically, the old pacemaker was a big can on your shoulder with leads to the heart, she says.

The new ones are like AAA batteries that sit beside the heart. As we miniaturise for treatment, there will be way more engagement with patients to pick up their symptoms early and get on top of them early. Discussions with IT academics at Monash will bridge the gap between our method and the digital platforms that are emerging.

Dr Kotschet also says womens heart health has not had the media profile and reach that breast cancer has, so public awareness is low.

I think breast cancer really got cancer into the spotlight 15 or 20 years ago with celebrities and endorsements, and they are now well-funded. Their [clinical]trials are travelling quite nicely. Now I think its time for heart disease as a big killer to find its spotlight.

This ability to more freely collaborate across university disciplines on an ostensibly "medical"issue is a priority for the heart specialists and clinicians.

The head of the School of Public Health and Preventive Medicine, Professor Sophia Zoungas, is a leading expert in screening and managing cardiovascular disease. She likens the new institute and hospital to a forward-thinking company such as Google that, when presented with a problem, draws in the best minds available to help solve it.

Despite the fact the institute and hospital have a physical location, it also provides a forum for virtual, broader work, bringing in areas across the University that may not be co-located, she says.

Whether thats public health, engineering, science hopefully it will also provide that core clinical training for clinicians and researchers in the one place. We will be able to track the best people, the best minds.

She says COVID-19 has put heart disease further under the radar.

People forget heart disease is still our biggest killer. We have all been a little distracted this year with the global pandemic but even within the context of this pandemic, one of the most serious outcomes of it [for patients]has been the cardiovascular complications.

For Professor Peter Currie, the head of Monashs renowned Australian Regenerative Medicine Institute (ARMI), the future of heart health relies on scientists understanding the fundamental building blocks of the heart. Despite the heart being unable to repair itself (unlike other organs), and the fact that stem cells do not work, Professor Currie has an optimistic view of how regenerative medicine can create the heart disease solutions of the future. He sees this is as the challenge, and the new institute and hospital as the place to do it.

I think the great opportunity is to cherry-pick interested and dedicated researchers who have an interest in this area of biology and medicine to work on these big problems.

Everyone thinks cardiovascular disease has been hit on the head because weve got a few drugs, he says, but nothing could be further from the truth.

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A Review of Stem CellBased Therapies for Parkinson Disease – AJMC.com Managed Markets Network

By daniellenierenberg

Researchers discuss the development and potential of stem cellbased therapies in the treatment of Parkinson disease.

In assessing treatment for Parkinson disease (PD), the current standard of care involves levodopa, potentially in combination with carbidopa, to address the loss of dopamine known to occur as the condition progresses. However, several innovations in therapy for PD have occurred in recent years, particularly deep brain stimulation and the potential use of stem cells.

Discussing in a review published in International Journal of Molecular Sciences, researchers Zhaohui Liu, PhD, MSc, and Hoi-Hung Cheung, PhD, sought to discuss the development of new therapeutic strategies that have led to the initiation of exploratory clinical trials, particularly the application of stem cells for the treatment of PD.

Delving into the use of stem cellbased treatments in PD, the researchers say that several important pathways have emerged as targets for potential therapeutic intervention.

Conventional therapeutic strategies for relieving the symptomatic stages of PD remain, but with new genetic insights, it may be possible to use preventive neuroprotective treatments for people at risk of developing PD, they highlight. In parallel with efforts to prevent and control symptomatic PD, researchers are also investigating stem cells as replacements for diseased neurons or degenerated tissues.

As they note, dopaminergic (DA) cell transplantation is believed to be the most promising cell replacement therapy. Aligned with this approach, a recent novel treatment showcased the plausibility of reprogramming the skin cells of a single patient with PD to take on the characteristics of DA neurons and replace damaged brain cells. In their findings, the patient exhibited improvements in quality of life and day-to-day activities requiring motor skills. However, as this treatment was performed on only 1 person, the researchers cautioned that larger, diverse clinical studies are needed to demonstrate further efficacy and long-term results.

Other notable stem cellbased treatments include:

Although we are not yet examining a disease-modifying treatment, stem cell transplantation has the potential to be at the forefront of such PD treatments in the future, conclude the researchers. The transplantation process and the procedures required for its optimization are still not fully understood, and further research is required to achieve treatment for PD.

Reference

Liu Z, Cheung H-H. Stem cell-based therapies for Parkinson disease. Int J Mol Sci. Published online October 29, 2020. doi:10.3390/ijms21218060

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Research providing better understand of graying hair | Health – Galveston County Daily News

By daniellenierenberg

You most likely have noticed this phenomenon. When someone you know gets critically injured, experiences a serious illness or a major stressor, they soon look older. Sound familiar? Next time you see your friend, their hair is gray or even white. Have you ever wondered why that happens?

A new study from scientists at Harvard now reveals our sympathetic nervous system likely plays a major role in this process. The sympathetic nervous system is also called the fight or flight system. When youre in a stressful or dangerous situation, the sympathetic nervous system releases hormones to increase your alert level and heart rate to prepare you to act.

It all starts in a small area of the brain called the amygdala, and it eventually activates adrenal glands in the kidneys, which release adrenaline to coordinate the response of your internal organs and heighten your mental focus.

We have stem cells in the bottom of each hair follicle. These stem cells mature into cells called melanocytes, which produce pigment for our hair and skin color. Using mice, researchers found stressful events can lead to damage in the stem cells in our hair follicles.

Stress caused these stem cells to mature much faster than normal, which meant the supply of stem cells in the hair follicles eventually ran out. Without the stem cells producing melanocytes, the hair in the follicle lost its color and looked gray.

But how did this happen? The researchers thought maybe the immune system attacked these melanocytes resulting in the graying or that a hormone called cortisol produced by the adrenal glands might be responsible. Neither of those turned out to be the cause of the graying hair.

In some intricate experiments, the scientists discovered sympathetic nerve cells associated with hair follicles were the key. These nerve cells released a hormone called noradrenaline into the melanocyte stem cells, which caused them to mature and eventually disappear from the base of the follicle and caused the graying of the hair.

In other experiments, noradrenaline treatment of human melanocytes in lab cultures lead to rapid growth, suggesting this same process likely occurs in humans leading to the graying of hair.

Other scientists also have shown depleting melanocytes leads to a progression of the first gray hairs to gray and then to white fur in dark-colored mice, which sounds like what we see in humans.

So heres a question: If you have rapid growth of these melanocytes, can you prevent the hair from graying? The answer appears to be yes, based on the treatment of these same mice with drugs that block the release of noradrenaline.

This treatment couldnt be used in humans because blocking the flight or flight response would have severe consequences in other areas of our lives. But this new information could be used in the future for more specific drugs that would just work on the hair follicles. This would be a great benefit to me if I only had enough hair.

Medical Discovery News is hosted by professors Norbert Herzog at Quinnipiac University, and David Niesel of the University of Texas Medical Branch. Learn more at http://www.medicaldiscoverynews.com.

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Global Anti-Ageing Drugs Market (2020) to Witness Huge Growth by 2026 | La Roche-Posay, Revision Optics, Calico, Nu Skin, L’ORAL, Unity Biotechnology,…

By daniellenierenberg

The latest research report on the Anti-Ageing Drugs Market Industry Analysis, Market Size, Opportunities and Forecast, 2020 2028 provides a comprehensive assessment of the Anti-Ageing Drugs market for the forecast period from 2020 to 2028, including market values for the years 2018 and 2019. The investigative report provides a detailed analysis of the impact of COVID-19 on various segments in the Anti-Ageing Drugs market based on product type, application, and end-use across numerous countries around the world. Further, the report also provides insights into market developments, trends, supply and demand changes across various regions across the globe. Thereby, the report provides a holistic view on the Anti-Ageing Drugs Market in order to help decision makers with various strategic insights and future outlook. The Anti-Ageing Drugs market is expected to witness continued growth during the forecast period from 2020 to 2028.

Leading companies reviewed in the Anti-Ageing Drugs Market report are: La Roche-Posay, Revision Optics, Calico, Nu Skin, LORAL, Unity Biotechnology

Get Free Exclusive Sample of this Premium Report at: https://www.zealinsider.com/report/6400/anti-ageing-drugs-market#sample

The report covers various aspects of the Anti-Ageing Drugs market segmented into product type, application and end-use. The report provides market numbers for the years 2018 and 2019 based on actual market findings also market estimates for forecasts for the period from 2020 to 2020 for each of the products types, applications and end-use segments.

Furthermore, the report includes a detailed competitive analysis among the market participants in the Anti-Ageing Drugs market. The report offers an in-depth comparative analysis of the competitors in the market based on their product offerings, market share and geographic presence. Some of the leading companies covered in the report include La Roche-Posay, Revision Optics, Calico, Nu Skin, LORAL, Unity Biotechnology

***NOTE***

We are continuously monitoring the market developments and changes occurring as a direct or indirect impact of the ongoing COVID-19 pandemic. Thereby, we are in a position to provide information on the market values and trends for both pre-COVID-19 and post-COVID-19 scenarios.

Target audiences for the report include:

Anti-Ageing Drugs Market Segmentation, By Product Type:Serums and supplements, Antioxidants and enzymes, Stem cells and drugs

Anti-Ageing Drugs Market Segmentation, By Application:Skin and hair, Skeletal and muscles, Age-related disorders, Others

Anti-Ageing Drugs Market Segmentation, By Geography:

Access Full Report information, here: https://www.zealinsider.com/report/6400/anti-ageing-drugs-market

Key Focus Areas in the Report:

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Global Anti-Ageing Drugs Market (2020) to Witness Huge Growth by 2026 | La Roche-Posay, Revision Optics, Calico, Nu Skin, L'ORAL, Unity Biotechnology,...

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Global Regenerative Medicine Market (2020 to 2025) – Industry Trends, Share, Size, Growth, Opportunity and Forecast – ResearchAndMarkets.com -…

By daniellenierenberg

DUBLIN--(BUSINESS WIRE)--The "Regenerative Medicine Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

The global regenerative medicine market grew at a CAGR of around 16% during 2014-2019. Looking forward, the publisher expects the global regenerative medicine market to continue its strong growth during the next five years.

Regenerative medicine refers to a branch of biomedical sciences aimed at restoring the structure and function of damaged tissues and organs. It involves the utilization of stem cells that are developed in laboratories and further implanted safely into the body for the regeneration of damaged bones, cartilage, blood vessels and organs. Cellular and acellular regenerative medicines are commonly used in various clinical therapeutic procedures, including cell, immunomodulation and tissue engineering therapies. They hold potential for the effective treatment of various chronic diseases, such as Alzheimer's, Parkinson's and cardiovascular disorders (CVDs), osteoporosis and spinal cord injuries.

The increasing prevalence of chronic medical ailments and genetic disorders across the globe is one of the key factors driving the growth of the market. Furthermore, the rising geriatric population, which is prone to various musculoskeletal, phonological, dermatological and cardiological disorders, is stimulating the market growth. In line with this, widespread adoption of organ transplantation is also contributing to the market growth. Regenerative medicine minimizes the risk of organ rejection by the body post-transplant and enhances the recovery speed of the patient.

Additionally, various technological advancements in cell-based therapies, such as the development of 3D bioprinting techniques and the adoption of artificial intelligence (AI) in the production of regenerative medicines, are acting as other growth-inducing factors. These advancements also aid in conducting efficient dermatological grafting procedures to treat chronic burns, bone defects and wounds on the skin. Other factors, including extensive research and development (R&D) activities in the field of medical sciences, along with improving healthcare infrastructure, are anticipated to drive the market further.

Companies Mentioned

Key Questions Answered in This Report:

Key Topics Covered:

1 Preface

2 Scope and Methodology

3 Executive Summary

4 Introduction

4.1 Overview

4.2 Key Industry Trends

5 Global Regenerative Medicine Market

5.1 Market Overview

5.2 Market Performance

5.3 Impact of COVID-19

5.4 Market Forecast

6 Market Breakup by Type

6.1 Stem Cell Therapy

6.1.1 Market Trends

6.1.2 Market Forecast

6.2 Biomaterial

6.2.1 Market Trends

6.2.2 Market Forecast

6.3 Tissue Engineering

6.3.1 Market Trends

6.3.2 Market Forecast

6.4 Others

6.4.1 Market Trends

6.4.2 Market Forecast

7 Market Breakup by Application

7.1 Bone Graft Substitutes

7.1.1 Market Trends

7.1.2 Market Forecast

7.2 Osteoarticular Diseases

7.2.1 Market Trends

7.2.2 Market Forecast

7.3 Dermatology

7.3.1 Market Trends

7.3.2 Market Forecast

7.4 Cardiovascular

7.4.1 Market Trends

7.4.2 Market Forecast

7.5 Central Nervous System

7.5.1 Market Trends

7.5.2 Market Forecast

7.6 Others

7.6.1 Market Trends

7.6.2 Market Forecast

8 Market Breakup by End User

8.1 Hospitals

8.1.1 Market Trends

8.1.2 Market Forecast

8.2 Specialty Clinics

8.2.1 Market Trends

8.2.2 Market Forecast

8.3 Others

8.3.1 Market Trends

8.3.2 Market Forecast

9 Market Breakup by Region

9.1 North America

9.2 Asia Pacific

9.3 Europe

9.4 Latin America

9.5 Middle East and Africa

10 SWOT Analysis

11 Value Chain Analysis

12 Porters Five Forces Analysis

13 Price Analysis

14 Competitive Landscape

14.1 Market Structure

14.2 Key Players

14.3 Profiles of Key Players

For more information about this report visit https://www.researchandmarkets.com/r/erd0e3

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Global Regenerative Medicine Market (2020 to 2025) - Industry Trends, Share, Size, Growth, Opportunity and Forecast - ResearchAndMarkets.com -...

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Here’s how to treat those brown spots on your face, chest and hands. – Mamamia

By daniellenierenberg

"It also hydrates and plumps up collagen making the skin appear more luminous." Go hard on it.

Retinol

You'll also need to have an exfoliant in your routine. We're talking chemical exfoliants, like retinol (can be in the form of a serum or cream - whatever works).

"Retinol (vitamin A) helps to improve cellular turnover, so that melanin patches are reduced in colour and some pigment may also be permanently shed too," said Dr D'Anna.

If retinol doesn't agree with your skin at this stage, try lactic acid or glycolic acid (again - can be used in a cleanser, toner, serum etc). These will do the job, too.

SPF

Okay. This is less of an option, and more of awe're-telling-you-to-do-it thing. Even Dr D'Anna agreed: "SPF is mandatory in reducing the darkening of the spots. Without the sun, the amount of melanin produced is very low."

"Prevention is much better than treatment. So, wear SPF every day, and cover your arms/hands and head/face with a good hat or long sleeves," added Dr D'Anna.

Prescription skincare

If all else fails on the skincare front - see your doctor for other topicaloptions.

"There are some pharmaceuticals that may be prescribed by your doctor too to help reduce the pigmentation," said Dr D'Anna. "This needs to be done under medical guidance and care to ensure that the skin has been examined and then diagnosed correctly."

Maybe. But you need to check in with a dermatologist or skin specialist first to make sure your skin type can be safely treated.

Dr D'Anna said, "The best treatment will vary according to your skin, the level of pigmentation and other skin considerations. It is best to get the advice of a qualified dermal therapist before proceeding."

Here are some professional treatments that can help reduce these melanin pigmentation patches:

Laser or IPL

These are good non-invasive solutions that will usually give you significant results after a course of treatments.

"A combination of topical treatments, light and laser therapies work best," said Dr Gunatheesa. "Broad-based light therapy, fractional ablative lasers will fade or remove existing liver spots."

Okay. How does that work?

"Laser or IPL also reduce the amount of colour in the skin," said Dr D'Anna. "The light energy heats the damaged and pigmented patches, so that the pigment is disrupted and lightened over a series of sessions."

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Gommage (Gentle Exfoliation) Benefits, Precautions, and How to Do It – Healthline

By daniellenierenberg

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Gommage is a French exfoliation technique thats been gaining popularity in the United States. The word gommage literally translates to erasing or exfoliation.

The concept of exfoliation isnt new in France. Marie Antoinette supposedly used a mask made of cognac, eggs, powdered milk, and lemon juice to cleanse and exfoliate her skin while ruling France in the 1700s.

Modern gommage uses a combination of ingredients that exfoliate your skin chemically through the combination of enzymes and scrubbing. Along with other exfoliating techniques, it has the potential to clear away dead skin cells and stimulate skin cell growth to keep your skin looking polished and smooth.

Keep reading to find out how gommage exfoliation works, why you may want to include it in your skin care routine, and who makes a good candidate.

Gommage products contain one of several enzymes that have proteolytic effects on dead skin cells. Proteolytic simply means they break down the proteins that make up these cells so that they can easily be removed.

The enzymes found in gommage products can be extracted from plant or animal sources. Some of the most commonly used enzymes include:

Gommage exfoliants also contain sticky ingredients like xanthan gum or paraffin that mechanically exfoliate your skin by pulling away dead skin cells when the product is removed from your skin.

When performing a gommage treatment at home, you can follow the instructions on the package. Most products will instruct you to:

Exfoliants are usually divided into two categories:

There are many types of exfoliation techniques, ranging from homemade scrubs to chemical peels. Most exfoliation products work by exfoliating your skin either mechanically or chemically, but gommage products do both when applied through gentle massage.

Theres no research available specifically comparing gommage products with other exfoliant techniques, but many products claim to be gentler on your skin than other exfoliants. Gommage formulas vary widely among products so some of these products are gentler and more effective than others.

Gommage exfoliants have the potential to have the same benefits as traditional exfoliating techniques, such as:

No matter what type of product youre using, over-exfoliating can lead to skin thats:

Gommage exfoliants often use plant-sourced enzymes in their formulas. If you have a known allergy to any fruits or plants, you may want to check the ingredients carefully before using a gommage product. Pineapples, papayas, and pumpkin are three common plants often used in these products.

Any time you start using a new skin care product, its a good idea to use it on only a small part of your skin to see how your body reacts before applying to your face or other sensitive areas.

Gommage products are gentle enough to be used by most adults. People with naturally oily skin can exfoliant as often as every day. If you have drier skin, you may only need to exfoliate once per week.

Gommage products are often marketed as gentler alternatives to traditional exfoliants. However, formulas vary among brands. If you have very sensitive skin, you may want to stick to a washcloth and mild chemical exfoliator.

If you have acne or are taking retinol or benzoyl peroxide for acne, its a good idea to talk to a dermatologist before using exfoliants since they can lead to worse breakouts

Its also a good idea to talk to a dermatologist if your skin is thinning, you have open wounds, or you have another skin condition you think could be affected.

Some aestheticians in your area may offer gommage treatments. You can also buy gommage exfoliants at some pharmacies and other places that sell cosmetics.

Shop for gommage exfoliants online.

Gommage comes from the French word for erasing. Gommage treatment involves applying a cream or paste to your skin and waiting for it to dry. As the product hardens, enzymes in the product break down dead skin cells. When you rub the product away, the friction rubs these dead skin cells away.

Gommage exfoliation is relatively safe and may be a good option if youre looking for a gentle exfoliant. Whenever you start using a new skin care product, its a good idea to use it on a small patch of skin before using it on your entire face to see how your body reacts first.

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The roles bone marrow plays in the body – Williston Daily Herald

By daniellenierenberg

Bones make up the skeletal system and serve the important function of giving the body support to be able to move. Whats inside the bones also is essential to personal health.

Bone marrow can be found in the center of bones. According to the online wellness resource Healthline, this viscous or spongy tissue comes in two types: red or yellow bone marrow. Both have specific functions in the body.

Red bone marrow is essential for a process called hematopoiesis, or blood cell production. Hematopoietic stem cells in the red bone marrow can develop into key blood cells, including red blood cells, which carry oxygen-rich blood to the body; platelets, which help blood to clot; and white blood cells, which are involved in immune system responses.

Yellow bone marrow is involved with the storage of fats. These fats can be used as an energy source as needed. Yellow bone marrow also contains mesenchymal stem cells that can develop into bone, fat, muscle, or cartilage cells.

Over time, yellow bone marrow replaces red bone marrow in most of the bones in the adult body. Only a few bones, such as the pelvis, skull, vertebrae, and ribs, will contain red bone marrow into adulthood.

According to Medical News Today, bone marrow makes more than 200 billion new blood cells every day. Most blood cells in the body develop from bone marrow cells.

Issues with bone marrow can produce a host of side effects. Fatigue or weakness, fever, increased infections, easy bleeding and bruising, and specific conditions like leukemia and anemia can develop as a result of bone marrow-related problems. In some cases, a bone marrow transplant may be needed to replace diseased or nonfunctioning bone marrow. It also may help regenerate a new immune system that can fight leukemia or other cancers.

Bone marrow transplants also may involve replacing existing bone marrow with genetically healthy bone marrow to prevent future damage from certain genetic diseases, according to Medical News Today. Bone marrow transplants can come from ones own stem cells, a twin, a sibling, parent, or an unrelated donor. Marrow transplants also may come from stored umbilical cord blood.

Bone marrow is vital to the overall health and function of the human body. Bone marrow affects just about every other cell due to its unique relationship with blood production and immune function.

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