Archive for December, 2020

Newswise Researchers from The University of Kansas Cancer Center are involved in the presentation of nearly 50 research studies at the 62ndAmerican Society of Hematology (ASH) Annual Meeting, to be held virtually Dec. 5-8 because of the COVID-19 pandemic. With more than 18,000 members from nearly 100 countries, the ASH is the world's largest professional society serving both clinicians and scientists around the world who are working to conquer blood diseases.

The KU Cancer Center is one of only 71 cancer centers designated by the National Cancer Institute because they meet rigorous standards for transdisciplinary, state-of-the-art research focused on developing new and better approaches to preventing, diagnosing and treating cancer. Its catchment area includes the state of Kansas as well as western Missouri.

These 49 research studies represent the hard work of our many researchers focused on blood diseases, said Roy Jensen, M.D., director of the KU Cancer Center. This includes innovations in immunotherapy, advances in leukemia and significant work in stem cell transplants. While the conference is virtual this year, the KU Cancer Center will be well represented.

While a full list of abstracts involving KU Cancer Center researchers can be found online, three of the most significant are listed below.

# # #

About The University of Kansas Cancer Center:

The University of Kansas Cancer Center is transforming cancer research and clinical care by linking an innovative approach to drug discovery, delivery and development to a nationally-accredited patient care program. Our consortium center includes cancer research and health care professionals associated with the University of Kansas Medical Center and The University of Kansas Health System; the University of Kansas, Lawrence; The Stowers Institute for Medical Research; Childrens Mercy; and in partnership with members of the Masonic Cancer Alliance.

About the University of Kansas Medical Center:

The University of Kansas Medical Centers mission is to educate exceptional health care professionals through a full range of undergraduate, graduate, professional, postdoctoral and continuing education programs in the schools of Medicine, Nursing and Health Professions. KU Medical Center also advances the health sciences through world-class research programs; provides compassionate and state-of-the-art patient care in an academic medical center environment; and works with communities in every Kansas county to improve the health of Kansans.

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Cancer center is a contributor to 49 research studies at the 62nd American Society of Hematology Annual Meeting - Newswise

NEW YORK, Dec. 03, 2020 (GLOBE NEWSWIRE) -- IN8bio, Inc., a clinical-stage biotechnology company focused on developing innovative allogeneic, autologous and genetically modified gamma-delta T cell therapies for the treatment of cancers (IN8bio or the Company), today announced an upcoming presentation that provides an update of the ongoing Phase I clinical trial of their product candidate INB-100 at the 62nd American Society of Hematology Annual Meeting & Exposition (ASH), which will take place virtually from December 5 to 8, 2020. INB-100 is designed for the treatment of patients with leukemia undergoing hematopoietic stem cell transplantation with haploidentical donors.

The poster and accompanying narrated slide presentation is titled, First-in-Human Phase I Trial of Adoptive Immunotherapy with Ex Vivo Expanded and Activated gamma delta T-Cells Following Haploidentical Bone Marrow Transplantation and Post-BMT Cyclophosphamide and reviews the study design and provides a brief update on enrollment and patient status.

The company reported that, as of abstract submission, three female subjects with acute leukemia had been enrolled in the INB-100 Phase 1 trial, of whom two had been dosed, and that no treatment-related adverse events had been recorded. The trial is continuing to enroll and treat patients. The abstract for the presentation can be found at https://ash.confex.com/ash/2020/webprogram/Paper142876.html.

The poster and slide presentation are jointly authored by the scientific and physician investigators from IN8bio and The University of Kansas Cancer Center (KU Cancer Center), and will be presented by the studys Principal Investigator, Dr. Joseph McGuirk, Schutte-Speas Professor of Hematology-Oncology, Division Director of Hematological Malignancies and Cellular Therapeutics and Medical Director, Blood and Marrow Transplant at KU Cancer Center.

This preliminary data report from KU Cancer Center with our allogeneic product candidate, INB-100, demonstrates the absence of significant GvHD in these initial patients, said William Ho, Chief Executive Officer of IN8bio. This suggests that gamma delta T-cells delivered as an off-the-shelf allogeneic cell therapy may be well tolerated and have significant potential to treat patients with serious and life-threatening cancers.

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Dr. McGuirk, commented, Potentially curative stem cell transplants using partially matched donors -- called haploidentical transplants have greatly expanded access to stem cell transplantation. The infusion of donor-derived gamma delta T-cells from the stem cell donor, offers the hope of diminishing this risk of relapse and curing more patients.

About IN8bioIN8bio is a clinical-stage biotechnology company focused on developing novel therapies for the treatment of cancers, including solid tumors, by employing allogeneic, autologous and genetically modified gamma-delta T cells. IN8bios technology incorporates drug-resistant immunotherapy (DRI), which has been shown in preclinical studies to function in combination with therapeutic levels of chemotherapy. IN8bio is currently conducting two investigator-initiated Phase 1 clinical trials for its lead gamma-delta T cell product candidates: INB-200 for the treatment of newly diagnosed glioblastoma, which is a difficult to treat brain tumor that progresses rapidly, and INB-100 for the treatment of patients with acute leukemia undergoing hematopoietic stem cell transplantation. For more information about the Company and its programs, visit http://www.IN8bio.com.

Forward Looking StatementsCertain statements herein concerning the Companys future expectations, plans and prospects, including without limitation, the Companys current expectations regarding the curative potential of its product candidates, constitute forward-looking statements. The use of words such as may, might, will, should, expect, plan, anticipate, believe, estimate, project, intend, future, potential, or continue, the negative of these and other similar expressions are intended to identify such forward looking statements. Such statements, based as they are on the current expectations of management, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond the Companys control. Consequently, actual future results may differ materially from the anticipated results expressed in such statements. Specific risks which could cause actual results to differ materially from the Companys current expectations include: scientific, regulatory and technical developments; failure to demonstrate safety, tolerability and efficacy; final and quality controlled verification of data and the related analyses; expense and uncertainty of obtaining regulatory approval, including from the U.S. Food and Drug Administration; and the Companys reliance on third parties, including licensors and clinical research organizations. Do not place undue reliance on any forward-looking statements included herein, which speak only as of the date hereof and which the Company is under no obligation to update or revise as a result of any event, circumstances or otherwise, unless required by applicable law.

Contact:IN8bio, Inc.Kate Rochlin, Ph.D.+1 646.933.5605info@IN8bio.com

Investor Contact:Julia Balanova+ 1 646.378.2936jbalanova@soleburytrout.com

Media Contact:Ryo Imai / Robert Flamm, Ph.D.Burns McClellan, Inc.212-213-0006 ext. 315 / 364Rimai@burnsmc.com / rflamm@burnsmc.com

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IN8bio announces first-in-human Phase 1 trial Update from The University of Kansas Cancer Center using INB-100, IN8bios Gamma Delta T-cell product...

Final Report will add the analysis of the impact of COVID-19 on this industry.

According to the latest industry research Bone Marrow Transplant Market share is predicted to gain better growth in upcoming years. Global Bone Marrow Transplant market report is one of the best sources of research data which provided by industry experts. Report gives facts about Covid-19 impact, geographical breakdown, top manufactures, type wise and applications wise segmentation. In Bone Marrow Transplant market report the growth rate, revenue, market shares, sales, production, consumption, manufacturers in particular areas are regionally analysed.

Experts also states challenges, risks, driving factors, trends, opportunities in Bone Marrow Transplant market so the investors, new participants, and stakeholders get good clarification with Bone Marrow Transplant market industry.

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Bone marrow transplant refers to the replacement of diseased or damaged bone marrow with healthy tissue or bone marrow stem cells in order to treat blood cancer or various cases of anemia. Depending on the source of bone marrow or stem cells, bone marrow transplant procedures are classified as peripheral stem cell transplant (PSCT) or conventional bone marrow transplant.

Bone Marrow Transplant Industry Segmentation:

Bone Marrow Transplant Market by Top Manufacturers:Lonza Group Ltd., Merck Millipore Corporation., Sanofi-Aventis LLC., AllCells LLC., STEMCELL Technologies., American Type Culture Collection (ATCC) Inc. By ProcedureAutologous Bone Marrow Transplant, Allogeneic Bone Marrow TransplantBy Disease IndicationLeukemia, Lymphoma, Myeloma, Myelodysplasia, Myeloproliferative Neoplasms, Aplastic Anemia, Solid tumors, Sickle cell Anemia, OthersBy End UserHospitals, Multispecialty Clinics, Ambulatory Surgical Centers

The study procedure elaborates the analysis of several features affecting the industry, with the government policy, Bone Marrow Transplant market forecast environment, technological innovation, competitive landscape, historical data, present trends in the market, forthcoming technologies and the technical progress in associated industry.

What the Bone Marrow Transplant Market Trend Report Offers:

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Bone Marrow Transplant market report is outcome of comprehensive primary and secondary research accepted by analysts having years of experience in the Bone Marrow Transplant industry. All the qualitative and quantitative aspects of the industry have been covered and the collected information has been examined and accessible in the form of easily understandable charts, graphs and tables.

In addition, report analyses Bone Marrow Transplant market size and forecast of product, region and application and different analysis essentials like type section, business section, channel segment etc. cover totally different segment market size, each volume and value. Moreover, cover different industries clients data, that is incredibly necessary for the manufacturers.

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Detailed TOC of 2019-2024 Global and Regional Bone Marrow Transplant Industry Production, Sales and Consumption Status and Prospects Professional Market Research Report

Chapter 1 Bone Marrow Transplant Industry Overview

1.1 Definition

1.2 Brief Introduction by Major Type

1.3 Brief Introduction by Major Application

1.4 Brief Introduction by Major Regions

1.4.1 United States

1.4.2 Europe

1.4.3 China

1.4.4 Japan

1.4.5 India

Chapter 2 Production Bone Marrow Transplant Market Analysis

2.1 Global Production Market Analysis

2.1.1 2012-2017 Global Capacity, Production, Capacity Utilization Rate, Ex-Factory Price, Revenue, Cost, Gross and Gross Margin Analysis

2.1.2 2012-2017 Major Manufacturers Performance and Market Share

2.2 Regional Production Market Analysis

2.2.1 2012-2017 Regional Market Performance and Market Share

2.2.2 United States Market

2.2.3 Europe Market

2.2.4 China Market

2.2.5 Japan Market

2.2.6 India Market

2.2.7 Rest Regions Market

Chapter 3 Bone Marrow Transplant Sales Market Analysis

3.1 Global Sales Market Analysis

3.1.1 2012-2017 Global Sales Volume, Sales Price and Sales Revenue Analysis

3.1.2 2012-2017 Major Manufacturers Performance and Market Share

3.2 Regional Sales Market Analysis

3.2.1 2012-2017 Regional Market Performance and Market Share

3.2.2 United States Market

3.2.3 Europe Market

3.2.4 China Market

3.2.5 Japan Market

3.2.6 India Market

3.2.7 Rest Regions Market

Chapter 4 Consumption Market Analysis

4.1 Global Consumption Market Analysis

4.1.1 2012-2017 Global Consumption Volume Analysis

4.2 Regional Consumption Market Analysis

4.2.1 2012-2017 Regional Market Performance and Market Share

4.2.2 United States Market

4.2.3 Europe Market

4.2.4 China Market

4.2.5 Japan Market

4.2.6 India Market

4.2.7 Rest Regions Market

Chapter 5 Production, Sales and Consumption Market Comparison Analysis

5.1 Global Production, Sales and Consumption Market Comparison Analysis

5.2 Regional Production, Sales Volume and Consumption Volume Market Comparison Analysis

5.2.1 United States

5.2.2 Europe

5.2.3 China

5.2.4 Japan

5.2.5 India

5.2.6 Rest Regions

Chapter 6 Major Manufacturers Production and Sales Market Comparison Analysis

6.1 Global Major Manufacturers Production and Sales Market Comparison Analysis

6.1.1 2012-2017 Global Major Manufacturers Production and Sales Market Comparison

6.2 Regional Major Manufacturers Production and Sales Market Comparison Analysis

6.2.1 United States

6.2.2 Europe

6.2.3 China

6.2.4 Japan

6.2.5 India

6.2.6 Rest Regions

Chapter 7 Major Type Analysis

7.1 2012-2017 Major Type Market Share

Chapter 8 Major Application Analysis

8.1 2012-2017 Major Application Market Share

Chapter 9 Bone Marrow Transplant Industry Chain Analysis

9.1 Up Stream Industries Analysis

9.1.1 Raw Material and Suppliers

9.1.2 Equipment and Suppliers

9.2 Manufacturing Analysis

9.2.1 Manufacturing Process

9.2.2 Manufacturing Cost Structure

9.2.3 Manufacturing Plants Distribution Analysis

9.3 Industry Chain Structure Analysis

Browse Complete TOC Here: https://www.industryresearch.co/TOC/13652362#TOC

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Bone Marrow Transplant Market Size, Segmented by Type of Deployment, Application, And Region Growth, Trends, And Forecast - The Haitian-Caribbean...

For the first time, researchers have demonstrated how the gut microbiome the community of microorganisms living in the gut can influence the immune system in humans. Their work could lead to new treatments for immune-related conditions.

The researchers at Memorial Sloan Kettering Cancer Center in New York, NY, tracked the recovery of patients gut microbiota and immune system after bone marrow transplants (BMTs) following treatment for blood cancers.

Healthcare professionals use chemotherapy and radiation therapy to destroy cancerous blood cells in conditions such as leukemia and lymphoma. After completion of the treatment, which also kills healthy immune cells, specialists inject patients with stem cells from a donors blood or bone marrow.

These donated cells slowly restore patients ability to make their own blood cells.

However, patients have to take antibiotics in the first few weeks after the transplant because they are still vulnerable to infections. These upset the balance of their gut microbiota, killing friendly bacteria and allowing dangerous strains to thrive.

Once patients immune systems are strong enough, they can stop taking the antibiotics, which allows their gut microbiota to recover.

The researchers at Sloan Kettering used this unique opportunity to study how the microbiota affects the immune system.

The scientific community had already accepted the idea that the gut microbiota was important for the health of the human immune system, but the data they used to make that assumption came from animal studies, explains systems biologist Joao Xavier, who is co-senior author of the paper with his former postdoc Jonas Schluter.

The parallel recoveries of the immune system and the microbiota, both of which are damaged and then restored, gives us a unique opportunity to analyze the associations between these two systems, says Dr. Schluter, who is now an assistant professor at NYU Langone Health in New York, NY.

Using blood and fecal samples from more than 2,000 patients treated at the cancer center between 20032019, the researchers were able to track daily changes in their gut microbiota and the number of immune cells in their blood.

Our study shows that we can learn a lot from stool biological samples that literally would be flushed down the toilet, says Dr. Xavier. The result of collecting them is that we have a unique dataset with thousands of data points that we can use to ask questions about the dynamics of this relationship.

The researchers used a machine-learning algorithm to identify patterns in the data, which included information about patients medications and the side effects they experienced.

One of the findings was that the presence of three types of gut bacteria called Faecalibacterium, Ruminococcus 2, and Akkermansia was associated with increased blood concentrations of immune cells called neutrophils.

By contrast, two types called Rothia and Clostridium sensu stricto 1, were associated with reduced numbers of these immune cells.

Computer simulations by the researchers predicted that enriching microbiota with the three friendly genera would speed up the recovery of patients immune systems.

This research could eventually suggest ways to make BMTs safer by more closely regulating the microbiota, says co-author Marcel van den Brink.

The study appears in Nature.

Concluding their paper, the authors write:

Our demonstration that the microbiota influences systemic immunity in humans opens the door toward an exploration of potential microbiota-targeted interventions to improve immunotherapy and treatments for immune-mediated and inflammatory diseases.

A previous study found that having a greater diversity of bacterial species in the gut is associated with a better chance of survival after a stem cell transplant. This research also found that a low diversity of bacteria increased the likelihood of potentially fatal graft-versus-host disease, when the donor immune cells attack the recipients tissues.

In 2018, the Sloan Kettering researchers published results from a clinical trial in which they used fecal transplants to restore patients microbiota after treatment for blood cancer.

They used the patients own fecal matter, which had been collected and frozen before the bone marrow transplant and antibiotic treatment disrupted their gut microbiota.

Original post:
Gut bacteria can help rebuild the immune system - Medical News Today

Vancouver, British Columbia--(Newsfile Corp. - December 1, 2020) - Hemostemix Inc. (TSXV: HEM) (OTCBB: HMTXF), a clinical stage biotechnology company with a patented stem cell technology platform, has successfully, following lengthy litigation, obtained its entire clinical trial database from Medrio Inc., which was hosting the database for Aspire Heath Science LLC. For more details on the litigation, please refer to today's news release.

For more information, please view the InvestmentPitch Media "video" which provides additional information about this news and the company. If this link is not enabled, please visit http://www.InvestmentPitch.com and enter "Hemostemix" in the search box.

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The company's principal business is to develop, manufacture and commercialize blood-derived stem cell therapies to treat various diseases not adequately addressed by current therapeutics. The company's process for harvesting stem cells is less invasive, as the stem cells are taken from a patient's blood, which is a simplified process as compared to taking stem cells from fatty tissue or bone marrow.

Critical limb ischemia or CLI, a severe blockage in the arteries of the lower extremities, which markedly reduces blood-flow, is deadliest form of peripheral arterial disease or PAD, with limited treatment options and no current approved drug treatments. The company's lead product, ACP-01 is the subject of a Phase II clinical trial of its safety and efficacy in patients with advanced CLI, who have exhausted all other options to save their limb from amputation.

Hemostemix owns 91 patents related to its products and manufacturing processes and has entered into a contract with a new clinical research organization which is completing the midpoint statistical analyses of the efficacy of ACP-01. A winner of the World Economic Forum Technology Pioneer Award, Hemostemix developed and is commercializing its lead product ACP-01.

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Hemostemix announced the abstract and interim results presented to the 41st annual Canadian Society for Vascular Surgery meeting, which noted healing of ulcers and resolution of ischemic rest pain occurred in 83 per cent of patients studied by lead investigators at the University of British Columbia and the University of Toronto with outcomes maintained for up to 4.5 years.

Thomas Smeenk, CEO, stated: "As every biotech investor knows, it is all about the data! Blinded, we will know in short order if our HS 12-01 midpoint results equal the interim clinical trial results that 83 per cent of patients followed for up to 4.5 years experienced. Fortunately, we have a lot of data of the efficacy of ACP. For example, we have the clinical trial results of the 41 patients treated for cardiomyopathy. And, we have the results of the 106 ischemic heart disease patients on maximal medical therapy who had no option for revascularization, who experienced significant improvement."

Hemostemix is addressing a huge potential market. According to The Sage Group LLC, in the United States alone, approximately 20 million people are affected by PAD, and it is estimated that approximately 7 to 8 million people in the United States and Europe suffer from CLI. The Sage Group estimates that in the United States, medical costs attributable to CLI amount to US$25 billion annually.

The company has also announced a non-brokered private placement. The company is looking to raise gross proceeds of up to $2.5 million from the placement of up to 250 million units priced at $0.01 per unit. The units consist of 1 share and 1 warrant, with each warrant exercisable at $0.05 for 12 months, subject to an acceleration clause. The shares are currently trading at $0.01. However, the company has obtained shareholder approval for a 20 for one share consolidation.

In addition to accredited investors, this placement is available under certain security exemptions to existing shareholders, friends and family, and those investors having received advice under the investment dealer exemption.

For more information, please visit the company's website at http://www.hemostemix.com, contact Thomas Smeenk, President, CEO and Co-Founder at 905-580-4170 or by email at TSmeenk@hemostemix.com.

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InvestmentPitch Media Video Discusses Hemostemix Successfully Obtaining all Clinical Trial Data and Announcement of $2.5 Million Unit Offering - Video...

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

The global multiple myeloma drugs market grew at a CAGR of around 9% during 2014-2019. Looking forward, the publisher expects the market to witness moderate growth during the next five years.

Multiple myeloma, or Kahler's disease, refers to a form of blood cancer that primarily affects the plasma cells. Some of the most common types of multiple myeloma drugs include chemotherapeutic agents, corticosteroids and immunomodulatory agents. These pharmaceutical drugs aid in promoting bone healing, prevent hypercalcemia, bone fracture, spinal cord compression and anemia, while minimizing the need for chemotherapy. The chemotherapeutic agents include various anthracycline antibiotics and alkylating agents, such as melphalan, doxorubicin, vincristine and liposomal doxorubicin. The targeted therapy drugs include proteasome inhibitor, such as bortezomib, and various other compounds, including dexamethasone, prednisone and thalidomide.

Significant developments in the healthcare sector, along with the increasing prevalence of hematological cancer, is one of the key factors driving the growth of the market. Multiple myeloma is usually caused by specific genetic abnormalities, and the treatment of this disease involves drugs that modulate the immune system and aid in enhancing the efficiency of chemotherapies, radiation therapies, stem cell transplants and platelet transfusion.

Furthermore, rising consumer awareness regarding the benefits of biologic therapy drugs, which utilize the body's immune system to identify and attack the myeloma cells, is also providing a boost to the market growth. Additionally, various technological advancements, such as the development of microRNA therapeutics and nanomedicines for the treatment of multiple myeloma, is acting as another growth-inducing factor. These medicines are used to facilitate the delivery of macromolecular agents into the bone marrow and catalyze antitumor responses. Other factors, including the rising healthcare expenditures and extensive research and development (R&D) activities in the field of medical sciences, are projected to drive the market further.

Companies Mentioned

Key Questions Answered in This Report:

Key Topics Covered:

1 Preface

2 Scope and Methodology

2.1 Objectives of the Study

2.2 Stakeholders

2.3 Data Sources

2.3.1 Primary Sources

2.3.2 Secondary Sources

2.4 Market Estimation

2.4.1 Bottom-Up Approach

2.4.2 Top-Down Approach

2.5 Forecasting Methodology

3 Executive Summary

4 Introduction

4.1 Overview

4.2 Key Industry Trends

5 Global Multiple Myeloma Drugs Market

5.1 Market Overview

5.2 Market Performance

5.3 Market Forecast

6 Market Breakup by Therapy

6.1 Targeted Therapy

6.1.1 Market Trends

6.1.2 Market Forecast

6.2 Biologic Therapy

6.2.1 Market Trends

6.2.2 Market Forecast

6.3 Chemotherapy

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 Drug Type

7.1 Immunomodulatory Drugs

7.1.1 Market Trends

7.1.2 Market Forecast

7.2 Proteasome Inhibitors

7.2.1 Market Trends

7.2.2 Market Forecast

7.3 Histone Deacetylase Inhibitors

7.3.1 Market Trends

7.3.2 Market Forecast

7.4 Monoclonal Antibody Drugs

7.4.1 Market Trends

7.4.2 Market Forecast

7.5 Steroids

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 Men

8.1.1 Market Trends

8.1.2 Market Forecast

8.2 Women

8.2.1 Market Trends

8.2.2 Market Forecast

9 Market Breakup by Distribution Channel

9.1 Hospital Pharmacies

9.1.1 Market Trends

9.1.2 Market Forecast

9.2 Retail Pharmacies

9.2.1 Market Trends

9.2.2 Market Forecast

9.3 Online Pharmacies

9.3.1 Market Trends

9.3.2 Market Forecast

9.4 Others

9.4.1 Market Trends

9.4.2 Market Forecast

10 Market Breakup by Region

10.1 North America

10.2 Asia Pacific

10.3 Europe

10.4 Latin America

10.5 Middle East and Africa

11 SWOT Analysis

12 Value Chain Analysis

13 Porters Five Forces Analysis

14 Price Indicators

15 Competitive Landscape

15.1 Market Structure

15.2 Key Players

15.3 Profiles of Key Players

For more information about this report visit https://www.researchandmarkets.com/r/8yriem

Excerpt from:
Outlook on the Multiple Myeloma Drugs Global Market to 2025 - by Therapy, Drug Type, End-user, Distribution Channel and Region -...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Imago BioSciences, Inc., (Imago) a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced the expansion of its global Phase 2b clinical study evaluating bomedemstat (IMG-7289) for the treatment of advanced myelofibrosis (MF) into Hong Kong, where the first patient has now been enrolled and dosed at the Department of Medicine, Queen Mary Hospital and the University of Hong Kong. Myelofibrosis is a rare bone marrow cancer that interferes with the production of blood cells.

In addition to Hong Kong, the Phase 2b study continues to actively enroll patients in the U.S., U.K., and E.U. The study is in the final stages of completing enrollment and continues to dose patients to evaluate safety, tolerability and efficacy.

Patients with myelofibrosis around the world are still in need of new treatment options, said Hugh Young Rienhoff, Jr. M.D., Chief Executive Officer, Imago BioSciences. We are progressing well with enrollment and are pleased to continue expanding our global Phase 2 study into new geographies like Hong Kong. We are encouraged by the signs of clinical activity and safety of bomedemstat as a treatment alternative for patients who do not benefit from the current standards of care.

Bomedemstat is an inhibitor of lysine-specific demethylase 1 (LSD1), an epigenetic regulator critical for self-renewal of malignant myeloid cells and the differentiation of myeloid progenitors. Data presented at the 25th European Hematology Association (EHA) Annual Congress in June demonstrated that the first-in-class LSD1 inhibitor was well tolerated with no dose-limiting toxicities or safety signals. Furthermore, recent data demonstrates the potential of bomedemstat as a monotherapy in intermediate-2 and high-risk patients with myelofibrosis who have become intolerant of, resistant to or are ineligible for a Janus Kinase (JAK) inhibitor.

Bomedemstat was recently granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA) for the treatment of MF. The EMA reviewed bomedemstat non-clinical and clinical data from the ongoing Phase 2 study. The PRIME initiative was launched by the EMA in 2016 to provide proactive and enhanced support to the developers of promising medicines with the view of accelerating their evaluation to reach patients faster.

About Bomedemstat (IMG-7289)

Bomedemstat is an orally available small molecule discovered and developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, bomedemstat demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other therapeutic agents. Bomedemstat is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185, NCT04262141, NCT04254978 and NCT04081220).

Bomedemstat has U.S. FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, Orphan Drug Designation for treatment of acute myeloid leukemia and PRIME designation by the European Medicines Agency for the treatment of MF.

Bomedemstat is being evaluated in two open-label Phase 2 clinical trials for the treatment of advanced myelofibrosis (MF) and essential thrombocythemia (ET), bone marrow cancers that interfere with the production of blood cells. MF patients who are resistant to a Janus Kinase (JAK) inhibitor are eligible for the study of bomedemstat. ET patients who have failed one standard of care treatment are eligible for the bomedemstat ET study.

About Imago BioSciences

Imago BioSciences is a clinical-stage biopharmaceutical company focused on discovering and developing novel therapeutics for the treatment of hematologic disorders targeting epigenetic enzymes. Imago has developed a series of compounds that inhibit LSD1, an epigenetic enzyme critical for cancer stem cell function and blood cell differentiation. Imago is advancing the clinical development of its first LSD1 inhibitor, bomedemstat, for the treatment of myeloid neoplasms. Imago BioSciences is backed by leading private, corporate, and public investors including Farallon Capital Management, LLC., funds and accounts advised by T. Rowe Price Associates, Inc., funds and accounts managed by Blackrock Advisors, LLC., Surveyor Capital (a Citadel company), Irving Investors, Kingdon Capital Management, a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital. The company is based in South San Francisco, California. To learn more, visit http://www.imagobio.com, http://www.myelofibrosisclinicalstudy.com, http://www.etclinicalstudy.com and follow us on Twitter @ImagoBioRx, Facebook and LinkedIn.

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Imago BioSciences Expands Phase 2 Clinical Trial of Bomedemstat (IMG-7289) for the Treatment of Myelofibrosis into Hong Kong - Business Wire

The mRNA technology used to create the Pfizer and Moderna vaccines for COVID is being applied to ... [+] many other medical treatments in addition to vaccines.

While the vaccines for Covid-19 seem to have been created in record time, the technology making them possible has been decades in development. The two vaccine candidates produced by Pfizer/BioNTech and Moderna are unlike any other vaccine thats come before. Should they achieve commercial success, it could usher in a new era of medical science not just for vaccines, but for cancer treatments, blood disorders, and gene therapy.

The two new vaccines are the first ever to use mRNA, which stands for messenger RNA, to generate immunity. Historically, vaccines have used dead or weakened viruses to imitate an infection, spurring the body to make antibodies against that virus without danger of getting sick. Measles, polio, and some seasonal flu shots are examples of vaccines made with whole virus particles.

Other vaccines use only certain fragments of the virus, called antigens, that provoke an immune response. To make this type of vaccine, the genetic code for the desired viral antigen molecule is put into yeast or bacteria cells. These microbes can be grown rapidly and inexpensively, and they can churn out massive quantities of antigen. Then the molecule must be purified to clinical standards so that its safe to inject into healthy people. Prevnar and Gardasil are examples of this type of vaccine.

These methods work well, but they require enormous research and development efforts. A laboratory could spend years optimizing the methods for producing one virus protein, but those methods wouldnt automatically translate to mass-producing a different protein.

For every new protein, you start over. Its a brand-new procedure every step of the way, explains immunologist Drew Weissman of the Perelman School of Medicine at the University of Pennsylvania. Weissman is one of the pioneering scientists behind the mRNA vaccine.

The way I see it, the mRNA platform is much better, its much quicker, and its cheaper, says Weissman. Thats the trilogy of what you need to improve vaccines. With mRNA, the steps are the same, no matter what virus the vaccine is targeting. This makes it easily customizable. Once an mRNA manufacturing facility is up and running, it can easily be deployed to make vaccines against any number of viral antigens.

A strand of mRNA carries the instructions for making one protein. Your cells normally make their own mRNA strands and use them as blueprints to manufacture all the proteins your body needs to function.

The vaccine slips a new strand of mRNA into the cell, like an extra page in the blueprint. This mRNA contains the instructions for making the coronavirus spike protein, and the cell reads it the same way it reads its own mRNAs, using it to build the viral protein. The immune system recognizes that protein as foreign, and starts making antibodies against it. Then, if youre exposed to the actual virus, those antibodies will be available to stop the infection. Astonishingly, in animal tests, mRNA vaccines appear to induce immunity that lasts much longer than live virus vaccines.

The beauty of mRNA is that its temporary. Your cells wont keep cranking out spike protein forever. Like an Instagram story, the mRNA fades away after a certain amount of time, because you dont need to keep making coronavirus protein forever in order to maintain the protective immunity.

Another big advantage of mRNA is that its rapidly customizable. Once scientists know the genetic sequence of a viral protein, they can make the mRNA in the lab and package it into a vaccine in a matter of weeks.

Originally envisioned as a way to deliver gene therapy, mRNA had to overcome some serious challenges before arriving at todays big moment. In 2005, Weissman and his colleague, Katalin Karik, solved one of the most difficult problems facing mRNA. In its natural form, the molecule sparks an excessive immune reaction, igniting inflammation that damages the body. To avoid this, they changed the structure of the mRNA just enough to fool the immune sentries.

Similar to DNA, RNA is made up of a series of chemical letters, a kind of code that the cell translates to make a protein. Modifying the chemical structure of one of those letters allowed the information to remain intact, and eliminated the signal that triggered the bodys immune alarms.

Before the coronavirus pandemic hit, Weissmans lab was working on vaccines for influenza, herpes, and HIV. Those will all be going into phase I clinical trials within the next year, he says. But vaccines are only the beginning of what mRNA can do.

Often in the case of genetic diseases, the problem is that a broken gene fails to produce a protein that the body needs for healthy function. The idea of gene therapy is simple: send in a healthy copy of the broken gene, which the cells can use to make the protein. Most times, researchers use viruses to deliver the gene, but viruses can cause problems of their own. Delivering mRNA to the cell without a virus circumvents some of these issues.

To ferry the mRNA into cells, it is encapsulated in a fatty coating called a lipid nanoparticle (LNP). Weissmans lab has been experimenting with ways to modify the LNP so that it can home in on certain cell types.

In sickle cell disease (SCD), a broken hemoglobin gene prevents blood cells from carrying oxygen ... [+] efficiently, and causes them to take on a rigid, sickle-shaped form.

My lab has figured out how to specifically deliver the LNP to bone marrow stem cells, Weissman says. This could lead to an inexpensive and practical cure for sickle cell anemia. An mRNA molecule can be programmed to encode the beta-hemoglobin gene, which is defective in sickle cell disease. That mRNA would be sent directly to the bone marrow cells using the specially targeted LNPs, enabling the bone marrow to produce healthy red blood cells that contain functioning beta-hemoglobin.

All that would need to be done is to give people a single intravenous injection of the mRNA LNP, and youll cure their sickle cell anemia, Weissman says. By contrast, the current FDA-approved gene-editing therapy for sickle cell requires the patients bone marrow be removed, treated, and then returned to the bodyan expensive and invasive procedure. The mRNA treatment could be simple enough to deliver in lower-income countries, where sickle cell disease impacts the health of millions of people.

An up-and-coming strategy for fighting cancer is a so-called cancer vaccine, which uses immune cells called dendritic cells (DCs). DCs perform surveillance for the immune system. When they detect something that shouldnt be there, whether its a virus, a bacteria, or even a cancer cell, the DCs chew it up, break it into its component molecules, and then show those foreign molecules to the immune cells that make antibodies.

Dendritic cells chew up viruses or other foreign bodies, and present the pieces to other immune ... [+] cells. T cells and B cells both play a role in mounting a long-lasting immunity against the pathogen.

When cancer grows slowly, though, it can slip past the DC surveillance network. To give the immune system a boost, a patients DCs are taken out and artificially loaded with tumor-specific proteins, or antigens. Back inside the body, the cells stimulate the generation of antibodies against the tumor.

Using mRNA to deliver the tumor antigen information to the DCs could provide a way to make this process easier, cheaper, and safer. BioNTech is currently conducting clinical trials on cancer vaccines for triple-negative breast cancer, metastatic melanoma, and HPV-positive head and neck cancers. Called FixVac, the vaccines include multiple tumor antigens that are frequently found across different patients. Early data published in September 2020 showed promise, suggesting that the mRNA therapy generates a lasting immune response, comparable to more expensive methods.

Karik, who is now a senior vice president at BioNTech, and Weissman both speak with an air of inevitability, as if they have only been waiting patiently for the world to catch up with their discovery. The two scientists told their stories recently at the 2nd annual mRNA Day celebration in San Diego, hosted by Trilink BioTechnologies in honor of their recently opened facility there. After hearing the tumultuous history of the technology and seeing promising new data, one attendee asked, what would you say was the turning point for mRNA therapeutics?

Karik responded simply, When people read our [2005] paper. We were waiting for somebody to respond, we did a lot of experiments, but we waited and waited. It was just too early for most people.

Weissman agreed. I think we were early, he said. It finally caught on, and it will hopefully change the world.

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If youre a woman in the menopause transition, hot flashes and night sweats arent the only things that should grab your attention. It turns out that the stage of fluctuating hormones leading to your final period is a risky time for your heart health.

Thats the consensus of nearly a dozen experts who reviewed the research on the topic for the American Heart Association. They published theirscientific statement on November 30 in the journal Circulation.

RELATED: 10 Ways to Beat Menopausal Belly Fat

This phase of a womans life is a time of accelerated cardiovascular risk at multiple dimensions, including changes in lipids, body fat, metabolic syndrome, and vascular health, saysSamar R. El Khoudary, PhD, MPH, an associate professor of epidemiology at the University of Pittsburgh and the chair of this AHA statement writing committee.

These changes significantly raise the potential for heart disease later in life, and this is why women and their doctors must see themenopause transitionas a crucial time for intervention, rather than wait until women are older and experiencing disease, Dr. El Khoudary says.

The AHA committee undertook their thorough review because many more studies on women going through the menopause transition are available now than previously, especially since the last AHA guidelines on treating women were written, in 2011.

Some of the best studies have followed women during their midlife years. This includes the Study of Womens Health Across the Nation (SWAN), the Melbourne Womens Midlife Health Project, and the Penn Ovarian Aging Study (POAS).

RELATED: 5 Things Weve Learned From the Most Important Menopause Study

Other, especially older, studies have tended to group women into before menopause and after, leaping over the important phase that occurs as they shift from one time of life to the other, El Khoudary says.

Still, even more research is needed, she says, especially studies testing specific cardiovascular interventions, such as medications, during this stage.

Understanding these processes is so important, because heart disease is the leading cause of death for women. If you didnt know that, youre not alone. Only 56 percent of women are aware of this fact, El Khoudary observes.

Cardiologists dont sufficiently focus on this, either. While most know that a womans risk of heart disease increases once the protective effects of estrogen cease after menopause, many dont intervene soon enough to make a difference, she says.

More cardiologists are becoming aware of this important window. But we are behind because of this notion that women dont get cardiovascular disease until later in life, and that they have different symptoms than men, says Ruwanthi Titano, MD, an assistant professor of cardiology at the Icahn School of Medicine at Mount Sinai in New York, who was not involved with the review.

Doctors need to view the menopause transition as a risk factor akin to high cholesterol and hypertension, Dr. Titano says.

RELATED: Coping With Hot Flashes and Other Menopausal Symptoms: What 9 Celebrities Said

A woman is considered post-menopausal only after she has gone a full year without a single period. During the transition period, known as perimenopause, a woman can go months without a menstrual bleed, but then menstruate, resetting the clock.

During this time, hormones fluctuate greatly. One of the main hormones is estrogen. It is this hormone that is thought to provide the key heart protection women experience earlier in life.

Once a woman becomes post-menopausal, her estrogen level remains low.

RELATED: What Are Irregular Periods?

And that's by no means the only change that happens in the body during the menopause transition. They cumulatively raise the potential for heart troubles down the road, El Khoudary says. Its not like a button you press that quickly increases your mortality. But, accumulated together, the changes women experience increase her risks for cardiovascular disease.

During this period, blood cholesterol levels start to rise. The carotid artery begins to thicken (a subclinical measure of atherosclerosis, El Khourdary says). Excess weight moves from being stored around the thighs and buttocks to around the abdominal organs and the heart more dangerous locations. And metabolic syndrome symptoms increase, the committee found.

All these factors can set the stage for later heart attacks or strokes.

RELATED:What Experts Want BIPOC Women Know About Menopause

Even once-healthy HDL cholesterol levels seem to take a destructive turn.

HDL, or good cholesterol, is considered to be cardioprotective. But once a woman transitions to menopause, it actually seems to become bad for you, El Khoudary says, citing her own soon-to-be published research.

This has led to the hypothesis that HDL experiences some dysfunctionality as women transition through menopause, causing it to stop functioning the way it used to, she says.

Doctors erroneously think of menopause as a point of demarcation, El Khoudary says. One day youre menstruating, then youre not. But menopause is actually a lengthy and varied process, with hormonal fluctuations sometimes lasting years.

Its connection to heart disease is especially complex. Age of menopause, the stage of menopause youre in, and even your race may play a role, the paper states. For instance, women who experience menopause at an earlier age, typically before 45, have a higher risk for later heart disease.

Socioeconomic indicators, prior cardiovascular health, and other factors are associated with a younger transition. Black, Hispanic, and Native Hawaiian women tend to experience the menopause transition at a younger age.

Women whose ovaries are surgically removed when theyre younger are also more likely to face increased cardiovascular problems, the paper states.

So, too, are women who suffer severe vasomotor symptoms, including hot flashes and night sweats.

The analysis reveals the importance of monitoring womens health during midlife and using the menopausal transition as a critical window for intervention.

Women and their doctors need to understand how the body changes during this time, and the ways that can boost your heart disease risk, El Khoudary says.

Your doctor might prescribe medications to keep blood pressure, cholesterol, and blood sugar in check, critical parts of the AHAs Lifes Simple 7 heart campaign.

My message for perimenopausal women is to engage in your healthcare. Get your yearly checkup with your physician or see your cardiologist if you have one. Your doctor should be looking more closely at your lipid panel, because maybe numbers that were acceptable before arent good enough anymore, Titano says.

RELATED:Fitness After 40 and Beyond: What to Know About Midlife Exercise Needs

Sufficient physical exercise is key. El Khoudary was stunned to learn that just 7.2 percent of women past menopause are meeting physical activity guidelines.

RELATED:7 Fun Ways to Move More in Midlife

Similarly, adopting a healthy eating plan, one that becomes part of your life rather than a short-term fad, can protect your heart. But post-menopausal women dont seem to be doing this either, with only 20 percent saying they consistently maintain a healthy diet.

Other important actions to protect your heart include getting sufficient sleep, adopting stress-reduction techniques, and quitting smoking if you havent already.

RELATED: Having Multiple Severe Menopause Symptoms Is Linked to Increased Risk for Heart Disease

While it might seem that properly dosed and timed menopausal hormone therapy (MHT) could lower womens heart disease risk, this has not been proved in research.

The last major study of hormone therapy, the Womens Health Initiative, was stopped in 2002 when participants were found to have a small increase in heart disease, blood clots, cancer, and stroke. But these women were older, generally many years past their menopause transition.

We dont know what would happen in terms of heart disease if you gave hormone therapy to women during the transition, El Khourdary says. We dont have the clinical trials for women in their forties or fifties.

Still, some smaller studies, many of them observational ones, indicate that newer formulations of hormone therapy, especially those taken via skin patches or sprays rather than pills, may offer some heart protection, she says.

RELATED: Timing and Type of Hormone Therapy Impacts Its Effectiveness in Preventing Heart Disease

The Circulation paper notes the promise, but also indicates that the AHA may not be ready to recommend that women take hormone therapy for their heart.

The literature supporting a critical role for the time of initiation of MHT use relative to menopause, with initiation at <60 years of age or within 10 years of menopause appearing to be associated with reduced CVD risk, strongly calls for further research assessing MHT use, including potential contrasts by form, route, and duration of administration, on cardiometabolic effects in women traversing menopause, the paper states.

RELATED: Hormone Therapy May Prevent UTIs in Post-Menopausal Women

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The Menopause Transition Poses Heart Disease Risk - Everyday Health

(UroToday.com) At the second prostate cancer session at the 2020 Annual Meeting of the Society of Urologic Oncology (SUO), Dr. Maha Hussain presented the results of the PROfound study, Study of Olaparib (Lynparza) Versus Enzalutamide or Abiraterone Acetate in Men With Metastatic Castration-Resistant Prostate Cancer (PROfound Study), which demonstrated the superiority of olaparib, a poly (adenosine diphosphate-ribose) polymerase inhibitor, for treatment of metastatic castrate-resistant prostate cancer (mCRPC).

After a brief discussion of the ToPARP trial and some of the rationale which lead to the genesis of the PROfound study, Dr. Hussain launched into a discussion of the trial itself. In this trial, published in the New England Journal of Medicine in May 20201 with an update in the same journal in September2, patients with the following characteristics were enrolled:

There were no restrictions based on ECOG status, volume or location of metastases, or prior taxane therapy.

Patients with mutations in BRCA1, BRCA2, ATM were called and cohort A and were the primary population for the analysis.

These patients were randomized to olaparib 300mg twice-daily vs the physician's choice of abiraterone or enzalutamide. The primary endpoint was imaging-based progression-free survival in cohort A based on blinded central review.

Overall the results were resoundingly positive, with olaparib showing superiority in progression-free survival (7.4 mo vs 3.6 mo, HR 0.34 0.25-0.47, P<0.001), the median time to pain progression (HR 0.44 0.22-0.91, P <0.02), andoverall survival (19.1 months vs 14.7 mo, HR 0.69 9.50-0.97 P =0.02)in cohort A. This overall survival difference is particularly impressive given an 81% cross over from the control to the olaparib arm after progression in these patients.

Results in the full population were similar, as were results from numerous prespecified subgroup analyses, including patients with and without prior taxane use, bone only and visceral metastases, and baseline ECOG score of 0 and 1.

This data lead to the United States Federal Drug Administration (FDA) to approve olaparib for patients with mCRCP with deleterious or suspected deleterious germline or somatic homologous recombination repair mutations who have progressed on enzalutamide or abiraterone. This recommendation was similarly adopted in theNational Comprehensive Cancer Network (NCCN) guidelines.

(Similar approval was given to rucaparib based on the TRITON2 study, though this was limited to tumors with deleterious BRCA mutations and men previously treated with taxane chemotherapy.)

As Dr. Daniel Spratt argued in his counterpoint, however, this approval may be overly broad.

When analyzing patients by which genes were altered, the 95% CI of patients with BRCA2 mutations was the only one not to cross 1 (HR 0.21 0.12-0.32) for progression-free survival despite there being similar numbers of patients with ATM and CDK12 mutations. The signal of benefit was generally much stronger in BRCA mutations for both OS and PFS, with very little signal of benefit in the remaining patients, so it may be wise to limit clinical use to patients with these mutations.

Dr. Spratt also pointed out that there now exists a large body of evidence suggesting there is very little benefit in using abiraterone after progression on enzalutamide (or similar) and vice versa. Thus, for the 44.7% (173/387) patients who received docetaxel only, and certainly for the 34% who had received no prior chemotherapy (133/387), olaparib cannot be said to have been compared to the current standard of care in this trial.

Thus even for patients with BRCA mutations, which seem to confer the most favorable responses to olaparib, it remains very much unclear whether olaparib should be chosen before exhausting the numerous alternative available therapies.

Presented by: Maha H.A. Hussain, MD, FACP, FASCO, Genevieve Teuton Professor of Medicine, Division of Hematology-Oncology, Department of Medicine, Northwestern University Feinberg School of Medicine

Daniel Spratt, MD, Professor, Laurie Snow Research Professor of Radiation Oncology, University of Michigan

Written by: Marshall Strother, MD, Society for Urologic Oncology Fellow, Division of Urologic Oncology, Fox Chase Cancer Center, Philadelphia PA, @mcstroth duringthe 2020 Society of Urologic Oncology Annual Meeting December 2-5, 2020 Washington, DC

References:

1. de Bono J, Mateo J, Fizazi K, Saad F, Shore N, Sandhu S, Chi KN, Sartor O, Agarwal N, Olmos D, Thiery-Vuillemin A, Twardowski P, Mehra N, Goessl C, Kang J, Burgents J, Wu W, Kohlmann A, Adelman CA, Hussain M. Olaparib for Metastatic Castration-Resistant Prostate Cancer. N Engl J Med. 2020 May 28;382(22):2091-2102. doi: 10.1056/NEJMoa1911440. Epub 2020 Apr 28. PMID: 32343890.

2. Hussain M, Mateo J, Fizazi K, et al. Survival with olaparib in metastatic castration-resistant prostate cancer. N Engl J Med. Published online September 20, 2020:NEJMoa2022485.

Related ContentPatients with Metastatic Castration-Resistant Prostate Cancer (mCRPC) Can Be Preselected to Maximize Benefit of Olaparib - Maha Hussain

Continued here:
SUO 2020: New Second Line Therapy for mCRPC: PARPi: Results from the PROfound Study - UroToday

Since 1901, Nobel Prizes have honored the worlds best and brightest and showcased the work of brilliant and creative minds, thanks to Swedish businessman Alfred Nobel, who made his fortune with the invention of dynamite.

The Prize in Physiology or Medicine often honors those whose discoveries led to medical breakthroughs, new drug treatments, or a better understanding of the human body that benefit us all.

The Prize in Literature celebrates those skilled in telling stories, creating poetry, and translating the human experience into words. The Prizes in Chemistry and Physics remind most of us how little we understand of genetics, atomic structures, or the universe around us, celebrating the scientists who further knowledge. A later addition to the award roster, the Nobel Memorial Prize in Economic Sciences is not an original Prize, but was established by the Central Bank of Sweden in 1968 as a memorial to Alfred Nobel. It applauds those who can unravel the mysteries of markets, trade, and money.

The Peace Prize celebrates, in Nobels words, the person who shall have done the most or the best work for fraternity between nations, the abolition or reduction of standing armies and for the holding and promotion of peace congresses, sometimes risking their lives to do so.

So precious are the awards that the medals of German physicists Max von Laue and James Franck, stored away for safekeeping in Copenhagen during World War II, were dissolved in acid to keep them away from approaching Nazi troops. After the war, the gold was reconstituted from the acid and recast into new medals.

But Nobel history has not been entirely noble. In 1939, British Prime Minister Neville Chamberlain, known for his policy of appeasement toward Nazi Germany, was nominated for the Peace Prize. In an act of irony and protest, members of the Swedish Parliament nominated Adolf Hitler. That nomination was withdrawn. Some recipients have ordered oppressive crackdowns on their own people or ignored genocides, either before or after receiving the Prize. The 1918 Nobel Prize in Chemistry was given to Germanys Fritz Haber, who invented a method of producing ammonia on a large scale, which was helpful in making fertilizer. But the same chemist helped develop the chlorine gas that was used as a chemical weapon in World War I.

Stacker looked at facts and events related to the Nobel Prizes each year from 1931 to 2020, drawing from the Nobel Committees recollections and announcements, news stories, and historical accounts.

Take a look, and see what was happening with the Nobel Prizes the year you were born.

You may also like: 100 years of military history

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Nobel Prize history from the year you were born - Albany Democrat Herald

Polycystic ovary syndrome (PCOS) is a medical condition that affects an estimated 8 to 13 percent of women who are of reproductive age.

PCOS can cause:

While these dont happen to every woman with PCOS, there are changes from a lifestyle perspective that can help reduce the likelihood these effects will occur.

This article will focus on some of the changes you can implement today, such as diet and exercise routines. As always, if you have specific questions, talk to your doctor who helps you manage your PCOS.

Women with PCOS experience higher rates of insulin resistance compared to women who dont have the condition. Insulin resistance affects your bodys ability to use blood sugar for energy.

Doctors have connected a lack of physical activity and excess body weight as potential contributing factors to insulin resistance, according to the National Institute of Diabetes and Digestive and Kidney Diseases.

Not all women with PCOS have overweight. The good news is physical exercise is something you can do for your health when you have PCOS, regardless of your weight.

A meta-analysis of 16 studies related to PCOS and exercise found that vigorous intensity aerobic exercise was the most likely to reduce body mass index (BMI) and insulin resistance in women with PCOS, according to an article in the journal Frontiers in Physiology.

The researchers compared moderate exercise to vigorous exercise. They also found that vigorous exercise and healthy diet interventions resulted in the greatest decreases in BMI.

A research review of lifestyle interventions in PCOS published in the journal Best Practice & Research Clinical Obstetrics & Gynaecology found that exercise helped to reduce weight, abdominal fat, and fasting insulin levels.

The review also found exercise could help women of all weight levels with PCOS either lose or maintain their weight to help them look and feel healthier.

A literature review of studies published in the journal Sports Medicine regarding types of exercises, such as strength training and aerobic activity, did not find one specific exercise type was the most beneficial to women with PCOS.

Some of the studies reviewed aerobic exercise and resistance training, riding a stationary bicycle versus riding a bicycle outside, and treadmill walking or jogging at a moderate intensity versus vigorous intensity. The authors did find there are many exercise types that could benefit women with PCOS.

The message from these and other studies is that exercise can usually help you when you have PCOS, and the best exercise is what you will do regularly. Bonus points if the exercise can be something you enjoy doing.

Here are some exercise types to consider:

These are just some examples of exercises you can do with minimal equipment and space.

Researchers have completed several studies regarding the best diet types for those with PCOS to follow. The Androgen Excess and PCOS Society used this research to make recommendations for women, which include:

If you arent sure where to start with incorporating these changes to your diet, talk to your doctor. Your doctor also may recommend seeing a dietitian to create an eating plan specific to women with PCOS.

Exercise to improve your PCOS doesnt have to take hours a week. Studies have found exercise sessions ranging from 30 minutes a day, three times a week, to three total hours per week improved metabolic and reproductive symptoms associated with PCOS.

The Androgen Excess and PCOS Society recommends getting at least 30 minutes of moderate to vigorous physical activity a day and increasing your activity effort when you can. Some of the ways you can incorporate exercise in your life include the following:

If boredom is a factor in your commitment to an exercise routine, utilize a combination of these exercise types, such as completing a different exercise session type three times a week.

Try to incorporate the following habits for healthy eating with each meal and snack:

Using these guidelines, you can often stay within recommended daily calorie requirements given your overall height, health level, and weight.

When you have PCOS, its always a good idea to talk to your doctor about the lifestyle changes you can make to improve your health. When it comes to exercise for your PCOS, its especially important you talk to your doctor if you have other medical conditions that could affect your ability to exercise. Examples include arthritis or heart-related conditions.

If you have been sedentary for some time or dont have a solid foundation in exercise safety or proper form, it may be a good idea to consult a personal trainer. Your personal trainer should have a fitness certification from an accredited organization. Examples include:

Your trainer should be experienced and emphasize safety.

Exercise can be an important part of your PCOS management. Not only does it improve your physical health, exercise can help you manage your stress levels.

If you arent sure where to start, seeing your doctor and finding a personal trainer can help set you on a safe pathway. Exercising at least three times a week and sticking with it can help you improve symptoms from PCOS.

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Exercise For PCOS: Sample Plan, Types of Exercise, and More - Healthline

SGF proudly welcomes Dr. Jennifer Hsu to its Woodbridge practice.

FAIRFAX, Va. (PRWEB) December 03, 2020

Shady Grove Fertility (SGF) announced today that reproductive endocrinologist, Jennifer Hsu, M.D., has joined the practice and will begin seeing patients in SGFs Woodbridge location late December 2020. As a University of Virginia Cavalier alumna, Dr. Hsu is proud to return to the state where her medical journey began.

The 85,000 babies and counting born at SGF is nothing short of a miracle, says Dr. Hsu. I am immensely proud to be a part of this team of people who are all 100% dedicated to the goal of building families and making dreams a reality.

Dr. Hsu will provide comprehensive fertility services to patients, including:

Dr. Hsu earned a full undergraduate merit scholarship from UVA, where she continued her graduate studies and earned a medical degree. As a medical student, Dr. Hsu extended her research efforts abroad in Quetzaltenango, Guatemala, and was ultimately honored with the Medical Alumni Association (MAA) Outstanding Medical Student Award. Her time in Central America allowed Dr. Hsu to exercise her fluency in Spanish.

She then completed her residency at Columbia University Medical Center, followed by a move to Boston where Dr. Hsu completed her fellowship in reproductive endocrinology and infertility at Massachusetts General Hospital. Today, she is board eligible in obstetrics and gynecology as well as reproductive endocrinology and infertility.

When patients are hurting, my vow is to stay by their side and help them through it, shares Dr. Hsu. Whether they need someone to listen, grieve with, or help them make a plan, I will be there. The journey to parenthood can be difficult, but I hope my patients never feel that they are alone.

Dr. Hsu is very active in the medical community. She is an experienced lecturer and oral examiner at Harvard Medical School and has spent time in Appalachia providing free medical and womens health services to residents. Additionally, Dr. Hsu is an accomplished researcher, having contributed to more than 15 presentations across the country on topics including intrauterine insemination (IUI), anti-Mullerian hormone (AMH), and polycystic ovary syndrome (PCOS). She has also served as a peer-reviewer for several publications including the Journal of Assisted Reproduction and Genetics.

Dr. Hsu is a very bright and compassionate physician who, on behalf of SGF, were very thankful to have part of our team, says Eric Widra, M.D., Chief Medical Officer at SGF.

Patients may schedule a new patient virtual physician consultation with Dr. Hsu by calling 1-877-761-1967 or submitting this brief form. Patients also have access to SGF Woodbridges unique financial options, such as the 100% refund program for IVF, that help make fertility treatment more affordable.

About Shady Grove Fertility (SGF)SGF is a leading fertility and IVF center of excellence with more than 85,000 babies born and 5,000+ 5-star patient reviews. With 37 locations throughout FL, GA, MD, NY, PA, VA, D.C., and Santiago, Chile, we offer patients virtual physician consults, deliver individualized care, accept most insurance plans, and make treatment affordable through innovative financial options, including 100% refund guarantees. More physicians refer their patients to SGF than any other center. Call 1-888-761-1967 or visit ShadyGroveFertility.com.

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Nearly 40 percent of Type-2 diabetics are meeting the amount of aerobic exercise recommended, but only 14 percent are meeting the resistance training standard.

NEW ORLEANS Mayoclinic.org says that type 2 diabetes also referred to as adult onset diabetes is a chronic condition that affects the way your body metabolizes glucose (blood sugar) resulting in your body either resisting the effects of the hormone insulin that regulates the movement of sugar into your cells, or doesn't produce enough insulin to maintain normal glucose levels.

Mayo comments that, type 2 diabetes develops, when the body becomes resistant to insulin or when the pancreas is unable to produce enough insulin. Exactly why this happens is unknown, although genetics and environmental factors, such as being overweight and inactive, seem to be contributing factors.

Research has demonstrated that exercise improves glycemic control in people with type 2 diabetes, alone, or the result of weight loss, improved insulin sensitivity, and modifications in cardiovascular risk factors like elevated blood pressure and triglycerides a blood fat associated with an excess of simple carbohydrates in the diet.

The American Diabetes Association recommends that people with type 2 diabetes attain a minimum of 150 minutes per week of aerobic exercise and at least two weekly resistance exercise sessions, while minimizing sedentary time.

A study Significant Dose-Response Between Exercise Adherence and Hemoglobin A1c Change which appears in the September 2020 issue of Medicine & Science in Sports & Exercise, examining the physical activity level in individuals with type 2 diabetes over the age of 65 years in the United States, found that only 25% met the American Diabetes Association 2007 guideline recommendations for total physical activity.

A separate study of physical activity levels in the United States reported that, 41.1% of individuals with type 2 diabetes met the aerobic exercise recommendations compared with only 12.4% for resistance training.

Canadian authors of this Medicine & Science study chose to examine, whether a doseresponse relationship existed between the level of adherence to prescribed exercise over a 6-month exercise intervention and glycemic control (specifically, change in hemoglobin A1c [HbA1c]) in patients with type 2 diabetes. And, if

this association was affected by any of the following factors: modality of exercise, age, sex, or glycemic control before participating in exercise training.

A HbA1c test, according to Medlineplus.gov, measures the amount of glucose attached to hemoglobin. Hemoglobin is the part of your red blood cells that carries oxygen from your lungs to the rest of your body. An HbA1c test shows what the average amount of glucose attached to hemoglobin has been over the past three months.

The Canadian researchers used data from the Diabetes Aerobic and Resistance Exercise (DARE) trial, a single-center, randomized controlled trial designed to evaluate in type 2 diabetes the effect of aerobic, resistance, and combined aerobic and resistance exercise training compared with no exercise training on glycemic control over 26 wk.

The aerobic exercise participants utilized a gradual intensity-based progression with a duration to 45 min at 75% of maximum heart rate per session, while the resistance participants worked up to three sets of eight repetitions of seven exercises at an intensity of eight maximal repetitions. The combined exercise training group completed both the aerobic and resistance training components.

The control group were encouraged to maintain their usual pretrial level of physical activity from baseline to the end of the intervention period.

Dietary guidelines were recommended from the Canadian Diabetes Association meeting 90% of the energy intake to maintain their weights during the study. HbA1c changes from baseline and the end of the 6-month supervised exercise intervention were evaluated.

It was concluded that, a doseresponse relationship between exercise adherence and change in HbA1c for aerobic and combined exercise but not for resistance exercise training. These results suggest that an increased volume of aerobic or combined aerobic and resistance exercise is associated with greater improvement in glycemic control.

If you have type 2 diabetes or are pre-diabetic, check with your physician first for guidelines, before you begin a new exercise program.

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Mackie: Type-2 diabetics need strength exercises in addition to aerobic - WWLTV.com

CARLSBAD, Calif., Dec. 2, 2020 /PRNewswire/ --Qualigen Therapeutics, Inc. (Nasdaq: QLGN) announcesthat it has completed the recertification of its supply of AS1411, and has begun final formulation and filling for use in the Company's proposed Phase 2a efficacy trials of AS1411 for the treatment of patients with COVID-19. Qualigen plans to seek U.S. Food and Drug Administration (FDA) approval for and to begin these trials in the first half of calendar 2021.

Qualigen's lead compound AS1411 is a nucleolin-targeting DNA aptamer drug candidate for the treatment of COVID-19 and other viruses. Preclinical studies at the University of Louisville's (UofL) Center for Infectious Disease have demonstrated the ability of AS1411 to protect cells from the damaging effects of the novel coronavirus. In addition, AS1411 holds potential as a broad antiviral therapeutic agent, which could expand its applications and market potential.

"Even with the roll-out of COVID-19 vaccines, we see this virus being around for many years to come in different forms. The mechanism by which AS1411 is believed to work, by blocking the ability of viruses to replicate in the body, may make our therapeutic effective against future mutations in COVID-19 as well as against other dangerous viruses including the flu," stated Michael Poirier, President, Chief Executive Officer and Chairman of Qualigen.

Mr. Poirier added: "Recertification of our AS1411 inventory represents another critical step in advancing our antiviral development program in a cost-efficient manner. We continue to move forward with our IND-enabling studies and are on track to commence the efficacy stage of human trials in the first half of next year, upon obtaining IND approval from the FDA. We believe there is great potential for this drug to be an effective therapeutic for COVID-19, in addition to other viral infections."

In June 2020, Qualigen signed an exclusive license agreement with UofL for U.S. patent rights covering the treatment of COVID-19 with AS1411, adding to Qualigen's other AS1411 license exclusivities. Qualigen intends to work with UofL to complete investigational new drug (IND)-enabling studies and plans to file an IND application with the FDA in early calendar 2021. Qualigen's aim is to initiate a Phase 2a clinical study in COVID-19 patients in the first half of calendar 2021.

About Qualigen Therapeutics, Inc.Qualigen Therapeutics, Inc. is a biotechnology company focused on developing novel therapeutics for the treatment of cancer and infectious diseases, as well as maintaining and expanding its core FDA-approved FastPack System, which has been used successfully in diagnostics for 20 years. The Company's cancer therapeutics pipeline includes ALAN (AS1411-GNP), RAS-F and STARS. ALAN (AS1411-GNP) is a DNA coated gold nanoparticle cancer drug candidate that has the potential to target various types of cancer with minimal side effects. The foundational nucleolin-targeting DNA aptamer of ALAN, AS1411, is also a drug candidatefor use in treating COVID-19 and other viral-based infectious diseases. RAS-F is a family of RAS oncogene protein-protein interaction inhibitor small molecules for preventing mutated RAS genes' proteins from binding to their effector proteins; preventing this binding could stop tumor growth, especially in pancreatic, colorectal and lung cancers. STARS is a DNA/RNA-based treatment device candidate for removal from circulating blood of precisely targeted tumor-produced and viral compounds. Because Qualigen's therapeutic candidates are still in the development stage, Qualigen's only products that are currently commercially available are FastPack System diagnostic instruments and test kits, used in physician offices, clinics and small hospitals around the world. The FastPack System menu includes rapid point-of-care diagnostic tests for cancer, men's health, hormone function, vitamin D status and antibodies against SARS-CoV-2. Qualigen's facility in Carlsbad, California is FDA and ISO Certified and its FastPack product line is sold worldwide by its commercial partner Sekisui Diagnostics, LLC. For more information on Qualigen Therapeutics, Inc., please visit https://www.qualigeninc.com/.

Forward-Looking StatementsThis news release contains forward-looking statements by the Company that involve risks and uncertainties and reflect the Company's judgment as of the date of this release. These statements include those related to potential future development, testing, efficacy, approval, manufacturing and commercialization of product candidates, including the possible effectiveness of AS1411 against COVID-19 or other viral infections and the approval and timing of clinical trials. Actual events or results may differ from the Company's expectations. For example, there can be no assurance that clinical trials will be approved to begin by or will proceed as contemplated by any projected timeline; that the Company will successfully develop any drugs or therapeutic devices; that preclinical or clinical development of the Company's drugs or therapeutic devices will be successful; that future clinical trial data will be favorable or that such trials will confirm any improvements over other products or lack negative impacts; that any drugs or therapeutic devices will receive required regulatory approvals or that they will be commercially successful; that patents will issue on the Company's owned and in-licensed patent applications; that such patents, if any, and the Company's current owned and in-licensed patents would prevent competition; that the Company will be able to procure or earn sufficient working capital to complete the development, testing and launch of the Company's prospective therapeutic products; that the Company will be able to maintain or expand market demand and/or market share for the Company's diagnostic products generally, particularly in view of COVID-19-related deferral of patients' physician-office visits and FastPack reimbursement pricing challenges; that adoption and placement of FastPack PRO System instruments (which are the only FastPackinstruments on which the Company's SARS-CoV-2 IgGtest kits can be run) will be widespread; that the Company will be able to manufacture the FastPack PRO System instruments and SARS-CoV-2 IgGtest kits successfully; that any commercialization of the FastPack PRO System instruments and SARS-CoV-2 IgGtest kits will be profitable; or that the FDA will ultimately approve an Emergency Use Authorization for the Company's SARS-CoV-2 IgG test. The Company's stock price could be harmed if any of the events or trends contemplated by the forward-looking statements fails to occur or is delayed or if any actual future event otherwise differs from expectations. Additional information concerning these and other risk factors affecting the Company's business (including events beyond the Company's control, such as epidemics and resulting changes) can be found in the Company's prior filings with the Securities and Exchange Commission, available atwww.sec.gov. The Company disclaims any intent or obligation to update these forward-looking statements beyond the date of this news release, except as required by law. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

SOURCE Qualigen, Inc.

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Less than two months after life sciences giant Bayer acquired N.C.-based AskBio, a gene therapy company, the healthcare giant launched a cell and gene therapy platform within its pharmaceutical division.

This morning, Bayer said the launch of the new platform is a deeply transformative move for its business. Stefan Oelrich, a member of Bayers Board of Management and president of the companys Pharmaceuticals Division, pointed to the impact cell and gene therapies have made in treating diseases and said the companys goal is to be at the forefront of this revolution in science. The company tapped Wolfram Carius, its current vice president of Pharmaceuticals Product Supply, to head the new program.

The C> field is growing at an unprecedented pace. With the establishment of Bayers own C> Platform our company will propel its presence in this area. This will complement our existing C> pipeline which already includes five advanced assets with at least three investigational new drugs annually for the next years, Oelrich said in a statement.

To boost its cell and gene therapy presence, Bayer said it is strengthening its internal capabilities and will also pursue external strategic collaborations, technology acquisitions and licensing. In October, the company acquired AskBio's AAV-based gene therapy pipeline of treatments and its Pro10 AAV manufacturing process, which has become something of a standard across the industry. The Pro10 AAV process is used by multiple companies, including Pfizer, Takeda and Viralgen Vector Core SA.Bayer now owns AskBios pipeline of treatments for Pompe disease, Parkinsons disease, as well as therapies for neuromuscular, central nervous system, cardiovascular and metabolic diseases.

The addition of AskBio complements Bayers other cell and gene therapy company, BlueRock Therapeutics, which itacquired last year. BlueRock is developing induced pluripotent stem cells (iPSC), with its most advanced program aimed at Parkinsons disease. In addition to the two companies, Bayer also acquired a contract manufacturing organization that specializes in gene therapy. Bayer said it has established a C> pipeline that includes five advanced assets and more than fifteen preclinical candidates.

The new C> Platform will combine multiple functions by providing support across the entire value chain for the research and development of cell and gene therapies, the company said. This includes support in preclinical development, CMC, clinical programs, project management and more. The platform will guide projects form concept through commercial launch. The goal is to build robust platforms with broad application across different therapeutic areas, the company said.

The emerging bio revolution represents a once-in-a-lifetime opportunity and a new era for Bayer, said Carius said in a statement. A dedicated C> Platform is vital to accelerate innovation at its source, and to ensure its translation into tangible therapies for patients who have no time to wait.

The C> Platform will allow its partners to operate autonomously to develop and progress their portfolio and technology. The role of Bayers C> Platform is to serve as a strategic guide to ensure the different parts of the organization complement each other and combine the best in Biotech and Pharma know-how.

The formation of the C> Platform comes one day after Bayer and Blackford Analysis entered into a development and license agreement to establish an artificial intelligence (AI) platform for medical imaging. The platform will enable the integration of AI applications into the medical imaging workflow which can support the complex decision-making process of radiologists and is intended to enhance diagnostic confidence, the companies said.

Also this week, Bayer sold most of its stake in Elanco Animal Health for $1.6 billion to cover legal bills from ongoing litigation over the weedkiller Roundup and its alleged role as a carcinogen. Bayer owned approximately 15.5% of Elanco, but faces nearly $11 billion in potential damages related to Roundup lawsuits. In June, the company proposed a $12 billion agreement to resolve Roundup litigation.

Last week, a federal judge rejected a $650 million settlement claim for pollution related to polychlorinated biphenyl, or PCB, which is used to cool heavy-duty electrical equipment.

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Bayer Launches Cell and Gene Therapy Platform to Maximize Recent Acquisitions - BioSpace

Inc () is a global biotech group focused on unlocking the potential of personalized therapies and closed processing systems through its cell and gene therapy platform.

The Germantown, Maryland-based companys aim is to provide life-changing treatments at the point-of-care to patients at low cost.

The group's Cell and Gene Therapy Biotech platform has three key elements.The first revolves around point-of-care therapeutics, which consists of a pipeline of licensed cell and gene therapies and scientific knowhow. The second aspect relates to point-of-care technologies, which include a suite of in-licensed technologies engineered to create customized processing systems for affordable therapies.

Finally, the third component rests on a point-of-care network, which is a collaborative, international ecosystem of leading research institutes and hospitals committed to supplying cell and gene therapies at the patient bedside. It is an intricate web of affiliated pre-clinical and clinical-stage biopharmaceutical companies, research institutions and hospitals through which is able to in-license technologies or advance therapy medicinal products (ATMPs) and co-develop them with its partners.

On February 11, 2020, Or completed the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO), to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

The successful sale has spotlighted Orgenesis boss Vered Caplans considerable leadership skills. She has since been named one of the top 20 inspirational leaders in the field of advanced medicine by The Medicine Maker, which creates an annual Power List of top global drugmakers.

Caplan acquired Masthercell in March 2015 and grew the CDMO segment revenue from a run-rate of $3 million to a run-rate of around $30 million at the end of 2019, reflecting a compound annual growth rate of 59% under her leadership, and a sale price of more than five times the initial purchase price of around $25 million.

Caplan has indicated that she plans to use the Masthercell sale proceeds to grow Orgenesis's evolving point-of-care cell therapy business and develop advanced therapy medicinal products.

In November, Orgenesis posted third-quarter results that saw its revenue jump 40% year-over-year on the back of growth in its point-of-care cell and gene therapy network. For the period ended September 30, 2020, the company reported revenue of $1.7 million, compared to $1.2 million in the same period in 2019.

The company also maintained a solid balance sheet with $88.8 million in cash and equivalents at the end of September, reflecting the successful sale of its Masthercell Global CDMO.

Orgenesis is using the Masthercell sale proceeds to expand the companys point-of-care cell therapy business.

In October, the group completed the acquisition of Koligo Therapeutics Inc, a regenerative medicine company, including all of the assets of Tissue Genesis LLC. The agreed consideration terms include $15 million in shares of Orgenesis stock valued at $7 share which will be issued to Koligos accredited investors (with certain non-accredited investors to be paid solely in cash) and an assumption of $1.3 million in Koligos liabilities, estimated to be nearly all of Koligos liabilities.

Koligos management team will be joining Orgenesis to continue commercial and development activities. Koligo CEO Matthew Lehman is an accomplished executive in the biotech and regenerative medicine fields. Orgenesis believes the acquisition will help to expand its therapeutic and technology resources, while adding a highly experienced US team to help further bolster Orgenesis point-of-care network in the US.

On the therapeutics front, Orgenesis is focused on several key verticals, including cell-based cancer immunotherapies, treatments for metabolic and neurodegenerative diseases and tissue regeneration, as well as antiviral therapies. The groups near-term goal is to expand the availability of KYSLECEL from the recent Koligo acquisition. KYSLECEL, is made from a patient's own pancreatic islets the cells that make insulin to regulate blood sugar and is commercially available in the US for chronic and recurrent acute pancreatitis.

Like many companies, Orgenesis is also focused on the coronavirus (COVID-19) space. It is planning patient recruitment for a phase 2 randomized clinical trial of Koligos KT-PC-301, subject to US Food and Drug Administration (FDA) review and clearance of an investigational new drug (IND) application. KT-PC-301 is an autologous clinical development-stage cell therapy candidate for COVID-19-related Acute Respiratory Distress Syndrome, which Orgenesis acquired as part of the Koligo acquisition.

Koligos KT-PC-301 is a cell therapy that is derived from a patients own adipose (fat) tissue. A small amount of fat is collected from the patient and sent to Koligos manufacturing facility in Indiana to make KT-PC-301. The product is made within hours and sent back to the hospital for intravenous administration. KT-PC-301 contains mesenchymal stem cells, vascular endothelial cells, and immune cells which migrate to the patients lungs and other peripheral sites of inflammation. Published nonclinical and clinical evidence demonstrates that KT-PC-301 may stabilize microcirculation to improve oxygenation; maintain T and B lymphocytes to support antibody production, and induce an anti-inflammatory effect.

In addition, Orgenesis is gearing up for a Phase 2 study of Ranpirnase for the treatment of conditions caused by human papillomavirus, pending a planned IND submission to the FDA.

Meanwhile, Orgenesis has advanced the development of a cell-based vaccine platform targeting COVID-19 and other viral diseases. It has launched BioShield to set up a first line of defense against COVID-19 through the discovery of neutralizing human antibodies to contain the spread of the viral pathogen. In addition, Orgenesis has signed an agreement with Fortune 500 science giant to develop, and potentially obtain FDA approval of Ranpirnase for treating COVID-19 patients. Ranpirnase has shown preclinical antiviral activity in diseases such as influenza, Ebola, SARS, and cytomegalovirus. Orgenesis acquired the broad-spectrum Ranpirnase antiviral as a part of its $12 million cash and stock deal to acquire Tamir Biotechnology.

Orgenesis intends to leverage its network of regional partners to advance the development of its pipeline. Towards this end, its partners have committed to funding the clinical programs. In turn, Orgenesis typically grants its partners geographic rights in exchange for future royalties, and a partnership with Orgenesis to support the supply of the targeted therapies. Caplan has said that through this unique business model, Orgenesis has already signed contracts, which could generate over $40 million in revenue over the next three years, if fully realized.

There are also plans to continue to develop, license and form partnerships around a variety of point-of-care technologies to support work in areas such as tumour-infiltrating lymphocytes, CAR-T, CAR-NK, dendritic cell therapies, and mesenchymal stem cell-based therapies.

Currently, the costs of cell and gene therapies are prohibitive, as illustrated by CAR-T therapies, which cost hundreds of thousands of dollars per patient. To lower costs, Orgenesis is switching from a high-cost centralized manufacturing model to a localized point-of-care model.

In April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous CGT Biotech platform to advance clinical trials. Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells as a source of exosomes and other cellular products. The companies are hoping to use Orgenesiss expertise and point-of-care platform, which include automated systems, 3D printing and bioreactor technologies.

Meanwhile, Orgenesis and ExcellaBio have developed a breakthrough manufacturing process for so-called bioxomes, which are synthetically made exosomes or extracellular vesicles (EVs). The latter are what transfer DNA, RNA, and proteins to other cells, thereby altering the function of targeted cells. Until now, exosome/EV production has been based on conventional, complex and costly methods of ultrafiltration. However, the two companies have demonstrated the scale-up of bioxomes through a proprietary technique, while generating consistent and repeatable results, including uniform particles sizes.

Orgenesis has added the University of California (UC), Davis to its point-of-care network. The first project with UC Davis Health is on developing a lentiviral manufacturing system which will address a significant unmet need in the market for an efficient and scalable manufacturing process.

Orgenesis CEO Vered Caplan noted that the company has dramatically transformed in 2020 helped by the Masthercell sale.

The sale was an important step in our strategy to leverage our unique capabilities that directly address the key challenges facing the cell and gene therapy industry, Caplan said in a statement.

Our goal is to transform the delivery of cell and gene therapy through our point-of-care therapeutics, technologies and network, thereby lowering costs and unlocking the power of cell and gene through a more decentralized and integrated approach, she added.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Orgenesis leads the charge at an exciting time for cell and gene therapy - Proactive Investors USA & Canada

Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act.

But a key analyst covering biopharma believes that theres a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, hes pointing to the 2 leaders in the field as the most likely players to come out ahead way, way ahead.

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Regeneron teams with gene therapy pioneer James Wilson, adapting its Covid-19 antibody cocktail to an AAV-based nasal spray - Endpoints News

Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act.

But a key analyst covering biopharma believes that theres a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, hes pointing to the 2 leaders in the field as the most likely players to come out ahead way, way ahead.

Unlock this story instantly and join 94,500+ biopharma pros reading Endpoints daily and it's free.

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UPDATED: Bayer continues its cell and gene therapy push, enveloping different projects under one strategic roof while hunting new deals - Endpoints...

4D Molecular Therapeutics raised $75 million in June to get several gene therapy programs into and through the clinic. Now, its adding a trio of executives to spearhead its work in heart, eye and lung diseases as it looks to shepherd treatments in those focus areas forward.

Robert Fishman, M.D. becomes 4Ds chief medical officer and therapeutic area head for pulmonology. He joins from Xoc Pharmaceuticals, where as chief medical officer he led phase 1 development for programs in Parkinsons disease and migraine. Before that, he headed clinical development at InterMune, overseeing the pivotal trial of Esbriet, an idiopathic pulmonary fibrosis drug now marketed by Roche.

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

RELATED: Restoring eyesight with genetically engineered stem cells

Raphael Schiffmann, M.D., signs on as senior vice president and therapeutic area head for 4Ds cardiology stable. He was previously director of the Institute of Metabolic Disease at the Baylor Research Institute and the lead investigator of the developmental and metabolic neurology branch at the NIHs National Institute of Neurological Disorders and Stroke.

Robert Kim, M.D., joins 4D as a senior vice president and clinical therapeutic area head of ophthalmology. Hes held multiple chief medical officer roles at ViewPoint Therapeutics, Apellis Pharma and Vision Medicines, and earlier in his career worked in ophthalmology at GlaxoSmithKline, Genentech and Novartis.

The three executives arrive six months after 4D topped up its coffers with a $75 million series C round. The capital, which came two years after a $90 million B round, was earmarked to push three programs into the clinic, including two that are partnered with Roche.

Those programs include 4D-310, a treatment for Fabry disease in which patients cells accumulate a type of fat called globotriaosylceramide, and 4D-125, a treatment for the eye disease X-linked retinitis pigmentosa. Roche has the exclusive right to develop and commercialize the latter. Roche has licensed the third prospect, 4D-110, a treatment for a type of vision loss called choroideremia.

RELATED: 4D raises $90M to move gene therapies into clinical testing with AstraZeneca and Roche

The funds will also bankroll the development of 4Ds preclinical pipeline, including IND-enabling studies for 4D-710, a program in cystic fibrosis, and other candidates for neuromuscular diseases and ophthalmology.

With the addition of Robert Fishman, Raphael Schiffmann and Robert Kim to our clinical R&D leadership team, 4DMT gains not only extensive experience in clinical development and translational medicine, but also unique and specific experience within each of the initial 4DMT therapeutic areas," said 4D CEO David Kim, M.D., in a statement.

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4D hires a trio of area heads as it ramps up its gene therapy pipeline - FierceBiotech

London, 2nd December 2020 Cell and Gene Therapy Catapult (CGT Catapult) today announces the launch of the Advanced Therapies Skills Training Network (ATSTN). It is designed to reach more people than ever before to propel them towards career opportunities in the advanced therapy and vaccine manufacturing industry by assessing their transferrable skills and providing access to training.

Driven by industry and coordinated by the CGT Catapult, the ATSTN delivers a platform consisting of dedicated online resources and national centres to upskill as well as the opportunity for people to identify how they can enter this industry. This is a significant contribution to ensuring continuous growth of the UK advanced therapies and vaccine manufacturing industry, developing the nations health resilience by creating opportunities for current and new skilled personnel.

The ATSTN initiative is funded by 4.7 million awarded from the Department for Business, Energy & Industrial Strategy (BEIS) through UK Research and Innovation (UKRI) to help grow the sector by creating economic opportunities for new jobs and industry driven learning. With the industry workforce expected to double to more than 6,000 by 2024, this investment is continuing the Governments commitment to expanding the UK expertise in advanced therapies.

Business Secretary Alok Sharma, said:

It is vital thatwe upskillourworkforce todevelopand manufacture vaccinesand treatmentsat scale,ensuring the advanced therapy and vaccine manufacturing sectors are stronger and more resilient in the future.

Backed with almost 5 million government investment, this first-of-its-kind skills network will equip people at all stages of their careersto pursueopportunities in advanced medicines and vaccine manufacturing. This will help to create highly skilled jobsacross the countrywhile putting the UK in the strongest position to respond to future pandemics.

Kate Bingham, Chair of the Vaccines Taskforce said:

One of the tasks of the governmentsVaccines Taskforceis to ensure the UK has the capabilities to respond to future health crises,and this includes scaling up our ability to manufacture safe and effective vaccines and treatments.

By upskilling and expanding the UKs vaccine manufacturing industry, this new skills network will help to ensure the UK has the capacity to produce successful vaccines at scale, including for coronavirus, helping to protect the UK population for generations to come.

Matthew Durdy, CEO of Cell and Gene Therapy Catapult commented:

The ATSTN initiative reiterates our commitment to upskill new and existing members of the advanced therapy workforce, building on the success and impact of the ATAC apprenticeship programme. Not only is it rewarding to work so closely with industry to deliver on this need, but it is also extremely motivating for all involved to think that this initiative is going to be the start of so many promising careers and additional job opportunities. We look forward to seeing the success stories from users of this ground-breaking platform

The ATSTN has been designed in collaboration with industry to address its needs to upskill current professionals as well as to leverage skills from other sectors, opening opportunities for people to join the cell and gene therapy industry from different ones. ATSTN users will be able to access learning materials, whether remotely or on-site, from a wealth of expertise across the entire industry, while tailoring their personal learning experience to their own needs.

There are three different components to the ATSTN platform: Online Training Platform, National Training Centres and Career Converter. These components allow users, whether they come from inside or outside the industry, to curate job matches based on their current skillset and identify what training they need to progress in that field.

The National Training Centres, that will provide on-site training, are to be announced later in December.

The ATSTN platform can be accessed from http://www.atskillstrainingnetwork.org.uk

- END-

Notes to editor:

About the ATSTN learning platform features

The networks Career Converter tool is a great place to start for people looking to enter the industry by identifying what skills they already have. Once they have built a comprehensive skills profile, the tool will match the candidate with the advanced therapy roles most suited to them as well as a breakdown of the training they would need.

The Online Training Platform is a place for candidates to enrol onto courses so they can start filling the gaps on their skills profile right away. Once they are logged in, they will be able to browse the online course catalogue and schedule their learning at their own pace. The platform, developed in collaboration with industry, can help build skills in areas specific to industry sectors as well as general training into leadership and people skills.

List of organisations that have been involved to date with the development of ATSTN

Achilles Therapeutics, Adaptimmune, Agylia, Allergan Biologics, Anthony Nolan Cell and Gene Therapy Services, AskBio, Autolus, Biogelx, Cancer Research UK, Cellular Therapies Facility Newcastle, Cell and Gene Therapy Catapult, Charles River, Cobra Biologics, CPI, Enginuity, EvOx Therapeutics, eXmoor, Freeline, Fujifilm, GOSH, GSK, Gyroscope, Immetacyte, Intertek, Medicines Discovery Catapult, John Goldman Centre for Cellular Therapy Imperial College, Merck SAFC, National Horizons Centre, NHSBT, HakoBio designed by OUAT!, Oxford BioMedica, Oxford University Clinical Biomanufacturing Facility, Pall Corporation, Phion Therapeutics, Replimmune, RoslinCT, Symbiosis, UKRI, VIMIC.

About Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

For further information please contact

FTI Consulting for the CGT Catapult:

Michael Trace +44 (0) 203 319 5674 / michael.trace@fticonsulting.com

George Kendrick + 44 (0) 203 727 1411 / george.kendrick@fticonsulting.com

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UK set to take advanced therapies and vaccine manufacturing industry to the next level: the Advanced Therapy Skills Training Network launches today -...

Dec. 2, 2020 06:00 UTC

LONDON--(BUSINESS WIRE)-- Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced that Jennifer Cook, David Fellows and Rene Gal have been appointed to the Gyroscope Board of Directors, effective immediately.

I am very pleased to welcome Jennifer, David, and Rene, three extremely accomplished executives with extensive biopharmaceutical backgrounds, to our board of directors, said Khurem Farooq, Chief Executive Officer. We look forward to their valuable contributions and unique perspectives as we continue our work to develop gene therapy beyond rare disease with the goal of delivering new medicines to people with retinal diseases.

Ms. Cook was most recently the Chief Executive Officer of GRAIL, a healthcare company focused on the early detection of cancer. Prior to joining GRAIL, she held a number of leadership positions during her 25-year tenure with Roche/Genentech, including leading Roche Pharmaceuticals European commercial business, Global Clinical Operations, U.S. and Global Product Portfolio Management, the U.S. Immunology and Ophthalmology Business Unit and Market Development. She currently serves on the boards of directors of Denali Therapeutics, BridgeBio Pharma and Ambys Medicines. Ms. Cook holds a B.A. in human biology and an M.S. in biology from Stanford University, as well as an MBA from the Haas School of Business at the University of California, Berkeley.

Mr. Fellows was most recently the Chief Executive Officer of Nightstar Therapeutics, a retinal gene therapy company acquired by Biogen in 2019. Prior to joining Nightstar, he was the Vice President of Johnson & Johnsons Vision Care Franchise where he led the global marketing, new product and licensing activities. Before joining Johnson & Johnson, Mr. Fellows held leadership positions at Allergan, Inc., for 25 years where he served primarily in sales and marketing in a number of capacities, including Regional President, Corporate Vice President, and Senior Vice President across North America, Europe and Asia. He currently serves as the chairman of the board for Oxular Limited and is a board member of the Glaucoma Foundation. Mr. Fellows holds a B.A. in psychology from Butler University.

Ms. Gal currently serves as the Executive Vice President and Chief Financial Officer of Jazz Pharmaceuticals, a global biopharmaceutical company. Prior to joining Jazz, she served as the Chief Financial Officer of GRAIL. Before joining GRAIL, Ms. Gal served as the Senior Vice President and Chief Financial Officer of Theravance Biopharma, where she led the companys spin-out transaction from Innoviva, Inc. Ms. Gal has also served in global treasury, pharmaceutical sales and corporate strategy/business development at Eli Lilly and Company. Prior to joining Eli Lilly, she spent seven years in the energy industry in positions focused on corporate finance, project finance and mergers and acquisitions. She currently serves on the board of directors of Gossamer Bio where she chairs its audit committee. Ms. Gal holds a B.S. in mathematics from Vanderbilt University and an MBA from Columbia Business School.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing gene therapy beyond rare disease to treat a leading cause of blindness, dry age-related macular degeneration (AMD). Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and Phase II clinical trials called EXPLORE and HORIZON.

Syncona Ltd, our lead investor, helped us create a leading retinal gene therapy company combining discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

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Gyroscope Therapeutics Announces Appointments of Industry Veterans in Gene Therapy, Ophthalmology and Biopharmaceuticals to Its Board of Directors -...

Researchers inserted genes into mouse eye cells to protect and restore the optic nerve (red fibers in microscope image of healthy mouse retina, above).

By Kelly ServickDec. 2, 2020 , 5:30 PM

Do old and damaged cells remember what it was like to be young? Thats the suggestion of new study, in which scientists reprogrammed neurons in mouse eyes to make them more resistant to damage and able to regrow after injurylike the cells of younger mice. The study suggests that hallmarks of aging, and possibly the keys to reversing it, lie in the epigenome, the proteins and other compounds that decorate DNA and influence what genes are turned on or off.

The idea that aging cells hold a memory of their young epigenome is very provocative, says Maximina Yun, a regenerative biologist at the Dresden University of Technology who was not involved in the work. The new study supports that [idea], but by no means proves it, she adds. If researchers can replicate these results in other animals and explain their mechanism, she says, the work could lead to treatments in humans for age-related disease in the eye and beyond.

Epigenetic factors influence our metabolism, our susceptibility to various diseases, and even the way emotional trauma is passed through generations. Molecular biologist David Sinclair of Harvard Medical School, who has long been on the hunt for antiaging strategies, has also looked for signs of aging in the epigenome.

The big question was, is there a reset button? he says. Would cells know how to become younger and healthier?

In the new study, Sinclair and his collaborators aimed to rejuvenate cells by inserting genes that encode reprogramming factors,which regulate gene expressionthe reading of DNA to make proteins. The team chose three of the four factors scientists have used for more than 10 years to turn adult cells into induced pluripotent stem cells, which resemble the cells of an early embryo. (Exposing animals to all four factors can cause tumors.)

The team focused specifically on neurons at the back of the eye called retinal ganglion cells. These cells relay information from light-sensitive photoreceptors to the brain using long tendrillike structures called axons, which make up the optic nerve. Theres a stark divide between youth and age in these cells: An embryonic or newborn mouse can regenerate the optic nerve if it gets severed, but that ability vanishes with time.

To test whether their treatment could bring back some of that resilience, Sinclair and colleagues crushed the optic nerves of mice using forceps and injected a harmless virus into the eye carrying the genes for the three reprogramming factors. The injection prevented some damaged retinal ganglion cells from dying and even prompted some to grow new axons reaching back to the brain, the team reports today in Nature.

When the researchers looked at methylation patternsthe DNA location of chemical tags called methyl groups that regulate gene expressionthey found that changes caused by the injury resembled those in aging mouse cells. In certain parts of the genome, the treatment reversed those changes. The researchers also found that the benefits of the introduced genes depended on cells ability to alter their methylation patterns: Mice lacking certain enzymes necessary to remove methyl groups from DNA saw no benefit to the treatment.

Thats really something special, says Leonard Levin, a visual neuroscientist at McGill University. The experiments suggest how the famous and well-studied reprogramming factors restore cells. But big questions remain, he says: How do these factors cause methyl groups to be added or removed? How does that process help retinal ganglion cells?

Sinclairs team also tested the approach in mice with a condition meant to mimic glaucoma, a leading cause of age-related blindness in humans. In glaucoma, the optic nerve gets damaged, often by a buildup of pressure in the eye. Sinclair and his colleagues injected tiny beads into the animals eyes that prevented normal drainage and increased pressure, which damaged retinal ganglion cells.

Four weeks later, the animals visual acuity had declined by about 25%, as measured by a vision test in which mice move their heads to track the movement of vertical bars displayed on computer monitors. But after the genetic treatment, the animals gained back roughly half of their lost acuitythe first demonstration of restored vision in mice after this glaucomalike injury.

Still, the improvement in acuity was small, Levin notes. And, he says, the treated mice were in a relatively early stage of damage, not the state of near or total blindness that people experience when glaucoma goes untreated for years. So its too early to say whether this approach could benefit people who have lost much of their vision. Levin adds that there are already very good treatments for early-stage glaucoma to prevent vision loss with medicated eye drops or surgery to lower eye pressure.

In a final set of experiments, Sinclair and colleagues injected the reprogramming-factor genes into the eyes of 1-year-old healthy mice, roughly the mouse equivalent of middle-age. By this stage, the animals had visual acuity scores about 15% lower than their 5-month-old counterparts. Four weeks after treatment, older mice had similar acuity scores to younger ones. In their cells, the researchers saw patterns of DNA methylation and gene expression resembling those of younger animals.

In the three sets of experiments, Sinclair says, the cells seemed to respond to the reprogramming factors by fine-tuning their gene expression to match a youthful state. He sees that behavior as a hint that cells keep a record of their epigenetic past, even though its not clear how that record is stored. A company Sinclair cofounded, Life Biosciences, is developing treatments for diseases associated with aging, including glaucoma, and he says hes now planning to test the safety of this gene therapy approach in larger animals.

Yun says that as a strategy for reversing aging or treating disease, resetting the epigenome is a very difficult one. Reprogramming cells to an earlier state carries a risk of prompting uncontrolled growth and cancer.Future studies should test how the three factors affect other types of cells and tissues and confirm that reprogrammed cells maintain their youthful state long-term, she says. There are a lot of roads to be traveled.

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Researchers restore lost sight in mice, offering clues to reversing aging - Science Magazine

"The need for reproducible, scalable, and economical production of cell and gene therapies is creating a demand for digital bioprocessing technologies," said Nitin Naik, Global Life Sciences Vice President at Frost & Sullivan. "These technologies are critical to realize the true commercial potential of cell and gene therapies in the next two to three years and serve as a conduit to improve market access and control the total cost of therapy."

Naik added: "From a market segment perspective, while the stem cell market is lucrative, the highest growth is expected to be in gene-modified cell therapies, with a pipeline of 269 products,* followed by gene therapies, which account for 182 assets in the pipeline.* Further, although allogeneic stem cell therapies dominate the marketed product catalogs, interest in disease-modifying CAR-T therapies, which are largely autologous, is driving demand for the evolution of manufacturing technologies, models, and capacity expansion investment by CDMOs." (*as of August 2020)

To tap into the growth prospects exposed by the CGT market, companies must focus on:

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026 is the latest addition to Frost & Sullivan's Healthcare research and analyses available through the Frost & Sullivan Leadership Council, which helps organizations identify a continuous flow of growth opportunities to succeed in an unpredictable future.

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Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026

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Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan - PRNewswire

Executive Summary Global Gene Therapy Market was valued at USD 1221. 84 Million in the year 2019. Escalating number of cases related to various chronic diseases including Cancer, Cardiovascular and Neurological Disorders, Alzheimers and Sickle Cell Diseases, with companies investing heavily in incorporating advanced technology supported by growing collaboration between bio-pharma companies and research institutes to advance in the field of Gene therapy, has been anticipated to infuse growth in the market for Gene Therapy during the forecast period of 2020-2025.

New York, Dec. 01, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Market Analysis By Vector Type, By Application, By Region, By Country (2020 Edition): Market Insights, Covid-19 Impact, Competition and Forecast (2020-2025)" - https://www.reportlinker.com/p05993452/?utm_source=GNW

Under the Vector Type segment, AAV vectors, followed by Retrovirus & Gammaretrovirus and Lentivirus, are anticipated to witness the largest market share owing to growing investment in adeno-associated viral (AAV) vectors to advance research programs against strategically selected cell targets. Increasing prevalence of various diseases across the globe will further accelerate the gene therapy market growth during the coming years.

Among the regions, North America, followed by Europe and Asia Pacific, will experience remarkable market share owing to the presence of various leading global companies including Orchard Therapeutics, Voyager Therapeutics, and Spark Therapeutics. With companies investing in adoption of advanced technology supported by enhanced focus on expanding product pipeline by manufacturers to advance in the field of Gene Therapy will further facilitate the market growth during the forecast period.

Scope of the Report The report analyses Gene Therapy market By Value. The report analyses Gene Therapy Market By Vector Type (Lentivirus, AAV, Retrovirus & Gammaretrovirus, Others). The report further assesses the Gene Therapy market By Application (Neurological Disorders, Cancer, Cardiovascular Diseases, Others). The Global Gene Therapy Market has been analysed By Region (North America, Europe, and Asia Pacific) and By Country (United States, Canada, Germany, France, United Kingdom, Italy, China, Japan, India, South Korea). The key insights of the report have been presented through the frameworks of Major Mergers & Acquisitions, Technological Innovations, and Role of Companies during COVID-19 Pandemic with and Product Pipeline of Leading Companies. Also, the attractiveness of the market has been presented By Region, Vector Type and Application. Additionally, the major opportunities, trends, drivers and challenges of the industry has been analysed in the report. The companies analysed in the report include Voyager Therapeutics, Novartis AG, Spark Therapeutics Inc., MoldMed S.P.A., Orchard Therapeutics PLC, Alnylam Pharmaceuticals Inc., AnGes Inc., Akcea Therapeutics, BlueBird Bio Inc., and Sarepta Therapeutics. The report presents the analysis of Gene Therapy market for the historical period of 2015-2019 and the forecast period of 2020-2025.

Key Target Audience Bio-Tech and Bio-Pharma Companies Hospitals, Clinics, and Healthcare Units Consulting and Advisory Firms Investment Banks and Equity FirmsRead the full report: https://www.reportlinker.com/p05993452/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Global Gene Therapy Market Analysis By Vector Type, By Application, By Region, By Country (2020 Edition): Market Insights, Covid-19 Impact,...