What Did the Winners of the Nobel Prize in Medicine Discover? – JSTOR Daily

Its Nobel Prize season! In the biological sciences, the prestigious prizes are often awarded for discoveries that unlock lifes most basic mechanisms. Nobel-prize-winning work often invokes concepts so fundamental that most people take them for granted. This years Nobel Prize for Physiology and Medicine is no exception. Awarded to William Kaelin Jr, Sir Peter Ratcliffe, and Gregg L. Semenza, the 2019 prize honors the discovery of how cells adapt to the presence of oxygen.

In a 2007 Science article, one of the winners, Gregg Semenza, describes portions of the groundbreaking work. The major issue, as Semenza describes it, is that while many of lifes essential functions depend on oxygen, the amount available is not always consistent. For example, oxygen availability has not been consistent throughout evolutionary history. Early organisms began utilizing oxygen for energy production in increasing degrees, starting around 2.5 billion years ago, about a billion years after life began. After photosynthetic organisms that produced oxygen as a byproduct emerged, the atmospheric oxygen concentration increased dramatically.

Early life, and the cells of more complicated organisms, had to get used to this newly abundant resource. Non-photosynthetic organisms became dependent on oxidative phosphorylation, a means by which the energy of sugar is unlocked for use by cells. Within the cell, in the face of changing external conditions, the amount of available oxygen must be relatively constant. Too little, and it will be impossible to generate energy; too much, and potentially deadly waste products can build up.

Enter HIF-1, aka Hypoxia Induced Factor 1, a protein known as a transcription factor. Transcription factors control the conditions under which a specific gene or genes is expressed, and HIF-1 controls hundreds, perhaps thousands, of genes. The amount of HIF-1 available varies depending on the available oxygen, increasing or decreasing how often the suite of genes controlled by HIF-1 gets expressed.

Semenza and his colleagues teased out the details of the incredibly complicated mechanism by which HIF-1 functions to maintain constant oxygen levels. There are more than a dozen steps, and related biochemical components, in the process. They have found versions of the HIF-1 process in much simpler animals, such as roundworms and flies, helping them trace the evolutionary origins of the adaptation to oxygen.

The work was doubly significant. Not only did the team come to a detailed understanding of one of lifes key processes, the discovery has great potential in medicine as well. Some of the most common cardiovascular diseases, such as stroke or heart attack, restrict the oxygen supply to cells. If a medication or procedure can be devised to control how cells respond to the drop in oxygen, it may be possible to prevent or even reverse the damage from cardiovascular disease. The mechanism may even be manipulated to limit the growth of tumors. Congratulations to doctors Kaelin, Ratcliffe, and Semenza!

JSTOR is a digital library for scholars, researchers, and students. JSTOR Daily readers can access the original research behind our articles for free on JSTOR.

By: Gregg L. Semenza

Science, New Series, Vol. 318, No. 5847 (Oct. 5, 2007), pp. 62-64

American Association for the Advancement of Science

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What Did the Winners of the Nobel Prize in Medicine Discover? - JSTOR Daily

Researchers identify genetic variations linked to oxygen drops during sleep – National Institutes of Health

News Release

Thursday, October 24, 2019

Researchers have identified 57 genetic variations ofagenestrongly associated withdeclinesinbloodoxygen levelsduring sleep. Low oxygen levels during sleep are a clinical indicator of the severity of sleep apnea, a disorder that increases the risk of heart disease, dementia, and death. The study, published today in theAmerican Journal of Human Genetics, was funded by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

A persons average blood oxygen levels during sleep are hereditary, and relatively easy to measure, said study author Susan Redline, M.D., senior physician in the Division of Sleep and Circadian Disorders at Brigham and Womens Hospital, and professor at Harvard Medical School, Boston. Studying the genetic basis of this trait can help explain why some people are more susceptible to sleep disordered breathing and its related morbidities.

When we sleep, the oxygen level in our blood drops, due to interruptions in breathing. Lung and sleep disorders tend to decrease those levels further, and dangerously so. But the range of those levels during sleep varies widely between individuals and, researchers suspect, is greatly influenced by genetics.

Despite the key roleblood oxygen levelsplayin health outcomes,theinfluenceof genetics on theirvariabilityremains understudied. The current findings contribute toa betterunderstanding, particularly because researcherslookedat overnight measurementsof oxygen levels. Thoseprovide more variability than daytime levelsdue to the stressesassociated withdisordered breathing occurring during sleep.

The researchers analyzed whole genome sequence data from the NHLBIs Trans-Omics for Precision Medicine (TOPMed) program. Tostrengthenthe data,they incorporated results of family-basedlinkage analysis, a method for mapping genes that carry hereditary traits to their location in the genome. Themethod usesdata fromfamilies with several members affected by aparticulardisorder.

This study highlights theadvantage of using family data in searching for rare variants, which is often missed in genome-wide association studies, said James Kiley, Ph.D., director of the Division of Lung Diseases at NHLBI. It showed that, when guided by family linkage data, whole genome sequence analysis can identify rare variants that signal disease risks, even with a small sample. In this case, the initial discovery was done with fewer than 500 samples.

The newly identified 57 variants of the DLC1 gene were clearly associated with the fluctuation in oxygen levels during sleep. In fact, they explained almost 1% ofthevariability in the oxygen levels in European Americans, which is relatively high for complex genetic phenotypes, or traits, that are influenced by myriad variants.

Notably,51 of the 57genetic variantsinfluence and regulate human lung fibroblast cells, a type of cell producing scar tissue in the lungs, according to study author XiaofengZhu, Ph.D., professor at the Case Western Reserve University School of Medicine, Cleveland.

This is important becauseMendelian Randomization analysis, a statistical approach for testing causal relationship between an exposure and an outcome, shows a potential causal relationship between how the DLC1 gene modifies fibroblasts cells andthechanges in oxygen levels during sleep, he said.

Thisrelationship,Kileyadded,suggests thata shared molecular pathway, or a common mechanism,may beinfluencing a persons susceptibility to the lack of oxygen caused by sleep disordered breathingand other lung illnesses such as emphysema.

The project was jointly led by Zhu and Redline, who also directs the National Sleep Research Resource, supported by NHLBI.

About theNational Heart, Lung, and Blood Institute (NHLBI): NHLBI is the global leader in conducting and supporting research in heart, lung, and blood diseases and sleep disorders that advances scientific knowledge, improves public health, and saves lives. For more information, visithttps://www.nhlbi.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Sequencing analysis at 8p23 identifies multiple rare variants in DLC1 associated with sleep related oxyhemoglobin saturation level.

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Researchers identify genetic variations linked to oxygen drops during sleep - National Institutes of Health

Vertex Announces Agreement with NHS England for Access to All Licensed Cystic Fibrosis Medicines – BioSpace

Oct. 24, 2019 09:23 UTC

LONDON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced an access agreement with NHS England for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines.

This means that within 30 days patients with CF in England ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can be prescribed ORKAMBI (lumacaftor/ivacaftor) by their doctor and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations can be prescribed SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor.

The agreement also offers expanded access to KALYDECO (ivacaftor) to include people ages 18 years and older who have the R117H mutation and those patients ages 12 months and older who have one of the nine licensed gating mutations.

Today is a significant day for the cystic fibrosis community in England. This important agreement, reached in collaboration and partnership with NHS England and NICE, will allow more than 5,000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease, said Ludovic Fenaux, Senior Vice President, Vertex International.

In addition to England, reimbursement agreements have also recently been announced in Scotland, Australia and Spain.

About CF in the UK Over 10,000 people in the UK have CF the second highest number in the world. Over 8,000 people in England have CF. CF is a debilitating, life-shortening inherited condition that causes progressive damage to organs across the body from birth. Currently, there is no cure for CF and half of people in the UK with CF die before they are 32. The daily impact of treatment is significant. It can take up to four or more hours involving, nebulizers, physiotherapy and up to 70 tablets a day. CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.

About ORKAMBI (lumacaftor/ivacaftor) and the F508del mutation In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

SYMKEVI is indicated for people with CF ages 12 and older who either have two copies of the F508del mutation or one copy of the F508del mutation and have one of the following 14 mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272-26AG, or 3849+10kbCT.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About KALYDECO (ivacaftor) KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

KALYDECO is indicated in people ages 12 months and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. KALYDECO is also indicated for the treatment of patients with CF ages 18 years and older who have an R117H mutation in the CFTR gene.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About Vertex

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

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including nine consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking Statements

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

(VRTX-GEN)

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ERT to Treat Pompe May Work Better in Combo with Blood Pressure Medication, Study Says – Pompe Disease News

Adding carvedilol, the active compound of a blood pressuremedicine, to enzyme replacement therapy (ERT) for Pompe disease can improve its effectiveness in reaching and strengthening skeletal muscles, a study in mice suggests.

This finding, Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease was published in Molecular Genetics and Metabolism.

At present, enzyme replacement therapy (ERT) is the only effective treatment for Pompe disease, a rare genetic disorder caused by the absence or deficiency of the acid alpha-glucosidase (GAA) enzyme.

When GAA activity is low, a sugar molecule called glycogen accumulates inside cells, damaging organs and tissues throughout the body, but primarily skeletal muscle, smooth muscle, and cardiac muscle. If left untreated, the accumulation of glycogen in cardiac and skeletal muscle leads to severe and progressive muscular weakness, risking heart and respiratory failure.

There is, however, a major limitation in ERT. Skeletal muscle is less accessible to it, meaning the therapy has trouble getting into this type of muscle cell. Skeletal muscles poor response to ERT has been attributed to a serious lack of a protein receptor called cation-independent mannose-6-phosphate receptor (CI-MPR) on its cells.

Animal studies suggest that an active compound common to blood pressure medications (with work to control hypertension) could increase the uptake of ERT by muscle cells, by increasing the amount of muscle (muscle hypertrophy), and therefore the amount of CI-MPR.

Investigators atDuke Universityevaluated the effects of ERT with and without three anti-hypertensive agents: carvedilol, losartan, and propranolol. All these compounds have different ways of working, or mechanisms of action, in the body. They experimented using a mouse model of Pompe disease called the GAA knockout (absent) mouse.

Animals were assigned to one of seven groups: no treatment, ERT alone, ERT with carvedilol, ERT with losartan, ERT with propranolol, or to only losartan or carvedilol. Drugs were given to the mice in drinking water, and one week after treatment initiation, recombinant human GAA was given by injection every week for a month. Five days following the last GAA injection, scientists examined the animals cardiac and muscle function.

The team reported that carvedilol uniquely increased muscle strength, while losartan uniquely decreased heart rate. GAA activity was also found to be significantly higher in the heart following either losartan or propranolol being added to enzyme replacement therapy, compared to mice left untreated as a control group.

Both carvedilol or propranolol significantly increased GAA activity in the animals quadriceps, the muscles in the front of the thigh, compared to control mice. However, only carvedilol administration significantly increased GAA activity in quadriceps, in comparison with ERT alone, the scientists wrote.

These findings indicate that the greatest rise in enzymatic activity occurred in response to carvedilol, the active substance in the blood pressure medication Coreg. Carvedilol is a beta-blocker that relaxes the smooth muscle that makes up blood vessels, leading to an overall reduction in blood pressure.

Because more than half (seven of 13) of the mice given losartan, either alone or in combination with ERT, died during the study, researchers thought this active molecule potentially toxic in Pompe, and suggested physicians should be mindful of it when prescribing high blood pressure medications to Pompe patients.

Currently we demonstrated unique toxicity from the administration of losartan in mice with Pompe disease, the researchers wrote.

Because of the benefits seen in diseased micegiven carvedilol during ERT, they recommended the compound be studied in a clinical trial in patients.

Carvedilol was well-tolerated, and the ability to use a -blocker [beta-blocker] in patients that will not interfere with ERT would be highly valuable for clinical use in patients with Pompe since they often require a -blocker to mitigate disease-associated hypertension, the investigators concluded.

A clinical trial of carvedilol in patients with Pompe disease should be considered to further evaluate its usefulness.

With over three years of experience in the medical communications business, Catarina holds a BSc. in Biomedical Sciences and a MSc. in Neurosciences. Apart from writing, she has been involved in patient-oriented translational and clinical research.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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ERT to Treat Pompe May Work Better in Combo with Blood Pressure Medication, Study Says - Pompe Disease News

BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference – GlobeNewswire

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwn NurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

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

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PR Phone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference - GlobeNewswire

Stem Cell Therapy Market Will Achieve 10.2% CAGR to Cross $15bn by 2025: Global Market Insights, Inc. – State-Journal.com

Global Stem Cell Therapy Market value will achieve over 10% CAGR to surpass USD 15 billion by 2025; according to a new research report by Global Market Insights, Inc. Rapid advancements in stem cell therapies in developed region such as North America and Europe will boost industry growth. Key industry players are indulged in introducing novel stem cell therapies that address limitations of existing therapies. Significant efficiency possessed by stem cell therapy has increased its preference in treatment of cancer that should positively impact the industry growth. However, high cost associated with stem cell therapies may hinder stem cell therapy industry growth to certain extent.

Increasing prevalence of chronic disease such as cancer, cardiovascular diseases will surge the demand for stem cell therapy business. Stem cell therapies have unique properties, such as immunosuppression, secretion of bioactive factors that fosters the development of tumor targeting technologies. Introduction of innovations in the regenerative medicine will help in conquering challenges in combating numerous diseases that should foster the industry growth.

Allogenic segment of stem cell therapy market accounted for over 39.5% revenue in 2018. Significant growth is attributed to the advantages associated with allogenic stem cell therapies. Allogenic stem cell involves transfer of stem cell from donor to patients. It overrules the limitations of autologous stem cell therapy such as difficulty in obtaining healthy and sufficient amount of stem cells from patients of diabetes, rheumatoid arthritis and other chronic diseases. Therefore, people have started relying more on the allogenic regenerative therapies that should augment the segmental growth.

Cardiovascular segment of stem cell therapy market is anticipated to witness around 9.5% CAGR throughout the forecast time frame. Increasing incidence of cardiovascular disease and growing healthcare concerns will surge the demand for stem cells employed in cardiovascular diseases. According to WHO, every year 17.9 million people die of cardiovascular disease. Stem cell therapies possess enormous potential to replace the conventional cardiovascular treatments that should increase its adoption over the forecast period.

Stem Cell Therapy Market Statistics, By Application

1.2. Oncology 1.2.1. Market size, by region, 2014-2025 (USD Million)

1.3. Orthopedic 1.3.1. Market size, by region, 2014-2025 (USD Million)

1.4. Cardiovascular 1.4.1. Market size, by region, 2014-2025 (USD Million)

1.5. Neurology 1.5.1. Market size, by region, 2014-2025 (USD Million)

1.6. Others 1.6.1. Market size, by region, 2014-2025 (USD Million)

Browse key industry insights spread across 130 pages with 91 market data tables & 8 figures & charts from the report, Stem Cell Therapy Market Forecast 2019 - 2025 in detail along with the table of contents:

Clinics segment of stem cell therapy industry was valued at USD 2.5 billion in 2018. Considerable revenue size is attributed to superior treatment provided by clinics to cure life threating disease. Clinics are equipped with advance equipment and skilled workforce that ultimately enhance treatment outcomes. Moreover, clinics are believed to provide sophisticated stem cell therapies that will stimulate the segmental growth.

North America stem cell therapy market held over 9.5% CAGR in 2018 owing to favorable regulatory scenario and demographic trends. Improving regulatory scenario for stem cell therapies in the U.S. and Canada should positively impact the market growth. For instance, FDA has framed pre-marketing authorization rules for the commercialization of stem cell therapies. This helps to minimize adverse events caused due to defective regenerative therapies. Moreover, several companies focus on R&D activities to develop innovative stem cell therapies that should surge the regional growth.

Notable industry players operational in industry are Astellas Pharma, Cellectis, Celyad, DiscGenics, Gamida Cell, Capricor Therapeutics, Novadip Biosciences, Cellular Dynamics, CESCA Therapeutics, OxStem, ReNeuron Group, Mesoblast, and Takeda Pharmaceuticals. Industry players are adopting strategic initiatives such as collaborations product launches, geographic expansions, mergers and acquisitions in order to sustain industry competition and acquire prominent market. For instance, in 2019 Gamida cell formed agreement with Lonza to commercialize omidubicel after its FDA approval. Omiducel is potential life-saving stem cell treatment for treating hematologic malignancies. Thus, such collaborations will boost the companys growth considerably.

Bioreactors Market Growth Report 2025: Biopharmaceutical products are individualized products with highly specific manufacturing requirements. Advanced biopharmaceutical manufacturing technologies have enabled development of effective drug delivery systems and drug device combination products. Some of the key industry players operating in the market include Eppendorf, GE Healthcare, Merck Millipore, Sartorius, and Thermo Fisher Scientific.

About Global Market Insights

Global Market Insights, Inc., headquartered in Delaware, U.S., is a global market research and consulting service provider; offering syndicated and custom research reports along with growth consulting services. Our business intelligence and industry research reports offer clients with penetrative insights and actionable market data specially designed and presented to aid strategic decision making. These exhaustive reports are designed via a proprietary research methodology and are available for key industries such as chemicals, advanced materials, technology, renewable energy and biotechnology.

Arun Hegde Corporate Sales, USA Global Market Insights, Inc. Phone:1-302-846-7766 Toll Free:1-888-689-0688 Email: sales@gminsights.com

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United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo – Pharmaceutical Online

The build-out is estimated at $9.5M.

United Therapeutics received a building permit Tuesday for a $9.5M build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4M building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo - Pharmaceutical Online

American Academy of Stem Cell Physicians Announced Today That Their Safety Panel Session is Open and Free to the Public – PRNewswire

MIAMI, Oct. 24, 2019 /PRNewswire/ -- The AASCP has recently created guidelines thatare current safety recommendations given to physicians who are using biologics in their medical practice. A highly anticipated and sought after Safety StandardsPanel session, hosted by AASCP on Nov. 2, 2019, will be moderated by The Alliance for Cell Therapy Now,with President Ms. Janet Marchbrody.The sessions normally are closed to the public but this particular SafetyStandard Panel discussion will be open to the public, covering the growing safety concerns of the industry.

Alliance for Cell Therapy Now is a coalition of organizations representing patients, health care providers and the academic and scientific community, who are working together to advance safe and effective regenerative cell therapies. The mission is to advance the development, manufacturing and delivery of safe and effective regenerative cell therapies through policy development, consensus and advocacy. Alliance for Cell Therapy Now is bringing together experts and stakeholders to gain consensus on and advocate for policies that will advance the science and the field, including those focused on promoting clinical research, assuring the adoption of consensus standards to promote safety and quality, building capacity and expertise within the workforce, and establishing a national outcomes database to advance the science, promote improvements in quality and safety, and inform regulatory, paymentand patient decision-making.

Alliance for Cell Therapy Now is guided by an Advisory Board comprised of leaders in the scientific, academicand patient communities; Ms.Janet M. MarchibrodaPresident, Alliance for Cell Therapy Now Fellow, Bipartisan Policy Center Senior Vice President, Health Policy, Bockorny Group, has agreed to join theAASCP as a moderator for their SafetyPanelat The Hyatt Regency in Miami. This particular coveted safetypanel session will be open to the public and broadcast live on YouTube at 3:00 p.m. on Nov. 2, 2019.

According to AASCP, if you are using biologics in your practice, whether you are using SVF, PRP, bone marrow, UCB, amniotic products,exosomes,xenografts, or peptides, there are key considerations to take into account to achieve the best safety for your patients. The AASCP also recommends communication with the Chief Scientific Officer from the laboratory you work with.AASCP advises that just talking to a sales agent is not sufficient enough when determining the quality of products for your patients. Sales agents typically do not have a medical or scientific background.

The spokesman for the AASCP, Dr. AJFarshchian,said earlier: "The American Academy of Stem Cell Physicians is a group of physicians, scientists and researchers who collectively represent the most authoritativenon-federal group advocating for guidelines and education on stem cell therapy and regenerative medicine. AASCP members are experts within all fields of stem cell therapy from: SVF, BM, UCB, Exosomes, Peptides, Xenografts, Allografts and Amniotic Fluids and are considered the most experienced leaders for proper advocacy in the field. The AASCP is involved directly with other authorities within the field and seeks only to bring knowledge and awareness for the ever growing regenerative medicine industry.My hope is that the SafetyPanel discussion on Nov.2, 2019, is to help get rid of the bad actors that are damaging the field for everyone."

AASCP is hosting their medical conference in Miami on Nov. 1-3 , 2019. Sessions are normally closed to the public and, therefore, require registration. The conference is taking place at the downtown MiamiHyatt Regency, located at 400 SE 2nd Ave, Miami, FL 33131.Becauseof limited seating, we encourage everyone to please RSVP ataascp.net andto register.

The American Academy of Stem Cell Physicians (AASCP) is an organization created to advance research and the development of therapeutics in regenerative medicine, including diagnosis, treatmentand prevention of disease related to or occurring within the human body. Secondarily, the AASCP aims to serve as an educational resource for physicians, scientistsand the public in diseases that can be caused by physiological dysfunction that areameliorableto medical treatment.

For further information, please contact Marie Barbaat AASCP 305-891-4686 and you can also visit us at http://www.aascp.net.

Related Images

dr-farshchian-teaching-at-aaoscp.jpeg Dr. Farshchian teaching at AAOSCP workshop Dr. Alimorad Farshchian speaking at AASCP

Related Links

AASCP Safety guidelines

AASCP website / registration

SOURCE The American Academy of Stem Cell Physicians

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American Academy of Stem Cell Physicians Announced Today That Their Safety Panel Session is Open and Free to the Public - PRNewswire

Global Stem Cell and Regenerative Therapy Market – Yahoo Finance

Report Scope: The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries.

New York, Oct. 24, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Stem Cell and Regenerative Therapy Market" - https://www.reportlinker.com/p05791357/?utm_source=GNW The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

The report details market shares of stem cell and regenerative medicines based on products, application, and geography.Based on product the market is segmented into therapeutic products, cell banking, tools and reagents.

The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes: - 28 data tables - An overview of global markets for stem cell and regenerative medicines - Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 - Details of historic background and description of embryonic and adult stem cells - Information on stem cell banking and stem cell research - A look at the growing research & development activities in regenerative medicine - Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals - Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

Summary The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018.The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024.

Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018.The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period.

The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications.New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future.

The biopharmaceutical companies are investing significantly in cell-based therapeutics.The government organizations are funding research and development activities related to stem cell research.

These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments.The market is impacted through adoption of stem cell therapy.

The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.Read the full report: https://www.reportlinker.com/p05791357/?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 Stem Cell and Regenerative Therapy Market - Yahoo Finance

GeoVax Vaccine To Be Used in a Phase 1 Trial of Genetically Modified Autologous Cell Therapy for HIV – GlobeNewswire

IND Application Submitted to FDA for Phase 1 Trial

ATLANTA, GA, Oct. 24, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE GeoVax Labs, Inc. (OTCQB: GOVX) announced today its participation in a planned clinical trial led by researchers at American Gene Technologies (AGT) (www.americangene.com), to develop a therapy aimed at eliminating HIV from infected people.

On October 18, 2019, AGT announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for AGTs lead HIV program, AGT103-T, a lentiviral vector-based gene therapy. Upon clearance by the FDA, this IND will allow AGT to initiate a Phase 1 clinical trial that will investigate the safety of AGT103-T in humans, measure key biomarkers, and explore surrogate markers of efficacy. AGT expects to begin recruiting patients for the Phase 1 study in January.

Pursuant to its collaboration agreement with AGT, GeoVax will provide its novel MVA-VLP HIV vaccine (MVA62B) for evaluation in an arm of the clinical trial in combination with AGT103-T. T cells obtained from vaccinated individuals will be reprogrammed by AGTs lentivirus vector and infused back into the study participants as a therapeutic cell product. MVA62B is the boosting component for GeoVaxs preventive HIV vaccine (GOVX-B11) which has successfully completed a Phase 2a clinical trial and is awaiting funding in support of a Phase 2b pivotal trial.

Farshad Guirakhoo, PhD, GeoVaxs Chief Scientific Officer, commented, In a previous therapeutic Phase 1 clinical trial conducted by GeoVax, we demonstrated that our vaccine can potently stimulate production of both CD8+ and CD4+ T cells in HIV-positive individuals the intended use of the MVA62B vaccine in the proposed AGT therapeutic study. Our vaccine also has a well-documented safety profile in >500 humans. We are hopeful that the combination of technologies in these studies will yield a promising regimen that eliminates the HIV reservoir from people infected with the disease in the absence of antiretroviral drugs.

David Dodd, GeoVaxs President and CEO, stated, Finding a cure for HIV/AIDS remains an elusive but critical goal, and GeoVax is pleased to be a contributor to this important effort. While current antiretroviral therapy is effective at suppressing the viral load of HIV-infected individuals and preventing progression to AIDS, the long-term use of ART can lead to loss of drug effectiveness and can come with severe side effects. Additionally, the financial burden of drug and other medical costs to both the individual and to society at large is staggering, with average lifetime medical costs of treating an HIV-positive individual in the U.S. estimated to exceed $350,000.

About GeoVax

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing human vaccines against infectious diseases and cancer using a novel patented Modified Vaccinia Ankara-Virus Like Particle (MVA-VLP) based vaccine platform. On this platform, MVA, a large virus capable of carrying several vaccine antigens, expresses proteins that assemble into VLP immunogens within (in vivo) the person receiving the vaccine. The production of VLPs in the person being vaccinated mimics virus production in a natural infection, stimulating both the humoral and cellular arms of the immune system to recognize, prevent, and control the target infection. The MVA-VLP derived vaccines elicit durable immune responses in the host similar to a live-attenuated virus, while providing the safety characteristics of a replication-defective vector.

GeoVaxs current development programs are focused on preventive vaccines against HIV, Zika Virus, hemorrhagic fever viruses (Ebola, Sudan, Marburg, and Lassa), and malaria, as well as therapeutic vaccines against chronic Hepatitis B infections and multiple cancers. The Company has designed the leading preventative HIV vaccine candidate to fight against the subtype of HIV prevalent in the commercial markets of the Americas, Western Europe, Japan, and Australia; this program is currently undergoing human clinical trials managed by the HIV Vaccine Trials Network (HVTN) with the support of the National Institutes of Health (NIH). For more information, visitwww.geovax.com.

Forward-Looking Statements

Certain statements in this document are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These statements are based on management's current expectations and are subject to uncertainty and changes in circumstances. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax can develop and manufacture its vaccines with the desired characteristics in a timely manner, GeoVax's vaccines will be safe for human use, GeoVax's vaccines will effectively prevent targeted infections in humans, GeoVaxs vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete vaccine development, there is development of competitive products that may be more effective or easier to use than GeoVax's products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control. GeoVax assumes no obligation to update these forward-looking statements and does not intend to do so. More information about these factors is contained in GeoVax's filings with the Securities and Exchange Commission including those set forth at "Risk Factors" in GeoVax's Form 10-K.

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GeoVax Vaccine To Be Used in a Phase 1 Trial of Genetically Modified Autologous Cell Therapy for HIV - GlobeNewswire