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BrainStorm Cell Therapeutics President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 – Yahoo Finance

NEW YORK, Oct. 24, 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, Chaim Lebovits, President and CEO, will serve as a Keynote Speaker at Cell Series UK.Cell Series UK, will be held October 29-30, 2019, at London Novotel West, London, UK. The Conference, organized by Oxford Global, is one of the foremost events in Europe focused on regenerative medicine and cellular innovation.

Ralph Kern MD, MHSc, Chief Operating and Chief Medical Officer of Brainstorm, who will also participate at Cell Series UK stated, We are very pleased to have Chaim Lebovits presenting at this prestigious conference where global leaders in stem cell and regenerative medicine will have the opportunity to learn more about NurOwn and the critical research being conducted by the Company. Mr. Lebovits Keynote Address, Stem Cell Therapeutic Approaches For ALS, will be presented to leading members of the scientific and business community including potential partners and investors.

About NurOwnNurOwn (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.

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BrainStorm Cell Therapeutics President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 - Yahoo Finance

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

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