Category Archives: Cell Medicine

Pulses of ultrasound aimed at a motion-sensitive brain area (from brown device, right) improved peoples ability to judge dots direction of motion.

By Kelly ServickJun. 24, 2020 , 1:30 PM

As a way to see inside the body, revealing a tumor or a fetus, ultrasound is tried and true. But neuroscientists have a newer ambition for the technology: tinkering with the brain. At frequencies lower than those of a sonogram but still beyond the range of human hearing, ultrasound can penetrate the skull and boost or suppress brain activity. If researchers can prove that ultrasound safely and predictably changes human brain function, it could become a powerful, noninvasive research tool and a new means of treating brain disorders.

How ultrasound works on the brain remains mysterious. But recent experiments have offered reassurance about safety, and small studies hint at meaningful effects in humansdampening pain, for example, or subtly enhancing perception. Ive seen a lot of tantalizing data, says Mark Cohen, a neuroscientist at the University of California, Los Angeles (UCLA). While the challenges are very large, the potential of this thing is so much larger that we really have to pursue it.

Scientists can already modulate the brain noninvasively by delivering electric current or magnetic pulses across the skull. The U.S. Food and Drug Administration (FDA) has approved transcranial magnetic stimulation (TMS) to treat depression, migraine pain, and obsessive-compulsive disorder (OCD).

But unlike magnetic or electric fields,sound waves can be focusedlike light through a magnifying glasson a point deep in the brain without affecting shallower tissue. For now, that combination of depth and focus is possible only with a surgically implanted wire. But ultrasound could temporarily disrupt a deep human brain regionthe almond-shaped amygdala, a driver of emotional responses, for example, or the thalamus, a relay station for pain and regulator of alertnessto test its function or treat disease.

Results in animals are encouraging. Experiments in the 1950s first showed ultrasound waves could suppress neural activity in a visual region of the cat brain. In rodents, aiming ultrasound at motor regions has triggered movements such as a twitch of a paw or whisker. And focusing it on a frontal region of monkey brains can change how the animals perform at eye movement tasks.

But its technically tricky to aim ultrasound through thick, dense skull bone and to show its energy has landed at the intended point. And ultrasounds effects on the brain can be hard to predict. How much it boosts or suppresses neural activity depends on many parameters, including the timing and intensity of ultrasound pulses, and even characteristics of the targeted neurons themselves. I have tremendous excitement about the potential, says Sarah Hollingsworth Lisanby, a psychiatrist at the National Institute of Mental Health who studies noninvasive neuromodulation. We also need to acknowledge that theres a lot we have to learn, she says.

For one thing, researchers are largely in the dark about how sound waves and brain cells interact. Thats the million-dollar question in this field, says Mikhail Shapiro, a biochemical engineer at the California Institute of Technology. At high intensities, ultrasound can heat up and kill brain cellsa feature neurosurgeons have exploited to burn away sections of brain responsible for tremors.

Even at intensities that dont significantly increase temperature, ultrasound exerts a mechanical force on cells. Some studies suggest this force alters ion channels on neurons, changing the cells likelihood of firing a signal to neighbors. If ultrasound works primarily via ion channels, Thats great news, Shapiro says, because that means we can look at where those channels are expressed and make some predictions about what cell types will be excited. In a preprint on bioRxiv last month, Shapiros team reported that exposing mouse neurons in a dish to ultrasound opens a particular set of calcium ion channels to render certain cells more excitable.

But these channels alone wont explain ultrasounds effects, says Seung-Schik Yoo, a neuroscientist at Harvard University. He notes that ultrasound also appears to affect receptors on nonneuronal brain cells called glia. Its very hard to [develop] any unifying theory about the exact mechanism of ultrasound, he says.

Regardless of mechanism, ultrasound is starting to show clear, if subtle, effects in humans. In 2014, a team at Virginia Polytechnic Institute and State University showed focused ultrasound could increase electrical activity in a sensory processing region of the human brain and improve participants ability to discern the number of points being touched on their fingers. Neurologist Christopher Butler at the University of Oxford and colleagues have tested ultrasound during a more complex sensory task: judging the motion of drifting, jiggling dots on a screen. Last month at the Cognitive Neuroscience Societys annual meeting online, he reported that stimulating a motion-processing visual region called MT improved subjects ability to judge which way the majority of the dots drifted.

Ultrasounds effects have so far been subtler than those of TMS, says Mark George, a psychiatrist at the Medical University of South Carolina, who helped develop and refine that technology. With TMS, you put it on your head and turn it on and your thumb moves, he says. But the ultrasound experiments that prompted paw twitches in mice used intensities so, so, so much higher than what were being allowed to use in humans.

Regulators have limited human studies in part because ultrasound has the potential to cook the brain or cause damage through cavitationthe creation of tiny bubbles in tissue. In 2015, Yoo and colleagues found microbleeds, a sign of blood vessel damage, in sheep brains repeatedly exposed to ultrasound. This was a huge speed bump, says Kim Butts Pauly, a biophysicist at Stanford University. But in February in Brain Stimulation, her group reported microbleeds in control animals as well, suggesting this damage might result from dissection of the brains. Butts Pauly and Yoo now say theyre confident the technology can be used safely.

Cohen and collaborators recently tested safety in people by aiming ultrasound at regions slated for surgical removal to treat epilepsy. With FDAs OK, they used intensities up to eight times as high as the limit for diagnostic ultrasound. As they reported in a preprint on medRxiv in April, they found no significant damage to brain tissue or blood vessels. However, to find the limit of safety, researchers will likely need to go all the way to levels that damage tissue, Cohen says.

Several teams are cautiously moving into tests of ultrasound as treatment. In 2016, UCLA neuroscientist Martin Monti and colleagues reported that a man in a minimally conscious state regained consciousness following ultrasound stimulation of his thalamus. Monti is preparing a publication on a follow-up study of three people with chronically impaired states of consciousness. After ultrasound, they showed increased responsiveness over a period of daysmuch faster than expected, Monti says, although the study included no control group.

That research and the tests in epilepsy patients used an ultrasound device developed by BrainSonix Corporation. Its founder, UCLA neuropsychiatrist Alexander Bystritsky, hopes ultrasound can disrupt neural circuits that drive symptoms of OCD. A team at Massachusetts General Hospital and Baylor College of Medicine is planning a study in humans using the BrainSonix device, he says.

Columbia University biomedical engineer Elisa Konofagou hopes to use ultrasound to treat Alzheimers disease. Before COVID-19 interrupted participant recruitment, she and colleagues were preparing a pilot study to inject tiny gas-filled bubbles into the bloodstream of six people with Alzheimers and use pulses of ultrasound to oscillate the microbubbles in blood vessels lining the brain. The mechanical force of those vibrations can temporarily pull apart the cells lining these vessels. The researchers hope opening this blood-brain barrier will help the brain clear toxic proteins. (Konofagous team and others are also exploring this ultrasound-microbubble combination to deliver drugs to the brain.)

In his first test of ultrasound after years of studying TMS, George looked to reduce pain. His team applied increasing heat to the arms of 19 participants, who tended to become more sensitive over repeated tests, reporting pain at lower temperatures by the last test. But if, between the first and last test, they had pulses of ultrasound aimed at the thalamus, their pain threshold dipped half as much. This is definitely a double green light to keep pursuing the technology, George says.

George regularly treats depressed patients with TMS and has seen the technology save lives. But everybody wonders if we could go deep with a different technologythat would be a game changer, he says. Ultrasound holds that promise, but the question is can it really deliver?

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Aiming ultrasound at the brain raises hope of new treatments - Science Magazine

It was once thought that the fat cell was fairly inactive, but we suspect that its active and controls a lot more than previously thought, says Niklas Mejhert, co-senior author of the paper and joint group leader with Rydn at the Department of Medicine, Huddinge, Karolinska Institutet. If we can regulate the accumulation of fat in a more controlled way, it could bring huge advantages.

The results of the study, which explain why adipose tissue becomes less effective and how lipolysis declines with age, are of interest to the efforts being made to find future treatments able to improve the function of adipose tissue.

We now plan to examine how different cells within the fat tissue are affected by age, continues Rydn. This is particularly interesting when it comes to stem cells, which have the unique and important ability to renew themselves and repair injury. Its something were keen to follow up on.

The study was conducted with grants from the Karolinska Institutet Strategic Research Programme in Diabetes, the Knut & Alice Wallenberg Foundation, the Swedish Research Council and the Novo Nordisk Foundation.

Age-Induced Reduction in Human Lipolysis: A Potential Role for Adipocyte Noradrenaline Degradation, Hui Gao, Peter Amer, Gallic Beauchef, Christelle Guere, Katell Vie, Ingrid Dahlman, Niklas Mejhert and Mikael Rydn. Cell Metabolism, online June 25, 2020, doi: 10.1016/j.cmet.2020.06.007

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Study gives insights into how human fat cells are affected by age - Mirage News

For someone with a food or drug allergy, the risk of life-threatening anaphylactic shock lurks around every corner. A new Northwestern Medicine study shows there might be a pill that can be taken proactively to prevent mild to life-threatening anaphylaxis, no matter the cause.Anaphylaxis is a severe, potentially life-threatening systemic allergic reaction that can occur within seconds or minutes of exposure to an allergen. It occurs in about one in 50 Americans, though many believe the rate is higher (closer to one in 20), according to the Asthma and Allergy Foundation of America. If a persons blood pressure drops so low during anaphylaxis or their airway closes up enough that they cant get enough oxygen to their organs, they enter anaphylactic shock.

The study used three different BTK inhibitors, which blocked allergic reactions when tested on human mast cells in a test tube. Additionally, the study used one U.S. Food and Drug Administration-approved oral drug, which successfully reduced or prevented allergic reactions, including severe, life-threatening anaphylactic reactions, in a new humanized mouse model of anaphylaxis. The mouses organs contained transplanted human cells that, over several months, matured into human mast cells, the primary cells that react during allergic reactions.

This would be the first known treatment to prevent anaphylaxis other than avoiding the allergen. The findings could pave the way for future human clinical trials of such oral drugs to be used as a preventive treatment to avoid serious allergic reactions, said senior and corresponding author Dr. Bruce Bochner, the Samuel M. Feinberg Professor of Medicine at Northwestern University Feinberg School of Medicine.

This pill could quite literally be life-changing and life-saving, Bochner said. Imagine being able to take medication proactively to prevent a serious allergic reaction.

Additionally, Bochner said people who are at high risk of allergic exposures to life-saving antibiotics or people about to undergo oral food desensitization (gradually eating foods to build up a threshold to an allergic reaction) could take the pill as a preventive measure. If such drugs turn out to be safe and cheap enough for daily use, theoretically anyone with a serious allergy, including food allergies, could take it and be able to eat the foods theyve been strictly avoiding, Bochner said.

For now, Bochner said the drug would likely be used preventatively, not for emergencies, like an EpiPen, which injects epinephrine into someone experiencing an allergic reaction to reverse the symptoms. But he and his team are considering exploring whether this sort of medication could be reformulated to be added to the EpiPen to be injected along with the epinephrine to see if it would better stop or abort anaphylaxis after it has begun.

Inhibition of skin tests is a kind of a surrogate test for whether the drug is actually working, Bochner said. So, one future goal is to give this medication to food- or drug-allergic subjects, show by skin testing that their allergic sensitivity has been blocked by the drugs effect and then give them the food or drug, expecting they will have little or no reaction.

BTK inhibitors are currently on the market for approximately $500 per day as a successful and less-toxic alternative to chemotherapy for patients with blood cancers like chronic lymphocytic leukemia and mantle cell lymphoma. They are not yet approved for use in children, who are more likely to have food allergies.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Food and Drug Allergy Anaphylaxis Could Be Prevented With a Pill - Technology Networks

Scientists have characterized the specific way SARS-CoV-2, which is the coronavirus that causes COVID-19, infects the nasal cavity to a great degree.

The new study published in the journal Cell suggests that the virus first tends to firmly establish itself in the nasal cavity, but it can then be aspirated into the lungs -- where it wreaks havoc and can lead to pneumonia.

Scientists at the UNC School of Medicine and the UNC Gillings School of Global Public Health believe their study supports the widespread wearing of masks as a way to prevent infection.

"If the nose is the dominant initial site from which lung infections are seeded, then the widespread use of masks to protect the nasal passages, as well as any therapeutic strategies that reduce virus in the nose, such as nasal irrigation or antiviral nasal sprays, could be beneficial," said study co-senior author Richard Boucher, a professor of medicine and director of the Marsico Lung Institute at the UNC School of Medicine, in a statement.

COVID-19 COULD BE SEASONAL ILLNESS THAT RETURNS AS HUMIDITY DECREASES, NEW STUDY SAYS

SARS-CoV-2 (red) infected ciliated cells in the COVID-19 patient's bronchi. (Takanori Asakura, PhD, UNC School of Medicine) (Takanori Asakura, PhD, UNC School of Medicine)

SARS-CoV-2, which first appeared in late 2019 in Wuhan, China and spread around the world, has now infected more than 6.4 million and killed 382,451. The U.S. accounts for almost one-third of the global infections and deaths.

"This is a landmark study that reveals new and unexpected insights into the mechanisms that regulate disease progression and severity following SARS-CoV-2 infection," said Ralph Baric, a professor of epidemiology at the UNC Gillings School of Public Health. "In addition, we describe a new reverse genetic platform for SARS-CoV-2 allowing us to produce key indicator viruses that will support national vaccine efforts designed to control the spread and severity of this terrible disease."

SOME SCIENTISTS FEAR 'SUPERSPREADERS' MAY PROMPT NEW COVID-19 OUTBREAKS AS STATES REOPEN

Scientists were hoping to gain a stronger understanding of which cells in the airways the virus infects and how it gets into patients' lungs when they develop pneumonia.

Researchers used different isolates of SARS-CoV-2 to see how efficiently they could infect cultured cells from different parts of the human airway for one of the study's experiments. They discovered a pattern of continuous variation, or gradient, from a relatively high infectivity of SARS-CoV-2 in cells lining the nasal passages, to less infectivity in cells lining the throat and bronchia, to relatively low infectivity in lung cells.

The scientists also found that ACE2, the cell surface receptor that the virus uses to get into cells, was more abundant on nasal-lining cells and less abundant on the surface of lower airway cells. This difference could explain, at least in part, why upper airway nasal-lining cells were more susceptible to infection.

The hypothesis that aspiration of oral contents into the lung is a strong contributor to COVID-19 pneumonia is consistent with observations that people at higher risk for severe lung disease -- the elderly, obese, and diabetic -- are more prone to aspiration (the process of drawing breath), especially at night.

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New map of SARS-CoV-2 infection in nasal cavity provides more support for wearing masks, researchers say - Fox News

Global Stem Cell Banking Market: Overview

The demand within the global stem cell banking market is growing on account of advancements in the field of regenerative medicine. The medical fraternity has become extremely focused towards the development of artificial tissues that can infuse with the human body. Furthermore, medical analysis and testing has gathered momentum across biological laboratories and research institutes. Henceforth, it is integral to develop stem cell samples and repositories that hold relevance in modern-day research. The need for regenerative medicine emerges from the growing incidence of internal tissue rupture. Certain types of tissues do not recover for several years, and may even be damaged permanently. Therefore, the need for stem cell banking is expected to grow at a significant pace.

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In a custom report, TMR Research digs into the factors that have aided the growth of the global stem cell banking market. The global stem cell banking market can be segmented on the basis of bank size, application, and region. The commendable developments that have incepted across the US healthcare industry has given a thrust to the growth of the North America stem cell banking market.

Global Stem Cell Banking Market: Notable Developments

The need for improved regenerative medication and anatomy has played an integral role in driving fresh developments within the stem cell banking market.

Gallant has emerged as a notable market entity that has remained as the torchbearer of innovation within the global stem cell banking market. The company has recently launched stem cell banking for dogs, and has attracted the attention of the masses. As people become increasingly concerned about their pets, the new move by Gallant shall help the company in earning the trust of the consumers. Moreover, it can move several notches higher on the innovation index.

Cells4Life has also remained at the forefront of developments within the global stem cell banking market. After suffering backlash for its error in cord blood stem cell promotion, the company is expected to use effective public relation strategies to regain its value in the market.

Global Stem Cell Banking Market: Growth Drivers

Development of improved facilities for storage of stem cells has played an integral role in driving market demand. Furthermore, the unprecedented demand for improved analysis of regenerative medications has also created new opportunities within the global stem cell banking market. Medical research has attracted investments from global investors and stakeholders. The tremendous level of resilience shown by biological researchers to develop stem cell samples has aided market growth. Henceforth, the total volume of revenues within the global stem cell banking market is slated to multiply.

Commercialization of stem cell banks has emerged as matter of concern for the healthcare industry. However, this trend has also helped in easy storage and procurement of cells stored during the yester years of children. Presence of sound procedures to register at stem cell banks, and the safety offered by these entities, has generated fresh demand within the global market. New regional territories are opening to the idea of stem cell banking. Several factors are responsible for the growth of this trend. Primarily, improvements in stem cell banking can have favourable impact on the growth of the healthcare industry. Moreover, the opportunities for revenue generation associated with the development of functional stem cell banks has aided regional market growth.

The global stem cell banking market is segmented on the basis of:

Source

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Stem Cell Banking Market Scope, Consumption and Opportunities Analysis 2018 2028 - Cole of Duty

First approvals for BRUKINSA in China and its first indication for relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma

BEIJING, China and CAMBRIDGE, Mass., June 03, 2020 (GLOBE NEWSWIRE) -- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, today announced that its BTK inhibitor BRUKINSA (zanubrutinib) has received approval from the China National Medical Products Administration (NMPA) in two indications the treatment of adult patients with chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL) who have received at least one prior therapy, and the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. Both new drug applications (NDAs) were previously granted priority review by the Center for Drug Evaluation (CDE) of the NMPA.

BRUKINSA received accelerated approval from the U.S. Food and Drug Administration (FDA) as a treatment for MCL in adult patients who have received at least one prior therapy in November 2019.

The concurrent approvals of BRUKINSA in R/R CLL/SLL and R/R MCL are a tribute to the collective expertise and hard work of the BeiGene team. With two product approvals covering four indications in China and one in the United States in merely seven months, we continue to focus on execution in advancing our broad portfolio, commented John V. Oyler, Co-Founder, Chief Executive Officer, and Chairman of BeiGene.

Following in the footsteps of tislelizumab, BRUKINSA is the second BeiGene internally-developed drug approved in China, another significant expansion in our growing commercial portfolio. These approvals would not have been possible without the many clinicians and patients who participated in our trials and the support of the health authorities, commented Xiaobin Wu, Ph.D., General Manager of China and President of BeiGene. We look forward to launching BRUKINSA in China, a potentially best-in-class BTK inhibitor with extensive global data from nine Phase 3 or potentially registration-enabling studies including a head-to-head comparison trial that was recently presented at the American Society of Clinical Oncology Annual Meeting.

The NMPA Approval in R/R CLL/SLL

The NMPA approval of BRUKINSA in patients with R/R CLL/SLL is based on results from a single-arm pivotal Phase 2 trial conducted in 91 patients (82 with R/R CLL; nine with R/R SLL) in China (NCT03206918; BGB-3111-205). Clinical efficacy data in the BRUKINSA label in China, as assessed by independent review committee (IRC) per iwCLL 2008 criteria for CLL and Lugano Classification 2014 for SLL, include an overall response rate (ORR) of 62.6%, including a complete response (CR) rate of 3.3%, a partial response (PR) rate of 59.3%, and the PR with lymphocytosis (PR-L) rate was 22%.

The most common adverse reactions reported in the label in China (10%) were neutropenia (68.1%), thrombocytopenia (40.7%), hematuria (35.2%), purpura (34.1%), anemia (23.1%), leukopenia (18.7%), pneumonia (18.7%), upper respiratory tract infection (15.4%), hemorrhage (14.3%), and rash (12.1%). The incidence of Grade 3 adverse reactions was 69.2%. The incidence of serious adverse reactions was 19.8%, and the most common serious adverse reaction was pneumonia (11.0%).

The approval of BRUKINSA will provide an important treatment option for Chinese patients with relapsed/refractory CLL/SLL. In addition to BRUKINSAs significant anti-tumor activity evidenced by an ORR of more than 60%, the drug demonstrated a favorable safety and tolerability profile, commented Jianyong Li, M.D., Professor and Director of the Department of Hematology and Director of the Pukou CLL Center at the First Affiliated Hospital of Nanjing Medical University.

The NMPA Approval in R/R MCL

The NMPA approval of BRUKINSA in patients with R/R MCL is based on results from a single-arm pivotal Phase 2 trial conducted in 86 patients in China (NCT03206970; BGB-3111-206). Clinical efficacy data in the BRUKINSA label in China, as assessed by IRC per Lugano Classification 2014, include the ORR of 83.7%, including a CR rate of 68.6% and a partial response PR rate of 15.1%.

The most common adverse reactions (10%) reported in the label in China were neutropenia (47.7%), rash (32.6%), leukopenia (31.4%), thrombocytopenia (30.2%), and anemia (11.6%). The incidence of serious adverse reactions was 15.1%, and common serious adverse reactions (2%) included pneumonia (8.1%), hemorrhage (2.3%), and thrombocytopenia (2.3%).

BRUKINSA has shown promise in hematologic malignancies including in patients with relapsed/refractory MCL. With robust results including a 68.6% CR rate in the MCL trial, we are optimistic and excited about the clinical benefits BRUKINSA can bring to these patients, said Jun Zhu, M.D., Ph.D., Professor and Director of the Department of Internal Medicine and Lymphoma, Peking University Cancer Hospital.

The recommended dose of BRUKINSA in Chinese Package Insert is 160 mg twice daily taken orally with or without food. The dose may be adjusted for adverse reactions, and reduced for patients with severe hepatic impairment and certain drug interactions.

About Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are forms of non-Hodgkin lymphoma, a type of blood cancer, that arise from B lymphocytes. CLL and SLL are essentially the same disease, with the only difference being the location where the cancer primarily occurs.i When most of the cancer cells are located in the peripheral blood and the bone marrow, the disease is referred to as CLL, although the lymph nodes and spleen are often involved. When the cancer cells are located mostly in the lymph nodes, the disease is called SLL.ii According to epidemiological statistics, 88,200 patients are newly diagnosed with lymphoma each year in China, with NHL accounting for approximately 91%. Among all the NHL incidences, patients with B-cell NHL account for 66%, and CLL or SLL accounts for approximately 6.4% of all B-cell NHL incidences, indicating that the number of patients with CLL/SLL reaches approximately 3,390 in all patients (88,200) newly diagnosed with lymphoma each year.iii

About Mantle Cell Lymphoma

Lymphoma is a diverse group of cancers that originate from B-, T- or NK-cells. MCL is typically an aggressive form of non-Hodgkins lymphoma (NHL) that arises from B-cells originating in the mantle zone.iv According to epidemiological statistics, 88,200 patients are newly diagnosed with lymphoma each year in China, with NHL accounting for approximately 91%. Among all the NHL incidences, patients with B-cell NHL account for 66%, and MCL accounts for approximately 5% of B-cell NHL incidences, equivalent to over 2,600 newly diagnosed cases each year.v MCL usually has a poor prognosis, with a median survival of three to four years,vi and is often diagnosed at a later stage of disease.

About BRUKINSA (zanubrutinib)

BRUKINSA (zanubrutinib) is a small molecule inhibitor of Brutons tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad pivotal clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies.

BRUKINSA was granted accelerated approval by the U.S. FDA to treat adult patients with MCL who have received at least one prior therapy in November 2019. This accelerated approval is based on overall response rate (ORR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. BRUKINSA was granted approval in China for the treatment of MCL in adult patients who have received at least one prior therapy and CLL or SLL in adult patients who have received at least one prior therapy in May 2020.

BRUKINSA is not approved for use outside of the United States and China.

IMPORTANT U.S. SAFETY INFORMATION FOR BRUKINSA (ZANUBRUTINIB)

Warnings and Precautions

Hemorrhage

Fatal and serious hemorrhagic events have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher bleeding events including intracranial and gastrointestinal hemorrhage, hematuria and hemothorax have been reported in 2% of patients treated with BRUKINSA monotherapy. Bleeding events of any grade, including purpura and petechiae, occurred in 50% of patients treated with BRUKINSA monotherapy.

Bleeding events have occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Co-administration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage.

Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding.

Infections

Fatal and serious infections (including bacterial, viral, or fungal) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher infections occurred in 23% of patients treated with BRUKINSA monotherapy. The most common Grade 3 or higher infection was pneumonia. Infections due to hepatitis B virus (HBV) reactivation have occurred.

Consider prophylaxis for herpes simplex virus, pneumocystis jiroveci pneumonia and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately.

Cytopenias

Grade 3 or 4 cytopenias, including neutropenia (27%), thrombocytopenia (10%) and anemia (8%) based on laboratory measurements, were reported in patients treated with BRUKINSA monotherapy.

Monitor complete blood counts during treatment and treat using growth factor or transfusions, as needed.

Second Primary Malignancies

Second primary malignancies, including non-skin carcinoma, have occurred in 9% of patients treated with BRUKINSA monotherapy. The most frequent second primary malignancy was skin cancer (basal cell carcinoma and squamous cell carcinoma of skin), reported in 6% of patients. Advise patients to use sun protection.

Cardiac Arrhythmias

Atrial fibrillation and atrial flutter have occurred in 2% of patients treated with BRUKINSA monotherapy. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher events were reported in 0.6% of patients treated with BRUKINSA monotherapy. Monitor signs and symptoms for atrial fibrillation and atrial flutter and manage as appropriate.

Embryo-Fetal Toxicity

Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity, including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for at least 1 week after the last dose. Advise men to avoid fathering a child during treatment and for at least 1 week after the last dose. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

Adverse Reactions

The most common adverse reactions in > 10% of patients who received BRUKINSA were neutrophil count decreased (53%), platelet count decreased (39%), upper respiratory tract infection (38%), white blood cell count decreased (30%), hemoglobin decreased (29%), rash (25%), bruising (23%), diarrhea (20%), cough (20%), musculoskeletal pain (19%), pneumonia (18%), urinary tract infection (13%), hematuria (12%), fatigue (11%), constipation (11%), and hemorrhage (10%). The most frequent serious adverse reactions were pneumonia (11%) and hemorrhage (5%).

Of the 118 patients with MCL treated with BRUKINSA, 8 (7%) patients discontinued treatment due to adverse reactions in the trials. The most frequent adverse reaction leading to treatment discontinuation was pneumonia (3.4%). One (0.8%) patient experienced an adverse reaction leading to dose reduction (hepatitis B).

Drug Interactions

CYP3A Inhibitors: When BRUKINSA is co-administered with a strong CYP3A inhibitor, reduce BRUKINSA dose to 80 mg once daily. For coadministration with a moderate CYP3A inhibitor, reduce BRUKINSA dose to 80 mg twice daily.

CYP3A Inducers: Avoid coadministration with moderate or strong CYP3A inducers.

Specific Populations

Hepatic Impairment: The recommended dose of BRUKINSA for patients with severe hepatic impairment is 80 mg orally twice daily.

INDICATION

BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Please see full U.S. Prescribing Information at beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at beigene.com/PDF/BRUKINSAUSPPI.pdf

About the Zanubrutinib Clinical Trial Program

Clinical trials of zanubrutinib include:

About BeiGene

BeiGene is a global, commercial-stage biotechnology company focused on discovering, developing, manufacturing, and commercializing innovative medicines to improve treatment outcomes and access for patients worldwide. Our 3,800+ employees in China, the United States, Australia, and Europe are committed to expediting the development of a diverse pipeline of novel therapeutics for cancer. We currently market two internally-discovered oncology products: BTK inhibitor BRUKINSA (zanubrutinib) in the United States, and anti-PD-1 antibody tislelizumab in China. We also market or plan to market in China additional oncology products licensed from Amgen Inc., Celgene Logistics Srl, a Bristol Myers Squibb (BMS) company, and EUSA Pharma. To learn more about BeiGene, please visit http://www.beigene.com and follow us on Twitter at @BeiGeneUSA.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeiGenes plans and expectations for the commercialization of BRUKINSA, the potential implications of clinical data for patients, and BeiGenes further advancement of, and anticipated clinical development, regulatory milestones and commercialization of BRUKINSA and its other products and product candidates. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on the Companys clinical development, commercial and other operations, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

Investor Contact Media Contact

Craig West Liza Heapes or Vivian Ni

+1 857-302-5189 + 1 857-302-7596

ir@beigene.com media@beigene.com

__________________________i Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma Fact Sheet, Lymphoma Research Foundation. Accessed at: https://www.lymphoma.org/wp-content/uploads/2018/04/LRF_FACTSHEET_CLL_SLL.pdf

ii Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma, Lymphoma Research Foundation. Accessed at: https://www.lymphoma.org/aboutlymphoma/cll/

iii Chen W, Zheng R , Baade P D , et al. Cancer statistics in China, 2015[J]. CA: A Cancer Journal for Clinicians, 2016, 66(2):115-132.

iv https://www.lls.org/sites/default/files/file_assets/mantlecelllymphoma.pdf

v Li, et al. Journal of Diagnostics Concepts & Practice, 2012,11(2): 111-115

vi Philip J. Bierman, James O. Armitage, in Goldman's Cecil Medicine (Twenty Fourth Edition), 2012.

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BeiGene Announces the Approval of BRUKINSA (Zanubrutinib) in China for Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia or Small...

A new treatment that helps to relieve coronavirus symptoms could be brought to market in three months' time if further trials go well, according to a researcher involved in the project.

"It's very early to say at this stage," said Dr.Fatima al-Kaabi, head of hematology and oncology at the Sheikh Khalifa Medical City in the United Arab Emirates.

"We've been happy that our initial safety results are promising, that's why we're heading into the next phase, of effectiveness of this treatment," she told CNBC's Hadley Gamble on Monday.

"If all ... went well and it worked well, then I would propose ... three months' time," she said, when asked how quickly the treatment, which was developed by doctors and researchersat the Abu Dhabi Stem Cell Center, could reach the market.

To date, there are no known vaccines or specific antiviral medicines against Covid-19.U.S. health officials say developing a vaccine will take at least 12 to 18 months.

The UAE has 14,163 cases and126 deaths due to the coronavirus, based on data from Johns Hopkins University.

The remedy uses a "minimally invasive" method where a Covid-19 patient's stem cells are extracted, activated and turned into a fine mist to be inhaled. This alleviates symptoms such as shortness of breath and possibly coughing, said Dr. al-Kaabi.

"It is hypothesized to have its therapeutic effect by regenerating lung cells and modulating the immune response to keep it from overreacting to the COVID-19 infection and causing further damage to healthy cells,"the UAE's ministry of health and preventionsaid a statement.

Some 73 patients with moderate to severe symptoms received this treatment, and all were "successfully treated and cured," the statement said, adding that none reported "immediate adverse effects." Around a quarter of these patients were intubated and in the intensive care unit.

The treatment was given along with "conventional medical intervention" and will not replace established protocols, according to the statement.

"We're hopeful," said Dr. al-Kaabi, noting that the results of further trials on the efficacy of the treatment will only be out a couple of weeks' time. "We've seen (a) favorable outcome."

Another treatment for the coronavirus, an antiviral drug from Gilead Sciences, has been in the spotlight following positive preliminary results from trials. America's Food and DrugAdministration granted the medicine emergency use authorization last week. That means doctors can administerremdesivir to patients hospitalized with Covid-19, even though the drug has not undergone the same FDA review as other treatments.

Separately, researchers cut short a study testing anti-malaria drug chloroquine as a potential Covid-19 treatment last month. The drug gained widespread international attention after two small studies published in France found the coronavirus infection cleared a lot faster for patients taking it when compared to a control group.

However, citing a high risk of death, scientists have now scrapped the trials, warning it should prompt some degree of skepticism from the public toward enthusiastic claims of the drug. President Donald Trump had touted chloroquine as a potential "game changer" in the fight against the virus.

CNBC'sBerkeley Lovelace Jr. andWilliam Feuer contributed to this report.

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Stem cell treatment in the UAE sees 'favorable' outcomes for coronavirus patients - CNBC

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, today announced that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), were published online via the ARVOLearn platform as part of the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting. The presentation entitled, Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results (Abstract # 3363764), was presented by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine. Dr. Riemanns presentation is available on the Media page of the Lineage website. Lineage will also host a live call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results of treatment with OpRegen. Interested parties can access the call on the Events and Presentations section of Lineages website.

This update is significant as it builds on our earlier reports of gains in visual acuity and provides a more comprehensive picture of treatment with OpRegen for dry AMD, with meaningful improvements in the progression of geographic atrophy, visual acuity, and reading speed observed in our first Cohort 4 patient and first Orbit SDS with thaw-and-inject formulation dosed patient, stated Brian M. Culley, Lineage CEO. As dry AMD is a slow and progressive disease, it takes many months to observe changes to retinal anatomy or visual acuity. With the benefit of longer follow-up, we now can report that some OpRegen treated patients are able to see better, have less growth in their area of GA, and are able to read faster, all of which represent significant enhancements to vision and quality of life metrics. In addition to these individual results, the pooled data continues to suggest a treatment effect in both visual acuity and GA progression. Notably, we also are reporting additional evidence that OpRegen cells remain present for at least 4 years and hope that longer follow-up periods will reinforce a growing body of evidence that OpRegen is well-tolerated and can provide sustained and clinically meaningful benefits with a single dose of RPE cells. Our near-term objective is to treat and monitor the final four patients in Cohort 4 of the current study and utilize these data to direct our clinical, regulatory, and partnership discussions. Our goal is to combine the best cell line, the best production process, and the best delivery system, to position OpRegen as the front-runner in the race to address the unmet need in the potential billion-dollar dry AMD market.

As a principal investigator on the OpRegen clinical study, I am excited to present this most recent update, where all Cohort 4 patients treated with OpRegen had improved Best Corrected Visual Acuity up to one year or at their last visit, demonstrating a substantial treatment response, stated Christopher D. Riemann, M.D. The pooled Cohort 4 data demonstrate a significant, greater than 10-letter sustained visual acuity improvement over the entire followup period. Reading center assessments of GA also suggest a reduction in GA progression in the OpRegen treated eye when compared to fellow eye in Cohort 4. I am encouraged by the results observed in patients treated to date with OpRegen and I look forward to dosing patients in this study at CEI.

KOL Call Information and Webcast

Lineage will host a conference call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results following treatment with OpRegen. A live webcast of the conference call will be available online in the Events and Presentations section of Lineages website. Interested parties may also access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through May 19, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 6597936.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC2, a cancer immunotherapy of antigen-presenting dendritic cells in Phase 1 development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to Lineages objectives with respect to OpRegen. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 12, 2020 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Originally posted here:
Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy - Business Wire

Dublin, May 05, 2020 (GLOBE NEWSWIRE) -- The "Cell Expansion Market to 2027 - Global Analysis and Forecasts By Product; Cell Type; Application; End User, and Geography" report has been added to ResearchAndMarkets.com's offering.

The global cell expansion market is projected to reach US$ 42,837.11 Mn in 2027 from US$ 11,929.43 Mn in 2018. The cell expansion market is expected to grow with a CAGR of 15.6% from 2019-2027.

Driving factors include increasing adoption of regenerative medicines, rising prevalence of cancer. However, the risk contamination during cell expansion is expected to hamper the market during the forecast period.

Cancer is one of the major cause of human death worldwide. In recent years, the cases of cancer have been increasing tremendously and the trend is anticipated to remain the same in the upcoming years. According to the World Health Organization in 2018, approximately 9.6 million deaths across the globe were due to cancer. Furthermore, the National Cancer Institute predicted that in 2018, approximately 1,735,350 new cancer cases would be diagnosed in the US.

Changes in lifestyle have resulted in more exposure to oncogenic factors. Cancer can be cured if diagnosed and treated at an initial stage. Cancer sequencing using next-generation sequencing (NGS) methods provides more information. Additionally, cell expansion related procedures also aids in research, diagnostics and treatment of cancer.

Furthermore, Asia Pacific region is also facing the problem of the growing prevalence of cancer. The top 15 countries with Cancer prevalence are Japan, Taiwan, Singapore, South Korea, Malaysia, Thailand, China, Philippines, Sri Lanka, Vietnam, Indonesia, Mongolia, India, Laos, and Cambodia. According to the National Institute of Cancer Prevention and Research (NICPR), in 2018, in India, total deaths due to cancer were 784,821.

The global Cell Expansion market is segmented by product, cell type, application, end user. Based on product, the cell expansion market is segmented into consumables and instruments. In 2018, the consumables accounted for the largest market share in the global cell expansion market by product. These consumables are essential components of any laboratory experiment hence they are expected to witness significant growth during the forecast period. Based on cell type, the cell expansion market has been segmented into human cell and animal cell. Furthermore based on application the cell expansion market has been segmented into Regenerative Medicine And Stem Cell Research, Cancer And Cell-Based Research and Other Applications. Based in end user market is segmented into Biopharmaceutical And Biotechnology Companies, Research Institutes, cell banks and others.

Some of the essential primary and secondary sources included in the report are the National Institute of Cancer Prevention and Research (NICPR), Association for Management Education and Development, Center for Cancer Research, International Society for Stem Cell Research (ISSCR), American Association of Blood Banks (AABB), National Institute of Cancer Prevention and Research and others.

Reasons to Buy

Key Topics Covered:

1. Introduction

2. Cell Expansion Market - Key Takeaways

3. Research Methodology

4. Cell Expansion- Market Landscape4.1 Overview4.2 PEST Analysis4.3 Expert Opinions

5. Global Cell Expansion Market - Key Market Dynamics5.1 Key Market Drivers5.1.1 Increasing Adoption of Regenerative Medicines5.1.2 Rising Prevalence of Cancer5.2 Key Restraints5.2.1 Risk Contamination During Cell Expansion5.3 Key Opportunity5.3.1 Middle Income Countries Creating Development Opportunities5.4 Future Trend5.4.1 Consistent Research in Drug Discovery Activities5.5 Impact Analysis

6. Cell Expansion Market - Global Analysis6.1 Global Cell Expansion Market Revenue Forecasts And Analysis6.2 Global Cell Expansion Market, By Geography - Forecasts And Analysis6.3 Market Positioning Of Key Players

7. Cell Expansion Market - Revenue And Forecasts To 2027 - Product7.1 Overview7.2 Global Cell Expansion Market, by Product , 2018 & 2027 (% Share)7.3 Consumables7.3.1 Overview7.3.2 Global Consumables Market Revenue and Forecast to 2027 (US$ Mn)7.3.3 Reagents, Media & Serum7.3.3.1 Overview7.3.3.2 Global Reagents, Media & Serum Market Revenue and Forecast to 2027 (US$ Mn)7.3.4 Disposables7.3.4.1 Overview7.3.4.2 Global Disposables Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.3 Culture Tissue Flasks7.3.4.3.1 Overview7.3.4.3.2 Global Culture Tissue Flasks Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.4 Bioreactor Accessories7.3.4.4.1 Overview7.3.4.4.2 Global Bioreactor Accessories Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.5 Other Disposables7.3.4.5.1 Overview7.3.4.5.2 Global Other Disposables Market Revenue and Forecast to 2027 (US$ Mn)7.4 Instruments7.4.1 Overview7.4.2 Global Instruments Market Revenue and Forecast to 2027 (US$ Mn)7.4.3 Cell Expansion Supporting Equipment7.4.3.1 Overview7.4.3.2 Global Cell Expansion Supporting Equipment Market Revenue and Forecast to 2027 (US$ Mn)7.4.4 Bioreactors7.4.4.1 Overview7.4.4.2 Global Bioreactors Market Revenue and Forecast to 2027 (US$ Mn)7.4.5 Automated Cell Expansion Systems7.4.5.1 Overview7.4.5.2 Global Automated Cell Expansion Systems Market Revenue and Forecast to 2027 (US$ Mn)

8. Cell Expansion Market Analysis and Forecasts to 2027 - Cell Type8.1 Overview8.2 Global Cell Expansion Market, by Cell Type, 2018 & 2027 (% Share)8.3 Human Cells8.3.1 Overview8.3.2 Global Human Cell Market Revenue and Forecast to 2027 (US$ Mn)8.3.3 Adult Stem Cells8.3.3.1 Overview8.3.3.2 Global Adult Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.4 Induced Pluripotent Stem Cells8.3.4.1 Overview8.3.4.2 Global Induced Pluripotent Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.5 Embryonic Stem Cells8.3.5.1 Overview8.3.5.2 Global Embryonic Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.6 Differentiated Cells8.3.6.1 Overview8.3.6.2 Global Differentiated Cells Market Revenue and Forecast to 2027 (US$ Mn)8.4 Animal Cells8.4.1 Overview8.4.2 Global Animal Cell Market Revenue and Forecast to 2027 (US$ Mn)

9. Cell Expansion Market Analysis And Forecasts To 2027 - Application9.1 Overview9.2 Global Cell Expansion Market Share by Application 2018 & 2027 (%)9.3 Regenerative Medicine And Stem Cell Research9.3.1 Overview9.3.2 Global Regenerative Medicine And Stem Cell Research Market Revenue and Forecast to 2027 (US$ Mn)9.4 Cancer And Cell-Based Research9.4.1 Overview9.4.2 Global Cancer And Cell-Based research Market Revenue and Forecast to 2027 (US$ Mn)9.5 Other Applications9.5.1 Overview9.5.2 Global Other Applications Market Revenue and Forecast to 2027 (US$ Mn)

10. Cell Expansion Market Analysis And Forecasts To 2027 - End User10.1 Overview10.2 Global Cell Expansion Market Share by End User 2018 & 2027 (%)10.3 Biopharmaceutical And Biotechnology Companies10.3.1 Overview10.3.2 Global Biopharmaceutical And Biotechnology Companies Market Revenue and Forecast to 2027 (US$ Mn)10.4 Research Institutes10.4.1 Overview10.4.2 Global Research Institutes Market Revenue and Forecast to 2027 (US$ Mn)10.5 Cell Banks10.5.1 Overview10.5.2 Global Cell Banks Market Revenue and Forecast to 2027 (US$ Mn)10.6 Other End Users10.6.1 Overview10.6.2 Global Other End Users Market Revenue and Forecast to 2027 (US$ Mn)

11. Cell Expansion Market - Geographic Analysis11.1 North America Cell Expansion Market, Revenue and Forecast to 202711.2 Europe Cell Expansion Market, Revenue and Forecast to 202711.3 APAC Cell Expansion Market, Revenue and Forecast to 202711.4 MEA Cell Expansion Market, Revenue and Forecast to 202711.5 South and Central America Cell Expansion Market, Revenue and Forecast to 2027

12. Cell Expansion Market - Industry Landscape12.1 Overview12.2 Growth Strategies In The Cell Expansion Market, 2017-201912.3 Organic Growth Strategies12.3.1 Overview12.3.1.1 Recent Organic Developments By Players In The Cell Expansion Market12.4 Inorganic Growth Strategies12.4.1 Overview12.4.2 Recent Developments By Players In The Cell Expansion Market

13. Global Cell Expansion Market-Key Company Profiles13.1 BD13.1.1 Key Facts13.1.2 Business Description13.1.3 Financial Overview13.1.4 Product Portfolio13.1.5 SWOT Analysis13.1.6 Key Developments13.2 Merck KGaA13.3 Thermo Fisher Scientific, Inc.13.4 Terumo Corporation13.5 General Electric Company13.6 Corning Incorporated13.7 Miltenyi Biotec13.8 Danaher13.9 Lonza13.10 STEMCELL Technologies, Inc.

14. Appendix14.1 About the Publisher14.2 Glossary Of Terms

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

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Insights on the Worldwide Cell Expansion Industry to 2027 - Analysis and Forecasts - GlobeNewswire

CRANBURY, N.J., May 06, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases today announced the acceptance of several abstracts for presentation at the American Society of Gene & Cell Therapy 23rd Annual Meeting being held virtually on May 12 15. Preclinical data from its Pompe gene therapy program, which Amicus is developing with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania, will be presented as an oral presentation. Preclinical data related to the CLN6 and CLN8 Batten disease programs, with our partners at Sanford Research and Nationwide Childrens Hospital, will be presented in respective posters.

Oral Platform Presentation: Thursday, May 14, 2020,4:45-5:00p.m. ET

Pompe Disease:

Poster Session: Tuesday, May 12, 2020, 5:30-6:30 p.m. ET

CLN6 Batten Disease:

Poster Session: Wednesday, May 13, 2020, 5:30-6:30 p.m. ET

CLN8 Batten Disease:

All abstracts for the American Society of Gene & Cell Therapy 23rd Annual Meeting are now available online.

About Pompe DiseasePompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which results in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

About Batten DiseaseBatten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cells ability to recycle certain molecules. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.

Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding, and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment, and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden, and die prematurely.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans or projections will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical products; and the potential that we will need additional funding to complete all of our studies and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2019. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors/Media:Amicus TherapeuticsAndrew FaughnanDirector, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

FOLDG

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UPDATE Amicus Therapeutics Announces Upcoming Presentations at the American Society of Gene & Cell Therapy 23rd Annual Meeting - GlobeNewswire