A 3D printer to produce human organs in space? Discover the experiments taking place in zero-gravity

Source Euronews Euronews space correspondent and European Space Agency (ESA) astronaut Luca Parmitano has been in space for a month now. His first mission was to “catch a Dragon” – a cargo vehicle which brings payloads for astronauts onboard the International Space Station (ISS). Once the dragon was “captured”, Luca and his team received new scientific equipment to start working on

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Health Technology Assessment of Gene Therapies in Europe and the USA: Analysis and Future Considerations

Gene therapies constitute a new concept of transformative therapies, administered once in a lifetime. The value assessment of these innovative therapies constitutes a challenge for health technology assessment (HTA) bodies. The HTA reports for all seven gene therapies that have to date been granted a market authorization in the European Union (EU) and/or the United

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Heart regeneration: Scientists uncloak one of nature’s deepest secrets

Scientists at the University of Texas Southwestern have uncloaked the molecular mechanisms underlying heart regeneration in the only mammals currently known to regrow a significant portion of their hearts after injury: neonatal mice. These newborns have long held the secret to regenerating approximately 15 percent of their ventricular tissue, a capability that occurs within a fleeting window of about

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Gene Therapy Shows Promise for AADC Deficiency But Requires Early Diagnosis, Study Says

Early clinical trials of a gene therapy for aromatic amino acid decarboxylase (AADC) deficiency have yielded encouraging results for its safety and efficacy, a study highlights. But researchers call attention to the importance of a timely diagnosis for treatment success, as only early interventions could fully prevent long-term brain damage and other outcomes. Their study, “Aromatic amino

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Immunotherapy for Pediatric Solid Tumors: What’s the Latest?

Immunotherapy can be effective in treating certain types of pediatric blood cancers, but researchers are still exploring how this type of treatment could work for pediatric solid tumors. In clinical trials, scientists are combining immunotherapy agents in an effort to jump-start the immune system against pediatric solid tumors. Source DANA-FARBER Cancer Institute New treatments that

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3D printed salt template for bioresorbable bone implants

With the help of a 3D printed salt template, ETH researchers have succeeded in producing magnesium scaffolds with structured porosity that are suitable for bioresorbable bone implants. For the treatment of complex bone fractures or even missing bone parts, surgeons typically deploy metal implants. In this context, an attractive alternative to the traditional materials like

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A robo-thread wiggles through some of the body’s most intricate spaces

Robots that are roughly half a millimetre wide veer around sharp corners and down tiny corridors. Source Nature A thread-shaped robot can worm through narrow spaces under the control of magnetic fields, raising hopes of its use in convoluted human organs. Doctors use snake-like robots in procedures on the heart, but these commercially available devices

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Using Electroporation and continuous-flow electrotransfection to Streamline Manufacturing of Next-Generation Cellular Therapies

A promising new treatment for cancer patients has received approval from the Centers for Medicare and Medicaid Services, paving the way for more patients to get the treatment known as CAR T-cell therapy. While this decision will likely expand access, particularly for some lymphoma and leukemia patients, treatment remains prohibitively expensive, prompting some patients to opt for experimental rather

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Deep learning enables scientists to identify cancer cells in blood in milliseconds

Technique could allow cells to be extracted in real time, help prevent cancer from spreading Researchers at UCLA and NantWorks have developed an artificial intelligence-powered device that detects cancer cells in a few milliseconds — hundreds of times faster than previous methods. With that speed, the invention could make it possible to extract cancer cells from blood immediately after

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$9 million grant funds trial of CAR T-cell therapy for metastatic breast cancer

California Institute for Regenerative Medicine awarded a $9.28 million grant will support a clinical trial at City of Hope to evaluate chimeric antigen receptor T-cell therapy for patients with HER2-positive breast cancer that spread to the brain. About 20% of breast cancers are HER2-positive, and nearly half of women with this type of breast cancer develop brain

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Now Available: ARM’s Searchable, Downloadable Database of Near-Term Anticipated RM/AT Data Readouts and Other Clinical Events

This new resource provides clear, simplified information on indication-, technology-, or company-specific upcoming data readouts and other clinical events. ARM has carefully curated all entries to ensure they fall within our narrowly defined criteria for regenerative medicines, and includes many milestones which are not reported in other clinical trial datasets, including anticipated product approvals. Currently, the database includes 188 anticipated events and includes a search-and-download function, and

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Superstar Athletes Popularize Unproven Stem Cell Procedures

Treatments for baseball pitcher Max Scherzer and other pros may mislead fans about costly, controversial, unapproved stem cell shots. Source KHN Baseball superstar Max Scherzer — whose back injury has prevented him from pitching for the Washington Nationals since he last played  on July 25 — is the latest in a long list of professional athletes to embrace unproven stem cell

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Centers for Medicare & Medicaid Services (CMS) Make CAR T-Cell Cancer Therapy Available to Medicare Beneficiaries Nationwide

Decision ensures consistency in access to the innovative new cancer therapy, and CMS is working closely with sister agencies to monitor outcomes for patients receiving the therapy Today the Centers for Medicare & Medicaid Services (CMS), under the leadership of President Trump and Secretary Azar, finalized the decision to cover FDA-approved Chimeric Antigen Receptor T-cell, or “CAR

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Masters of Science (MSc) degree in ATMPs has opened to enrolment at the University of Manchester

Under the guidance of the iMATCH team,  a new Masters of Science (MSc) degree in Advanced Therapy Medicinal Products (ATMPs) has opened to enrolment at the University of Manchester. This course is believed to be a world first focussing on this rapidly evolving area of medicine which is increasingly being used to treat a broad

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RESTORE Large Scale Research Initiative presents 1st Advanced Therapies Science Meeting

Shifting from treating symptoms to curing chronic diseases by making the transformative promise of Advanced Therapies a reality for the benefit of patients, that is the vision of the international project RESTORE Health by Advanced Therapies. For genetic diseases, immune diseases, cancer and tissue injury potential cures through Advanced Therapies exist – they are reality,

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bluebird bio Announces EU Conditional Marketing Authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype

ZYNTEGLO is the first gene therapy approved for transfusion-dependent β-thalassemia (TDT) European marketing authorization for ZYNTEGLO follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program ZYNTEGLO is bluebird bio’s first gene therapy to gain regulatory approval bluebird bio, Inc. announced today that the European Commission (EC)

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Are industry-funded charities promoting “advocacy-led studies” or “evidence-based science”?: a case study of the International Life Sciences Institute

Source The Guardian International Life Sciences Institute used by corporate backers to counter public health policies, says study An institute whose experts have occupied key positions on EU and UN regulatory panels is, in reality, an industry lobby group that masquerades as a scientific health charity, according to a peer-reviewed study. The Washington-based International Life Sciences

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Advanced Therapies News

Source ARM Organovo Collaborates With MCRI and Leiden University Medical Center to Develop Stem Cell-Based Bioprinted Tissue Treatments for Kidney DiseaseMay 30, 2019 – (Organovo) – This multi-organizational effort integrates Organovo’s leading bioprinting platform with MCRI’s advanced stem cell differentiation technology and LUMC’s cell lines and clinical expertise.  The collaboration has been made possible through generous

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Advanced therapies and carT cells in the next IMI2 calls

Future topics To give potential applicants as much time as possible to form consortia and prepare their proposals, IMI regularly publishes information on possible future topics well in advance of the official Call launch. To ensure you get the latest information on forthcoming Calls, sign up to our newsletter, follow us on Twitter, or join our LinkedIn group.

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ICER Comments on the FDA Approval of Zolgensma for the Treatment of Spinal Muscular Atrophy

In light of additional data from ongoing trials of onasemnogene abeparvovec (Zolgensma®, Novartis/AveXis), as well as the treatment’s final FDA label and just-announced launch price, the Institute for Clinical and Economic Review (ICER) today published an addendum to its Final Evidence Report on treatments for spinal muscular atrophy (SMA). Since the publication of ICER’s Final Evidence Report on

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Vascularized Kidney Tissue Engineered by WFIRM Scientists

Wake Forest Institute for Regenerative Medicine (WFIRM) researchers have shown the feasibility of bioengineering vascularized functional renal tissues for kidney regeneration, developing a partial augmentation strategy that may be a more feasible and practical approach than creating whole organs. In the proof-of-concept study published online this month in Acta Biomaterialia journal, the scientists created a novel

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5G In Healthcare

Source: TMF The next telecommunication revolution is just around the corner: the promises of high bandwidth, low latency and low-power-low-cost of 5G will open the gate to a flood of new inventions and the implementation of ideas, which have already been for long in the public consciousness – such as stable augmented reality or truly

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Most Healthcare, Fitness Tracker, And Wellness Apps Are Not Covered By HIPAA And HHS’s New Faqs Makes That Clear

Individuals who use healthcare apps such as fitness trackers, weight loss, wellness, exercise, etc., BEWARE!A couple of recent developments have highlighted the fact that most apps are not subject to HIPAA, which means that with broadly-worded privacy policy these healthcare apps can and do readily share healthcare and other data collected by the apps with third

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Artificial intelligence could select heart failure patients for expensive treatment

Machine learning algorithm predicts sudden death in heart failure patients for the first time Artificial intelligence (AI) has shown promise to select heart failure patients for expensive treatments to prevent lethal arrhythmias, reports a study presented today at ICNC 2019. (1) The study is the first to use a machine learning algorithm to predict sudden

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German Ethics Council: germline interventions currently too risky, but not ethically out of the question

Last year, the birth of the first genetically modified babies shook the world. The German Ethics Council now presents a comprehensive ethical investigation into possible interventions in the genome of human embryos or germ cells. The Council does not deem the human germline to be inviolable. It does, however, consider germline interventions to be ethically

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JAMA Counteracting Health Misinformation A Role for Medical Journals?

A recent editorial in the Journal of the American Medical Association (JAMA) addresses the growing problem of medical misinformation, such as anti-vaccine views, fake treatments, unproven alternative products and services, and others. The authors, Armstrong and Naylor, make some good recommendations, but unfortunately are about 20 years behind the times when it comes to confronting scientific misinformation.

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USC-led advance in groundbreaking cancer treatment eliminates severe side effects

An advance in the groundbreaking cancer treatment known as CAR T-cell therapy appears to eliminate its severe side effects, making the treatment safer and potentially available in outpatient settings, a new USC study shows. “This is a major improvement,” said Si-Yi Chen of the USC Norris Comprehensive Cancer Center, professor in the Department of Molecular Microbiology

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Synthetic Speech Generated from Brain Recordings

A state-of-the-art brain-machine interface created by UC San Francisco neuroscientists can generate natural-sounding synthetic speech by using brain activity to control a virtual vocal tract – an anatomically detailed computer simulation including the lips, jaw, tongue and larynx. The study was conducted in research participants with intact speech, but the technology could one day restore the

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UK named the ‘heartland of European healthcare AI’ in new report

Germany and France are two flourishing AI hubs, but the UK has been named the European AI “powerhouse” in a new report from London-based investment firm MMC Ventures. Published earlier this month, the analysis sheds light on the hype around the technology, but identifies healthcare as an area of increasing focus for AI entrepreneurs as systems

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Augmented Reality: The Future of Medicine

Augmented Reality can change brain surgery thanks to powerful diagnostic platforms, revolutionize radiology, and open new doors to reconstructive surgery. Augmented Reality (AR), also known as spatial computing –a merging of digital and physical spaces,– is one of the current technology trends that, together with Virtual Reality (VR) and Mixed Reality (MR), is changing all industries, includinghealthcare and medical education. 

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Pig brains kept alive outside body for hours after death

Researchers have developed a high-tech support system that can keep a large mammalian brain from rapidly decomposing in the hours after death, enabling study of certain molecular and cellular functions. With funding through the National Institutes of Health BRAIN Initiative, researchers developed a way to deliver an artificial blood supply to the isolated postmortem brain of

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Gene therapy restores immunity in infants with rare immunodeficiency disease

NIH scientists and funding contributed to development of experimental treatment A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined

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Two Patients Treated with CRISPRed Cells in Immunotherapy Trial

Researchers have infused cells edited using CRISPR-Cas9 into two patients in a trial conducted at the University of Pennsylvania, NPR reports today (April 16). A university spokesperson confirmed in an emailed statement to The Scientist that the trial is underway and that two patients, one with multiple myeloma and one with sarcoma, have been treated so far. The study is

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Study: Half of people on statins don’t hit healthy cholesterol levels

Cardiovascular disease (CVD) remains the in the United States. And one of the most effective preventive measures to lower the risk of a CVD event are statins — a class of cholesterol-lowering medications. It’s well established that statins save lives, but a recent study finds that even after two years, half of all people prescribed statins don’t achieve healthy cholesterol levels.

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RESTORE Consortium – CURING CHRONIC DISEASES BY “LIVING DRUGS”

OVERVIEW Shifting from treating symptoms to curing chronic diseases by making the transformative promise of Advanced Therapies a reality for the benefit of patients and society and by making Europe a spearhead of Advanced Therapies in Science,Clinics and Biomedical Industry, that is the vision of the international project RESTORE Health by Advanced Therapies, coordinated by

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Researchers conduct largest study of childhood cancer after conception by IVF

In the past three decades, in vitro fertilization (IVF) has gone from an experimental procedure to being more common. Pregnancies enabled by IVF frequently have more difficulties, with children born earlier and smaller even among singleton births. University of Minnesota researchers conducted the largest study of childhood cancer after conception by IVF to date. This

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First gene therapy to treat rare blood disease nears European approval

The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency.

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Launch of six European initiatives with potential for transformational impact on society and the economy

The European Commission is announcing the winners of a pan-European competition open to researchers addressing grand scientific and technological challenges that could change our future. The six preparatory actions now launched will have one year to prepare science and technology agendas in areas of strategic importance for Europe. These may contribute to defining and launching

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Stem Cell Therapy for Heart Failure

With modern, guideline-directed therapy,1 which includes angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers (ARBs), β-receptor blockers, mineralocorticoid receptor blockers, and angiotensin receptor neprilysin inhibitors, up to 40% of patients with dilated cardiomyopathy (heart failure with reduced ejection fraction) may experience a return of left ventricular ejection fraction (LVEF) and ventricular geometry to normal.2 This encouraging result

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Google AI research group shows that AI can improve physicians’ diagnostic accuracy.

As artificial intelligence continues to evolve, diagnosing disease faster and potentially with greater accuracy than physicians, some have suggested that technology may soon replace tasks that physicians currently perform. But a new study from the Google AI research group shows that physicians and algorithms working together are more effective than either alone. It’s one of

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Study finds old brains still make neurons

https://www.nature.com/articles/s41591-019-0375-9 Reports of old brains’ decrepitude have been greatly exaggerated, scientists reported on Monday, unveiling results that contradict a much-discussed 2018 study and instead support the idea that human gray matter is capable of generating new neurons up to the ninth decade of life. The research, published in Nature Medicine, also found that old brains from

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A special edition of The American Statistician presents over 40 papers on “Statistical inference in the 21st century: a world beyond P < 0.05”

Statistical significance has become an infallible doctrine of scientific research. However, many scientists and statisticians argue that long-held beliefs about statistical significance have, in fact, harmed the scientific community. In hypothesis testing, the p-value gives the probability or likelihood that the null hypothesis is true and is frequently used as a measure of “statistical significance.”

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Giant study shows Apple Watch can spot heart rhythm changes — but it’s far from ‘medical-grade technology’

New results of a gigantic study testing how well the Apple Watch detects important heart-rhythm changes offer a tantalizing glimpse of how consumer devices might change medicine and lead to new ways to conduct science — but little more. Unfortunately, the data don’t tell us much about whether anyone should buy an Apple Watch, or

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Large Scale Research Initiative RESTORE New presentation

SUPPORT RESTORE AND DIFFUSE INFORMATION USING DOWNLOAD MATERIAL AT https://www.restore-h2020.eu/documents/download/restore_pr_slides_short_update_14_03_2019.pdf Large Scale Research Initiative RESTORE is a “place” where medicine, basic research, technology development and engineering meet, communicate and work together. Advanced Therapies (Advanced Therapies Medicinal Products and Biologised Medical Devices – ATMP/bio-MD) are a potential game changer in health care: They aim to transform

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The human reference genome falls short in ways that have become embarrassing, misleading, and emblematic of the white European dominance of science.

Here’s why that’s undermining personalized medicine In a paper published last week, scientists led by Dr. Pui-Yan Kwok of the University of California, San Francisco, analyzed 154 genomes from 26 ethnic populations, from Han Chinese and Tuscans to Yoruba, Esan, Puerto Ricans, and Peruvians. They found 60 million bases in one or more of these populations

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Scientists find method to boost CRISPR efficiency

Discovery made while editing genetic defect behind Duchenne muscular dystrophy Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases. The finding stemmed from research at UT Southwestern in which a single-cut gene-editing

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Scaffold-free cell patches possible with simple protocol for engineered adipose-derived stem cells

Cell sheets have previously shown greater efficacy for tissue repair than injections of single stem cells. New research conducted by scientists at the Los Angeles Biomedical Research Institute and Emmaus Life Sciences, Inc. (both CA, USA) has demonstrated the possibility of producing scaffold-free engineered cell sheets. These cell ‘patches’ could enable better organ repair follow

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Today marks the official start of RESTORE – Health by Advanced Therapies, entering the preparatory phase to become a Large-Scale Research Initiative.

Advanced Therapies are new treatment modalities (e.g. cell and gene therapy, tissue engineering) that aim to consign the never-ending treatment of chronic ailments to the past and instead offer sustained improvement and even cures. To enable Europe to become a competitive leader in this field, RESTORE is defining an Advanced Therapies roadmap 2021-2030 for Europe.

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Unique augmented reality concept for image-guided minimally invasive therapies

February 27 2019 P.M.Fornasari At the MWC Barcelona, one of the largest mobile events in the world, Royal Philips (NYSE: PHG, AEX: PHIA), a global leader in health technology today unveiled a unique mixed reality concept developed together with Microsoft Corp. (NASDAQ: MSFT) for the operating room of the future. Based on the state-of-the-art technologies

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How gut bacteria controls gene expression through “interspecies communication”

Highlights •Microbiome-derived NO promotes widespread S-nitrosylation of the host proteome •Interspecies S-nitrosylation regulates miRNAs, gene expression and host development •Microbiota control host function by shaping the post-translational landscape Summary Bioactive molecules can pass between microbiota and host to influence host cellular functions. However, general principles of interspecies communication have not been discovered. We show here

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RESTORE encourages your contribution to promote the project. Support RESTORE consortium

Advanced Therapies (Advanced Therapies Medicinal Products and Biologised Medical Devices – ATMP/bio-MD) are a potential game changer in health care: They aim to transform the current focus on “treatment of disease” into one that concentrates on “regeneration of health”. The unifying goal of RESTORE is the implementation of newly developed Advanced Therapies in clinical routine

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‘Off-the-shelf’ CAR-Ts have an important role to play – but are they ready for prime time?

Allogeneic CAR-Ts could serve an important niche – particularly for patients who can’t receive autologous CAR-Ts – but the earliness of the data makes conclusions tough to draw. Since their transition from small, academic clinical trials to commercial production, CAR-T cell therapies have won recognition as a novel and highly effective therapeutic modality, particularly in

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AI applications in healthcare grow, opening channel prospects

The hype around AI applications in healthcare spans decades, but the latest wave of AI tech suggests real-world traction. Artificial intelligence is already promising big things at Massachusetts General Hospital and Brigham and Women’s Hospital in Boston, where researchers have been building machine learning models over the past couple of years. Those models now help

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RHS is supporting RESTORE large scale research initiative

REGENHEALTHSOLUTIONS is a strong supporter of RESTORE, one of the six large scale research initiatives selected by European Commission in areas from health and energy to artificial intelligence and cultural heritage—to compete to become one of its next billion-euro ‘flagship’ science initiatives. On 1 March, each team will receive €1 million (US$1.1 million) to develop a

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Boston University researchers create new protocol to improve gene therapy tool production

Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus

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Multiple myeloma treatment granted FDA Orphan Drug Designation

Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Glenmark Pharmaceuticals’ bispecific antibody candidate GBR 1342 for the treatment of patients with multiple myeloma who have received prior therapies. “Patients who are struggling with diseases like

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FDA Grants Breakthrough Therapy Designation for Genentech’s Gazyva (Obinutuzumab) in Lupus Nephritis

The designation is based on the results of the Phase II NOBILITY study that showed Gazyva, in combination with standard of care, helped more people achieve a complete renal response than standard of care alone Genentech today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Gazyva® (obinutuzumab) for

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SanBio Granted Regenerative Medicine Advanced Therapy Designation from the U.S. FDA for SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury

The SanBio Group, a scientific leader in regenerative medicine for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation for SB623 cell therapy for the treatment of chronic neurological motor deficits secondary to traumatic brain injury (TBI). The designation is based on clinical results

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Research Continues to Drive Advances Against Cancer following AACR Cancer Progress Report 2019

AACR Cancer Progress Report 2019 details 27 new cancer treatments, a record high number of U.S. cancer survivors, and the benefit of increasing federal investments in medical research through the National Institutes of Health and the National Cancer Institute The American Association for Cancer Research (AACR) today released its annual Cancer Progress Report highlighting how research largely

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Myelofibrosis in 2019: moving beyond JAK2 inhibition

The authors outline the latest discoveries in the biology of myelofibrosis (MF), discuss current clinical management of patients with MF, and summarize the ongoing clinical trials that hope to change the landscape of MF treatment. Myelofibrosis (MF) is a myeloproliferative neoplasm characterized by ineffective clonal hematopoiesis, splenomegaly, bone marrow fibrosis, and the propensity for transformation

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