A validated composite model to predict risk of curve progression in adolescent idiopathic scoliosis

A newly published study on @EClinicalMed by @CUHKofficial aims to formulate a composite model composed of clinical parameters and circulating markers in the prediction of curve progression. In adolescent idiopathic scoliosis (AIS), the continuous search for effective prognostication of significant curve progression at the initial clinical consultation to inform decision for timely treatment and to

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Hematopoietic stem cell marker: A key player in the ontogeny of hematopoiesis

A group of researchers at Osaka University revealed that endothelial cell-selective adhesion molecule (ESAM), a surface marker for hematopoietic stem cells (HSCs) and vascular endothelial cells (ECs), played an important role in the ontogeny of hematopoiesis in mice, particularly in the development of adult-type erythropoiesis. Their research results were published in Stem Cell Reports. In hematopoiesis,

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Current Immunotherapy: Progress With Adoptive Cellular Therapies and Combinations

Source Targeted Oncology The clinical development and application of cancer immunotherapy over the past decade has translated the long-standing knowledge of the close relationship between cancerous tissues and lymphoid immune cells, dating back to the late 19th century.1,2 Today, cancer immunotherapies, all of which recruit the body’s own immune system to target and eliminate cancer cells,

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Engineered 3D Polymer and Hydrogel Microenvironments for Cell Culture Applications

In the recently published ‘Engineered 3D Polymer and Hydrogel Microenvironments for Cell Culture Applications,’ authors Daniel Fan, Urs Staufer, and Angelo Accardo explore the world of bioengineering and microenvironments, reviewing the best types of methods, materials, and challenges that must be met. Material properties are one of the most highly discussed topics today in 3D

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Production and Quality Requirements of Human Platelet Lysate: A Position Statement from the Working Party on Cellular Therapies of the International Society of Blood Transfusion

Human platelet lysate (HPL), rich in growth factors, is an efficient alternative supplement to fetal bovine serum (FBS) for ex vivo propagation of stromal cell-based medicinal products. Since 2014, HPL has been a focus of the Working Party for Cellular Therapies of the International Society of Blood Transfusion (ISBT). Currently, as several Good Manufacturing Practice

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The Alliance for Regenerative Medicine Outlines Recommendations for Increasing the Number of European-Based ATMP Clinical Trials

Europe has become less competitive than other regions in attracting new ATMP clinical trials. Faster and more streamlined review processes for clinical trials by regulatory authorities are fundamental to increase clinical research and development in Europe. There is considerable country-to-country variability within Europe: the UK has the highest absolute number of new ATMP clinical trials,

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NIH launches new collaboration with Bill & Melinda Gates Foundation to develop gene-based cures for sickle cell disease and HIV on global scale

Source NIH Initial investment aims to advance accessible and scalable candidate interventions into clinical trials within 10 years. The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates

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NIH researchers create new viral vector for improved gene therapy in sickle cell disease

Source NIH ‘Forward-oriented’ design might boost treatment effectiveness and broaden use. Researchers at the National Institutes of Health have developed a new and improved viral vector—a virus-based vehicle that delivers therapeutic genes—for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times

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RESTORE Large Scale Research Initiative community presents 1st Advanced Therapies Science Meeting (1st ATSM) in Berlin, 25-26 November 2019

What are the research challenges slowing development of curative treatments and what are the roadblocks for the wider adoption of Advanced Therapies in clinical routine? Join the conversation at the1st Advanced Therapies Science Meeting (1st ATSM)in Berlin, 25-26 November 2019 to explore the latest trends in the field and to discuss the Advanced Therapies Roadmap 2030 that

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Future and Emerging Technologies Flagships and Large Research Initiatives

European Commission, Digital Single Market has published a new Factsheet/Infographic on FET Flagships and Large Research Initiative. These visionary, long-term, large-scale research initiatives address major science and technology challenges in Europe. They involve hundreds of research teams with the aim of boosting the EU’s scientific and industrial landscape.


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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Tumor neoantigenicity assessment with CSiN score incorporates clonality and immunogenicity to predict immunotherapy outcomes

Immunotherapy has unleashed a revolution in care for some cancer patients. But most immunotherapies help only a small subset of patients, meaning doctors often have to resort to a trial-and-error process to determine who might actually benefit from the novel treatments. Now, scientists have developed a new metric they believe can help predict whether patients will respond

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Covid-19 incubation could be as long as 27 days, Chinese provincial government says

Source Reuters A 70-year-old man in China’s Hubei Province was infected with coronavirus but did not show symptoms until 27 days later, the local government said on Saturday, meaning the virus’ incubation period could be much longer than the presumed 14 days. A longer incubation period could complicate efforts to contain spread of the epidemic

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Plant-based relatives of cholesterol could help gene therapy for cystic fibrosis, other diseases

Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol, new research shows. That’s because the shape and structure of the phytosterols help the genes get where they need to be inside cells. The findings by Oregon State University researchers, published today in Nature Communications, are important because many illnesses that

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BioMarin’s Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020

If approved, 1st Gene Therapy in U.S. for the Treatment of Any Type of Hemophilia BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia

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Egypt, Algeria and Republic of South Africa, main gates for coronavirus importation in Africa

Egypt, Algeria and Republic of South Africa are the African countries most at risk for coronavirus COVID-19 importation in the continent, due to high air traffic with the contaminated Chinese provinces. But these countries are also among the best equipped on the continent to quickly detect and deal with new cases.  In other African countries,

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New Institute Launched to Ensure the U.S. Healthcare System Is Ready for Gene Therapies

Industry leaders, patient advocates, researchers unite to maximize the incredible potential of transformative gene therapies The Institute for Gene Therapies (IGT) launched today with a focus on advocating for a modernize the U.S. regulatory and reimbursement framework so that gene therapies can deliver their significant potential to patients. IGT will educate stakeholders across the healthcare

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