Mon.Jun 10, 2024

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Improved prime editing system makes gene-sized edits in human cells at therapeutic levels

Broad Institute

Improved prime editing system makes gene-sized edits in human cells at therapeutic levels By Allessandra DiCorato June 10, 2024 Breadcrumb Home Improved prime editing system makes gene-sized edits in human cells at therapeutic levels The gene-editing approach uses prime editors and evolved enzymes called recombinases, and could pave the way to effective one-size-fits-all gene therapies for diseases such as cystic fibrosis.

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Preclinical systemic pharmacokinetics, dose-proportionality, and CNS distribution of the ATM inhibitor WSD0628, a novel radiosensitizer for the treatment of brain tumors [Metabolism, Transport, and Pharmacogenetics]

ASPET

Radiation therapy, a standard treatment option for many cancer patients, induces DNA double strand breaks (DSBs), leading to cell death. Ataxia telangiectasia mutated (ATM) kinase is a key regulator of DSB repair, and ATM inhibitors are being explored as radiosensitizers for various tumors, including primary and metastatic brain tumors. Efficacy of radiosensitizers for brain tumors may be influenced by a lack of effective drug delivery across the blood-brain barrier (BBB).

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What to know about endometrial cancer

Antidote

Also known as endometrial carcinoma or uterine cancer, endometrial cancer occurs when the cells that line the uterus begin growing uncontrollably. Though the mortality rates are relatively low due to the high rate of early detection, it is a cancer that impacts nearly 70,000 people every year.

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Therapeutic targeting of full-length interleukin-33 protein levels with cell-permeable decoy peptides attenuates fibrosis in the bleomycin model in vivo [Gastrointestinal, Hepatic, Pulmonary, and Renal]

ASPET

Interleukin (IL)-33 has been shown to centrally regulate, among other processes, inflammation and fibrosis. Both intracellular full-length (FLIL33) precursor and extracellular mature cytokine (MIL33) forms exert such regulation, albeit differentially. Drug development efforts to target the IL-33 pathway have focused mostly on MIL33 and its specific cell-surface receptor, ST2, with limited attempts to negotiate the pathophysiological contributions from FLIL33.

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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

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Moderna Announces Good Results From Trial of Combo COVID/Flu Vaccine

Drugs.com

MONDAY, June 10, 2024 -- An experimental vaccine that could offer one-stop prevention for both COVID-19 and influenza is showing positive results among older adults in trials, maker Moderna announced Monday.The shot — for now called mRNA-1083 — "ha.

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Long-term treatment with the streptococcal exotoxin streptolysin O inhibits vascular smooth muscle contraction by inducing iNOS expression in endothelial cells [Cardiovascular]

ASPET

Streptolysin O (SLO), a bacterial toxin produced by common hemolytic streptococci, including Streptococcus pyogenes and resident microbiota, may be associated with inflammation in the cardiovascular system. We previously reported that short-term treatment with SLO at relatively high concentrations (10-1000 ng/mL) diminished acetylcholine-induced, endothelial-dependent relaxation in a concentration-dependent manner.

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Two-Drug Treatment Could Curb Meth Addiction

Drugs.com

MONDAY, June 10, 2024 — Though overdose deaths continue to surge, there is no approved medication to treat methamphetamine use disorder.Now, an experimental two-drug therapy has yielded promising results, UCLA researchers report."These findings h.

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Knock, Knock – FDA Issues Guidance on Best Processes and Practices During BIMO Inspections

FDA Law Blog: Biosimilars

By Sarah Wicks & Anne K. Walsh — Among FDA-regulated establishments and stakeholders, there is one word that makes everyone go on edge – the dreaded FDA “inspection.” In an effort to clarify for industry and alleviate some of the stress associated with these activities, last week the FDA issued a draft guidance aimed at providing recommendations on how to handle inspections under FDA’s Bioresearch Monitoring (BIMO) program.

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Protocol for creating 'wired miniature brains'

Science Daily: Pharmacology News

Researchers have developed -- and shared -- a process for creating brain cortical organoids -- essentially miniature artificial brains with functioning neural networks.

Research 105
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Microneedle Patch Might Restore Hair Growth After Alopecia

Drugs.com

MONDAY, June 10, 2024 -- Researchers have developed a novel way to promote hair growth in people who have the autoimmune disease alopecia areata.It's a skin patch that delivers immune-system controllers through an array of tiny needles.In alopecia.

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Health economics and outcomes research: Biopharma and the shift to value-based care

BioPharma Drive: Drug Pricing

Of the changes sparked by value-based care, perhaps the most valuable for the biopharmaceutical industry to embrace is the use of health economics and outcomes research (HEOR) to better understand a product’s value.

Research 102
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FDA Gives Nod to RSV Vaccine, Arexvy, for People in Their 50s

Drugs.com

MONDAY, June 10, 2024 -- The U.S. Food and Drug Administration on Friday has for the first time approved the use of a respiratory syncytial virus (RSV) vaccine for people in their 50s who are at increased risk for the illness. Drugmaker GSK's.

Vaccine 105
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Four-legged, dog-like robot 'sniffs' hazardous gases in inaccessible environments

Science Daily: Pharmacology News

Nightmare material or truly man's best friend? A team of researchers equipped a dog-like quadruped robot with a mechanized arm that takes air samples from potentially treacherous situations, such as an abandoned building or fire. The robot dog walks samples to a person who screens them for potentially hazardous compounds.

Research 100
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Experimental GLP-1 Med Might Be Breakthrough Against Fatty Liver Disease

Drugs.com

MONDAY, June 10, 2024 -- An experimental 'supercharged' form of popular GLP-1 weight-loss meds could help ease fatty liver disease, a new trial suggests. The drug under development, survodutide, helped up to 83% of patients gain real improvements.

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.

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Improved prime editing system makes gene-sized edits in human cells at therapeutic levels

Science Daily: Pharmacology News

Scientists have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough to be potentially useful for therapeutic applications. The advance could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

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Tips to Making Your Home Safer for People With Alzheimer's

Drugs.com

MONDAY, June 10, 2024 -- Bright lights, loud sounds and trip hazards can make a person with Alzheimer's uncomfortable in the home and even pose real dangers. The Alzheimer's Foundation of America (AFA) says a few easy fixes can change all.

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Lone Star State: Tracking a low-mass star as it speeds across the Milky Way

Science Daily: Pharmacology News

Astronomers have discovered a rare hypervelocity L subdwarf star racing through the Milky Way. More remarkably, this star may be on a trajectory that causes it to leave the Milky Way altogether.

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FDA Gives Nod to RSV Vaccine for People in Their 50s

Drugs.com

MONDAY, June 10, 2024 -- The U.S. Food and Drug Administration on Friday has for the first time approved the use of a respiratory syncytial virus (RSV) vaccine for people in their 50s who are at increased risk for the illness. Drugmaker GSK's.

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Clinical Data Like You´ve Never Seen It Before: Why Spotfire Is the Leading Tool for Clinical Analytics

Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies. The whitepaper also features a success story from Ambrx, a leading biopharmaceutical company, detailing how it has leveraged Spotfire to tackle data quality and collaboration challenges in clinic

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Statins for heart disease prevention could be recommended for far fewer Americans if new risk equation is adopted

Science Daily: Pharmacology News

If national guidelines are revised to incorporate a new risk equation, about 40% fewer people could meet criteria for cholesterol-lowering statins to prevent heart disease. The study examines the potential impact of widespread adoption of the PREVENT equations, which were released by the American Heart Association in November 2023 to update physicians' go-to calculators for assessing patients' 10-year risk of heart attack or stroke.

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Moderna says combination flu, COVID shot succeeds in study

BioPharma Drive: Drug Pricing

Positive results from the trial could position Moderna to bring the two-in-one vaccine to market in 2025.

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Brain's structure hangs in 'a delicate balance'

Science Daily: Pharmacology News

Researchers examined anatomy of neurons from humans, mice and fruit flies. They discovered that the cellular structure of the brain is at a critical point, poised between two phases. New insights could help design computational models of the brain's complexity.

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4-1BB and the critical importance of local control

SugarCone Biotech

Pathology stain of tertiary lymphoid structures (arrows) found on the margin of, and formed within, a colon carcinoma sample. [link] Immunity is complex and can be dangerous when exploited clinically, as demonstrated by the lethal administration of TNF, or anti-CD40L antibody (Biogen) or CAR-T cells expressing the CD16 Fc-receptor (Unum), among many other examples.

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Novel Genetic Clock discovers oldest known marine plant

Science Daily: Pharmacology News

An international research team has discovered the oldest known marine plant using a novel genetic clock. This 1400-year-old seagrass clone from the Baltic Sea dates back to the Migration Period. The research project is a significant step towards better understanding and protecting marine ecosystems.

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Lilly Alzheimer’s drug gets unanimous backing of FDA panel

BioPharma Drive: Drug Pricing

The expert committee concluded donanemab’s benefits outweigh its risks, despite some concern over the drug’s safety.

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Elephants have names for each other like people do, new study shows

Science Daily: Pharmacology News

Wild African elephants address each other with name-like calls, a rare ability among nonhuman animals, according to a new study. Researchers used machine learning to confirm that elephant calls contained a name-like component identifying the intended recipient, a behavior they suspected based on observation. The study suggests elephants do not imitate the receiver's call to address one another but instead use arbitrary vocal labels like humans.

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Crucial Insights on Patent Litigation Strategies in Pharma Industry

Drug Patent Watch

In the “Patent Effect” podcast, Mustafa introduces his guest, Hakan Yum, the IP Director of Exrain Pharma, based in Sweden.

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Planetary Health Diet associated with lower risk of premature death, lower environmental impact

Science Daily: Pharmacology News

People who eat a healthy, sustainable diet may substantially lower their risk of premature death in addition to their environmental impact, according to a new study. This large study directly evaluates the impacts of adherence to recommendations in the landmark 2019 EAT-Lancet report. The researchers have named the dietary pattern outlined in the report -- which emphasizes a variety of minimally processed plant foods but allows for modest consumption of meat and dairy foods -- the Planetary Heal

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Time for the Children Gala in Detroit: Making a Difference with Friends of the Children

Perficient: Drug Development

It is an honor and privilege to impact another person and make their lives better with more opportunities, and the Time for the Children Event Gala in Detroit is one of the greatest examples. I was able to attend this event last week with my colleagues and support an organization called Friends of the Children which is making generational impacts with at-risk children in the city.

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Hubble finds surprises around a star that erupted 40 years ago

Science Daily: Pharmacology News

Astronomers have used new data from NASA's Hubble Space Telescope and the retired SOFIA (Stratospheric Observatory for Infrared Astronomy) as well as archival data from other missions to revisit one of the strangest binary star systems in our galaxy -- 40 years after it burst onto the scene as a bright and long-lived nova. A nova is a star that suddenly increases its brightness tremendously and then fades away to its former obscurity, usually in a few months or years.

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Just 18% of People Who Need Lung Cancer Screening Get It

Drugs.com

MONDAY, June 10, 2024 -- Only a fraction of Americans are getting recommended lung cancer screenings, new research shows.While rates overall are up slightly, fewer than 1 in 5 people who are eligible for screening are up-to-date with it, according.

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Optimism wards off procrastination

Science Daily: Pharmacology News

People with an optimistic outlook on the future are less likely to be severe procrastinators, according to new research. While procrastinators often admonish themselves for their 'bad habit,' it turns out that their worries for the future are more to blame. Through a survey of nearly 300 young people, researchers found that those who had a positive view about their stress levels decreasing in the future, compared to the past or present, were less likely to experience severe procrastination.

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CDD Vault Update (June 2024)

Collaborative Drug

We are excited to announce a new round of features associated with our Curves Add-on. If you are not yet subscribed to Curves, please contact your CDD Vault account manager or the CDD Vault Support Team to discuss enabling these features in your Vault.

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Researchers engineer new approach for controlling thermal emission

Science Daily: Pharmacology News

If a material absorbs light, it will heat up. That heat must go somewhere, and the ability to control where and how much heat is emitted can protect or even hide such devices as satellites. An international team of researchers has published a novel method for controlling this thermal emission in Science.