Sat.Jun 08, 2024 - Fri.Jun 14, 2024

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Some CRISPR screens may be missing cancer drug targets

Broad Institute

Some CRISPR screens may be missing cancer drug targets By Allessandra DiCorato June 14, 2024 Breadcrumb Home Some CRISPR screens may be missing cancer drug targets Current CRISPR guides don’t work equally well in cells from people of all ancestries, which could lead to false negative results. By Allessandra DiCorato June 14, 2024 Credit: Ricardo Job-Reese, Broad Communications CRISPR/Cas9 gene editing has made possible a multitude of biomedical experiments including studies that systematically t

Drugs 145
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Scientists preserve DNA in an amber-like polymer

Science Daily: Pharmacology News

With their 'T-REX' method, researchers developed a glassy, amber-like polymer that can be used for long-term storage of DNA, such as entire human genomes or digital files such as photos.

DNA 135
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AAV9-Mediated Intra-Striatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice [Chemotherapy, Antibiotics, and Gene Therapy]

ASPET

Mutations in the GNAO1 gene, which encodes the abundant brain G-protein Gα o , result in neurologic disorders characterized by developmental delay, epilepsy, and movement abnormalities. There are over 50 mutant alleles associated with GNAO1 disorders; the R209H mutation results in dystonia, choreoathetosis, and developmental delay without seizures.

Therapies 100
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Brusatol induces ferroptosis to inhibit hepatocellular carcinoma progression by targeting ATF3

Chemical Biology and Drug Design

In this study, we explored the mechanism of action of brusatol in hepatocellular carcinoma. Using RNA-seq and through in vitro and in vivo studies, we determined that brusatol suppresses hepatocellular carcinoma progression by inducing ATF3-mediated ferroptosis. Therefore, our research revealed the biological effect of brusatol treatment and provided ATF3 as a novel therapeutic target and prognostic biomarker for HCC therapy.

RNA 100
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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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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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Studies uncover the critical role of sleep in the formation of memories

Science Daily: Pharmacology News

A lack of sleep can make it extraordinarily difficult to retain information. Two new studies uncover why this is and what is happening inside the brain during sleep and sleep deprivation to help or harm the formation of memories.

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Avidity drug for muscular dystrophy shows promise; Syntis takes new approach to weight loss

BioPharma Drive: Drug Pricing

Study results indicated Avidity’s medicine muted target genes. Elsewhere, Syntis revealed obesity drug plans and Nodthera chalked up more positive data for its NLRP3 inhibitor.

Drugs 114
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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.

Vaccine 119
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Would astronauts' kidneys survive a roundtrip to Mars?

Science Daily: Pharmacology News

The structure and function of the kidneys is altered by space flight, with galactic radiation causing permanent damage that would jeopardise any mission to Mars, according to a new study led by researchers from UCL.

Research 129
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OTC Hearing Aids: “Nothing to See Here” Says GAO Report

FDA Law Blog: Biosimilars

By Sara W. Koblitz & Jeffrey N. Gibbs — It’s been over a year and a half since Over-the-Counter (“OTC”) hearing aids became legal, and it’s not clear that they’ve made the difference in hearing loss treatment that Congress anticipated. (FDA once estimated that OTC hearing aids would save patients over $3000.) A recent GAO Report hasn’t found that OTC hearing aids have had much impact.

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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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Ipsen drug for rare liver disease approved by FDA

BioPharma Drive: Drug Pricing

The medicine will join Intercept’s Ocaliva as a treatment option for primary biliary cholangitis. Another drug, from the now Gilead-owned CymaBay, could be cleared for the condition by August.

Disease 113
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FDA Tells Vaccine Makers to Target New COVID Variant for Fall

Drugs.com

FRIDAY, June 14, 2024 -- COVID vaccine makers will be advised to update their shots to target the KP.2 variant, an offshoot of the JN.1 variant that spread widely last winter, the U.S. Food and Drug Administration announced Thursday. It's a.

Vaccine 115
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AI-powered simulation training improves human performance in robotic exoskeletons

Science Daily: Pharmacology News

Researchers have demonstrated a new method that leverages artificial intelligence (AI) and computer simulations to train robotic exoskeletons to autonomously help users save energy while walking, running and climbing stairs.

Research 126
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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.

FDA 111
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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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Pfizer gene therapy for Duchenne fails to meet goals of key trial

BioPharma Drive: Drug Pricing

The negative results from the Phase 3 study, called Ciffreo, come a little more than a month after Pfizer said a patient died in another trial of the gene therapy.

Therapies 117
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ADHD Patients Could Face Disrupted Access to Meds Following Fraud Case

Drugs.com

FRIDAY, June 14, 2024 -- The two top officers of a telehealth company that began to distribute ADHD drugs widely during the pandemic have been charged with health care fraud, the U.S. Department of Justice announced Thursday. The arrests will.

Drugs 111
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Swimming microrobots deliver cancer-fighting drugs to metastatic lung tumors in mice

Science Daily: Pharmacology News

Engineers have developed microscopic robots, known as microrobots, capable of swimming through the lungs to deliver cancer-fighting medication directly to metastatic tumors. This approach has shown promise in mice, where it inhibited the growth and spread of tumors that had metastasized to the lungs, thereby boosting survival rates compared to control treatments.

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Up Close and Personal With Ashley (Schnider) Mahoney, Study Director, Scientist

Alta Sciences

Up Close and Personal With Ashley (Schnider) Mahoney, Study Director, Scientist nbartlett Wed, 06/12/2024 - 16:09 Ashley joined Altasciences in 2022 as Study Director. Along with managing non-GLP nonhuman primate (NHP) studies at our Sacramento, CA, site, Ashley coordinates study logistics with sponsors and internal departments, monitors study conduct, and maintains steady communication with sponsors.

Doctors 98
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Regenxbio CEO to step down after 15 years

BioPharma Drive: Drug Pricing

Kenneth Mills will become chair of the gene therapy developer's board while Curran Simpson, the current chief operating officer, will take his place as company head.

Therapies 106
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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.

Disease 105
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Are mixed emotions real? New research says yes

Science Daily: Pharmacology News

Neuroscientists found that brains display distinct neural activity when experiencing emotions such as bitter-sweetness. The advance could help solve a longstanding scientific debate: whether 'mixed emotions' arise from unique activity in the brain, or if we're just flip-flopping back and forth between positive and negative feelings.

Research 122
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Addgene's 20th Anniversary Party

addgene Blog

This year marks Addgene's 20th anniversary! We've been celebrating throughout the year, and on Sunday, June 9th, we hosted our 20th anniversary party for all Addgenies to celebrate together. We were even able to have many of our remote workers travel to Addgene's headquarters in Watertown, Massachusetts, for the occasion!

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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 104
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J&J Settles Talcum Powder Lawsuits From States for $700 Million

Drugs.com

THURSDAY, June 13, 2024 -- Johnson & Johnson will pay $700 million to settle claims from 42 states and the District of Columbia that the company continued to market its talcum powder products even as evidence tied them to a heightened risk for.

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Tiny New Zealand bird delivers a lesson in birdsong evolution

Science Daily: Pharmacology News

Scientists are rethinking the evolution of vocal learning in birds. New Zealand's smallest bird, the rifleman or titipounamu, may have a rudimentary version of the talent possessed by parrots, hummingbirds and songbirds.

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How long does a patent last for drugs?

Drug Patent Watch

Sure, here’s a more detailed explanation with examples: Drug Patent Duration Drug patents are granted by the United States Patent and Trademark Office (USPTO) for a period of 20 years… Source

Drugs 90
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Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision

BioPharma Drive: Drug Pricing

The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.

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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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Pair plasmas found in deep space can now be generated in the lab

Science Daily: Pharmacology News

Researchers have experimentally generated high-density relativistic electron-positron pair-plasma beams by producing two to three orders of magnitude more pairs than previously reported.

Research 114
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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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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.

Vaccine 120
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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.

Treatment 111
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3D-printed mini-actuators can move small soft robots, lock them into new shapes

Science Daily: Pharmacology News

Researchers have demonstrated miniature soft hydraulic actuators that can be used to control the deformation and motion of soft robots that are less than a millimeter thick. The researchers have also demonstrated that this technique works with shape memory materials, allowing users to repeatedly lock the soft robots into a desired shape and return to the original shape as needed.

Research 114
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#WhyIScience Q&A: A biochemist uses mass spectrometry to find proteins involved in cancer

Broad Institute

#WhyIScience Q&A: A biochemist uses mass spectrometry to find proteins involved in cancer By Corie Lok June 11, 2024 Breadcrumb Home #WhyIScience Q&A: A biochemist uses mass spectrometry to find proteins involved in cancer Moe Haines talks about how his open-minded approach to science led him to pursue a career in proteomics. By Claire Hendershot June 11, 2024 Credit: Allison Colorado, Broad Communications Moe Haines is a senior research associate in the Proteomics Platform at Broad.

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