2023

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Potentially game-changing schizophrenia drug enters Phase I trials

Drug Discovery World

A potential schizophrenia treatment, discovered at Vanderbilt University in Tennessee in the US, has been cleared by the US Food and Drug Administration for use in Phase I clinical trials. NMRA-266, an allosteric modulator that works through a mechanism that has been clinically validated in the treatment of disorders like schizophrenia, will be developed by Neumora Therapeutics.

Trials 299
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GDC-6599

Drug Hunter

A First-in-Class TRPA1 Antagonist Overcomes Toxicity Hurdles to Become Cough Candidate Genentech’s GDC-6599 is the first oral TRP Ankyrin 1 (TRPA1) antagonist to reach Ph. IIa ( NCT05660850 ) for chronic cough after preclinical studies and a Ph. I trial showed it was well-tolerated, in contrast to prior molecules. The transient receptor potential (TRP) family of ion channels has been the subject of intensive drug discovery efforts due to their critical role in the development and progression of

Trials 315
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Companies Seek To Increase Life Span By A Decade Or More

Forbes: Drug Truths

But keep in mind that it’s going to be a long while before such drugs are actually available.

Drugs 325
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New method tags cells with location coordinates for single-cell studies

Broad Institute

New method tags cells with location coordinates for single-cell studies By Corie Lok December 13, 2023 Breadcrumb Home New method tags cells with location coordinates for single-cell studies The technique, called Slide-tags, allows scientists to map the location of cells within tissues in standard single-cell experiments. By Sarah C.P. Williams December 13, 2023 Credit: Andrew Russell Different cell types (each represented by a different color) are mapped to their native location in human brain

RNA 145
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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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World’s first engineered B cell therapy enters human trials

Drug Discovery World

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

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As ALS research booms, one treatment center finds itself in the spotlight

BioPharma Drive: Drug Pricing

The Healey center is at the front of ALS research and care, earning acclaim from patients, doctors and scientists. Still, the complexities of the disease and of drug development have brought hard-felt losses.

Research 142

More Trending

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Neanderthals were the world's first artists, research reveals

Science Daily: Pharmacology News

Recent research has shown that engravings in a cave in La Roche-Cotard (France), which has been sealed for thousands of years, were actually made by Neanderthals. The findings reveal that the Neanderthals were the first humans with an appreciation of art.

Research 140
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Social Connection and Worker Well-being

NIOSH Science Blog: Drugs

In May, the U.S. Surgeon General, Vice Admiral Vivek Murthy, MD, MBA, released Our Epidemic of Loneliness and Isolation: The U.S. Surgeon General’s Advisory on the Healing Effects of Social Connection and Community calling for a whole-of-society approach to address the epidemic of loneliness and isolation.1 Below we briefly highlight information from the Advisory and its implications for worker well-being.

Research 136
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Antibody Drug Conjugates: windows of opportunity

Drug Target Review

Over the last two decades, an increasing number of Antibody Drug Conjugate (ADC) therapeutics have been approved for oncology indications. These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Through their unique modes of action, ADCs have been heralded as having the potential to positively impact not only cancer patient outcomes but also quality of life, an increasingly important factor in treatment decisions for t

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Keypoint Newsletter: Welcoming the New Fellows Class of 2024

keypoint

Keystone Symposia is pleased to introduce the Keystone Symposia Fellows Class of 2024! This year we welcome seven early-career investigators and seven post-doctoral fellows, a new addition to the program this year.

Doctors 134
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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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Buprenorphine now replaces methadone as the most common medicine for opioid dependence’: 10-year trends in opioid agonist treatment medicines in Australia

National Drug & Alcohol Research Centre Blog

Kendal Chidwick NDARC’s Kendal Chidwick discusses the findings from a recently published study that examined how patterns of opioid agonist treatment medicines have changed over the past decade. In Australia, both methadone and buprenorphine have been subsidised by the Pharmaceutical Benefit Scheme (PBS) for the treatment of opioid dependence (termed ‘opioid agonist treatment’ or OAT) for several decades.

Treatment 133
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The Power of Personalization Amid the Changing CRO Landscape

Conversations in Drug Development Trends

Amidst a shifting clinical landscape characterized by increasingly complex trial designs and growing patient subpopulations, many contract research organizations (CROs) have adopted a “one-stop-shop” strategic approach. As a result, various CROs have undergone significant consolidations and acquisitions of specialized capabilities to address the escalating complexity in clinical trials.

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Common Stomach Bug Is Linked to Higher Alzheimer's Risk

Drugs.com

THURSDAY, Dec. 28, 2023 -- A common stomach bug may play a part in Alzheimer's disease risk.New research found that older folks infected with Helicobacter pylori (H. pylori) had greater odds for developing Alzheimer's, the most common type of.

Disease 131
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New patient-partnered research project will study heart disease and diabetes risk in South Asian populations

Broad Institute

New patient-partnered research project will study heart disease and diabetes risk in South Asian populations By Corie Lok October 23, 2023 Breadcrumb Home New patient-partnered research project will study heart disease and diabetes risk in South Asian populations The OurHealth study aims to increase representation of people of South Asian ancestry in cardiometabolic research.

Disease 145
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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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CRUK and Guardant Health collaborate on precision cancer drugs

Drug Discovery World

Cancer Research UK (CRUK), Cancer Research Horizons, and precision oncology company Guardant Health have launched a collaboration to advance cancer treatment. The agreement will enable the parties to discuss opportunities for collaboration to support the charity’s research and clinical development activities, focusing primarily on its clinical trials run by its Centre for Drug Development.

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‘No tolerance for failure’: An oral history of the first CRISPR medicine

BioPharma Drive: Drug Pricing

A new sickle cell disease therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals is now approved in the U.S. and U.K. This is the story of how it came to be.

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Plasmids 101: Choosing an Antibiotic Resistance Gene

addgene Blog

Plasmids need antibiotic resistance genes so that you can specifically isolate bacteria containing your constructs of interest. But does it matter which antibiotic resistance gene you select? In this blog, we’ll break down the mechanism of action of the most common antibiotic resistance genes and which applications are best (or worst) for each antibiotic.

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AI's memory-forming mechanism found to be strikingly similar to that of the brain

Science Daily: Pharmacology News

An interdisciplinary team consisting of researchers has revealed a striking similarity between the memory processing of artificial intelligence (AI) models and the hippocampus of the human brain. This new finding provides a novel perspective on memory consolidation, which is a process that transforms short-term memories into long-term ones, in AI systems.

Research 138
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A guide to clinical trial phases [infographic]

Antidote

Before any new treatment or therapy is able to be used by the patient population, it must go through the process of a clinical trial — this is the case for any new prescription drug, but also true for over-the-counter medications, medical devices, and more.

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New molecular insights on medical cannabis

Drug Target Review

Autotaxin (ATX) is a 99-125 kDa lysophospholipase D involved in a large range of physiological and pathological processes. 1 This critical enzyme is part of the nucleotide pyrophosphatase/ phosphodiesterase family and is also referred to as ENPP2. Five ATX isoforms have been identified, with ATX-b expressed in most human tissues and ATX-g specific to the central nervous system.

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Getting Real with Molecular Property Prediction

Practical Cheminformatics

Introduction If you believe everything you read in the popular press, this AI business is easy. Just ask ChatGPT, and the perfect solution magically appears. Unfortunately, that's not the reality. In this post, I'll walk through a predictive modeling example and demonstrate that there are still a lot of subtleties to consider. In addition, I want to show that data is critical to building good machine learning (ML) models.

Drugs 130
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Bloodborne Pathogen Exposures Continue in Operating Room Settings

NIOSH Science Blog: Drugs

Despite legislation and improved technology, data from Massachusetts hospitals show that sharps injuries have increased in the operating room (OR) [1]. These injuries place healthcare workers at risk of exposure to bloodborne pathogens (BBPs). There is an urgent need to renew efforts to protect healthcare workers inside the operating room. The Massachusetts data highlight a gap and the need to establish a national surveillance program that would help hospitals develop further measures to prevent

Hospitals 123
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Using Rendering Parameters on a XM Cloud component

Perficient: Drug Development

What about Rendering Parameters? I recently did a POC using rendering parameters in a component in XM Cloud. In this blog i will describe my approach, issues faced and how i overcame them. Adding a rendering to a component, is relatively straight forward in Sitecore development, i.e.( [link] ). However, there are some circumstances where things are a little bit more complex (than they need to be).

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Counterfeit Pills Fuel Rising Number of Fatal Drug Overdoses

Drugs.com

FRIDAY, Sept. 1, 2023 -- A growing number of overdose deaths in the United States involve counterfeit pills, health officials reported Thursday. Overdose deaths involving counterfeit pills were twice as common in the latter half of 2021 as they were.

Drugs 135
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Evolved prime editors are smaller and more efficient for therapeutic applications

Broad Institute

Evolved prime editors are smaller and more efficient for therapeutic applications By Corie Lok August 31, 2023 Breadcrumb Home Evolved prime editors are smaller and more efficient for therapeutic applications Researchers have evolved the editing machine at the heart of the prime editing system, which can make a wide range of changes to the genome. By Sarah C.P.

DNA 145
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AI/ML-enabled Medical Devices Have Everyone’s Attention, Including FDA’s

FDA Law Blog: Biosimilars

By Gail H. Javitt & Steven J. Gonzalez — The Wall Street Journal (WSJ) recently published a series of articles as part of its special report “What’s Ahead for Artificial Intelligence.” Three of these articles focus on medical applications of Artificial Intelligence and Machine Learning (AI/ML) and explore FDA’s role in regulating such products. The first article—“Is the Eye the Window to Alzheimer’s?

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CRISPR therapy for sickle cell approved by FDA in gene editing milestone

BioPharma Drive: Drug Pricing

In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.

FDA 139
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New optogenetic tools for cytoskeleton and membrane control

addgene Blog

Optogenetics gives you the power to control cells or organisms with the flip of a switch. You may be familiar with popular light-sensitive ion channels used to control activities like neuronal signaling — think of a mouse with an LED brain implant or a worm wiggling back and forth under blue light — but there’s a lot more to optogenetics than neuronal activity.

Research 126
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Octopuses rewire their brains to adapt to seasonal temperature shifts

Science Daily: Pharmacology News

Octopuses don't thermoregulate, so their powerful brains are exposed to -- and potentially threatened by -- changes in temperature. Researchers report that two-spot octopuses adapt to seasonal temperature shifts by producing different neural proteins under warm versus cool conditions. The octopuses achieve this by editing their RNA, the messenger molecule between DNA and proteins.

RNA 144
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Clinical trial phases: What’s the difference?

Antidote

Clinical trials are how researchers advance their knowledge about potential new treatments, including medications, medical devices, and lifestyle interventions. Clinical trials are divided into phases , each with a distinct duration, purpose, and number of volunteers needed. Before any new treatment becomes available on the market, it must go through this process, as required by the FDA.

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New biomarkers found for diabetic kidney disease

Drug Target Review

Scientists in China have published their findings 1 regarding novel biomarkers, which they hope will benefit patients by identifying the disease at an earlier stage. Minjia Tan’s group from the Shanghai Institute of Materia Medica, Chinese Academy of Sciences, and Shichun Du’s group from Xinhua Hospital affiliated to Shanghai Jiaotong University School of Medicine, analysed urinary and exosome proteome profiling and discovered biomarkers for diabetic kidney disease (DKD).

Disease 126
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The power of protective layers: Employers advancing whole-person health

National Institute on Drug Abuse: Nora's Blog

The power of protective layers: Employers advancing whole-person health mfleming Fri, 10/06/2023 - 17:56 Nora's Blog October 11, 2023 This blog was originally posted on LinkedIn.com. Image @GettyImages/ SolStock There is a growing movement among employers to holistically recognize employees’ mental, financial, and physical health through a "whole-person" approach.

Treatment 119
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Family Farms: When Working From Home Can Put Children at Risk

NIOSH Science Blog: Drugs

Agriculture ranks among the most hazardous industries for workers,[1] but since many farms share work and home spaces, it is also one of the few industries in which family members are also at risk for fatal and nonfatal injuries. Every day, 33 children in the United States are seriously injured in agricultural-related incidents, and every three days a child dies on a farm.[2] However, over 60% of children impacted by a farm incident were not working when the injury occurred.[3] Adult supervision

Research 123
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Suspend Reality – 6 Steps to Move from Fantasy to Execution

Perficient: Drug Development

We’re just now finishing up the holiday season. At this time of year, I’m always intrigued by the magic of the holidays we celebrate, and the optimistic goals we set for the new year. It amazes me how we collectively like to suspend reality for a bit, reflect, and forecast. And I wonder why we don’t drift into thoughts of the extraordinary more often.