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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The first major set of genetic associations found in long COVID

Drug Target Review

Why has it been challenging to identify genetic risk factors for long COVID? There is an extensive array of symptoms associated with long COVID, with the most common being fatigue and post-exertional malaise, cognitive dysfunction, mood disturbances and respiratory problems. This is likely indicative of the heterogeneous nature of the disorder, and it is this complexity and diversity of clinical presentation and effects across multiple organ systems, that has made efforts to identify genetic ris

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Early Career Researcher Toolbox: Free Online Molecular Biology Tools

addgene Blog

Primer design. Plasmid mapping. DNA sequence analysis. We all have our favorite tools for tackling these particular tasks, but they tend to be scattered about the internet. To help you keep your virtual molecular biology toolbox organized, today’s post features a list of free online molecular biology tools all in one place.

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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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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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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 133
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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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Comparing Classification Models - You’re Probably Doing It Wrong

Practical Cheminformatics

In my last post , I discussed benchmark datasets for machine learning (ML) in drug discovery and several flaws in widely used datasets. In this installment, I’d like to focus on how methods are compared. Every year, dozens, if not hundreds, of papers present comparisons of ML methods or molecular representations. These papers typically conclude that one approach is superior to several others for a specific task.

Packaging 133
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Breakthrough in Functional Annotation with HiFi-NN

Nvidia Developer: Drug Discovery

Enzymes are vital biological catalysts for a multitude of processes, from cellular metabolism to industrial manufacturing. The applications of artificial. Enzymes are vital biological catalysts for a multitude of processes, from cellular metabolism to industrial manufacturing. The applications of artificial intelligence for enzyme generation is an exciting field of research with direct applications in the life sciences.

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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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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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‘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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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 131
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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 136
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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 133
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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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In search of the perfect assay description

The ChEMBL-og

Credit: Science biotech, CC BY-SA 4.0 Assays des cribe the experimental set-up when testing the activity of drug-like compounds against biological targets; they provide useful context for researchers interested in drug-target relationships. Ver sion 33 of ChEMBL contains 1.6 million diverse assays spanning ADMET, physicochemical, binding, functional and toxicity experiments.

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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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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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Scientists reveal cellular changes unique to early Alzheimer’s disease

Broad Institute

Scientists reveal cellular changes unique to early Alzheimer’s disease By Allessandra DiCorato September 28, 2023 Breadcrumb Home Scientists reveal cellular changes unique to early Alzheimer’s disease A study of brain tissue from living adults provides a rare look into the earliest stages of the neurodegenerative disease and highlights cell types involved in plaque production.

Disease 144
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The epigenetic edge: Harnessing precision medicine’s potential 

Drug Discovery World

HKG Epitherapeutics is a biotechnology company developing novel tools for the early detection of cancer and promoting healthy aging. Founder Dr Moshe Szyf Founder shares the potential of harnessing precision medicine. Embarking on a new era of medicine The advent of genomics has ushered in the era of personalised medicine, enabling us to analyse the genetic makeup of individuals with unprecedented accuracy.

DNA 289
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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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A decade later, biotech’s CRISPR revolution is still going strong

BioPharma Drive: Drug Pricing

Once the specialty of a few select drugmakers, CRISPR gene editing is now an essential technology for a growing group of biotechs, many led by former students of the field's pioneering scientists.

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What Biology Can Learn from Physics

Codon

I. Moonshots Physics was long dominated by solitary celebrities. Newton formulated laws of motion, Einstein developed a theory of relativity, and Dirac sculpted a general theory of quantum mechanics. But then, World War II changed the equation. The Manhattan project employed 130,000 people and cost $2.2 billion , or more than $25 billion in today’s dollars.

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Humans are disrupting natural 'salt cycle' on a global scale, new study shows

Science Daily: Pharmacology News

A new paper revealed that human activities are making Earth's air, soil and freshwater saltier, which could pose an 'existential threat' if current trends continue. Geologic and hydrologic processes bring salts to Earth's surface over time, but human activities such as mining and land development are rapidly accelerating this natural 'salt cycle.

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Rheumatoid Arthritis Drug, Baricitinib (Olumiant), Could Put Brakes on Type 1 Diabetes

Drugs.com

THURSDAY, Dec. 7, 2023 -- A drug long used to curb rheumatoid arthritis may be a potent foe against another immune disorder, type 1 diabetes. Australian researchers report that baricitinib (Olumiant) appears to help patients newly diagnosed with.

Drugs 126
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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 127
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Chemical con artists foil drug discovery

Molecular Design

One piece of general advice that I offer to fellow scientists is to not let the fact that an article has been published in Nature (or any other ‘elite’ journal for that matter) cause you to switch off your critical thinking skills while reading it and the BW2014 article (Chemistry: Chemical con artists foil drug discovery) that I’ll be reviewing in this post is an excellent case in point.

Drugs 119
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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.