December, 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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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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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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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
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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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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 135
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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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Scientists map the locations of hundreds to thousands of cell types across a mammalian brain

Broad Institute

Scientists map the locations of hundreds to thousands of cell types across a mammalian brain By Leah Eisenstadt December 13, 2023 Breadcrumb Home Scientists map the locations of hundreds to thousands of cell types across a mammalian brain Two Broad research teams use spatial transcriptomics to create detailed atlases of the mouse nervous system, paving the way for similar efforts in humans.

DNA 140
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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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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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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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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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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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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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New “dictionary” of immune responses reveals far more complexity in the immune system than previously thought

Broad Institute

New “dictionary” of immune responses reveals far more complexity in the immune system than previously thought By Allessandra DiCorato December 6, 2023 Breadcrumb Home New “dictionary” of immune responses reveals far more complexity in the immune system than previously thought Scientists map the effects of 86 immune-signaling molecules called cytokines on every major immune cell type, creating a reference for studying the inner workings of the immune system.

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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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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 138
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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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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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Cancer drug discovery company wins £50K Franklin research residency

Drug Discovery World

Oxford Target Therapeutics, a company developing a promising therapy for triple negative breast cancer, is one of three UK life science companies awarded research prizes by the Rosalind Franklin Institute, based in Oxfordshire, UK. The prizes, awarded in collaboration with the Science and Technology Facilities Council (STFC), a part of UKRI, enable the winners to benefit from the Franklin’s top-level technologies and support to move their work forward.

Research 266
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More than a meteorite: New clues about the demise of dinosaurs

Science Daily: Pharmacology News

What wiped out the dinosaurs? A meteorite plummeting to Earth is only part of the story, a new study suggests. Climate change triggered by massive volcanic eruptions may have ultimately set the stage for the dinosaur extinction, challenging the traditional narrative that a meteorite alone delivered the final blow to the ancient giants.

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Q&A: How to jump-start new psychiatric and neurological drug development

Broad Institute

Q&A: How to jump-start new psychiatric and neurological drug development By Tom Ulrich December 18, 2023 Breadcrumb Home Q&A: How to jump-start new psychiatric and neurological drug development Stanley Center director Steve Hyman talks about a new report he co-authored that explores the obstacles in developing new treatments and how to overcome them By Tom Ulrich December 18, 2023 Steven Hyman Related people Steven Hyman Related programs Stanley Center for Psychiatric Research Related News Scien

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Tome Biosciences debuts with $213M and a new way to edit the genome

BioPharma Drive: Drug Pricing

Based on the work of MIT scientists, the well-funded startup is developing ways to insert large sizes of genetic material anywhere in the genome without damaging or breaking DNA.

DNA 129
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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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FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

Drugs.com

FRIDAY, Dec. 8, 2023 -- The U.S. Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. Casgevy, developed by Vertex.

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Discovery lights the way for new secondary breast cancer drugs

Drug Discovery World

Using a novel approach, researchers from The Institute of Cancer Research, London have uncovered details of secondary breast cancer in the brain and spinal cord that may help with developing effective treatments. The study revealed for the first time that breast cancer leptomeningeal metastasis (BCLM) cells spread early from the primary breast tumour and that they acquire features typically associated with lobular breast cancer, which develops in the glands that produce breast milk.

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10 achondroplasia facts to note

Antidote

Achondroplasia, a term meaning “without cartilage formation,” is a genetic disorder leading to disproportionate short stature. Sometimes called achondroplastic dwarfism or ACH , this condition has few treatment options and no known cure. However, individuals diagnosed with this condition can typically lead fulfilling, healthy lives with proper management.

Treatment 121
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New protein linked to early-onset dementia identified

Science Daily: Pharmacology News

Scientists have identified abnormal aggregates of a protein called TAF15 in the brains of individuals with early-onset dementia, known as frontotemporal dementia, where the cause was not previously known.

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AbbVie to buy Cerevel in $8.7B bet on brain drugs

BioPharma Drive: Drug Pricing

The deal hands AbbVie a portfolio of psychiatric medicines that originated within Pfizer, among them a closely watched schizophrenia treatment that’s in late-stage testing.

Treatment 122
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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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Marijuana Use Increasingly Linked to Addiction, Psychosis

Drugs.com

WEDNESDAY, Dec. 27, 2023 (HealthDay news) -- A rising tide of cannabis-related mental health problems is resulting from the widespread legalization of recreational weed in the United States, warns a new evidence review.Nearly one in five Americans.

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Gene therapy could reduce seizures in childhood epilepsy

Drug Discovery World

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. Focal cortical dysplasia is caused by areas of the brain that have developed abnormally and is among the most common causes of drug-resistant epilepsy in children. Epilepsy in focal cortical dysplasia is associated with comorbidities, including learning disabilities.

Therapies 264
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What is chronic spontaneous urticartia (chronic hives)?

Antidote

Chronic spontaneous urticaria, also called chronic idiopathic urticaria, is a type of chronic hives that come and go unexpectedly. These hives persist daily for a minimum of six weeks without a clear cause or trigger. While this condition can affect anyone at any point, women experience it twice as often as men , and it’s most common between the ages of 20 and 40.

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How a drought led to the rise of skateboarding in 1970s California

Science Daily: Pharmacology News

Why did professional skateboarding arise in southern California in the 1970s? Was it a coincidence, or was it a perfect storm of multiple factors? It's fairly well-known that a drought in southern California in the mid-1970s led to a ban on filling backyard swimming pools, and these empty pools became playgrounds for freestyle skateboarders in the greater Los Angeles area.

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Apellis eye drug likely to be rejected in Europe, company says

BioPharma Drive: Drug Pricing

According to Apellis executives, reviewers in Europe are skeptical of the benefits of its geographic atrophy medicine, Syfovre, and appear poised to recommend against approval at a meeting next month.

Drugs 122
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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.