2023

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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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Gene Accessibility May Explain Why Alzheimer’s Gene, APOE4, is Less Dangerous for People of African Descent

Drug Discovery Today

(MIAMI) Researchers at the John P. Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine have found that variations in chromatin accessibility, and thus gene expression, may explain why people of European descent with APOE4 gene variants have a greater risk of developing Alzheimer’s disease than people of African descent with similar genetics.

Disease 201
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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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Parthenolide as a therapeutic for disseminated canine neoplasms [Drug Discovery and Translational Medicine]

ASPET

This study provides a unique translational research opportunity to help both humans and dogs diagnosed with diseases that carry dismal prognoses in both species: histiocytic sarcoma (HS), hemangiosarcoma (HSA), and disseminated mastocytosis/mast cell tumor (MCT). While exceedingly rare in humans, these so called "orphan diseases" are relatively more common in dogs.

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Berberine attenuates cognitive dysfunction and hippocampal apoptosis in rats with prediabetes

Chemical Biology and Drug Design

Prediabetes rat model was induced by high-fat diet and a normal diet was used as a control. They were fed for 20 weeks. At week 13, the model rats were given 100 mg/kg Berberine by gavage for 7 weeks. OGTT, fasting blood glucose, blood lipid, insulin level, and insulin resistance index were measured in each group. The cognitive function of rats was observed by a water maze test and new object recognition experiment.

Disease 189

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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 143
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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.

Therapies 142
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Prescribing Red Flags and Suspicious Controlled Substance Orders: Current Cautionary Tales

FDA Law Blog: Biosimilars

By Larry K. Houck — Separate decisions by federal district courts in Texas and Puerto Rico in the past two months provide cautionary tales for every pharmacy and wholesale distributor dispensing or distributing controlled substances. On October 10th, based on ability to pay, the U.S. District Court for the Western District of Texas imposed a $275,000 civil penalty on Zarzamora Healthcare LLC, in San Antonio, and its pharmacist-owner.

Pharmacy 139
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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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Phase III is Coming!

Drug Hunter

With initial signs of efficacy and tolerability, Drug Hunter is launching into Phase III! To address your helpful feedback, we’ve rebuilt the platform from scratch to support new features and enhancements. In a few days, you’ll see the changeover to the new version go live. Show Me the Molecules! Fig. 1. The new homepage, which you can preview at v2.drughunter.com.

Trials 247
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NY Times Gets It Wrong On Impact Of Price Controls On New Drugs

Forbes: Drug Truths

But, make no mistake – the IRA will have a major impact on biopharma R&D and, as a result, fewer important medicines will be available.

Drugs 274
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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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Longevity Biotech Startup Gero Demonstrates the Power of Quantum Computing in Drug Design

Drug Discovery Today

Research published in Nature Scientific Reports builds on earlier work and demonstrates that commercially available quantum computing services can dramatically accelerate discovery of a new universe of unique chemical structures that are synthetically feasible and possess drug-like properties for tackling complex diseases and aging

Drugs 184
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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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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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Exendin?4: A potential therapeutic strategy for Alzheimer's disease and Parkinson's disease

Chemical Biology and Drug Design

The figure depicts the neuroprotective role of exendin-4 in Alzheimer's disease and Parkinson's disease. Abstract Neurodegenerative disorders, which affect millions worldwide, are marked by a steady decline of neurons that are selectively susceptible. Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders.

Disease 189
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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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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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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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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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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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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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ACS Fall 2023 First Disclosures  

Drug Hunter

The First Disclosures sessions at the 2023 ACS Fall Meeting in San Francisco, organized by Nikki Goodwin , presented a variety of new orally available small molecules. The standing room-only sessions were so highly attended that many attendees were unfortunately kicked out due to the fire code. In case you missed any of these exciting molecules, from Scorpion’s mutant-selective PI3Kα inhibitor to Nurix’s CNS-penetrant BTK degrader, here we share the structures and targets of all the novel molecu

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To Really Impact Healthcare Costs Look Beyond Drug Prices

Forbes: Drug Truths

At a time when the opportunities for discovering and developing new medicines is the greatest in our history, we will be doing less. What we really should be doing is investing more in order to generate new medicines thus reducing downstream healthcare costs.

Drugs 264
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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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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 131
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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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BSV163/DOPE?mediated TRAIL gene transfection acts synergistically with chemotherapy against cisplatin?resistant ovarian cancer

Chemical Biology and Drug Design

Ovarian cancer is the most lethal gynecologic cancer with recurrent or resistant disease observed in 70% of patients. We described herein the anti-cancer effect of a novel lipid vector BSV163/DOPE-mediated TRAIL gene transfection in association with cisplatin in—sensitive and—resistant ovarian cancer cells. The combined treatment successfully enhanced cisplatin cytotoxicity and helped to overcome cisplatin resistance in CR-Caov3 cells.

Treatment 190
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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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Long-Acting, Injected HIV Meds Can Help Tough-to-Treat Patients

Drugs.com

WEDNESDAY, July 5, 2023 -- For nearly three decades, daily antiretroviral pills have offered patients living with HIV a highly effective way to keep their infection under control. But some patients, particularly those beset by homelessness, drug.

Drugs 131
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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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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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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 128
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More LRRK2 On-Target Toxicity?

Drug Hunter

LRRK2 has been a hotly pursued drug target for Parkinson’s Disease based on human genetics. Clinical advancement of LRRK2 inhibitors was initially stalled by concerns about on-target lung findings in primates, but these were ameliorated by a Merck/Genentech/Pfizer/MJFF study showing that these lung changes were reversible, and Biogen/Denali has currently a small molecule (BIIB122/DNL151) in Ph.