Trending Articles

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Why Mallory Freeberg is creating a perturbation catalogue

The Open Targets Blog

How can you work out the function of a particular gene or protein across different cell types and functions? If you perturb it—modify it and observe the consequences—you can start to unravel what its biological role might be, and determine whether the effects are unique to one cell type or whether they propagate across different cell contexts.

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The structure of KRASG12C bound to divarasib highlights features of potent switch-II pocket engagement

Covalent Modifiers

Fernando, M. C., Craven, G. B., & Shokat, K. M. Small GTPases, 2024 15(1), 1–7. [link] KRAS is the most frequently mutated oncogene in human cancer. In multiple types of cancer, a missense mutation at codon 12 substitutes a glycine for a cysteine, causing hyperactivation of the GTPase and enhanced MAPK signalling. Recent drug discovery efforts culminating from work during the past decade have resulted in two FDA-approved inhibitors, sotorasib and adagrasib, which target the KRASG12C mutant a

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CRISPR uncovers gene that supercharges vitamin D—and stops tumors in their tracks

Science Daily: Pharmacology News

A gene called SDR42E1 has been identified as a key player in how our bodies absorb and process vitamin D. Researchers found that disabling this gene in colorectal cancer cells not only crippled their survival but also disrupted thousands of other genes tied to cancer and metabolism. This opens the door to highly targeted cancer therapies—by either cutting off vitamin D supply to tumors or enhancing the gene’s activity to boost health.

Therapies 277
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Multifaceted In Silico Screening Strategies Identifies Potent Inhibitors Facilitating Inhibition of ZNF726 Activity in Breast Cancer

Chemical Biology and Drug Design

Zoledronic acid was identified as a lead hit targeting ZNF726 activity, inhibiting its proliferative effects on breast cancer cells. Zoledronic acid inhibits cellular cholesterol level through the inhibition of ZNF726 activity in breast cancer cells. Nine phytochemicals were identified from docking studies that might be used as potential therapeutics for targeting ZNF726 activity.

Treatment 100
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Bridging Innovation & Patient Care: The Growing Role of AI

Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health

AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis. But its impact goes far beyond research. As AI-driven innovation reshapes the clinical trial process, it’s also influencing broader healthcare trends, from personalized medicine to patient outcomes. Join this new webinar featuring Simran Kaur for an insightful discussion on what all of this means for the future of healthcare!

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Prime editing treats childhood brain disease in mice

Broad Institute

Prime editing treats childhood brain disease in mice By Corie Lok July 21, 2025 Breadcrumb Home Prime editing treats childhood brain disease in mice Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

Disease 141
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The future of CNS drug development: signs of real progress

Drug Target Review

Hundreds of life-altering conditions attack the central nervous system (CNS). Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. According to the World Health Organization (WHO) , neurological conditions are now the number one cause of ill health and disability across the globe, 1 highlighting the

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Cracking the carbene code: A 100x faster path to life-saving drugs

Science Daily: Pharmacology News

Using a clever combo of iron and radical chemistry, scientists have unlocked a safer, faster way to create carbenes molecular powerhouses key to modern medicine and materials. It s 100x more efficient than previous methods.

Drugs 123
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Recap Of The Collaborative Drug Discovery's Inaugural Canadian User Group Meeting

Collaborative Drug

The 2025 CDD User Group Meeting (UGM) Canada brought together researchers, industry experts, and thought leaders to discuss the latest advancements in drug discovery, data management, and collaborative research.

Drugs 147
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Fixing failed drugs: AI solutions for toxicity in drug discovery – part 2

Drug Target Review

In Part 2 of our conversation with Layla Hosseini-Gerami, Chief Data Science Officer at Ignota Labs , we explore the many forms of toxicity and how AI-powered tools like omics and cell painting are transforming early prediction in pre-clinical research. The conversation revealed important distinctions between different types of toxicity and our current ability to predict them: “For certain types of toxicity like liver and cardio, I think those are quite well understood in terms of the mech

Drugs 72
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Transforming Cancer Therapy: Insights from 2025 H1 FDA Approvals and Preclinical Drug Discovery

Crown Bioscience

Oncology drug approvals in H1 2025 In the first half of 2025, the FDA’s Center for Drug Evaluation and Research (CDER) approved a total of 16 novel drugs , with half of these drugs related to the treatment of cancer. Both the novel and supplemental oncology approvals reflected recent developmental innovations, including an increased focus on targeted, immunologically driven, and personalized oncology therapies, targeting a broad range of cancers.

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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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Scientists just discovered a secret code hidden in your DNA

Science Daily: Pharmacology News

What scientists once dismissed as junk DNA may actually be some of the most powerful code in our genome. A new international study reveals that ancient viral DNA buried in our genes plays an active role in controlling how other genes are turned on or off, especially during early human development. These sequences, originally from long-extinct viruses, have evolved to act like tiny genetic switches.

DNA 228
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Drug Discovery at the Broad Institute

Broad Institute

Drug Discovery at the Broad Institute By Rose Circeo July 18, 2025 Breadcrumb Home Drug Discovery at the Broad Institute Broad Institute is home to a community of drug development experts who collaborate closely with other Broad scientists to turn discoveries about the biological roots of disease into new medicines. Broad researchers use innovative approaches to accelerate the development of new drugs, tackling projects that might be viewed as too risky or early-stage for a company to take on.

Drugs 52
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Optimising T-cell expansion to improve early-stage cell therapy

Drug Target Review

Maria Knaub, Senior Medical Affairs Operations Lead at Terumo Blood and Cell Technologies (Terumo BCT), brings a unique blend of frontline experience and scientific expertise to the fore of cell therapy innovation. Her background as a paramedic and PhD in cancer biology have led Maria to lead global medical affairs efforts focused on advancing cell and gene therapies (CGTs).

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Cell Line-Derived Xenografts: A Reliable Platform for Preclinical Cancer Drug Testing

Crown Bioscience

In the complex landscape of oncology research and drug development, reliable preclinical models are crucial. Among various available platforms, cell line-derived xenografts (CDX) stand out as a robust and highly valuable model system, offering unique advantages for understanding tumor biology, drug efficacy, and therapeutic potential. This article explores the pivotal role of CDX models in preclinical cancer research, emphasizing their utility in drug discovery and development processes.

Drugs 52
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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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New Thiadiazole‐Benzenesulfonamide Hybrids as Dual B‐Raf/VEGFR‐2 Inhibitors With Promising Anti‐Hepatic Cancer Activity

Chemical Biology and Drug Design

New thiadiazole-benzenesulfonamide hybrids having the pharmacophoric features of both B-Raf and VEGFR-2 inhibitors were designed and synthesized as potent anti-cancer agents. ABSTRACT A new group of thiadiazole-benzenesulfonamide hybrids was designed, synthesized, and biologically evaluated as potential dual inhibitors targeting B-Raf and VEGFR-2 for cancer therapy.

Therapies 100
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Three-person DNA IVF stops inherited disease—eight healthy babies born in UK first

Science Daily: Pharmacology News

In a groundbreaking UK first, eight healthy babies have been born using an IVF technique that includes DNA from three people—two parents and a female donor. The process, known as pronuclear transfer, was designed to prevent the inheritance of devastating mitochondrial diseases passed down through the mother’s DNA. The early results are highly promising: all the babies are developing normally, and the disease-causing mutations are undetectable or present at levels too low to cause harm.

DNA 217
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Some Newer Antiseizure Meds Safer During Pregnancy

Drugs.com

THURSDAY, July 17, 2025 — Some newer antiseizure medications appear to be safer for pregnant women to take without risk of birth defects, a new study says. Second-generation antiseizure drugs like levetiracetam, oxcarbazepine, gabapentin and.

Drugs 283
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Outsmarting immune suppression through GPCR innovation

Drug Target Review

Immuno-oncology has transformed cancer treatment, but for many patients, tumours remain resistant to even the most advanced immune checkpoint inhibitors. Domain Therapeutics is addressing this challenge by focusing on G protein-coupled receptors (GPCRs) – a well-validated and the largest family of drug targets that remains underexplored in cancer and inflammation.

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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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Mastering Clinical Trial Submissions in Europe: A Guide for Drug Developers

thought leadership

Launching a clinical trial in Europe is a complex but rewarding endeavor, requiring careful navigation of regulatory frameworks, ethical requirements, and submission processes. Recent regulatory changes, including the transition from the EU Clinical Trial Directive to the EU Clinical Trial Regulation (EU CTR) , have harmonized approval pathways, enabling Sponsors to submit a single application covering both the applicable regulatory authorities and ethics committees through the Clinical Trial In

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Computer-Aided Drug Design and Workload Automation

Fierce BioTech

Computer-Aided Drug Design and Workload Automation scox Fri, 07/18/2025 - 11:17 Tue, 08/26/2025 - 13:00 Resource Type Webinar Nikhil Venkat Computer-aided drug design (CADD) combining computational chemistry, molecular modeling, and rational drug design together with artificial intelligence (AI) and machine learning are rapidly changing the technological landscape of drug discovery.

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This flat chip uses twisted light to reveal hidden images

Science Daily: Pharmacology News

Using advanced metasurfaces, researchers can now twist light to uncover hidden images and detect molecular handedness, potentially revolutionizing data encryption, biosensing, and drug safety.

Research 194
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Case study: an Open Targets Platform MCP server

The Open Targets Blog

This blog post is part of a series that will explore applications and expansions of the Open Targets informatics ecosystem, particularly the Open Targets Platform , through conversations with our users. Nick Ren is an engineer with a lot of experience working alongside geneticists. In 2019, as part of David Goldstein’s group, he created ATAV , a population-scale genomic-analysis platform aiming to speed up gene discovery and improve care for patients at Columbia University Medical Center.

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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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COVID Boosters Protect People With Cancer

Drugs.com

WEDNESDAY, July 23, 2025 — Cancer patients can be very vulnerable to a severe COVID infection, but COVID-19 vaccine boosters can be lifesavers for them, a new study says. COVID boosters reduced cancer patients’ hospitalizations and ICU.

Vaccine 162
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Interactive Module: Demo Workflow for Starting with a Human Gene/Protein Identifier

Broad Institute

Interactive Module: Demo Workflow for Starting with a Human Gene/Protein Identifier By Rose Circeo July 16, 2025 Breadcrumb Home Interactive Module: Demo Workflow for Starting with a Human Gene/Protein Identifier This video demonstrates a sample workflow for mapping annotations (mutations/scores/features) onto protein sequences and structures, starting with a human gene/protein identifier.

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Repurposing Drugs for Cancers of the Breast, Colon, and Ovary using Active Machine Learning and Biobank of DTC’s

Predictive Oncology

Drug repurposing using the Predictive Oncology machine learning approach and proprietary biobank of frozen dissociated tumor cells (DTCs) is outlined in this new white paper. An expedient workflow for identifying drugs that are good candidates for repurposing is demonstrated in this brand-new white paper. The Predictive Oncology machine learning approach paired with our proprietary biobank of frozen dissociated tumor cells (DTCs) offers a screening opportunity for many repurposing candidates to

Drugs 52
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Winning Regulatory Trust: Six Smart Strategies for Emerging Biotechs

PPD

Effective communication with regulatory authorities is critical for small biotech companies, as it is often the key to success in clinical trials. When small and emerging companies are able to build strong, trust-based relationships with regulators, they often see benefits like reduced development risk and accelerated timelines. However, to achieve these benefits, sponsors must have experience in and knowledge of the regulatory landscape.

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ACTO LAUNCHES CXZONE: THE FIRST AI-SIMULATED ROLEPLAY SOLUTION BUILT EXCLUSIVELY FOR LIFE SCIENCES FIELD PROFESSIONALS AND INTEGRATED WITH VEEVA PROMOMATS AND MEDCOMMS

ACTO

A Specialized Practice Environment to Sharpen Clinical Fluency, Master Product Messaging, and Drive Continuous Improvement Through a Gamified Experience BTORONTO and NEW YORK– July 24, 2025 — ACTO , a leader in AI-first commercial learning, enablement, and customer engagement solutions for life sciences and a Veeva Product Partner and member of the Veeva AI Partner Program, today announced the launch of CxZone , the first and only AI-simulated roleplay solution exclusively designed for pharmaceu

Science 52
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Highlights From the 2025 American Society for Mass Spectrometry Conference | By Jean-François Larocque and Benjamin Charron

Alta Sciences

Highlights From the 2025 American Society for Mass Spectrometry Conference | By Jean-François Larocque and Benjamin Charron aasimakopoulos Tue, 07/22/2025 - 18:05 We were thrilled to represent Altasciences at this year’s American Society for Mass Spectrometry (ASMS) conference in Baltimore. With 6,000 scientists, students, and industry professionals from around the globe in attendance, it was an excellent opportunity to learn about the latest developments in mass spectrometry and share insights

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ICH E6(R3) Guidance: Why Clinical Trial Teams Need More Than Just a Process Update

Quanticate

This article is based on the final, Step 4 version of the ICH E6(R3) Good Clinical Practice guidance, officially adopted on 6 January 2025 by the International Council for Harmonisation. This Step 4 document is the version recommended for regulatory adoption across all ICH member regions.

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Contezolid

New Drug Approvals

Contezolid MRX-I 1112968-42-9 MRX-1 B669M62ELP (5 S )-5-[(1,2-oxazol-3-ylamino)methyl]-3-[2,3,5-trifluoro-4-(4-oxo-2,3-dihydropyridin-1-yl)phenyl]-1,3-oxazolidin-2-one 4(1H)-Pyridinone, 2,3-dihydro-1-(2,3,6-trifluoro-4-((5S)-5-((3-isoxazolylamino)methyl)-2-oxo-3-oxazolidinyl)phenyl)- (5S)-5-[(1,2-oxazol-3-ylamino)methyl]-3-[2,3,5-trifluoro-4-(4-oxo-2,3-dihydropyridin-1-yl)phenyl]-1,3-oxazolidin-2-one 4(1H)-Pyridinone, 2,3-dihydro-1-[2,3,6-trifluoro-4-[(5S)-5-[(3-isoxazolylamino)methyl]-2-oxo-3-o

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New Pyrazole‐Based Tetrazole Derivatives: Synthesis, Characterization, and Their Vasorelaxant and α‐Amylase Inhibition Activities

Chemical Biology and Drug Design

A family of hybrid pyrazole tatrazole compounds was synthesized and characterized. They show a good vasorelaxant and a-amylase inhibition activities. ABSTRACT A series of new linear pyrazole-based tetrazole derivatives 1 – 10 were synthesized and characterized. The structures of the intermediate compounds were confirmed using 1 H and 13 C NMR spectroscopy, as well as high-resolution mass spectrometry (HRMS).

Research 100
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Overcoming Formulation Challenges in Generic Drug Development

Drug Patent Watch

"Breaking the Patent Barrier: The Unseen Hurdles in Generic Drug Development As the pharmaceutical industry grapples with rising costs and accessibility concerns, generic drug development has emerged as a beacon of hope. However, behind the scenes, generic manufacturers are facing a daunting challenge: overcoming formulation hurdles that can make or break the success of a generic drug.