Trending Articles

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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 276
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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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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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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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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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FDA Approves Prefilled Syringe Presentation for Shingles Vaccine

Drugs.com

MONDAY, July 21, 2025 -- The U.S. Food and Drug Administration has approved a prefilled syringe presentation of Shingrix (zoster vaccine recombinant, adjuvanted) for the prevention of shingles (herpes zoster). The existing vaccine consists of two.

Vaccine 248
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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 118

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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 130
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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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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 210
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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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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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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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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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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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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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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 185
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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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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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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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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.

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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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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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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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CDMO Price Benchmarking: Unlocking Market Domination Through Strategic Manufacturing Partnerships

Drug Patent Watch

"Unlocking Market Domination: How Strategic Manufacturing Partnerships Can Give Your CDMO an Edge in a Competitive Landscape As the pharmaceutical industry continues to evolve, Contract Development and Manufacturing Organizations (CDMOs) are under increasing pressure to deliver high-quality products at competitive prices. But what sets the leaders apart from the rest?

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CMC Expectations During Drug Substance Transfer from Ex-US Manufacturers

thought leadership

Regulatory Drivers for US-based API Manufacturers Recent shifts in US tariff policies have introduced new pressure points in the global pharmaceutical supply chain, particularly for manufacturers sourcing drug substances and active pharmaceutical ingredients (APIs) from overseas—most notably from China. In response, many firms are now evaluating the feasibility of transferring manufacturing operations to US facilities.

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Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks

BioPharma Drive: Drug Pricing

Skip to main content CONTINUE TO SITE ➞ Dont miss tomorrows biopharma industry news Let BioPharma Dives free newsletter keep you informed, straight from your inbox. Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy.

Therapies 301
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Few Babies Getting RSV Antibody Shot, Beyfortus, Study Says

Drugs.com

THURSDAY, July 17, 2025 — A new antibody shot that protects babies against RSV infection could be struggling to gain traction, researchers report. Only about a third (35%) of babies eligible for nirsevimab got the injection during the 2023-24.

Research 288
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ChEBI 2.0 Data Products

The ChEMBL-og

Image generated using DALLE-3 Introduction Dear ChEBI users, If you have visited the ChEBI website recently, you have been able to intuit that something really great is just about to come. Our team has been working for the last three years on the redevelopment of ChEBI , which includes: a new website, modern infrastructure, a new submission and curator tool and improvements in the ChEBI data products (ontology, TSV flat files, SDF files, database dump).

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AI Meets Drug Discovery – But Who Gets the Patent?

Drug Patent Watch

"The Patent Paradox: As AI Revolutionizes Drug Discovery, Who Owns the Breakthroughs? The pharmaceutical industry is on the cusp of a revolution, with artificial intelligence (AI) poised to transform the way new medicines are discovered. But as AI-powered tools begin to yield groundbreaking results, a pressing question arises: who will hold the patent rights to these innovations?

Drugs 53
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Turning Sites into Your Enrollment Superpower

Antidote

For all the focus placed on patient recruitment metrics, it's easy to forget where actual enrollment happens: at the site. Yet all too often, sponsors and CROs overlook the incredible potential that lies in empowering research sites to become active, agile, and confident engines of recruitment.

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2024 Gross-to-Net Realities at 9 Top Drugmakers: A New Era of Market Access

Drug Channels

It’s time for Drug Channels ’ annual update on drug pricing trends at the largest pharmaceutical manufacturers. This year’s review includes the following nine companies: Bristol Myers Squibb, Eli Lilly and Company, Genentech, GlaxoSmithKline, Johnson & Johnson, Sanofi, Takeda, Teva, and UCB. You can find links to each company’s data below. These data highlight divergent trends reshaping the gross-to-net bubble: Rebates, discounts, and other fees reduced the selling prices of brand-name drugs

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July Fellow's Spotlight on Alicer Andrew

keypoint

This month's Fellows Spotlight goes to Keystone Symposia Fellow Dr. Alicer Andrew! Born and raised on the Caribbean island of Antigua, Dr. Andrew is a postdoctoral scholar at the UCSF Gladstone Institutes, where she studies HIV in the lab of Dr. Nadia Roan. She works in the Institute of Virology, where she characterizes HIV-specific T cell responses in people living with HIV, and endometrial immune responses in the context of infectious diseases and infertility.