April, 2025

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The rising impact of biomarkers in early clinical development

Drug Target Review

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Advanced strategies and tools are being implemented to establish the safety and efficacy of new therapeutic modalities, with the development of new biomarkers becoming one of the most relevant approaches for enhancing the precision and utility of early-stage studies.

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What if we find nothing in our search for life beyond Earth?

Science Daily: Pharmacology News

What if we spend decades building advanced telescopes to search for life on other planets and come up emptyhanded? A recent study exploring what we can learn about life in the universe -- even if we don't detect signs of life or habitability. Using advanced statistical modeling, the research team sought to explore how many exoplanets scientists should observe and understand before declaring that life beyond Earth is either common or rare.

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Identification of a phenyl ester covalent inhibitor of caseinolytic protease and analysis of the ClpP1P2 inhibition in mycobacteria

Covalent Modifiers

Genhui Xiao, Yumeng Cui, Liangliang Zhou, Chuya Niu, Bing Wang, Jinglan Wang, Shaoyang Zhou, Miaomiao Pan, Chi Kin Chan, Yan Xia, Lan Xu, Yu Lu, Shawn Chen mLife , 2025 DOI: [link] The caseinolytic protease complex ClpP1P2 is crucial for protein homeostasis in mycobacteria and stress response and virulence of the pathogens. Its role as a potential drug target for combating tuberculosis (TB) has just begun to be substantiated in drug discovery research.

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Novo says it’s submitted obesity pill for FDA approval

BioPharma Drive: Drug Pricing

A spokesperson confirmed the Danish drugmaker has asked U.S. regulators to clear an oral version of its weight loss drug Wegovy two years after reporting initial Phase 3 results.

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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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Novavax COVID Vaccine Moves Closer to Full FDA Approval

Drugs.com

FRIDAY, April 25, 2025 Novavaxs COVID-19 vaccine could soon receive full approval from the U.S. Food and Drug Administration (FDA), the company said Wednesday.The update sent Novavaxs stock up more than 21% in early trading. It also helped ease.

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Reviewing the IDH1 Mutation‐Mediated Mechanism of Drug Resistance and Revisiting Its Overcoming Strategies

Chemical Biology and Drug Design

Isocitrate dehydrogenase (IDH) is a key metabolic enzyme that catalyzes the conversion of isocitrate to -ketoglutaric acid (-KG). At present, there are few studies on the drug resistance of mIDH1 inhibitors. Therefore, this article first summarizes terms of IDH1 function from the molecular mechanism of IDH1, the mechanism of mutation, and the metabolism of substances due to mutations.

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Researchers use machine learning to engineer 'bespoke enzymes' for gene editing

Science Daily: Pharmacology News

Genome editing has advanced at a rapid pace with promising results for treating genetic conditions -- but there is always room for improvement. A new paper showcases the power of scalable protein engineering combined with machine learning to boost progress in the field of gene and cell therapy. In their study, authors developed a machine learning algorithm -- known as PAMmla -- that can predict the properties of about 64 million genome editing enzymes.

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Proteomic Ligandability Maps of Phosphorus(V) Stereoprobes Identify Covalent TLCD1 Inhibitors

Covalent Modifiers

Hayden A. Sharma, Michael Bielecki, Meredith A. Holm, Ty M. Thompson, Yue Yin, Jacob B. Cravatt, Timothy B. Ware, Alex Reed, Molhm Nassir, Tamara El-Hayek Ewing, Bruno Melillo, J. Fernando Bazan, Phil S. Baran, and Benjamin F. Cravatt Journal of the American Chemical Society 2025 DOI: 10.1021/jacs.5c01944 Activity-based protein profiling (ABPP) of stereoisomerically defined sets of electrophilic compounds (stereoprobes) offers a versatile way to discover covalent ligands for proteins in native b

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US risks losing biotech edge to China, report warns

BioPharma Drive: Drug Pricing

“There will be a ChatGPT moment for biotechnology,” a bipartisan commission wrote in the new report, which calls for the U.S. to invest at least $15 billion into the sector over the next five years.

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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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HHS Announces Plans to Pay Moderna $176 Million for mRNA Flu Vaccine

Drugs.com

FRIDAY, April 25, 2025 -- On Tuesday, the U.S. Department of Health and Human Services announced that the government will invest $176 million in Moderna to expedite the creation of a pandemic influenza vaccine, according to a report from the.

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AI in drug discovery: faster, smarter, better

Drug Target Review

In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. At the forefront of this revolution stands Claudia Zylberberg, a scientist, entrepreneur, and advocate for accessible and advanced healthcare solutions. Zylberberg has built an impressive career at the intersection of science, technology, and business.

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Network Pharmacology Integrated Experimental Validation Uncover Quercetin as the Key Ingredient of Hedyotis diffusa Anti‐BRCA

Chemical Biology and Drug Design

Quercetin is the core component of Hedyotis diffusa against breast cancer (BRCA). CDK1, BIRC5, and HSP90AA1 play key roles in the anti-BRCA effect of Hedyotis diffusa. ABSTRACT Hedyotis diffusa , a famous Traditional Chinese Medicine (TCM), has been extensively used clinically for thousands of years. Although the therapeutic effect of Hedyotis diffusa on tumors has attracted wide attention, components, and mechanisms against breast cancer (BRCA) have not been fully understood.

Treatment 100
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Our DNA is at risk of hacking, warn scientists

Science Daily: Pharmacology News

According to new research next-generation DNA sequencing (NGS) -- the same technology which is powering the development of tailor-made medicines, cancer diagnostics, infectious disease tracking, and gene research -- could become a prime target for hackers.

DNA 360
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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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Discovery and Optimization of a Covalent AKR1C3 Inhibitor

Covalent Modifiers

R. Justin Grams, Wesley J. Wolfe, Robert J. Seal, James Veccia, and Ku-Lung Hsu Journal of Medicinal Chemistry 2025 [link] Aldo-keto reductase family 1 member C3 (AKR1C3) is a member of the AKR superfamily of enzymes that metabolize androgen, estrogen, and prostaglandin substrates that drive proliferation in hormone-dependent cancers. Interest in developing selective inhibitors has produced tool compounds for the inactivation or degradation of AKR1C3 with varying degrees of selectivity among the

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New research kindles excitement around stem cell therapies for Parkinson’s

BioPharma Drive: Drug Pricing

Two studies published in Nature found stem cell-derived products can not only be safely transplanted into the brain, but also show promising — albeit unproven — signs of efficacy.

Therapies 312
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Supreme Court Case Targets Free Preventive Care Provided By The Affordable Care Act

Drugs.com

MONDAY, April 21, 2025 -- Colonoscopy for younger adults. Chest CT scans for lung cancer. Statin meds to lower cholesterol. Screening for drug addiction.More than 150 million Americans might lose no-cost access to these and other preventive care.

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CDD Vault Update (April 2025 #2): Pharmacokinetic (PK) and Michaelis-Menten Kinetics (Km/Kd) Curve Fit Equations, Donut Charts, TIFF Image Previews, and Parallel Reactions

Collaborative Drug

CDD Update March #2 2025 Full Atom Layout of Macromolecules New Dataset for AI Similarity Searching Import Inventory Locations by Unique Box Names:

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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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SAIAME: Semi‐Parameter Adaptation Information‐Assisted Multi‐Objective Evolutionary for Protein‐Ligand Docking

Chemical Biology and Drug Design

A multi-objective optimization algorithm incorporating the strategies of dynamic semi-parameter adaptation updating, gradient enhancement, and population size reduction is adopted to solve the problem of protein-ligand docking. ABSTRACT Molecular docking, which simulates the binding pose of a drug molecule to target proteins and predicts the binding affinity, is an important computational tool in structure-based drug discovery.

Drugs 100
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Sunscreen, clothes and caves may have helped Homo sapiens survive 41,000 years ago

Science Daily: Pharmacology News

A study suggests that Homo sapiens may have benefited from the use of ochre and tailored clothing during a period of increased UV light 41,000 years ago, during the Laschamps excursion.

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Substrate Trapping in Polyketide Synthase Thioesterase Domains: Structural Basis for Macrolactone Formation

Covalent Modifiers

Tyler M. McCullough, Vishakha Choudhary, David L. Akey, Meredith A. Skiba, Steffen M. Bernard, Jeffrey D. Kittendorf, Jennifer J. Schmidt, David H. Sherman, and Janet L. Smith ACS Catalysis 2024 14 (16), 12551-12563 DOI: 10.1021/acscatal.4c03637 Emerging antibiotic resistance requires continual improvement in the arsenal of antimicrobial drugs, especially the critical macrolide antibiotics.

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FDA plans to phase out animal testing for some drugs

BioPharma Drive: Drug Pricing

The initiative, which will start with monoclonal antibodies, is the first big regulatory shift from the FDA since Martin Makary was sworn in.

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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 Antibiotic, Blujepa, May Be Effective in Treating Gonorrhea

Drugs.com

TUESDAY, April 15, 2025 — A newly approved antibiotic to treat urinary tract infections may also help fight drug-resistant gonorrhea, a new study shows. The medication, called gepotidacin, sold under the brand name Blujepa, could become the.

Drugs 288
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CDD Vault Update (April 2025 #3): AI+ Folding and Docking

Collaborative Drug

CDD Update March #2 2025 Full Atom Layout of Macromolecules New Dataset for AI Similarity Searching Import Inventory Locations by Unique Box Names:

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Fitusiran

New Drug Approvals

Fitusiran 1711.0g/mol, C 78 H 139 N 11 O 30 FDA APPROVED 3/28/2025, Qfitlia , To prevent or reduce the frequency of bleeding episodes in hemophilia A or B Press Release CAS 1499251-18-1 EX-A12034 DA-53206 N-[1,3-Bis[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]-12-[(2R,4R)-4

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New gene editing tool shows promise for treating diseases with multiple mutations

Science Daily: Pharmacology News

Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations. The system can be formulated completely as RNA, dramatically simplifying delivery logistics compared to traditional systems that use both RNA and DNA. By inserting an entire gene, the tool offers a one-and-done approach that overcomes hurdles from CRISPR gene editing technology -- which is programmed to correct individual mutations -- offerin

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Factors affecting irreversible inhibition of EGFR and influence of chirality on covalent binding

Covalent Modifiers

Pasquale A. Morese, Ayaz Ahmad, Mathew P. Martin, Richard A. Noble, Sara Pintar, Lan Z. Wang, Shangze Xu, Andrew Lister, Richard A. Ward, Agnieszka K. Bronowska, Martin E. M. Noble, Hannah L. Stewart & Michael J. Waring Commun Chem 8 , 111 (2025). [link] The discovery of targeted covalent inhibitors is of increasing importance in drug discovery.

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How integrated real-world data helps identify and improve outcomes for rare disease patients

BioPharma Drive: Drug Pricing

Pharmaceutical companies are turning to integrated RWD approaches to overcome challenges in rare disease research.

Disease 278
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A New UV Light May Help Stop Future Pandemics

Drugs.com

THURSDAY, April 10, 2025 -- Five years after COVID-19 first hit the United States, scientists are already brainstorming how to stop the next big virus.One possible solution? A special kind of ultraviolet light called far-UVC, CBS News.

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CDD Vault Update (April 2025): Biphasic Curve Fit, Import Parser Sections, Custom Calculation Functions, Generate Inventory Labels, Inventory Admin Permission

Collaborative Drug

CDD Update March #2 2025 Full Atom Layout of Macromolecules New Dataset for AI Similarity Searching Import Inventory Locations by Unique Box Names:

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