Sat.Dec 02, 2023 - Fri.Dec 08, 2023

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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 347
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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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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 321
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Discovery of YSR734: A Covalent HDAC Inhibitor with Cellular Activity in Acute Myeloid Leukemia and Duchenne Muscular Dystrophy

Covalent Modifiers

Yasir S. Raouf, Abootaleb Sedighi, Mulu Geletu, Geordon A. Frere, Rebecca G. Allan, Nabanita Nawar, Elvin D. de Araujo, and Patrick T. Gunning Journal of Medicinal Chemistry 2023 DOI: 10.1021/acs.jmedchem.3c01236 Histone deacetylases (HDACs) have emerged as powerful epigenetic modifiers of histone/non-histone proteins via catalyzing the deacetylation of ε-N-acetyl lysines.

Disease 200
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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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Radiopharma startup Artbio raises $90M in sign of field’s momentum

BioPharma Drive: Drug Pricing

The funding is indicative of investor interest in an area of drug research that involves at least a dozen startups and multiple publicly traded companies.

More Trending

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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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Covalent targeting of non-cysteine residues in PI4KIII?

Covalent Modifiers

Brett Cosgrove, Emma K. Grant, Sophie Bertrand, Kenneth D. Down, Don O. Somers, John P. Evans d, Nicholas C. O. Tomkinson and Michael D. Barker RSC Chem. Biol. , 2023, 4, 1111-1122 DOI: 10.1039/D3CB00142C The synthesis and characterisation of fluorosulfate covalent inhibitors of the lipid kinase PI4KIIIβ is described. The conserved lysine residue located within the ATP binding site was targeted, and optimised compounds based upon reversible inhibitors with good activity and physicochemical profi

Drugs 189
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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 305
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Harvesting water from air with solar power

Science Daily: Pharmacology News

Researchers have developed a promising new solar-powered atmospheric water harvesting technology that could help provide enough drinking water for people to survive in difficult, dryland areas: They synthesized a super hygroscopic gel capable of absorbing and retaining an unparalleled amount of water. One kilogram of dry gel could adsorb 1.18 kilograms of water in arid atmospheric environments and up to 6.4 kilograms in humid atmospheric environments.

Research 328
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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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Diabetes Meds Like Ozempic, Mounjaro Might Also Lower Risks for Colon Cancer

Drugs.com

FRIDAY, Dec. 8, 2023 -- Could blockbuster diabetes and weight-loss meds such as Ozempic, Wegovy, Mounjaro and Zepbound also lower users' odds for colon cancer? New research suggests they might.All of these medications (and more) fall into a class.

Research 307
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Covalent Modifiers - Untitled Article

Covalent Modifiers

Ahmed Fnaiche, Hwai-Chien Chan, Alexis Paquin, Narjara González Suárez, Victoria Vu, Fengling Li, Abdellah Allali-Hassani, Michelle Ada Cao, Magdalena M. Szewczyk, Albina Bolotokova, Frédéric Allemand, Muriel Gelin, Dalia Barsyte-Lovejoy, Vijayaratnam Santhakumar, Masoud Vedadi, Jean-François Guichou, Borhane Annabi, and Alexandre Gagnon ACS Medicinal Chemistry Letters 2023 DOI: 10.1021/acsmedchemlett.3c00386 The transcription factor YAP–TEAD is the downstream effector of the Hippo pathway which

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Pricey new gene therapies for sickle cell pose access test

BioPharma Drive: Drug Pricing

Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1 million.

Therapies 294
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Stimulating nerves connected to the pancreas regenerates insulin-producing cells, mouse study shows

Science Daily: Pharmacology News

Decreasing pancreatic beta cell numbers -- the only cells that produce insulin -- is a leading cause of diabetes. In a promising development, a research group has revealed that stimulating autonomic vagal nerves connected to the pancreas can improve the function and also increase the number of pancreatic beta cells in mice.

Research 325
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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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STD Specialists Warn of Shortage of Vital Syphilis Drug, Bicillin L-A

Drugs.com

MONDAY, Dec. 4, 2023 -- As syphilis cases surge across America, a group representing the nation's STD specialists says members are reporting shortages of a drug essential to fighting the disease. In a survey from the National Coalition of STD.

Drugs 306
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Covalent PROTAC design method based on a sulfonyl pyridone probe

Covalent Modifiers

Qinhong Luo, Yaqi Wang, Zhanfeng Hou, Huiting Liang, Licheng Tu, Yun Xing, Chuan Wan, Jianbo Liu, Rui Wang, Lizhi Zhu, Wei Han, Jianlong Wu, Fei Lu, Feng Yin and Zigang Li Chem. Commun. , 2024 DOI: 10.1039/D3CC05127G Covalent proteolysis-targeting chimeras (PROTACs) offer enhanced selectivity, prolonged action, and increased efficacy against challenging target proteins.

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Novartis gets FDA approval of closely watched rare disease drug

BioPharma Drive: Drug Pricing

The pharma is developing Fabhalta, now cleared for paroxysmal nocturnal hemoglobinuria, for several other rare, complement-driven diseases.

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Snail-inspired robot could scoop ocean microplastics

Science Daily: Pharmacology News

Inspired by a small and slow snail, scientists have developed a robot protype that may one day scoop up microplastics from the surfaces of oceans, seas and lakes.

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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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One Food Could Boost Health of Colon Cancer Survivors

Drugs.com

MONDAY, Dec. 4, 2023 -- Colon cancer survivors can give their health a boost by eating more navy beans, a new clinical trial finds.Small, white navy beans are full of gut-supporting fibers, amino acids and other nutrients that can help the.

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Structure of Staphylococcus aureus ClpP Bound to the Covalent Active Site Inhibitor Cystargolide A

Covalent Modifiers

Illigmann, Astrid , Vielberg, Marie-Theres , Lakemeyer, Markus , Wolf, Felix , Dema, Taulant , Stange, Patrik , Kuttenlochner, Wolfgang , Liebhart, Elisa , Kulik, Andreas , Staudt, Nicole , Malik, Imran , Grond, Stephanie , Sieber, Stephan A. , Kaysser, Leonard , Groll, Michael , Brötz-Oesterhelt, Heike , Angew. Chem. Int. Ed. 2023 , e202314028. [link] The caseinolytic protease is a highly conserved serine protease, crucial to prokaryotic and eukaryotic protein homeostasis, and a promising antib

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Overcoming scalability challenges in real-world data processing for transformative patient care

BioPharma Drive: Drug Pricing

Accurate and timely processing of data is imperative to create robust analytical datasets that can be used in the RWE setting.

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ChatGPT often won't defend its answers -- even when it is right

Science Daily: Pharmacology News

ChatGPT may do an impressive job at correctly answering complex questions, but a new study suggests it may be absurdly easy to convince the AI chatbot that it's in the wrong.

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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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Spotting Epilepsy in Kids Isn't Always Easy: Know the Signs

Drugs.com

WEDNESDAY, Dec. 6, 2023 -- Neurologist Dr. Deborah Holder says she often has parents come to her with kids who've experienced what they call "funny spells." “Sometimes I start talking to a parent and find out the parent has [also] had.

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A simple method for developing lysine targeted covalent protein reagents

Covalent Modifiers

Gabizon, Ronen; Tivon, Barr; Reddi, Rambabu N.; van den Oetelaar; Maxime C. M.; Amartely, Hadar; Cossar, Peter J.; Ottmann, Christian London, Nir Nat Commun 14 , 7933 (2023). [link] Peptide-based covalent probes can target shallow protein surfaces not typically addressable using small molecules, yet there is a need for versatile approaches to convert native peptide sequences into covalent binders that can target a broad range of residues.

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Roche joins obesity drug chase with $2.7B deal for startup Carmot

BioPharma Drive: Drug Pricing

The acquisition of Carmot, which had been planning an IPO, will hand Roche three weight loss medicines in early clinical testing.

Drugs 288
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Fossil CO2 emissions at record high in 2023

Science Daily: Pharmacology News

Global carbon emissions from fossil fuels have risen again in 2023 -- reaching record levels, according to new research.

Research 318
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Cognitive Decline May Come Earlier for People With Epilepsy

Drugs.com

TUESDAY, Dec. 5, 2023 -- People with epilepsy suffer quicker declines in thinking than people without the brain disorder, particularly if they also have risk factors like high blood pressure or diabetes, a new study finds.The difference was.

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