Thu.Nov 16, 2023

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Great Britain first to approve CRISPR-based gene therapy

Drug Discovery World

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy (exagamglogene autotemcel, or exa-cel) is the first medicine to be licensed that uses gene editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

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??elemene enhances cisplatin sensitivity of non?small cell lung cancer cells via the miR?17?5p/STAT3 axis

Chemical Biology and Drug Design

β-elemene regulates miR-17-5p/STAT3 axis to sensitize NSCLC cells to cisplatin, suggesting that it is a potential drug for the palliative treatment of NSCLC. Abstract In China, β-elemene, a sesquiterpene compound derived from Curcuma wenyujin , is clinically used to treat many human malignancies, including non-small cell lung cancer (NSCLC). Nonetheless, the role of β-elemene in regulating cisplatin sensitivity of NSCLC cells and the related mechanisms are not clear.

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AI and transcriptomics identify dual targets for ageing and cancer

Drug Discovery World

An international research team is the first to use artificial intelligence (AI) analysis to identify dual-purpose target candidates for the treatment of cancer and ageing. Researchers from the University of Oslo, University of Chicago Pritzker School of Medicine, and Insilico Medicine used Insilico’s AI target discovery engine, PandaOmics, to analyse transcriptomic data derived from 16,740 healthy samples and 11,303 tumours (across 11 solid cancers).

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World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia

BioPharma Drive: Drug Pricing

Clearance of Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy in the U.K. comes ahead of expected regulatory decisions in the U.S. and Europe.

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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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A comprehensive approach to immunophenotyping for targeted drug discovery and development

Drug Discovery World

With high costs of therapeutic development, success relies on optimising preclinical and early clinical studies. Download this content-rich digital article to read about considerations for implementing and standardising high-dimensional immune profiling methods into drug development programs. Learn about different applications that demonstrate the robustness and reproducibility of a mass cytometry-based analysis to deeply interrogate complex biological systems in drug development strategies, inc

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Antimicrobial resistance with Recce Pharmaceuticals

Drug Target Review

What is the thesis underlying Recce Pharmaceutical’s approach? Our approach is to combat the increasing global health threat posed by antimicrobial resistance (AMR) by advancing a new class of synthetic anti-infectives with a unique mechanism of action. AMR results from the overuse of antimicrobials, contributing to the development of multi-drug resistant bacterial species, often referred to as superbugs.

More Trending

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FDA approves new Bristol Myers drug for lung cancer

BioPharma Drive: Drug Pricing

Acquired via a $4 billion biotech buyout, Augtyro is one of an array of new products the pharma hopes will offset patent expirations for current top sellers.

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University spin-out secures £4.3M to develop precision antibiotics

Drug Discovery World

Glox Therapeutics has raised £4.3M ($5.2M) in seed funding to develop targeted therapeutics against antibiotic-resistant Gram-negative bacteria. The company will use the funding to establish laboratories in Oxford and Glasgow and expand its team to accelerate its bacteriocin development programme. Glox Therapeutics is developing precision antibiotics using engineered protein bacteriocins.

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West Coast investor Red Tree on its San Diego expansion and staying bullish in biotech

BioPharma Drive: Drug Pricing

Red Tree Venture Capital plans to tap into San Diego’s biotech pipeline as it works to build a West Coast competitor to Boston-area investors.

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Isomer FOMO? Unlock hidden isomers in your LC-MS analysis

Drug Discovery World

MOBILion Systems is pioneering the evolution of separation science. High-resolution ion mobility (HRIM) on the MOBIE platform offers deeper, more accurate, and efficient characterisation of complex molecules than traditional methods. Download the whitepaper “High-Throughput Monitoring of Biotherapeutic Critical Quality Attributes with High-Resolution Ion Mobility-Mass Spectrometry (HRIM-MS)” to learn more about: Enhancing your analysis of post-translational modifications (PTMs) with reduced runt

Science 130
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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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Homology enters reverse merger with Q32 Bio

BioPharma Drive: Drug Pricing

The deal, which will result in a combined company operating under Q32's name, comes months after Homology laid off much of its staff and halted research.

Research 107
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New whitepaper: Unlock hidden isomers in your LC-MS analysis

Drug Discovery World

Feeling isomer FOMO? Download this new whitepaper from MOBILion Systems ‘ High-Throughput Monitoring of Biotherapeutic Critical Quality Attributes with High-Resolution Ion Mobility-Mass Spectrometry (HRIM-MS)’ MOBILion Systems is pioneering the evolution of separation science. High-resolution ion mobility (HRIM) on the MOBIE platform offers deeper, more accurate, and efficient characterisation of complex molecules than traditional methods.

Science 130
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Paper details Astellas gene therapy study that led to patient deaths

BioPharma Drive: Drug Pricing

The company, which is working with regulators to lift a clinical hold, said the treatment could still help people with X-linked myotubular myopathy.

Therapies 107
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Realistic talking faces created from only an audio clip and a person's photo

Science Daily: Pharmacology News

A team of researchers has developed a computer program that creates realistic videos that reflect the facial expressions and head movements of the person speaking, only requiring an audio clip and a face photo. DIverse yet Realistic Facial Animations, or DIRFA, is an artificial intelligence-based program that takes audio and a photo and produces a 3D video showing the person demonstrating realistic and consistent facial animations synchronised with the spoken audio (see videos).

Research 101
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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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The mind’s eye of a neural network system

SCIENMAG: Medicine & Health

WEST LAFAYETTE, Ind. – In the background of image recognition software that can ID our friends on social media and wildflowers in our yard are neural networks, a type of artificial intelligence inspired by how own our brains process data.

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Breeding Better Butternut Squash – and a Thanksgiving Recipe

PLOS: DNA Science

After a long career as a science writer, textbook author, and genetic counselor, I’ve become an accidental authority on squashes. I began volunteering at the largest food pantry in my small city in June 2020, where my husband Larry had been in charge of the plant and fungal kingdoms for years. It was the height of the pandemic. So gloved and masked, we shoved fruits and veggies into plastic bags, filled shopping carts with the bags, and wheeled them over to a window at which another masked

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Drug manufacturers use FDA, patent strategies to keep insulin prices high

SCIENMAG: Medicine & Health

Over the last four decades, insulin manufacturers have extended their periods of market exclusivity on brand-name insulin products by employing several strategies, including filing additional patents on their products after FDA approval and obtaining many patents on delivery devices for their insulin products.

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Birds set foot near South Pole in Early Cretaceous, Australian tracks show

Science Daily: Pharmacology News

The discovery of 27 avian footprints on the southern Australia coast -- dating back to the Early Cretaceous when Australia was still connected to Antarctica -- opens another window onto early avian evolution and possible migratory behavior.

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Top FDA official Woodcock to retire early next year

BioPharma Drive: Drug Pricing

The 37-year veteran helmed the agency’s pandemic response, and was a key decision-maker in controversial calls on opioids, an Alzheimer's therapy and muscular dystrophy drugs.

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Plants that survived dinosaur extinction pulled nitrogen from air

Science Daily: Pharmacology News

Ancient cycad lineages that survived the extinction of the dinosaurs may have done so by relying on symbiotic bacteria in their roots to fix atmospheric nitrogen. The finding came from an effort to understand ancient atmospheres, but became an insight into plant evolution instead.

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Bear genes show circadian rhythms even during hibernation

SCIENMAG: Medicine & Health

PULLMAN, Wash. – The internal clocks of grizzly bears appear to keep ticking through hibernation, according to a genetic study. This persistence highlights the strong role of circadian rhythms in the metabolism of many organisms including humans. Credit: Washington State University PULLMAN, Wash.

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New patent for Sk Life drug XCOPRI

Drug Patent Watch

Annual Drug Patent Expirations for XCOPRI Xcopri is a drug marketed by Sk Life and is included in one NDA. It is available from one supplier. There are two patents… The post New patent for Sk Life drug XCOPRI appeared first on DrugPatentWatch - Make Better Decisions.

Drugs 59
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GTEX Portal: Downloading Open Access Files from the GTEx Portal

Broad Institute

GTEX Portal: Downloading Open Access Files from the GTEx Portal By Rose Circeo November 16, 2023 Breadcrumb Home GTEX Portal: Downloading Open Access Files from the GTEx Portal This video demonstrates how to download open access data files from the GTEx Portal. For more information visit: [link].

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New patent for Avid Radiopharms drug TAUVID

Drug Patent Watch

Annual Drug Patent Expirations for TAUVID Tauvid is a drug marketed by Avid Radiopharms Inc and is included in one NDA. It is available from one supplier. There is one… The post New patent for Avid Radiopharms drug TAUVID appeared first on DrugPatentWatch - Make Better Decisions.

Drugs 59
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Concerns Regarding Unregulated Clinical Decision Support Software

Common Sense for Drug Policy Blog

Concerns Regarding Unregulated Clinical Decision Support Software "While we do not know exactly which data elements are included in NarxCare, evidence suggests the algorithm differentially identifies individuals on the basis of work status, disability, and insurance, among other characteristics. 42 Cochran et al. 34 found patients with higher pain severity or interference, those who were widowed, on leave, retired, or disabled, were most likely to have artificially elevated Narx Scores (“false p

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New tentative approval for Rising drug acetylcysteine

Drug Patent Watch

Acetylcysteine is the generic ingredient in seven branded drugs marketed by Cumberland Pharms, Aspen, Eugia Pharma, Exela Pharma, Fresenius Kabi Usa, Indoco, Rising, Sagent Pharms Inc, Steriscience, Zydus Pharms, Alvogen,… The post New tentative approval for Rising drug acetylcysteine appeared first on DrugPatentWatch - Make Better Decisions.

Drugs 59
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Meet the Expert: Brian Wellins

thought leadership

Our "Meet the Expert" series introduces you to our team of experts around the world. This "behind the curtain" view will help you get to know who we are on a professional and personal level, and highlight how our colleagues work together on our higher purpose to improve patient health and safety throughout the complete product lifecycle.

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New patent for Deciphera Pharms drug QINLOCK

Drug Patent Watch

Annual Drug Patent Expirations for QINLOCK Qinlock is a drug marketed by Deciphera Pharms and is included in one NDA. It is available from one supplier. There are fifteen patents… The post New patent for Deciphera Pharms drug QINLOCK appeared first on DrugPatentWatch - Make Better Decisions.

Drugs 59
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Beyond cfDNA: Harnessing the Power of Exosomes to Improve Sensitivity for Cancer Mutation Detection

Fierce BioTech

Beyond cfDNA: Harnessing the Power of Exosomes to Improve Sensitivity for Cancer Mutation Detection Liquid biopsy markers are advantageous because they can be used to assess overall prognosis, response to therapy, and likelihood of relapse or progression. This insightful whitepaper explores the combination of exosomal RNA (exoRNA) and cfDNA in cancer diagnosis and treatment.

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New patent for Vanda Pharms drug HETLIOZ

Drug Patent Watch

Annual Drug Patent Expirations for HETLIOZ Hetlioz is a drug marketed by Vanda Pharms Inc and is included in two NDAs. It is available from one supplier. There are twenty-two… The post New patent for Vanda Pharms drug HETLIOZ appeared first on DrugPatentWatch - Make Better Decisions.

Drugs 59
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The evolution of clinical trial recruitment

Antidote

As the medical research industry is ever-changing, so too is the continuous evolution of effective patient recruitment strategies. To gain more perspective on what these changes have looked like through the years, we chatted with Tom Ruane, the Global Head of Patient Recruitment at Parexel.

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New patent for Karyopharm Theraps drug XPOVIO

Drug Patent Watch

Annual Drug Patent Expirations for XPOVIO Xpovio is a drug marketed by Karyopharm Theraps and is included in one NDA. It is available from one supplier. There are eight patents… The post New patent for Karyopharm Theraps drug XPOVIO appeared first on DrugPatentWatch - Make Better Decisions.

Drugs 59
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VoiceOver Your TalkBack To Me: A Mobile Screen Reader Overview

Perficient: Drug Development

Accessibility testing on Websites and apps with automated accessibility scanner tools are a great help but they only get you so far. Screen Readers will bridge the gap in your accessibility goals to reach your user base spanning various impairments. Using Screen Readers Text-to-Speech Screen Reader technology (generally referred to as simply “Screen Readers” ) can be used to help people who are blind or otherwise visually impaired to use and enjoy websites and applications.

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New patent for Vanda Pharms drug HETLIOZ LQ

Drug Patent Watch

Annual Drug Patent Expirations for HETLIOZ+LQ Hetlioz Lq is a drug marketed by Vanda Pharms Inc and is included in one NDA. It is available from one supplier. There are… The post New patent for Vanda Pharms drug HETLIOZ LQ appeared first on DrugPatentWatch - Make Better Decisions.

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