Fri.Dec 08, 2023

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Can vertical AI advance cell and gene therapies? 

Drug Discovery World

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The once-fictional artificial intelligence (AI) depicted in literature and movies has transitioned into a tangible and easily accessible tool in our modern world. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

Therapies 163
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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 139
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Researchers identify potential therapeutic target for early-onset dementia

Drug Discovery World

Scientists have identified abnormal aggregates of a protein called TAF15 in the brains of individuals with early-onset dementia, known as frontotemporal dementia, where the cause was not previously known. The research led by scientists from the Medical Research Council (MRC) Laboratory of Molecular Biology, in Cambridge, UK, could provide a target for the future development of diagnostic tests and treatments.

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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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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 week in drug discovery (4-8 December) 

Drug Discovery World

News round-up for 4-8 December by DDW Digital Content Editor Diana Spencer. The news highlights this week are mostly focused around neuroscience, with new guiding principles and a drug repurposing fund launched for motor neuron disease research, a new drug target found for early-onset dementia, and a possibly game-changing schizophrenia drug entering human trials.

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Botulinum toxin type A ameliorates rat dorsal root ganglia neuron pyroptosis in postherpetic neuralgia by upregulating cathelicidin antimicrobial peptide to inhibit neutrophil elastase

Chemical Biology and Drug Design

BoNT/A (30 U/kg) promoted cl-SNAP-25 expression in DRG of RTX-induced rats and reversed RTX-induced decrease of rat paw withdrawal thresholds and CAMP expression and increase of pyroptosis-associated protein and inflammatory factor expression in rat DRG. CAMP interacted with ELANE in rat DRG neurons. BoNT/A (1 nM) attenuated LPS/ATP-stimulated inhibition of viability and CAMP expression and upregulation of inflammatory mediators, pyroptosis-related proteins, and ELANE expression in rat DRG neuro

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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.

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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 122
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Major breakthrough for severe asthma treatment

Science Daily: Pharmacology News

A landmark study has shown that severe asthma can be controlled using biologic therapies, without the addition of regular high-dose inhaled steroids which can have significant side effects.

Treatment 116
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Merck suffers bad week as 3 Keytruda combination trials fail

BioPharma Drive: Drug Pricing

The setbacks for Merck demonstrate the challenge drug developers face in improving on Keytruda, which has brought in at least $18 billion so far this year for its maker.

Trials 116
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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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When is an aurora not an aurora?

Science Daily: Pharmacology News

While auroras occur at high latitude, the associated phenomena Steve and the picket fence occur farther south and at lower altitude. Their emissions also differ from aurora. A physics graduate student has proposed a physical mechanism behind these emissions, and a rocket launch to test the theory. She argues that an electric field in the upper atmosphere parallel to Earth's magnetic field could explain the green picket fence spectrum and perhaps Steve and the enhanced aurora.

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Suicide Rates Have Risen Steadily for Black Girls and Women

Drugs.com

FRIDAY, Dec. 8, 2023 -- Suicide rates for Black women and girls ages 15 to 24 have more than doubled over the past two decades, a new report finds.“Suicides are rapidly increasingly among young, Black females in the U.S.,” said study first.

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Qualification vs. Validation: Understanding the Distinctions in Scientific Processes

biobide

Ensuring the reliability and accuracy of processes and equipment is paramount In scientific research and manufacturing, Two key concepts that play a crucial role in this assurance are qualification and validation. While these terms are often used interchangeably, they signify different procedures with unique purposes and methods. Qualification and validation are crucial processes in various industries, especially those involving R&D, manufacturing, and regulated sectors such as Pharmaceutica

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Salmonella Illnesses Tied to Cantaloupes Have Doubled: CDC

Drugs.com

FRIDAY, Dec. 8, 2023 (Healthday News) -- A salmonella outbreak tied to tainted cantaloupes keeps expanding, with cases doubling since the last tally, U.S. health officials reported Thursday. "Since the last update [on] November 30, 2023, an.

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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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Transforming Medication Access with the Next Generation of Patient Support Services

Drug Channels

Today’s guest post comes from Kelly Martin, Vice President of Product Management at CoverMyMeds. Kelly discusses the obstacles that patients face when attempting to access, afford, and adhere to specialty therapies. She describes how CoverMyMeds improves the patient experience by blending technology with human expertise and connection. Click here to learn about CoverMyMed’s next-generation patient support services.

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Hospitals in Ukraine Seeing Surge in Drug-Resistant Infections: CDC

Drugs.com

FRIDAY, Dec. 8, 2023 -- As the war in the Ukraine rages on, new research shows that hospitals there are waging a battle of their own against a different kind of enemy: antibiotic-resistant "superbugs" that are spreading at an alarming rate.In a.

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Suburban backyard home to more than 1,000 species

Science Daily: Pharmacology News

A challenge among three housemates to identify species around their inner-Brisbane home has resulted in an academic research paper, showcasing the rich biodiversity in urban landscapes.

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FDA Approves Landmark Sickle Cell Gene Therapies

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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Patient perspectives: Meeting the wonderful “e-patient Dave”

Antidote

At Antidote, matching individuals to clinical trials is our driving force — and one of the key ways we do this is by prioritizing our connection to patients. Understanding the real-world impact of our work allows us to continually improve our processes and approach clinical trial recruiting from a patient-centric perspective.

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Lymphedema Left her 'Miserable, Depressed' Until Specialized Surgery Changed Everything

Drugs.com

FRIDAY, Dec. 8, 2023 -- Sydnee Meth survived breast cancer, but she wasn’t prepared for the aftereffects of her treatment.Doctors removed the lymph nodes from Meth’s right armpit during her second bout with breast cancer in 2014, and as a.

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Coral reefs in peril from record-breaking ocean heat

Science Daily: Pharmacology News

Record breaking marine heatwaves will cause devastating mass coral bleaching worldwide in the next few years, according to a coral reef scientist.

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Doctors discover many patients at UNC’s Inflammatory Bowel Disease Clinic screen positive for malnutrition

SCIENMAG: Medicine & Health

CHAPEL HILL, NC — Eating food and absorbing its nutrients is an everyday occurrence, but this normal activity can look different for someone who suffers from inflammatory bowel disease. IBD, which includes Crohn’s disease and ulcerative colitis, can cause chronic inflammation of the digestive tract – which for many reasons can lead to malnutrition.

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New genes can arise from nothing

Science Daily: Pharmacology News

The complexity of living organisms is encoded within their genes, but where do these genes come from? Researchers resolved outstanding questions regarding the origin of small regulatory genes, and described a mechanism that creates their DNA palindromes. Under suitable circumstances, these palindromes evolve into microRNA genes.

DNA 91
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40 years after the discovery of HIV, research raises hopes of remission

SCIENMAG: Medicine & Health

Nearly 39 million people worldwide are carriers of human immunodeficiency virus (HIV), and 1.3 million people contracted HIV in 2022[1]. Forty years after the virus was discovered at the Institut Pasteur, HIV research is still active – the aim is to elucidate the mechanisms of infection so that the virus can be eradicated.

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Scholars say it's time to declare a new epoch on the moon, the 'lunar Anthropocene'

Science Daily: Pharmacology News

According to anthropologists and geologists, it's time to acknowledge humans have become the dominant force shaping the moon's environment by declaring a new geological epoch for the moon: the Lunar Anthropocene. They argue the new epoch may have dawned in 1959 when the USSR's unmanned spacecraft Luna 2 alighted on the lunar surface.

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New source of stem cells in injury-affected brains of patients

SCIENMAG: Medicine & Health

Researchers from Helmholtz Munich and the LMU have discovered that, in the case of brain injuries, specific cells in the brain become active in disease situations, exhibiting properties of neural stem cells.

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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.

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Engaging heterosexual men more effectively could slash HIV infections in Uganda

SCIENMAG: Medicine & Health

A study looking at 15 years of HIV transmission and suppression in Uganda reveals how closing gender gaps in treatment could slash infection rates. Credit: Rakai Health Sciences Program A study looking at 15 years of HIV transmission and suppression in Uganda reveals how closing gender gaps in treatment could slash infection rates.

Science 83
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Night Shifts Are Triggering Sleep Disorders in Workers

Drugs.com

FRIDAY, Dec. 8, 2023 -- More than half of night shift workers have at least one sleep disorder, as nocturnal labor plays havoc with body rhythms, a new study shows.

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A dynamic picture of how we respond to high or low oxygen levels

SCIENMAG: Medicine & Health

SAN FRANCISCO—December 8, 2023—It only takes holding your breath for slightly too long to understand that too little oxygen is bad for you. But can you also have too much? Indeed, breathing air with a higher oxygen level than your body needs can cause health problems or even death.

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E-Scooter on Your Kids' Holiday Gift List? Experts Have Warnings, Safety Tips

Drugs.com

FRIDAY, Dec. 8, 2023 -- An electric scooter might be on your kid’s wish list for Christmas, but pediatricians say parents should think twice before buying one.Even taking a child on a ride with an e-scooter is a dicey proposition, said Dr. Ashley.

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Mothers need more “exclusive breastfeeding” support

SCIENMAG: Medicine & Health

Exclusive breastfeeding for the first six months of life is proven to protect both mother and child health. According to the World Health Organization (WHO), between 2015 and 2021, 48% of mothers exclusively breastfed, meaning that their babies were not given any other food or liquids.

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Immersive VR goggles for mice unlock new potential for brain science

Science Daily: Pharmacology News

New miniature virtual reality (VR) goggles provide more immersive experiences for mice living in laboratory settings. By more faithfully simulating natural environments, the researchers can more accurately and precisely study the neural circuitry that underlies behavior. Compared to current state-of-the-art systems, which simply surround mice with computer or projection screens, the new goggles provide a leap in advancement.

Science 67
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Milestones in Europe: First results in using new tools to tackle respiratory syncytial virus

SCIENMAG: Medicine & Health

Each year, an estimated 101,000 children below the age of 5 years die across the globe due to infections caused by respiratory syncytial virus (RSV). Worldwide, RSV is a main cause of hospitalisation among young children.

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