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

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Potentially game-changing schizophrenia drug enters Phase I trials

Drug Discovery World

A potential schizophrenia treatment, discovered at Vanderbilt University in Tennessee in the US, has been cleared by the US Food and Drug Administration for use in Phase I clinical trials. NMRA-266, an allosteric modulator that works through a mechanism that has been clinically validated in the treatment of disorders like schizophrenia, will be developed by Neumora Therapeutics.

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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.

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The Power of Personalization Amid the Changing CRO Landscape

Conversations in Drug Development Trends

Amidst a shifting clinical landscape characterized by increasingly complex trial designs and growing patient subpopulations, many contract research organizations (CROs) have adopted a “one-stop-shop” strategic approach. As a result, various CROs have undergone significant consolidations and acquisitions of specialized capabilities to address the escalating complexity in clinical trials.

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Buprenorphine now replaces methadone as the most common medicine for opioid dependence’: 10-year trends in opioid agonist treatment medicines in Australia

National Drug & Alcohol Research Centre Blog

Kendal Chidwick NDARC’s Kendal Chidwick discusses the findings from a recently published study that examined how patterns of opioid agonist treatment medicines have changed over the past decade. In Australia, both methadone and buprenorphine have been subsidised by the Pharmaceutical Benefit Scheme (PBS) for the treatment of opioid dependence (termed ‘opioid agonist treatment’ or OAT) for several decades.

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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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CRUK and Guardant Health collaborate on precision cancer drugs

Drug Discovery World

Cancer Research UK (CRUK), Cancer Research Horizons, and precision oncology company Guardant Health have launched a collaboration to advance cancer treatment. The agreement will enable the parties to discuss opportunities for collaboration to support the charity’s research and clinical development activities, focusing primarily on its clinical trials run by its Centre for Drug Development.

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Keypoint Newsletter: Welcoming the New Fellows Class of 2024

keypoint

Keystone Symposia is pleased to introduce the Keystone Symposia Fellows Class of 2024! This year we welcome seven early-career investigators and seven post-doctoral fellows, a new addition to the program this year.

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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.

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Study shows osteoarthritis could be reversible

Drug Discovery World

Osteoarthritis may be treatable and reversible, according to a new University of Adelaide study. The researchers have discovered a novel population of stem cells – marked by the Gremlin 1 gene – responsible for the progression of osteoarthritis. Treatment with fibroblast growth factor 18 (FGF18) stimulated the proliferation of Gremlin 1 cells in joint cartilage in mice, leading to significant recovery of cartilage thickness and reduced osteoarthritis.

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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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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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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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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.

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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.

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New protein linked to early-onset dementia identified

Science Daily: Pharmacology News

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.

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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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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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What is chronic spontaneous urticartia (chronic hives)?

Antidote

Chronic spontaneous urticaria, also called chronic idiopathic urticaria, is a type of chronic hives that come and go unexpectedly. These hives persist daily for a minimum of six weeks without a clear cause or trigger. While this condition can affect anyone at any point, women experience it twice as often as men , and it’s most common between the ages of 20 and 40.

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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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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.

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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.

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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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Organ-on-a-chip data supports clinical progression of liver disease drug

Drug Discovery World

Inipharm has initiated Phase I dosing of its lead candidate, INI-822, which is a small-molecule inhibitor for HSD17B13, a gene implicated in nonalcoholic steatohepatitis (NASH). Loss-of-function variants of HSD17B13 are known to be associated with reduced incidence risk and severity of multiple liver diseases. CN Bio’s PhysioMimix Organ-on-a-Chip (OOC) Systems and NASH ‘in-a-box’ (NIAB) kit was used to provide human-relevant data on compound efficacy for INI-822.

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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.

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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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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.

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Changes to UK Statutory Scheme go ahead despite pharma criticism

Drug Discovery World

The UK government has published new terms for the Statutory Scheme for branded medicines, which controls the prices of medicines paid by the NHS. The announcement follows the agreement in mid-November of a new Voluntary Scheme for Pricing, Access and Growth (VPAG), a negotiated alternative scheme which companies can opt into over the Statutory Scheme.

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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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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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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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DNA therapy effective against chemo-resistant ovarian cancer

Drug Discovery World

A novel DNA vaccine in combination with chemotherapy has demonstrated promising clinical results against a deadly form of ovarian cancer. The investigational compound Elenagen is an experimental DNA therapy that consists of a circular piece of DNA called a plasmid that includes a gene for a human protein called p62/SQSTM1. In a new paper, a team of scientists from CureLab Oncology, the NN Alexandrov National Cancer Center and Minsk City Oncology Center have demonstrated a clear clinical benefit

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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.

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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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Approach studies how rare gene variant pairs contribute to disease

Broad Institute

Approach studies how rare gene variant pairs contribute to disease By Tom Ulrich December 7, 2023 Breadcrumb Home Approach studies how rare gene variant pairs contribute to disease Strategy infers whether variants that appear in the same gene are on the same copy of that gene, could improve genetic diagnosis of disease By Tracy Hampton, Massachusetts General Hospital December 7, 2023 Credit: Susanna Hamilton, Broad Communications Related Programs Program in Medical and Population Genetics Relate

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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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Top 10 climate science insights unveiled

Science Daily: Pharmacology News

A new report equips policymakers with the latest and most pivotal climate science research from the previous 18 months, synthesized to help inform negotiations at COP28 and policy implementation through 2024 and beyond.

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Advances in personalised medicine, leave data processing behind

Drug Target Review

According to the Personalised Medicine Coalition , 34 percent of all new drugs approved by the US Food and Drug Administration (FDA) in 2022 are personalised medicines. What’s more, investment in this sector is predicted to rise by 70 percent by 2030. The reason for all this attention and investment is clear: significant benefits for patients. Tailoring medical care to a patient’s unique genetic makeup, alongside the close analysis of disease progression, leads to more effective treatments, redu

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