December, 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.

Trials 299
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New method tags cells with location coordinates for single-cell studies

Broad Institute

New method tags cells with location coordinates for single-cell studies By Corie Lok December 13, 2023 Breadcrumb Home New method tags cells with location coordinates for single-cell studies The technique, called Slide-tags, allows scientists to map the location of cells within tissues in standard single-cell experiments. By Sarah C.P. Williams December 13, 2023 Credit: Andrew Russell Different cell types (each represented by a different color) are mapped to their native location in human brain

RNA 145
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As ALS research booms, one treatment center finds itself in the spotlight

BioPharma Drive: Drug Pricing

The Healey center is at the front of ALS research and care, earning acclaim from patients, doctors and scientists. Still, the complexities of the disease and of drug development have brought hard-felt losses.

Research 142
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AI's memory-forming mechanism found to be strikingly similar to that of the brain

Science Daily: Pharmacology News

An interdisciplinary team consisting of researchers has revealed a striking similarity between the memory processing of artificial intelligence (AI) models and the hippocampus of the human brain. This new finding provides a novel perspective on memory consolidation, which is a process that transforms short-term memories into long-term ones, in AI systems.

Research 138
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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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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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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.

Doctors 134

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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.

Treatment 133
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‘No tolerance for failure’: An oral history of the first CRISPR medicine

BioPharma Drive: Drug Pricing

A new sickle cell disease therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals is now approved in the U.S. and U.K. This is the story of how it came to be.

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Common Stomach Bug Is Linked to Higher Alzheimer's Risk

Drugs.com

THURSDAY, Dec. 28, 2023 -- A common stomach bug may play a part in Alzheimer's disease risk.New research found that older folks infected with Helicobacter pylori (H. pylori) had greater odds for developing Alzheimer's, the most common type of.

Disease 131
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New biomarkers found for diabetic kidney disease

Drug Target Review

Scientists in China have published their findings 1 regarding novel biomarkers, which they hope will benefit patients by identifying the disease at an earlier stage. Minjia Tan’s group from the Shanghai Institute of Materia Medica, Chinese Academy of Sciences, and Shichun Du’s group from Xinhua Hospital affiliated to Shanghai Jiaotong University School of Medicine, analysed urinary and exosome proteome profiling and discovered biomarkers for diabetic kidney disease (DKD).

Disease 126
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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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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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World’s first engineered B cell therapy enters human trials

Drug Discovery World

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

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10 achondroplasia facts to note

Antidote

Achondroplasia, a term meaning “without cartilage formation,” is a genetic disorder leading to disproportionate short stature. Sometimes called achondroplastic dwarfism or ACH , this condition has few treatment options and no known cure. However, individuals diagnosed with this condition can typically lead fulfilling, healthy lives with proper management.

Treatment 121
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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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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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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 128
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Suspend Reality – 6 Steps to Move from Fantasy to Execution

Perficient: Drug Development

We’re just now finishing up the holiday season. At this time of year, I’m always intrigued by the magic of the holidays we celebrate, and the optimistic goals we set for the new year. It amazes me how we collectively like to suspend reality for a bit, reflect, and forecast. And I wonder why we don’t drift into thoughts of the extraordinary more often.

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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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Cancer drug discovery company wins £50K Franklin research residency

Drug Discovery World

Oxford Target Therapeutics, a company developing a promising therapy for triple negative breast cancer, is one of three UK life science companies awarded research prizes by the Rosalind Franklin Institute, based in Oxfordshire, UK. The prizes, awarded in collaboration with the Science and Technology Facilities Council (STFC), a part of UKRI, enable the winners to benefit from the Franklin’s top-level technologies and support to move their work forward.

Research 266
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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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Tome Biosciences debuts with $213M and a new way to edit the genome

BioPharma Drive: Drug Pricing

Based on the work of MIT scientists, the well-funded startup is developing ways to insert large sizes of genetic material anywhere in the genome without damaging or breaking DNA.

DNA 131
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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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Navigating the neuroscientific landscape with Dr Judy Illes

Drug Target Review

In light of eco-anxiety in our society, how can neuroscientists shoulder the responsibility of contributing to the discourse surrounding environmental challenges and their impact on individual and collective well-being? I don’t think it is a question of neuroscientists having to shoulder the responsibility of responding to and addressing questions of eco-anxiety, climate change, and environmental change, but rather a matter of upping the interest in this space and delivering more evidence throug

Bioethics 115
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How technology and economics can help save endangered species

Science Daily: Pharmacology News

A lot has changed in the world since the Endangered Species Act (ESA) was enacted 50 years ago in December 1973. Experts are now discuss how the ESA has evolved and what its future might hold.

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Discovery lights the way for new secondary breast cancer drugs

Drug Discovery World

Using a novel approach, researchers from The Institute of Cancer Research, London have uncovered details of secondary breast cancer in the brain and spinal cord that may help with developing effective treatments. The study revealed for the first time that breast cancer leptomeningeal metastasis (BCLM) cells spread early from the primary breast tumour and that they acquire features typically associated with lobular breast cancer, which develops in the glands that produce breast milk.

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Patient perspectives: From public relations executive to chronic pain patient

Antidote

Because Antidote’s key focus is connecting patients to clinical trials , hearing directly from individuals serves as a great reminder of why we do what we do. One patient we’ve had the pleasure of speaking with is Linda VandeVrede, a former high-tech PR executive who shared her story of chronic pain, clinical trials, and personal experience.

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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 124
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Marijuana Use Increasingly Linked to Addiction, Psychosis

Drugs.com

WEDNESDAY, Dec. 27, 2023 (HealthDay news) -- A rising tide of cannabis-related mental health problems is resulting from the widespread legalization of recreational weed in the United States, warns a new evidence review.Nearly one in five Americans.

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The future of lymphoma treatment

Drug Target Review

How does Araris’ linker technology platform enable improved development of ADCs? Our technology platform allows for a one-step, easy to use method to efficiently conjugate a molecule to any off-the-shelf antibody. We use hydrophilic linkers, which prevent ADC aggregation and generate highly stable ADCs, in combination with a unique attachment site on the antibody to create ADCs that retain pharmacokinetic properties similar to the original unconjugated antibody.

Treatment 114
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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 121
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Gene therapy could reduce seizures in childhood epilepsy

Drug Discovery World

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. Focal cortical dysplasia is caused by areas of the brain that have developed abnormally and is among the most common causes of drug-resistant epilepsy in children. Epilepsy in focal cortical dysplasia is associated with comorbidities, including learning disabilities.

Therapies 264
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6 Reasons Financial Institutions Are Embracing Risk and Regulation Tactics

Perficient: Drug Development

In the fast-paced realm of finance, the significance of regulatory risk and compliance management practices cannot be overstated. This blog post delves into the pivotal role these practices play in ensuring the stability and success of financial institutions and banks. By adopting a proactive stance and leveraging technological advancements, financial entities can navigate the regulatory landscape with greater resilience and efficacy.

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Apellis eye drug likely to be rejected in Europe, company says

BioPharma Drive: Drug Pricing

According to Apellis executives, reviewers in Europe are skeptical of the benefits of its geographic atrophy medicine, Syfovre, and appear poised to recommend against approval at a meeting next month.

Drugs 124
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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 122
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Decoding Pfizer’s Portfolio: A New Model for Precision in Pharma Investments

Drug Patent Watch

In the ever-evolving landscape of the biotech and pharmaceutical sectors, accurate valuation of post-revenue drug assets stands as the linchpin for informed and strategic decision-making. The significance of this valuation… The post Decoding Pfizer’s Portfolio: A New Model for Precision in Pharma Investments appeared first on DrugPatentWatch - Make Better Decisions.