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Category: Clinical Trials


Clinical Trials, Research and Development

Pfizer’s haemophilia B gene therapy meets phase 3 primary endpoint

January 3, 2023

Via: Pharmaphorum

Haemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot because of a deficiency in one or several blood clotting factors. It is almost exclusively found in males. According to the World […]


Clinical Trials, Research and Development

CStone gets a win for sugemalimab in oesophageal cancer

January 3, 2023

Via: Pharmaphorum

The Hong Kong-listed cancer biotech said this morning that anti-PD-L1 inhibitor sugemalimab given in combination with chemotherapy showed “statistically significant and clinically meaningful” improvements in both progression-free survival (PFS) and overall survival (OS) in previously-untreated patients with unresectable locally advanced, […]


Clinical Trials, Research and Development

Newron says pivotal treatment-resistant schizophrenia trial is a go

January 3, 2023

Via: Pharmaphorum

The Italian biopharma said it had decided to press ahead with the new study after assessing results from 100 patients with TRS in open-label studies who received the glutamate modulator drug as an add-on to their current therapy for more […]


Clinical Trials, Research and Development

Upholding regulatory compliance in decentralised trials

December 30, 2022

Via: Pharmaphorum

These are all critical consideration for sponsors, as failing to conduct trials in accordance with GCP and other applicable regulations can lead to regulatory authorities rejecting data that is generated to support new treatments and regulatory applications, as well as […]


Clinical Trials, Research and Development

Pfizer says hemophilia B gene therapy controlled bleeding in key study

December 29, 2022

Via: Biopharma Dive

Drugmakers have long studied hemophilia gene therapies, seeking to replace the infusions that people with the disease take for life to control their bleeding. In recent years, their research has delivered clinical successes in both the “A” and “B” forms […]


Clinical Trials, Research and Development

Study Suggests Pain Due to GI Disorders Might Be Treated by Targeting Touch Receptor

December 28, 2022

Via: GEN

A receptor that is responsible for our sense of touch and temperature, and which researchers have now found to be present in our colon, could provide a target for treating chronic pain associated with gastrointestinal disorders such as irritable bowel […]


Clinical Trials, Research and Development

Germinal Center Insights Can Lead to Better Understanding of Immunity

December 28, 2022

Via: GEN

Germinal centers form in the body’s lymphatic tissues shortly after vaccination or infection. Once inside a germinal center, B cells undergo rapid mutations and, through a process of natural selection, only B cells with antibodies that most effectively bind to […]


Clinical Trials, Research and Development

Precision Phenotyping for Obesity Pharmacotherapy

December 28, 2022

Via: GEN

Despite a plethora of interventions, including diet, exercise, and medications, obesity continues to be a significant health problem. The fight against obesity currently involves substantial trial-and-error, out-of-pocket costs, and adverse effects. Obesity medications cause gastrointestinal side effects in nearly 73% […]


Clinical Trials, Research and Development

Cellular Reprogramming Leads to the Creation of Neural Networks with Characteristics of Human Cells

December 27, 2022

Via: GEN

Scientists report that they have used a cellular reprogramming methodology to allow the creation of neural networks that reproduce unique characteristics of human cells—different from those obtained from rodent cells—with temporary dynamics that recall human brain development. The researchers, who […]


Clinical Trials, Research and Development

Temperature-Regulating Neurons Identified, Hint at Treatment Strategies for Heat Stroke, Obesity

December 27, 2022

Via: GEN

The results of a study in rodents carried out by scientists at Nagoya University suggest that a group of neurons, called EP3 neurons, in the preoptic area (POA) of the brain play a key role in regulating body temperature in […]


Clinical Trials, Research and Development

How the Brain Consolidates Remote Fear Memories Uncovered

December 27, 2022

Via: GEN

A new mouse study by scientists at UC Riverside (UCR) demonstrates that remote fear memories formed in the distant past are permanently stored in connections between memory neurons in the prefrontal cortex or PFC. Their findings provide insights and may […]


Clinical Trials, Research and Development

Link Between Pain Perception and Specific Brain Region Could Point to Neurostimulation Strategies

December 23, 2022

Via: GEN

A new study in mice, carried out by researchers at Heidelberg University Medical Faculty Heidelberg, has helped illuminate how the brain’s primary motor cortex (a region known as M1) helps to suppress dimensions of neuropathic pain. M1 is a brain […]


Clinical Trials, FDA, Regulations, Research and Development

FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies

December 21, 2022

Via: PMLiVE

The clinical studies were placed on a partial hold in December 2021 relating to an investigation by bluebird into an adolescent patient with persistent, non-transfusion-dependent anaemia following treatment with lovo-cel. Results from a detailed investigation of the case were presented […]


Clinical Trials, Research and Development

Intelligence Predictions Improve with Whole-Brain Connectivity Profiles

December 21, 2022

Via: GEN

The most accurate predictions of intelligence consider global profiles of whole-brain connectivity rather than spatially localized regional networks, claims a predictive modeling study based on fMRI and cognitive tests that aimed to evaluate the effectiveness of various neuroscientific theories of […]


Clinical Trials, Research and Development

Colorectal Cancer Genetic Risk Factor Findings Could Help Identify Individuals at Risk

December 21, 2022

Via: GEN

A comprehensive analysis of more than 100,000 colorectal cancer (CRC) cases has identified upwards of 100 new genetic risk factors strongly linked with the disease. Findings from the study, which was led by researchers at the Fred Hutchinson Cancer Center, […]


Clinical Trials, Research and Development

Tracking Resistant Breast Cancer Cells May Lead to Novel Therapies

December 21, 2022

Via: GEN

Researchers at the Cancer Research UK Cambridge Institute at the University of Cambridge report that they have developed a new technique for identifying the different types of cells in a tumor. Developed in mice, the method allows them to track […]


Clinical Trials, Research and Development

Cardiology Leaders Call for Reinvention of Randomized Clinical Trials

December 19, 2022

Via: GEN

The current model for randomized clinical trials must be redesigned for the 21st century, according to the European Society of Cardiology (ESC), American Heart Association (AHA), World Heart Federation (WHF) and American College of Cardiology (ACC). Their joint statement “Randomized […]


Clinical Trials, Research and Development

Monkeypox Virus DNA Polymerase Structure Solved Using Cryo-EM

December 16, 2022

Via: GEN

In July 2022, the World Health Organization (WHO) announced that monkeypox was a public health emergency of international concern. And, as of early December 2022, more than 82,000 human monkeypox cases have been confirmed in 110 countries worldwide. New preventative […]


Clinical Trials, Research and Development

Bacterial Defense Mechanism May Lead to Rise of New Antibiotics

December 16, 2022

Via: GEN

Princeton Engineering researchers report they have isolated a compound that kills bacteria that can cause incurable infections. The compound, called cloacaenodin, is a short, slip-knotted chain of amino acids known as a lasso peptide, encoded by gut-dwelling bacteria as a […]


Clinical Trials, Research and Development

Higher Alzheimer’s Incidence in Women Linked to Modified Immune System Protein

December 16, 2022

Via: GEN

Scientists at Scripps Research and Massachusetts Institute of Technology (MIT) have found a clue to the molecular cause of Alzheimer’s disease (AD), which may also explain why women are at greater risk for the disease than men. The team found […]