Neuroendocrine carcinomas, such as neuroendocrine prostate cancer and small-cell lung cancer, originate in hormone-releasing cells and can develop in various organs, including the prostate and lungs. While they are not the most prevalent cancer type in these organs, they often have a poor prognosis and limited therapeutic options. Current treatments for these cancers include chemotherapy, radiation, and immunotherapy combinations. Neuroblastoma, predominantly found in young children, develops from immature nerve cells, often in the adrenal glands or nerve tissue along the spine, chest, abdomen, or pelvis. Despite treatment efforts, these therapies only extend survival by a few months, underscoring the need for better therapeutic targets and less invasive diagnostic approaches for these malignancies. Investigators from the UCLA Health Jonsson Comprehensive Cancer Center (Los Angeles, CA, USA) have identified UCHL1, a protein found in aggressive neuroendocrine carcinomas and neuroblastoma, as a potential molecular biomarker for diagnosing these cancers and predicting and monitoring ...
The lack of non-invasive methods for monitoring brain status is a significant challenge in psychiatric care. Using genetic material from human blood and lab-grown brain cells, researchers have now made advances in developing a blood test to detect brain-related changes associated with postpartum depression and other psychiatric and neurological disorders. The research by investigators at Johns Hopkins Medicine (Baltimore, MD, USA) focused on tracing brain cell-derived mRNAs in the bloodstream. These extracellular vesicles (EVs), which are tiny sacs containing genetic material, are crucial for cell communication and carry messenger RNA (mRNA) from the brain. This method allows for the detection of changes in gene activity within the brain. The team’s interest in this area grew from an earlier study that found altered EV communication in pregnant women who developed postpartum depression after childbirth. The latest study used the human placenta as a model to identify 26 placental mRNAs in maternal ...
The US Food and Drug Administration (FDA) has granted orphan drug designation to Ionis Pharmaceuticals’ olezarsen, an investigational ligand conjugated antisense medicine for familial chylomicronemia syndrome (FCS). This rare genetic disease is characterised by extremely high triglyceride levels and can lead to recurrent acute pancreatitis (AP). Olezarsen is an RNA-targeted therapy aimed at reducing the production of apolipoprotein C-III (ApoC-III), a protein that regulates triglyceride metabolism. It is currently undergoing evaluation for individuals at risk of diseases caused by elevated triglyceride levels, including FCS. In January 2023, the US regulator awarded fast track designation to olezarsen for FCS. Ionis is also investigating its potential to treat severe hypertriglyceridemia (sHTG) in Phase III clinical trials. FCS patients experience triglyceride levels that are significantly higher than normal, leading to life-threatening conditions such as AP and potential damage to vital organs. No currently FDA-approved therapies exist for FCS, and standard treatments for lowering ...
Dragonfly Therapeutics has entered a clinical collaboration with Gilead Sciences to assess the potential of its investigational drug candidate DF1001 with the latter’s Trodelvy for two cancer indications. The study of the combination regimen will focus on metastatic breast cancer (mBC) and non-small cell lung cancer (NSCLC). DF1001 is designed to act on natural killer (NK) cells and T-cell activation signals, leveraging co-stimulation of NK receptor NKG2D and CD16 for NK cell activation. Trodelvy is a Trophoblast cell-surface antigen 2-directed antibody-drug conjugate. Dragonfly will maintain operational control of the trial, with the first patients receiving the combination treatment in the second quarter of 2024. Study sites are already operational in the US, Belgium, France, Denmark and the Netherlands. The study is set to expand, with additional sites in Europe, North America and Asia Pacific. Developed using Dragonfly’s TriNKET platform, DF1001 is being evaluated in adult patients for the treatment of ...
Sanofi and Regeneron Pharmaceuticals have received yet another approval from the Japanese Ministry of Health, Labor and Welfare (MHLW) for Dupixent (dupilumab), this time as a treatment of chronic spontaneous urticaria (CSU). The Japanese agency approved Dupixent in CSU patients ages 12 and older whose symptoms are inadequately controlled by H1-antihistamines. This is the first approval for the therapy in this indication. Dupixent, a monoclonal antibody that inhibits the signalling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways, has been a high-grossing drug for Sanofi and Regeneron. In 2023, the therapy netted $11.59bn in global sales in 2023, as per Regeneron’s financials. GlobalData forecasts Dupixent sales to maintain their upward trajectory and generate $20.4bn in sales in 2030. Dupixent has been approved by the US Food and Drug Administration (FDA) and European Commission (EC) for five indications: atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis and prurigo nodularis. ...
Australian biotech QBiotics has won an orphan drug designation for a drug based on a chemical obtained from the blushwood tree, which is being investigated as a treatment for a rare group of cancers called soft tissue sarcoma. Qbiotics will now be in line for tax credits for US-based clinical trials and, if the therapy is approved, the potential for seven years of market exclusivity for the given indication. A veterinary formulation of the drug, tigilanol tiglate, is already approved for use in the US, UK, and Australia under the trade name Stelfonta. QBiotics started life as EcoBiotics, which was established in 2000 to discover potential drugs from Queensland’s tropical rainforest. QBiotics itself was founded in 2004 as a subsidiary of EcoBiotics, and the two groups merged in 2017. The company has signed several biodiscovery agreements covering the rainforest areas in the country in the past decade. The company has ...
After a high-profile failure in an Alzheimer’s related agitation trial earlier this week, Otsuka has garnered a positive update with a breakthrough therapy designation for its rare kidney disease drug sibeprenlimab. The US Food and Drug Administration (FDA) designation is for sibeprenlimab’s use in immunoglobulin A nephropathy (IgAN), sometimes referred to as Berger’s disease, a disorder that occurs when antibody immunoglobin A builds up in the kidneys, leading to inflammation and damage. This results in blood and protein in urine, as well as high blood pressure and kidney failure over time. The FDA made the decision following positive results from the Phase II ENVISION trial (NCT04287985), announced in November 2023. The 155-patient study evaluated the dose response of different doses of sibeprenlimab by measuring proteinuria or high levels of protein in the urine. Data from the trial, published in The New England Journal of Medicine, showed that after 12 months, ...
Following an eventful couple of months with Elevidys (delandistrogene moxeparvovec-rokl), Sarepta Therapeutics has announced that the efficacy supplement for its biologics license application (BLA) of the Duchenne muscular dystrophy (DMD) gene therapy has received priority review by the FDA. As per the 16 February press release, the purpose of the efficacy supplement is to change Elevidys’ accelerated approval to a traditional approval while also expanding the therapy’s label to treat all DMD patients with a confirmed mutation in the DMD gene. The FDA has set a priority review goal date of 21 June 2024 and will not discuss the supplement in an advisory committee meeting. Elevidys, which was granted approval in June 2023, is currently indicated to treat ambulatory pediatric DMD patients between the ages of four and five years with a confirmed mutation in the DMD gene. The single-dose gene transfer therapy uses a recombinant adeno-associated virus vector serotype ...
BHF data reveals early heart disease deaths rise to highest level In 2022, over 39,000 people in England died prematurely of cardiovascular conditions The British Heart Foundation (BHF) has revealed new data showing that early heart disease deaths in England have risen to the highest level seen since 2008. New figures show that over 39,000 people in England died prematurely of cardiovascular conditions, including heart attacks, coronary heart disease and stroke, in 2022. Cardiovascular disease is a general term for conditions that affect the heart or blood vessels in the body. In the UK, there are currently around 7.6 million people living with heart and circulatory diseases. Before 2012, the number and rate of deaths from these types of conditions among people under the age of 75 were falling. However, recent statistics have shown that the rate of premature deaths from cardiovascular disease has increased in England for three years. ...
CAD is responsible for around 68,000 deaths per year in the UK PlaqueTec and the Babraham Institute’s Flow Cytometry facility have announced a collaboration to develop and improve treatment for coronary artery disease (CAD). Both will develop a bespoke cell phenotyping assay for human blood to be used to analyse the blood of patients with CAD to catalogue cell types at the coronary disease sites. Responsible for around 68,000 deaths per year in the UK, CAD occurs when the arteries that supply blood to the heart are narrowed or blocked by atherosclerotic plaques. The Babraham Institute’s Flow Cytometry facility is currently one of the UK’s leading facilities, pioneering the latest flow cytometry technology and expertise. PlaqueTec aims to focus on understanding the biological mechanisms of CAD to advance the development of precision medicine. Current treatment approaches for CAD are general, as opposed to tailored for individuals, and can be ineffective ...
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