Scientists from the UK’s Dementia Research Institute at University College London (UCL) and VIB-KU Leuven have discovered the cause of the death of neurons in Alzheimer’s disease (AD), opening up potential avenues to develop new treatments for the condition. The researchers found that a programmed form of cell death, known as necroptosis, is initiated when neurons are exposed to amyloid plaques and tau tangles. The researchers created a new model to replicate and connect AD hallmark features – amyloid plaques, tau tangles, and death of neurons – by implanting both healthy human and mouse neurons into the brains of AD mouse models. They discovered that only human neurons displayed Alzheimer’s features, including tau tangles and neuronal cell loss. These findings suggest that humans have specific factors that play in Alzheimer’s that standard mouse models cannot replicate, as their neurons are more resilient to amyloid pathology. Upon further research, the team ...
Researchers from the Institute of Cancer Research’s (ICR) Centre for Cancer Drug Discovery have developed a new test, RKD2, which makes the early stages of drug discovery for cancer more efficient. Funded by Cancer Research UK, the researchers created an investigative procedure that can help scientists identify new biologically active compounds to be used to develop effective medications. Fragment-based drug discovery uses various techniques to screen libraries of compounds, or fragments, with a low molecular weight. During the initial stage, scientists introduce a potential anti-cancer target and see which fragments interact with it. Previously, researchers could only determine that ‘yes, an interaction occurred’ or ‘no, there was no interaction’. However, it was difficult to apply this to weak interactions, and the team could not rule out nonspecific interactions. The new RKD2 test allows researchers to quantify interactions by determining their strength, identifying those that are truly active and worth following ...
By Connor Lynch Pictured: FDA sign in its headquarters/iStock, Grandbrothers Taysha Gene Therapies has decided to drop its lead experimental AAV-based gene therapy candidate after the FDA reiterated calls for the company to put the treatment through a randomized, double-blind placebo-controlled trial. The Dallas-based company announced on Tuesday that it would no longer be developing its TSHA-120 treatment for giant axonal neuropathy (GAN) based on the FDA’s Type C meeting feedback regarding the therapy’s potential pathway. “FDA continues to recommend a randomized, double-blind, placebo-controlled trial as the optimal path to demonstrate efficacy in TSHA-120,” the company said in a statement. Taysha noted in the announcement that the FDA “provided a potential path for a single-arm trial with an external control group matched with to-be treated patients by multiple prognostic factors and recommended longer term follow up to account for potential bias.” CEO Sean Nolan said in a statement that the ...
Dive Brief Johnson & Johnson has begun a clinical trial of another investigational pulsed field ablation (PFA) device, opening a new front in its pursuit of a potentially significant cardiovascular opportunity. The treatment of the first patients with the Omnypulse catheter comes months after J&J posted data on Varipulse, another PFA device to treat atrial fibrillation. J&J also has a PFA and radiofrequency dual energy device in late-phase development. Omnypulse expands J&J’s portfolio of investigational devices with a catheter that gathers contact force data to support mapping. Analysts have identified the combination of PFA and mapping, which J&J sees as a key feature of its systems, as a threat to Abbott’s electrophysiology unit. Dive Insight It has been only three weeks since Boston Scientific notched a milestone in the race for the first-generation PFA market with positive clinical trial results. Boston Scientific and Medtronic are leading the way. Stifel analysts ...
In response to the persistent health challenges of herpes simplex virus 1 (HSV-1) and HSV-2, today the National Institutes of Health released the Strategic Plan for Herpes Simplex Virus Research. An NIH-wide HSV Working Group developed the plan, informed by feedback from more than 100 representatives of the research and advocacy communities and interested public stakeholders. The plan outlines an HSV research framework with four strategic priorities: improving fundamental knowledge of HSV biology, pathogenesis, and epidemiology; accelerating research to improve HSV diagnosis; improving strategies to treat HSV while seeking a curative therapeutic; and, advancing research to prevent HSV infection. HSV-1 and HSV-2 are among the most common viral infections in the United States. HSV can cause recurring blisters or sores. In severe cases HSV may lead to life-threatening or long-term complications, typically in the central nervous system. There are no licensed preventive HSV vaccines, and there is no cure. Up ...
Auden Mckenzie and Actavis UK—now known as Accord-UK—will remain on the hook to pay hefty fines for their roles in introducing costly price hikes on hydrocortisone tablets.In a judgment published Monday, the U.K.’s Competition Appeal Tribunal (CAT) upheld previous findings by the country’s Competition and Markets Authority (CMA) that multiple drugmakers had charged “excessive and unfair prices” for generic hydrocortisone over the course of 10 years. Hydrocortisone tablets treat adrenal insufficiency, including serious conditions such as Addison’s disease. In the U.K., the medicine is funded by England’s National Health Service. The tribunal’s decision comes a little more than two years after CMA doled out fines worth more than 260 million pounds sterling ($360 million) to more than 10 pharmaceutical companies. The punishment followed a long-running investigation by the U.K. into the steep cost of hydrocortisone. From 2008 to 2016, the med’s price grew by 10,000%, costing taxpayers hundreds of millions ...
Kinnate Biopharma plans to sack around 70% of its workforce and stop the development of three programmes as a result of its strategic review. The company expects to retain 28 full-time employees after the layoffs are concluded, per the 18 September press release. The US-based oncology company has also decided to consider strategic alternatives for the exarafenib monotherapy programme and the asset KIN-3248, which is designed for use in cancers with FGFR2 and FGFR3 alterations. Despite the clearing of KIN-7136’s investigational new drug application (IND), Kinnate will also not be going ahead with its Phase I trial. The strategic review reflects multiple factors that include its cash runway and the commercial potential of its programmes, based on the 18 September update. Although exarafenib was well tolerated by patients in a Phase I trial (NCT04913285), it displayed limited anti-tumour activity in subjects with Class II alterations. The company has decided to ...
BeiGene will utilise Nona’s Harbour Mice platform as part of the agreement BeiGene and Nona Biosciences have announced an agreement to expand their strategic collaboration for antibody discovery. The collaboration will allow BeiGene to utilise Nona’s Harbour Mice platform – a fully human transgenic mouse platform – to further improve therapeutic antibody discovery efficiency and flexibility. BeiGene first obtained the rights to use the proprietary Harbour Mice H2L2 platform for multiple antibody programmes in 2018 as part of the now expanded collaboration. Harbour Mice works to generate fully human monoclonal antibodies in classical two light and two heavy chain (H2L) formats and heavy chain only (HCAb) formats. The H2L platform allows mice to bear fully human immunoglobulin genes with robust B cell development and antibody maturation. The HCAb platform is the world’s first human HCAb transgenic mouse platform that works to generate fully human heavy chain-only antibodies. It allows the ...
CSL Seqirus has unveiled new data highlighting the potential impact of influenza vaccines on reducing the burden seasonal flu has on hospitals and broader healthcare systems. The analyses were presented at this year’s European Scientific Working Group on Influenza (ESWI) Conference. Seasonal flu is an acute respiratory infection caused by influenza viruses, which circulate in all parts of the world. Although most people can recover within a week without requiring medical attention, the flu can cause severe illness among high-risk groups such as young children, older adults, pregnant individuals and those with serious medical conditions. In France, approximately 2 to 6 million people are affected by influenza every year, leading to over 20,000 hospitalisations and 10,000 excess deaths, 90% of which occur in older adults. According to findings from a dynamic transmission model, standard-dose quadrivalent flu vaccines for older adults could reduce hospitalisations by 11.1% to 30.0% and cut ICU ...
By Tristan Manalac Pictured: Sanofi’s Germany office at night/iStock, Panama7 Sanofi on Monday offloaded 11 brands in its central nervous system portfolio to U.K-based company Pharmanovia, including epilepsy drugs Frisium and Gardenal. The companies did not disclose specific terms of the agreement, but revealed that the medicines span four therapeutic areas—all with ongoing unmet need: psycholeptics, anxiolytics, anti-epileptics and anti-psychotics. Though not all these drugs are approved in every market, they still have “clear synergy” with Pharmanovia’s existing portfolio and will fit well with the company’s “core sub-therapy focuses of mental health and epilepsy, where there continues to be significant unmet need,” CEO James Burt said in a statement. Among the divested drugs are several brands of the benzodiazepine clobazam, including Sentil, Urbanol, Noiafren, Castilium and Frisium. All of these are for the treatment of anxiety in certain patient subgroups; Frisium can also be used as an adjunctive treatment in ...
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