A uniQure gene therapy for Huntington’s disease has interim clinical data showing an 80% slowing of disease progression. An accelerated approval pathway is one of the topics the company wants to discuss with the FDA. By Frank Vinluan on July 09, 2024A uniQure gene therapy for Huntington’s disease has interim data showing the one-time treatment slows progression of this rare neurodegenerative disorder that has no FDA-approved therapies. Based on these encouraging data, the company said Tuesday it now plans to discuss with regulators the potential for a faster clinical and regulatory path forward. Huntington’s stems from a genetic mutation that leads to abnormal versions of the huntingin protein. The disease leads to motor dysfunction, behavioral changes, and cognitive decline. The uniQure gene therapy, code-named AMT-130, uses an engineered virus to deliver microRNA that silences the huntingin gene and the toxic protein fragment that drives the disease. Amsterdam-based UniQure is evaluating ...
Bacterial drug resistance is a global problem and challenge in anti-infective treatment, and multi-drug-resistant bacterial infections are facing the situation of having no choice of drugs in the clinic. (hereinafter referred to as “Consensus”) was authoritatively released, aiming to provide clinicians with an authoritative treatment guideline to help them choose treatment options more scientifically and rationally when facing multidrug-resistant bacterial infections. Multi-drug resistant bacteria (MDRB) refers to bacteria that are resistant to three or more classes of commonly used antibacterial drugs that are usually sensitive. In the treatment of MDRB infections, polymyxins, such as polymyxin B and polymyxin E (CMS), have become a powerful weapon against drug-resistant bacteria due to their unique antimicrobial mechanisms and remarkable efficacy. These drugs exert their antimicrobial effects by binding to lipopolysaccharide on the bacterial cell membrane and disrupting the integrity of the cell membrane. Polymyxin E sodium methanesulfonate has been widely used in the ...
Recently, results of the phase 2 study of HLX22, an innovative anti-HER2 monoclonal antibody (mAb), in combination with HANQUYOU (HLX02, trastuzumab, trade name: HERCESSI™️ in the U.S., Zercepac® in Europe) and chemotherapy for the first-line treatment of HER2-positive advanced gastric/gastroesophageal junction (G/GEJ) cancer were presented on 2024 ESMO Gastrointestinal Cancers Congress (ESMO GI) and MED, a flagship clinical and translational research monthly journal by Cell Press. The leading principal investigator for this study is Professor Jin Li from Shanghai East Hospital, School of Medicine, Tongji University. The results showed that the addition of HLX22 to HLX02 (trastuzumab) plus chemotherapy as first-line therapy improved efficacy in HER2-positive G/GEJ cancer patients with manageable safety. The results of HLX22-GC-201 were first presented at the 2024 American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO GI). The study data and updated results with another 7.8 months of follow-up were accepted for publication on MED ...
The Scottish Medicines Consortium (SMC) has accepted Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) for use within NHS Scotland to treat adult patients living with chronic kidney disease (CKD). The regulator has indicated the therapy for adult CKD patients who are receiving optimised standard care, including angiotensin converting enzyme inhibitors or angiotensin 2 receptor blockers, with either an estimated glomerular filtration rate (eGFR) of 20ml/min/1.73m2 up to 45ml/min/1.73m2 or an eGFR of 45ml/min/1.73m2 up to 90ml/min/1.73m2. Additionally , patients in Scotland in the 45ml to 80ml group will now be eligible to receive Jardiance if they have a urine albumin-to-creatine ratio of 22.6mg/mmol or more or type 2 diabetes (T2D) mellitus. Affecting nearly 850 million people worldwide, CKD is a serious, progressive condition that is caused by decreased kidney function and is often triggered by diabetes, hypertension and glomerulonephritis. Already approved in the EU and US, Jardiance is an oral, ...
The Francis Crick Institute and Imperial College London (ICL) cancer therapy spinout, Myricx Bio, has raised £90m in series A financing to advance its novel cancer treatments into clinical development. The investment will help to develop the company’s therapies to treat a range of different tumour types, including breast, lung and colorectal cancer, to advance into clinical testing. Currently the largest series A round to ever be raised by an EU academic biotech spinout, the round was co-led by life science investors Novo Holdings and Abingworth, with additional investors including British Patient Capital, Cancer Research Horizons and Eli Lilly and Company, as well as founding investors Brandon Capital and Sofinnova Partners. The spinout is focused on the discovery and development of a novel class of payloads for antibody-drug conjugates (ADCs), which involves antibodies that bind to the surface of certain tumour types to deliver a drug to its target. The ...
Organizer: IPHEB Company, Russia Date: April 8-10, 2025 Venue: Peterburgskoye sh. 64/1, St. Petersburg, Russia Exhibition hall:ExpoForum Convention and Exhibition Centre Product range: Pharmaceutical raw materials, intermediates, and formulations; antibiotic, vitamin, and amino acid-based raw materials for pharmaceuticals and veterinary drugs; Food ingredients and additives, feed additives; Pharmaceutical machinery, laboratory equipment, packaging equipment, materials, and new technologies; Contract manufacturing services, biomedical products Exhibition Introduction: The International Pharmaceutical and Healthcare Exhibition (IPhEB Russia), held in St. Petersburg, Russia, is the most influential exhibition for pharmaceutical raw materials in the country. It presents a unique opportunity for businesses in the pharmaceutical industry of Russia and the Commonwealth of Independent States (CIS) to conduct business and forge partnerships, making it the leading pharmaceutical event in the region.
A team of researchers is developing advanced solutions to rapidly test for drugs and improve the treatment of patients presenting to the hospital. The researchers from the Colonial Foundation Healthy Ageing Centre (Melbourne, VIC, Australia) are employing mass spectrometry technology to rapidly detect any medications or drugs that might complicate a patient’s treatment. This rapid identification is crucial to ensure that patients receive appropriate care and to minimize the effects of any previously administered medications or drugs. Mass spectrometry identifies and quantifies chemicals in a sample using charged particles, or ions, proving to be more precise than traditional immunoassays, which can sometimes yield false positives. In their recent research, published in the Medical Journal of Australia, the research team demonstrated that traditional testing methods could mistakenly show toxic levels of a heart failure medication due to interference from a common prostate cancer drug. However, when mass spectrometry was used for ...
Progressive supranuclear palsy (PSP) is a severe neurological disorder classified under frontotemporal dementia (FTD) that impairs cognition, movement, and behavior. Thought to be caused by an accumulation of tau proteins that damage and kill cells, PSP presents symptoms such as poor balance with frequent backward falls and difficulties moving the eyes vertically. Often misdiagnosed as Parkinson’s disease, PSP progresses more rapidly and does not respond to Parkinson’s treatments. Most individuals with PSP pass away approximately seven years after symptoms begin, and frequently, the disease is only identified posthumously during an autopsy. Unlike Alzheimer’s, there are no tau scans, blood tests, or MRIs that can conclusively diagnose PSP, a fact that has hampered the development of treatments. Now, the discovery of a unique pattern of proteins in the spinal fluid of patients could lead to earlier diagnosis and the development of new treatments for PSP. In a study published in Neurology ...
By Don Tracy, Associate Editor Results from the Phase III STREAM Stage 2 study show the efficacy and safety of an all-oral bedaquiline-containing regimen for multidrug-resistant pulmonary tuberculosis. The FDA has granted a full approval to Johnson & Johnson’s (J&J) Sirturo for use in combination therapy in treating pulmonary tuberculosis (TB) caused by Mycobacterium tuberculosis resistant to rifampicin and isoniazid. According to the company, the approval by the FDA effectively removes previous label restrictions that were part of the medicine’s accelerated approval in December 2012. The regulatory action was based on promising results from the Phase III STREAM Stage 2 study.1 STREAM Stage 2 was the first large-scale, randomized, multi-country open-label clinical study to evaluate the efficacy and safety of an all-oral bedaquiline-containing regimen for treatment of MDR-TB. Between March 28, 2016, and January 28, 2020, 1436 participants were screened with 588 individuals randomly assigned to either the 2011 WHO ...
Eli Lilly’s Morphic acquisition brings an oral small molecule that blocks the same target as Entvyio, an injectable inflammatory bowel disease drug from Takeda Pharmaceutical. The Morphic deal follows Lilly’s 2023 acquisition of Dice Therapeutics, another company developing oral immunology medications. By Frank VinluanEli Lilly has immunology and inflammation products administered by injection, but the pharmaceutical giant is among the companies interested in bringing patients more convenient pill formulations. Its $3.2 billion acquisition of Morphic comes with a lead drug candidate that’s an oral small molecule in mid-stage development for inflammatory bowel disease. According to deal terms announced Monday, Lilly is paying $57 in cash for each share of Waltham, Massachusetts-based Morphic. That price represents a 79% premium to Morphic’s closing stock price on Friday. The deal, which has been approved by the boards of directors of both companies, is expected to close in the third quarter of this year. ...
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