From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
Researchers from Xiangya Hospital of Central South University published data from a study that explored potential treatment targets for cutaneous squamous cell carcinoma (cSCC) by investigating the regulatory mechanisms of follistatin-like 1 (FSTL1), which has been shown to be implicated in a variety of cancers.
Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex.
The recent cyberattack on Change Healthcare, a part of the Unitedhealth Group, crippled reimbursement claims processing for thousands of providers for several weeks and potentially exposed troves of patient data. Congressional committees are investigating the attack, its scope, and Unitedhealth’s response. Are there downstream impacts that are being overlooked in the biopharmaceutical and/or medical technology sectors? Please take a few minutes to fill out this short survey to help BioWorld understand how your company is responding to these threats. You can click through to the questions here.
Crispr Therapeutics AG has expanded its in vivo pipeline with two new programs, which utilize lipid nanoparticle (LNP)-based delivery of CRISPR/Cas9 gene-editing cargo to the liver.
Université de Montréal has disclosed serine/threonine-protein kinase A-Raf, (ARAF) and/or B-Raf (BRAF) and/or RAF proto-oncogene serine/threonine-protein kinase (c-Raf) inhibitors reported to be useful for the treatment of cancer.
Researchers from Sichuan Baili Pharmaceutical Co. Ltd. and Systimmune Inc. presented preclinical data for the novel CD33-targeting antibody-drug conjugate (ADC) being evaluated for the treatment of hematologic malignancies.
Recent findings discovered a mutation in the METTL23 gene, which encodes methyltransferase-like protein 23, in a pedigree of normal-tension glaucoma (NTG). The aim of researchers from the Institute for Vision Research, The University of Iowa Roy J and Lucille A Carver College of Medicine was to...
New treatment options for treating Mycobaterium abscessus infections are needed. Previous findings had identified the leucyl-tRNA synthetase inhibitor MRX-6038 to have significant activity against M. abscessus. The aim of this new study was to focus on the activity of MRX-5, the oral prodrug of...
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in...
Researchers from the Icahn School of Medicine at Mount Sinai and affiliated organizations presented the discovery and preclinical characterization of MS-8535, a novel spindlin-1 (SPIN1) inhibitor being developed as chemical tool anticancer agent.
The Global Health Innovative Technology (GHIT) Fund has announced a total investment of approximately ¥1.64 billion (US$10.8 million) in four projects for the development of new drugs for malaria and neglected tropical diseases.
Sepsis occurs when there is a dysregulated host response to infection, resulting in organ dysfunction. According to the Global Burden of Disease study, about 50 million people annually worldwide develop severe sepsis or septic shock, and 11 million of them die. Growing evidence suggests that...
There is a compelling need to produce new antibiotics that hamper severe opportunistic infections, especially in immunocompromised individuals, such as those caused by gram-negative Pseudomonas aeruginosa.
Avicenna Biosciences Inc. has introduced an extension to its machine learning (ML) technology platform to enhance medicinal chemistry and expedite clinical-stage drug discovery.
Saniona AB has initiated preclinical development of SAN-2355, a potential best-in-class and new-generation Kv7.2/Kv7.3 activator for the treatment of focal onset seizures.
Scientists at Centre Hospitalier Regional Universitaire de Lille, INSERM and University of Lille have divulged diphenylpyrazole compounds acting as β-amyloid (Aβ) production inhibitors and autophagy marker ratio modulators reported to be useful for the treatment of Alzheimer’s disease.
Based on its analysis of a large cohort of individuals homozygous for the ε4 variant of apolipoprotein E (APOE4), a multinational team of researchers is arguing that homozygosity for APOE4 should be considered a genetic form of Alzheimer’s disease. However, not everyone agrees that the findings...
Researchers at Biodol Therapeutics SAS, Centre National de la Recherche Scientifique, INSERM and Université de Strasbourg have divulged new n-heteroarylbenzamide derivatives acting as FLT3 (FLK2/STK1) inhibitors and reported to be useful for the treatment of pain.
Researchers from Tan Tock Seng Hospital presented data from a study investigating the utility of soluble suppressor of tumorigenicity-2 (sST2) as early prognostic biomarker of severe dengue.
Severe fever with thrombocytopenia syndrome (SFTS) is a tick-borne disease that results from an infection with the SFTS virus, with a case fatality rate of 6.1% to 21.8%. Identifying critically ill patients at the early stages is crucial for clinical management.
About 25% of subjects with West Nile virus (WNV) infection develop fever and about 1% have neuroinvasive disease. Recent research has proposed measuring CD169 in peripheral blood (monocyte/lymphocyte ratio) as a marker of viral infections. The usefulness of monocyte CD169 (mCD169) in peripheral...
Pfizer Inc. has divulged 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17- β-HSD 13) inhibitors and/or degraders reported to be useful for the treatment of hepatocellular carcinoma, biliary cirrhosis, hepatitis B and hepatitis C viral...
Virginia Commonwealth University has identified cholestenoic acid derivatives reported to be useful for the treatment of cancer, sepsis, atherosclerosis and nonalcoholic fatty liver disease (NAFLD; metabolic dysfunction-associated steatotic liver...
Synedgen Inc. has completed IND-enabling studies for its lead candidate, MIIST-305, which is being developed for ulcerative colitis and as a medical countermeasure for gastrointestinal acute radiation syndrome. Within a year, Synedgen plans to...
Ochre Bio Ltd. has established a partnership with Boehringer Ingelheim Pharma GmbH & Co. KG focused on the discovery and development of novel first-in-class regenerative treatments for chronic liver diseases, such as late-stage metabolic...
New York University has described protein spinster homolog 2 (SPNS2) inhibitors reported to be useful for the treatment of acute lung injury, autoimmune disease, colitis, Alzheimer’s disease, fibrosis, inflammatory bowel disease, multiple...
Palisade Bio Inc. has completed its analysis evaluating ex vivo bioactivation of PALI-2108, an orally administered, locally acting colon-specific phosphodiesterase-4 (PDE4) inhibitor prodrug in development for ulcerative colitis.
Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.