The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
Researchers from Yale School of Medicine, Ionis Pharmaceuticals Inc. and the National Institutes of Health have published data from a study that aimed to identify the signaling pathways involved in cilia-dependent cyst activation (CDCA) in autosomal dominant polycystic kidney disease (ADPKD).
Breast cancer is a heterogenous disease in terms of its prognosis and treatment response. Metabolic reprogramming is a potential therapeutic target because of its repercussion on oncogenesis.
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. 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.
Hepaitech (Beijing) Biopharma Technology Co. Ltd. has patented compounds reported to be useful for the treatment of anemia, fungal infections and HIV infections, among others.
Immunotherapy-based cancer vaccines could permanently kill tumors by stimulating immune cells in multiple ways. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), researchers presented their advances in this field with different techniques in the scientific symposium...
Annually in the U.S., about 40,000 people with corneal endothelial cell dystrophy receive a corneal transplant. The ligand of melanocortin MC1 receptor (MC1R) has been shown to protect the corneal endothelial cells from stress and injury in several models.
Previous studies have linked variants of complement factor H (CFH) and its alternative splicing isoform, FHL-1, with increased complement activation and risk of age-related macular degeneration (AMD).
Ascidian Therapeutics Inc. recently provided preclinical data for ACDN-01, an AAV-encoded RNA exon editor targeting ABCA4, being developed for the treatment of ABCA4-related retinopathies, including Stargardt disease.
By isolating a single clone from human isolates, researchers from the National Taiwan University College of Medicine aimed to enhance the immunogenicity of the Zika virus (ZIKV).
Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized 3C-like proteinase (3CLpro, Mpro) (coronavirus) inhibitors reported to be useful for the treatment of coronavirus acute respiratory syndrome infections.
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.
4M Therapeutics Inc. has announced the presentation of preclinical data on its small-molecule glycogen synthase kinase-3β (GSK-3β) inhibitors in models of bipolar disorder.
Macrophage colony-stimulating factor 1 receptor (CSF-1R) is a transmembrane tyrosine kinase receptor expressed in brain microglia, and mutations in the CSF1R gene have been linked to adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
A Tisento Therapeutics Inc. patent describes new soluble guanylate cyclase (sGC) activators reported to be useful for the treatment of Alzheimer’s and Parkinson’s diseases, vascular dementia, ischemic stroke, Leigh syndrome, Pearson syndrome, creatine deficiency syndrome and Alpers syndrome, among...
Norwegian University of Science and Technology (NTNU) has synthesized macrophage colony-stimulating factor 1 receptor (CSF-1R; CD115; c-Fms) inhibitors reported to be useful for the treatment of cancer, amyotrophic lateral sclerosis, Charcot-Marie-Tooth disease, macular degeneration,...
Avicenna Biosciences Inc. has introduced an extension to its machine learning (ML) technology platform to enhance medicinal chemistry and expedite clinical-stage drug discovery.
Centogene NV and Evotec SE have announced the discovery of a new small molecule with potential to treat patients with type 2 and type 3 Gaucher disease, or neuronopathic Gaucher disease.
Researchers from Tokyo Medical University presented data from a study that assessed microRNA (miRNA) profiles in extracellular vesicles (EVs) isolated from vitreous humor and serum of patients with ocular sarcoidosis and vitreoretinal lymphoma (VRL).
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...
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...
Aqilion AB has expanded its pipeline through the nomination of protein kinase C θ (PKCθ) as the target of a new immunology project. The PKCθ kinase is believed to be centrally involved in the pathogenesis of T-cell-mediated inflammatory and...
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...
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.