Spirits were high at the 2023 Annual Meeting of the American Society of Hematology (ASH), buoyed by U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego. Read More
Axonal degeneration is a key early pathogenic driver of amyotrophic lateral sclerosis and other neurodegenerative diseases. Activation of calpain-2 (CAPN2) is thought to be the critical effector behind axonal degeneration. Amylyx Pharmaceuticals Inc. has presented data on their CAPN2 inhibitor antisense oligonucleotide (ASO) AMX-0114 as a potential therapeutic for axonal degeneration. Read More
Korro Bio Inc. has nominated its first development candidate, KRRO-110, for the potential treatment of α1-antitrypsin deficiency. KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant. Read More
Researchers from Delix Therapeutics Inc. recently presented preclinical pharmacology data for DLX-001, a novel orally bioavailable, nonhallucinogenic neuroplastogen being developed for the treatment of neuropsychiatric diseases. Read More
Touchstone Innovations Ltd. has disclosed eukaryotic translation initiation factor 2-α kinase 4 (GCN2) inhibitors reported to be useful for the treatment of cancer, myocardial ischemia, diabetic cardiopathy, cardiotoxicity, nonalcoholic fatty liver disease and diabetic retinopathy. Read More
Researchers from Anhui University presented the discovery and preclinical evaluation of novel negative allosteric modulators (NAMs) of the metabotropic glutamate mGlu5 receptor. Read More
Vittoria Biotherapeutics Inc. has received FDA clearance of its IND application to initiate a first-in-human phase I trial with VIPER-101, a gene-edited, autologous, chimeric antigen receptor (CAR) T-cell therapy for treatment of patients with relapsed or refractory T-cell lymphoma. Read More
Simcere Zaiming Pharmaceutical Co. Ltd. has synthesized antibody-drug conjugates (ADC) comprising humanized monoclonal antibodies targeting CDH6 covalently linked to a camptothecin analogue through a linker. Read More
Nextpoint Therapeutics Inc. has received FDA clearance of its IND application for NPX-887, a fully human monoclonal antibody targeting HHLA2 (B7-H7), a novel immune checkpoint and tumor target antigen highly expressed in many cancers independently of PD-L1. Read More
Previous research has demonstrated that systemic administration of a P2X7 agonist improved motor performance in a mouse model of amyotrophic lateral sclerosis (ALS) through enhancing satellite cells and the muscle pro-regenerative activity of infiltrating macrophages. Read More
Shanghai Meiyue Biotech Development Co. Ltd. has identified MAPK p38 inhibitors reported to be useful for the treatment of cancer, arthritis, psoriasis, systemic lupus erythematosus, diabetes, atherosclerosis and Alzheimer’s disease. Read More
Subcommisural organ (SCO)-spondin is a brain-specific glycoprotein essential for neurogenesis with a high impact on neuronal development; thus, reduction in the production of SCO-spondin may lead to a lack of regeneration in neurological disorders. Read More
Guangzhou Imd Therapeutics Co. Ltd. has divulged proteolysis targeting chimeric (PROTAC) compounds comprising a protein cereblon (CRBN) binding moiety covalently bound to a eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1) and/or bromodomain-containing protein 2 (BRD2) targeting moiety through a linker. Read More
Foghorn Therapeutics Inc. has described probable global transcription activator SNF2L2 (SMARCA2; BRM) and transcription activator BRG1 (SMARCA4) degradation inducers acting as BRG1- or BRM-associated factor (BAF) complex modulators reported to be useful for the treatment of cancer and viral infection. Read More
Breast cancer patients receiving aromatase inhibitors as antihormone therapy are at major risk of cardiovascular disease. Previous observations suggest that, since estrogen biosynthesis is regulated by aromatase and aldosterone biosynthesis is modulated by aldosterone synthase, a dual inhibitor could have anticancer efficacy while reducing cardiovascular risks in breast cancer patients. Read More
Are you trying to stay on top of developments in CAR T therapies? Drug resistance? Emerging compounds? Updates from certain regulatory agencies? Want to be the first to learn about new biomarkers or business deals? You can still find these articles in the daily BioWorld e-newsletters or on the website, but if you’re laser-focused on a specific topic, we have a way to highlight the news you need as it happens, whether we’ve published a full-length feature story or a brief update. You asked and we listened. Now, in addition to the daily news lineup, you can create topic alerts to be delivered directly to your inbox or via an RSS reader. It’s easy to set up. Instructions can be found here: https://www.bioworld.com/featured-feeds. Happy holidays! Read More
