The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments. Read More
Oligomeric forms of α-synuclein are increasingly recognized as the primary neurotoxic species in Parkinson’s disease (PD) and other synucleinopathies, contributing to synaptic dysfunction, mitochondrial impairment and the prion-like propagation of pathology. Targeting these early aggregates represents a promising strategy for disease modification. Read More
Glycovax Pharma Inc. has entered into a strategic collaboration with the National Research Council of Canada (NRC) and the Université de Montréal to develop a glycoconjugate vaccine against Pseudomonas aeruginosa infections, with expected utility for patients with cystic fibrosis and hospitalized individuals at risk of nosocomial infections.
Boehringer Ingelheim Pharma GmbH & Co KG has signed an asset purchase agreement with Accent Therapeutics Inc. for Accent’s preclinical small-molecule program that offers a novel approach for treating tumors with high interferon-stimulated gene (ISG) expression. Read More
BioWorld Staff Writer Randy Osborne reflects on the transformative progress in gene therapy – from tools like CRISPR and AAV vectors to major breakthroughs in diseases like hemophilia, sickle cell and beta-thalassemia. Read More
Tessera Therapeutics Inc. has been awarded up to $41.3 million from the Advanced Research Projects Agency for Health (ARPA-H) as part of its EMBODY (Engineering of immune cells inside the body) program to support the development of Tessera’s in vivo CAR T therapy efforts. Read More
A new study aimed to investigate the therapeutic effect of ISM012-042 in a chronic T-cell transfer-induced colitis model in mice that mimicked Crohn’s disease. Read More
Zenas Biopharma Inc. and Innocare Pharma Ltd. have entered into a license agreement granting Zenas rights for three autoimmune product candidates. Read More
Researchers at Hefei Amvite Pharmaceutical Co. Ltd. reported the discovery and preclinical evaluation of a series of structurally modified ziritaxestat derivatives, leading to the identification of more potent autotaxin inhibitors. Read More
Pfizer Inc. has identified new indole analogues acting as sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatotic liver disease (NAFLD; MASLD), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism-spectrum disorders, among others. Read More
Shenzhen Bay Laboratory has synthesized fused ring derivatives acting as dihydrofolate reductase (DHFR) (bacterial) inhibitors reported to be useful for the treatment of cancer, bacterial, fungal, nematode and viral infections. Read More
Confo Therapeutics NV has secured a 2-year €1 million (US$1.2 million) grant from Flanders Innovation & Entrepreneurship (VLAIO) to advance research and development of ultra-long-acting medicines targeting G protein-coupled receptors (GPCRs), including bi- and multispecific antibody formats for obesity and other metabolic and endocrine disorders. Read More
Convalife Pharmaceuticals Co. Ltd. and Zhejiang Convalife Pharmaceutical Co. Ltd. have discovered sulfanilamide compounds acting as kinesin-like protein KIF18A inhibitors reported to be useful for the treatment of cancer, inflammatory disorders and ciliopathy. Read More
Simon Fraser University, TRIUMF and the University of British Columbia have disclosed radiolabeled compounds reported to be useful for the diagnosis of cancer. Read More
Nilo Therapeutics Inc. has launched with a $101 million series A financing and a focus on harnessing neural circuits to restore immune homeostasis in disease. Read More
Casma Therapeutics Inc. has developed bicyclic heteroaromatic compounds acting as mucolipin-1 (MCOLN1; TRPML1) activators reported to be useful for the treatment of Duchenne muscular dystrophy, neurodegeneration, atherosclerosis, cancer, infections, liver, lysosomal storage and inflammatory bowel diseases. Read More
Additional early-stage research and drug discovery news in brief, from: Circio, IGC Pharma, JCR Pharmaceuticals, Nurexone Biologic, Resalis Therapeutics, Telomir Pharmaceuticals. Read More
