While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity. Read More
Basal-like breast cancer is a particularly aggressive subtype of the malignancy, for which it has been difficult to identify good therapeutic targets. In collaboration, European researchers mined public transcriptome datasets to show that the aldehyde dehydrogenase 1A isoform ALDH1A3 was predominantly expressed in epithelial cells within basal-like tumors, whereas the isoforms ALDH1A1 and ALDH1A2 were predominantly expressed in stromal and immune-associated cells. Read More
Allterum Therapeutics Inc. has obtained IND clearance from the FDA for 4A10, paving the way for initiation of a first-in-human phase I trial in patients with relapsed or refractory acute lymphoblastic leukemia (ALL). 4A10 is a monoclonal antibody with a human immunoglobulin G subclass 1 (IgG1) backbone that specifically binds to CD127 (IL-7Rα). Read More
Recurv Pharma Inc. has developed a novel taxoid compound formulated in an oil-in-water nanoemulsion, named RP-001, as a potential therapeutic approach for the management of pancreatic ductal adenocarcinoma (PDAC), either as a single agent or combined with immune checkpoint inhibitors. 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
IKZF2, a transcription factor in the Ikaros family, plays a key role in stabilizing regulatory T (Treg) cells and promoting immune suppression within the tumor microenvironment (TME). Selective targeting of IKZF2 in intratumoral Tregs has the potential to enhance antitumor immunity and improve the efficacy of immunotherapy, while minimizing immune-related toxicities associated with systemic Treg depletion. Read More
Primary sclerosing cholangitis is an autoimmune disease affecting the liver in which there is reduced expression and function of the bile acid receptor GPBAR1 in the cholangiocytes. Read More
In their efforts to develop next-generation antibiotics, researchers from Hunan University and collaborators designed amidated oligopyridinium peptidomimetics. Read More
Sichuan Kelun Pharmaceutical Research Institute Co. Ltd. and Suzhou Kelun Pharmaceutical Co. Ltd. have identified carboxylic acid ester compounds reported to be useful for anesthesia. Read More
Antimicrobial peptides such as human β-defensin 3 (hBD3) have shown promise as a treatment for periodontitis; however, their poor stability and high production costs have hindered their clinical translation until now. Read More
Beijing Avistone Pharmaceuticals Biotechnology Co. Ltd. has disclosed spiro/bridged ring compounds acting as HER2 (erbB2) (exon 20 insertion [Ex20Ins] [Ala775_Gly776insTyrValMetAla mutant]) inhibitors reported to be useful for the treatment of atherosclerosis, cancer and pulmonary fibrosis. Read More
Myrobalan Therapeutics Nanjing Co. Ltd. has divulged uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-like) antagonists reported to be useful for the treatment of multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease. Read More
While immunotherapy targeting PD-1 or its ligand PD-L1 has transformed cancer treatment by overcoming T-cell exhaustion, only a minority of patients achieves durable responses after treatment. Read More
Beijing Chempion Biotechnology Co. Ltd. has synthesized antibody-drug conjugates comprising antibodies targeting folate receptor α (FOLR1; FR-α) covalently linked to a cytotoxic drug through a linker reported to be useful for the treatment of cancer. Read More
Polo-like kinase 4 (PLK4), which regulates centriole duplication and mitotic progression, is overexpressed in several cancers, including neuroblastoma as well as breast, lung and colorectal cancers, making it an attractive target. Several PLK4 inhibitors have been developed, but only one has entered clinical trials, which involve patients with acute myeloid or chronic myelomonocytic leukemia. Read More
Parkinson’s disease (PD) is a progressive disease characterized by loss of dopaminergic neurons in the substantia nigra, which lead to motor symptoms. Aromatic-L-amino-acid decarboxylase (AADC) converts levodopa into dopamine and glial cell line-derived neurotrophic factor (GDNF) promotes the survival, growth and regeneration of dopaminergic neurons. VGN-R09b is an adeno-associated viral vector (AAV)-based AADC and GDNF combination gene therapy that is delivered to the striatum for the treatment of PD. Read More
Shandong Simcere Medgenn Bio-Pharmaceutical Co. Ltd. has described CDK4/cyclin D1 inhibitors reported to be useful for the treatment of cancer. Read More
