Some rare skin diseases not only reduce the quality of life of patients, but also can be devastating conditions, leading to amputations or death. At the 31st annual congress of the European Society of Gene and Cell Therapy (ESGCT), held last week in Rome, different laboratories showcased their approaches to editing mutations related to this group of diseases. Read More
Researchers from Immunome Inc. and Zentalis Pharmaceuticals Inc. presented preclinical data for IM-1021, a novel tyrosine kinase-like orphan receptor 1 (ROR1)-targeted antibody-drug conjugate with a new topoisomerase 1 (TOP1) linker payload, being developed for the treatment of solid tumors and B-cell malignancies. Read More
AKT1 E17K is the most frequent gain of function mutation of the AKT1 gene. This mutation promotes pathologic localization of AKT1 to the plasma membrane and has been shown to induce leukemia in mice. Current options for AKT1 E17K-driven tumors are limited. Read More
Ottimo Pharma Ltd. has emerged from stealth with a focus on developing innovative cancer therapies for solid tumors. The company’s lead program is jankistomig, a PD-1/VEGFR-2 bifunctional antibody. Read More
Researchers at Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have disclosed crystalline salts of known tyrosine-protein kinase ABL1 (ABL) inhibitors reported to be useful for the treatment of leukemia. Read More
Ideaya Biosciences Inc. has announced FDA clearance of an IND application for IDE-275 (GSK-959), a small-molecule inhibitor of Werner helicase (WRN) discovered by Ideaya in collaboration with GSK plc. Read More
Researchers from Universidad Carlos III de Madrid presented the development and preclinical characterization of a novel specific radiotracer for the diagnosis of Clostridioides difficile infection (CDI). Read More
Wigen Biomedicine Technology (Shanghai) Co. Ltd. has synthesized compounds acting as membrane-associated tyrosine- and threonine-specific Cdc2-inhibitory kinase (PKMYT1) inhibitors reported to be useful for the treatment of cancer. Read More
Suzhou Ansailong Pharmaceutical Technology Co Ltd. has identified mitochondrial ATP-dependent Clp protease proteolytic subunit (ClpP) activators reported to be useful for the treatment of cancer, AIDS, nonalcoholic fatty liver disease (metabolic dysfunction-associated steatotic liver disease [MASLD]) and neurological disorders. Read More
The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic syndrome caused by choline acetyltransferase (CHAT) gene deficiency. The FDA awarded the orphan drug designation to AVCHAT-01X (AAV serotype 9 human choline acetyltransferase). Read More
Haisco Pharmaceutical Group Co. Ltd. has divulged cholesterol side-chain cleavage enzyme, mitochondrial (CYP11A1) inhibitors reported to be useful for the treatment of cancer. Read More
The advantages of affibodies vs. antibodies are that they have a smaller size, better penetration and faster extravasation, can be produced both recombinantly and synthetically, and show robustness regarding protein scaffold. Read More
Bivision Biomedical Technology (Nanjing) Co. Ltd. has described radiolabeled-metal complexes targeting fibroblast activation protein α (FAP) reported to be useful for the diagnosis and treatment of cancer. Read More
