Parkinson’s disease is a progressive neurodegenerative disorder best known for its motor symptoms. However, a proportion of patients also develop dementia as the condition advances. Yet the biological divide between those who experience this cognitive decline and those who do not has remained an open question. Are they different conditions or simply stages of the same disease? Read More
Forx Therapeutics AG presented a comprehensive preclinical medicinal chemistry and translational profiling program describing the optimization of potent and selective poly(ADP-ribose) glycohydrolase inhibitors, which led to the identification of FORX-428, currently in phase I clinical trials. The company also disclosed the chemical structure of the compound. Read More
The use of CAR T-cell therapy has transformed outcomes for relapsed or refractory B-cell malignancies, but access to it remains extremely limited in some countries. Cartogene Therapeutics Pvt Ltd. aimed to address this need by in-licensing CGT-19, a CD19 CAR T construct from Vector Biomed Inc. Read More
In both acute myeloid leukemia (AML) and synovial sarcoma (SS), targeting BRD9 disrupts oncogenic transcriptional programs, including MYC, leading to reduced proliferation and induction of apoptosis. Researchers from Pamplona Therapeutics (Shenzhen) Co. Ltd. reported the discovery and preclinical efficacy profile of XYD-270, a BRD9-targeting PROTAC, in models of SS and AML. Read More
A University of Texas System patent describes new radiolabeled positron emission tomography (PET) imaging agents targeting receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1). As such, they are potentially useful for the diagnosis of cancer, retinal and autoimmune diseases, neurological and inflammatory disorders. Read More
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by the progressive degeneration of motor neurons, leading to muscle wasting and paralysis, among others. Due to its high clinical heterogeneity, effective therapies are still not available. Read More
Rapt Therapeutics Inc. has patented new fused ring imidazole compounds acting as TNF-α modulators and thus reported to be useful for the treatment of cancer, pain, metabolic and autoimmune disease, inflammation, neurological, cardiovascular and eyes disorders. Read More
Work at Ventus Therapeutics US Inc. has led to the identification of new caspase-4 allosteric and/or caspase-5 inhibitors reported to be useful for the treatment of sepsis, stroke, myocardial infarction, acute respiratory distress syndrome, inflammatory bowel disease, hidradenitis suppurativa, diabetic nephropathy and diabetic retinopathy, among others. Read More
Using Iambic Therapeutics’ AI/HTE platform, researchers at the company have engineered a next-generation HER2 inhibitor with superb selectivity for HER2, as well as broad mutant coverage and brain penetrance. The compound, IAM-1363, binds to HER2 in a type II DFG-out conformation, which represents the first reported type II HER2 tyrosine kinase inhibitor. Read More
Nanjing Sanhome Pharmaceutical Co. Ltd. has synthesized new Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors potentially useful for the treatment of cancer. Read More
Researchers from Blueprint Medicines Corp. and F. Hoffmann-La Roche Ltd. presented the preclinical characterization of BLU-852, a selective MAP4K1 inhibitor developed as a potential immunotherapeutic agent for cancer treatment. Read More
