Cerebral cavernous malformations (CCMs) are vascular lesions in the central nervous system and in the brain that are tied with intracerebral bleeds and seizures, with an occurrence ratio of 0.4% to 0.9%. Seizure is the second most common symptom and >25% of CCMs are identified following an epilepsy diagnosis.
Ichnos Sciences Inc. has presented data on the first-in-class trispecific BCMA and CD38 T-cell engager ISB-2001, which is based on the company’s TREAT (Trispecific Engagement by Antibodies based on the TCR) technology. The compound targets CD3 on T cells and cotargets BCMA and CD38 on multiple myeloma (MM) cells.
Ovarian cancer is ordinarily associated with poor survival; patients diagnosed with high-grade serous ovarian carcinoma (HGSC) have an overall survival of about 40% at 5 years and 15% at 10 years. Despite having similar histologic features, HGSC patients often experience highly variable outcomes and the underlying determinants for long-term survival (LTS) are largely unknown. In a study published online in Nature Genetics, a multi-institutional group of researchers tried to determine the molecular differences that drive LTS in patients with HGSC.
Shanghai Pharmaceuticals Holding Co. Ltd. has described quinazoline compounds acting as TNF-α and/or IL-6 and/or IL-1β production inhibitors reported to be useful for the treatment of cancer, asthma, malaria, viral infections, autoimmune diseases, cardiovascular disorders, chronic obstructive pulmonary disease (COPD) and transplant rejection.
Quralis Corporation’s clinical trial application (CTA) for QRL-201, a first-in-class Stathmin-2 (STMN2) precision medicine for amyotrophic lateral sclerosis (ALS), has been authorized by Health Canada.
Wigen Biomedicine Technology (Shanghai) Co. Ltd. has divulged pyrrolopyrimidine derivatives containing a pyrazine structure acting as Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of cancer.
Opna Bio AG has identified transcriptional coactivator YAP1/transcriptional enhancer factor (TEAD) interaction inhibitors reported to be useful for the treatment of arrhythmogenic right ventricular cardiomyopathy, cancer, Holt-Oram syndrome, neurofibromatosis type 2, polycystic kidney and Alzheimer's disease.
Sionna Therapeutics Inc. has received FDA clearance of its IND application for SION-638, a small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A phase I study is now dosing healthy volunteers.
The archetypal UbiB protein COQ8 has human homologues COQ8A and COQ8B, both with well-established connection to human disease, with inactivating mutations in COQ8A resulting in autosomal recessive cerebellar ataxia. Researchers from the University of Wisconsin-Madison and affiliated organizations have now recently reported the discovery of small-molecule inhibitors of COQ8A.
RSS
