Beam Therapeutics Inc. has offered a progress update on its genetic disease franchise. BEAM-302, the company’s priority genetic disease program, is a potential treatment for α1-antitrypsin deficiency (AATD).
Olema Pharmaceuticals Inc. has advanced its program targeting KAT6, an epigenetic target dysregulated in breast and other cancers, with the selection of a development candidate, OP-3136. The orally bioavailable, potent KAT6A/B-selective inhibitor has been developed by Olema in collaboration with Aurigene Oncology Ltd.
Pulmonary arterial hypertension (PAH), characterized by vasoconstriction and pulmonary vascular remodeling, has a 10% annual mortality rate among patients due to right heart failure. There are genetic variants known to impact the risk of PAH, but susceptibility from epigenetic changes is poorly understood.
Hinova Pharmaceuticals Inc. has divulged proteolysis targeting chimeric (PROTAC) compounds comprising a protein cereblon (CRBN) binding moiety covalently bound to an estrogen receptor (ER)-targeting moiety through a linker.
Boehringer Ingelheim Pharma GmbH & Co. KG has identified 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) inhibitors reported to be useful for the treatment of nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH).
GSK plc has synthesized nitrogen-containing condensed 2,3-dihydroquinazolinone compounds acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of atrial fibrillation, osteoarthritis, and postoperative and neuropathic pain.
Chengdu Baiyu Pharmaceutical Co. Ltd. has disclosed pyrrolidine derivatives acting as IL-6 inhibitors and Toll-like receptor 7 (TLR7) and/or TLR8 antagonists reported to be useful for the treatment of autoimmune disease.
Researchers from China Pharmaceutical University have reported the discovery and preclinical evaluation of novel bifunctional molecules inducing DNA damage and targeting PARP for the treatment of cancer.
Sana Biotechnology Inc. has obtained FDA clearance of its IND application to conduct a study of SC-262 in patients with relapsed or refractory B-cell malignancies, initially in patients who have received prior CD19-directed chimeric antigen receptor (CAR) T therapy.
RSS
