Heptares Therapeutics Ltd. has divulged pyrrolidine-2-carboxamide derivatives and morpholine-3-carboxamide derivatives acting as prostaglandin E2 receptor EP4 subtype (PTGER4; EP4) agonists reported to be useful for the treatment of gastrointestinal and respiratory disorders.
Scientists at Johns Hopkins University and The Lieber Institute for Brain Development have identified peripherally restricted GABA(A) receptor subunits α3β2γ2S (GABRA3) positive allosteric modulators reported to be useful for the treatment of inflammatory bowel disease, lactose intolerance and abdominal pain.
Arvinas Operations Inc. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN)-binding moiety covalently linked to a non-receptor tyrosine-protein kinase TYK2-targeting moiety via linker.
Iambic Therapeutics Inc. has disclosed quinazoline compounds acting as cyclin-dependent kinase (CDK) inhibitors, particularly CDK2 and/or CDK4 and/or CDK6 inhibitors reported to be useful for the treatment of ovarian and breast cancer.
Pulsesight Therapeutics SAS has launched with seed financing, with the aim of developing disruptive non-viral gene therapies with minimally invasive delivery technology.
PARP inhibitors such as olaparib are used for the treatment of metastatic castration-resistant prostate cancer (mCRPC) harboring homologous recombination deficiency (HRD), but a proportion of these patients do not respond to therapy or eventually develop resistance.
Arthex Biotech SL has received FDA clearance to initiate the phase I/IIa Arthemir study of ATX-01 for the treatment of myotonic dystrophy type 1 (DM1).
Researchers from Macquarie University have detailed the discovery of a novel gene therapy vector targeting pathological TAR-binding protein 43 (TDP-43), CTx-1000, as a potential therapeutic candidate for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) – two diseases characterized by cytoplasmic deposition of the nuclear TDP-43.