An analysis of more than 1,000 small molecules has identified dozens of compounds that could be effective to treat Marfan syndrome (MFS), an inherited disorder affecting connective tissue, primarily in the heart and blood vessels, the skeleton, and the eyes. In particular, the researchers from Cambridge University found that glycogen synthase kinase-3β (GSK-3β) could be a target to develop new therapies based on its inhibition. Read More
A genome-wide association study (GWAS) from The University of Queensland has linked blood cell traits (BCTs) and neurological and psychiatric disorders (NPDs), providing a tool to improve patient treatments or repurposing different drugs. The researchers also found a cause-effect relationship between Parkinson's and platelet distribution width. In their study, published Jan. 25, 2023, in Cell Genomics, the scientists observed the genetic overlap between common NPDs and 29 BCTs, including functional genes, regulatory elements and new genetic correlations linked to hematological data and for these diseases. Read More
Genetically modified derivatives of the chimeric oncolytic virus (OV) CF33 have previously shown cancer selectivity and oncolytic potency against various solid tumors.Read More
Outcomes after therapy with DNA-damaging agents such as irinotecan or oxaliplatin have been seen to improve in patients with homologous recombination-deficient colorectal cancer (HRD CRC) compared to those who have HR proficient CRC (HRP CRC). Read More
The FDA has cleared Cullinan Oncology Inc.'s IND application for CLN-978, a CD19/CD3 T-cell engaging antibody construct with a human serum albumin (HSA) binding domain to increase serum half-life. Read More
Shanghai Allist Pharmaceuticals Co. Ltd. has synthesized nitrogen-containing heterocyclic compounds acting as GTPase KRAS (G12D mutant) and (G12V mutant) inhibitors reported to be useful for the treatment of cancer. Read More
Immpact Bio USA Inc. has announced clearance of its IND application by the FDA for IMPT-314, a bispecific OR-Gate autologous chimeric antigen receptor (CAR) T-cell therapy targeting the B-cell antigens CD19 and CD20. Read More
Sunshine Lake Pharma Co. Ltd. has divulged benzazepine heterocyclic compounds acting as nuclear receptor RORγT (T cell-specific isoform 2) agonists reported to be useful for the treatment of cancer, rheumatoid arthritis, psoriasis, asthma, systemic lupus erythematosus, multiple sclerosis, inflammatory bowel disease and Kawasaki disease, among others. Read More
Decibel Therapeutics Inc. has received clearance of its clinical trial application (CTA) by the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of gene therapy DB-OTO in pediatric patients with congenital hearing loss due to an otoferlin deficiency. Read More
West China Hospital, Sichuan University has described heteroatom-substituted aromatic compounds reported to be useful for the treatment of pain and as general anesthetics. Read More
