Fibrodysplasia ossificans progressiva is a rare and life-threatening genetic disease caused by gain-of-function mutations in the ALK2 gene, which encodes activin receptor-like kinase 2. Blueprint Medicines Corp. has elucidated the discovery of their ALK2 inhibitor BLU-782, which is now in phase II studies at Ipsen for the treatment of FOP. Read More
Researchers from National Health Research Institutes reported the discovery and preclinical characterization of a novel long half-life Aurora A (AURKA) inhibitor being developed for the treatment of MYC-amplified solid tumors. Lead optimization of a previously reported series of small-molecule Aurora kinase inhibitors led to the discovery of AURKA kinase inhibitor 6K465, which was then developed into the acyl-based prodrug DBPR-728. Read More
Aurobac Therapeutics SAS, a biopharmaceutical company founded by Boehringer Ingelheim Pharma GmbH & Co KG, Biomérieux SA and Evotec SE, has announced its strategy to target bacterial infections, antimicrobial resistance and their consequences in acute hospital settings, such as hospital-acquired and ventilator-associated bacterial pneumonia and sepsis. Read More
Merck KGaA has announced it is advancing M-3554, a potential first-in-class anti-GD2 antibody-drug conjugate (ADC), toward the clinic. M-3554 links an exatecan payload with an anti-GD2 antibody. Read More
7 Hills Pharma Inc. has been awarded $4.7 million from the Cancer Prevention and Research Institute of Texas (CPRIT) to support its cell/gene therapy development program. Read More
Flagship Pioneering Innovations VI LLC has disclosed ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors reported to be useful for the treatment of fibrosis, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), Parkinson’s disease and cancer. Read More
Fibrogen Inc. has received IND clearance from the FDA allowing the company to initiate a phase I trial of FG-3165, a galectin-9 (Gal9)-targeted monoclonal antibody under development for treatment of solid tumors characterized by high levels of Gal9 expression. Read More
Researchers from Sun Yat-sen University Cancer Center (SYSUCC) presented data from a study that aimed to assess the role of trophoblast cell-surface antigen-2 (TROP-2) in penile squamous cell carcinoma (PSCC). Read More
Evopoint Biosciences Co. Ltd. has synthesized probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) and/or transcription activator BRG1 (SMARCA4; BAF190A; SNF2-β) inhibitors reported to be useful for the treatment of cancer. Read More
Beijing Danatlas Pharmaceutical Technology Co. Ltd. has identified thiadiazolyl derivatives acting as DNA polymerase θ (POLθ, POLQ) inhibitors reported to be useful for the treatment of cancer. Read More
Bluesphere Bio Inc. has received FDA clearance of its IND application for BSB-1001 for patients with relapsed or refractory acute myeloid leukemia (AML), acute lymphocytic leukemia and myelodysplastic syndromes, in conjunction with allogeneic hematopoietic stem cell transplantation (alloHSCT). Read More
Beijing Primegene Therapeutics Co. Ltd. has divulged thioether compounds acting as transient receptor potential cation channel subfamily V member 3 (TRPV3) antagonists reported to be useful for the treatment of pain, pruritus, inflammation, fever, cystitis, irritable bowel syndrome, cough and alopecia. Read More
Parmedics Inc. has described proteinase-activated PAR2 receptor antagonists reported to be useful for the treatment of asthma, cancer, osteoarthritis, migraine, chronic pain, inflammatory and vascular disorders. Read More
One in every 500 newborns worldwide are affected by nonsyndromic hearing loss (NSHL). Whole-exome sequencing may help in identifying the genetic cause of NSHL. Read More
