Cure Rare Disease has successfully completed a pre-IND meeting with the FDA regarding its investigational gene therapy program for limb-girdle muscular dystrophy type 2I/R9 (LGMDR9).
Bispecific antibodies mimicking coagulation factor VIIIa (FVIIIa) have yielded successful results in the treatment of hemophilia A, but there remain some issues in regard to their production.
Supercede Therapeutics Inc. has released preclinical efficacy data on its small-molecule inhibitor of the Activin receptor type II (ACTRII) to treat obesity.
Cue Biopharma Inc. has received pre-IND feedback from the FDA to discuss the proposed development plan for CUE-401, its lead autoimmune asset. Based on FDA feedback, the company intends to file an IND pending completion of final IND-enabling studies.
At the recent meeting of the Society of Nuclear Medicine and Molecular Imaging, Chinese Academy of Medical Sciences and Peking Union Medical College scientists co-presented a carbonic anhydrase IX (CAIX)-targeting 68Ga/177Lu theranostic pair for clear cell renal cell carcinoma (ccRCC) and its preclinical evaluation.
Work at Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has led to the identification of new protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer.
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