The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that causes this genetic or infectious disorder could be achieved thanks to new gene-silencing techniques. At a special meeting of the American Society of Gene & Cell Therapy, in “AAV-mediated epigenetic editing for prion disease,” Sonia Vallabh presented not just the data of her research, but the impact of this disease on her family and on herself. Read More
Myrio Therapeutics Pty Ltd. has been able to accomplish something no other company has yet been able to crack: to develop binders where both the affinity and the specificity can be increased. Read More
CSPC Pharmaceutical Group Ltd. has gained clinical trial clearance from China’s National Medical Products Administration (NMPA) for JMT-206 for weight management in individuals with obesity or overweight and at least one weight-related comorbidity. Read More
SK Biopharmaceuticals Co. Ltd. has signed a license agreement with the Wisconsin Alumni Research Foundation (WARF) to acquire rights to WARF’s WT-7695, a preclinical-stage radiopharmaceutical therapy candidate developed in collaboration with the University of Wisconsin-Madison. Read More
Researchers from Tango Therapeutics Inc. presented preclinical efficacy data on TNG-456, a selective MTA-cooperative PRMT5 inhibitor under development for the treatment of glioma and other tumors that frequently metastasize to the brain, such as non-small-cell lung cancer. Read More
Blueprint Medicines Corp. has disclosed PROTAC compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a cyclin-dependent kinase 4 (CDK4)-targeting moiety potentially useful for the treatment of cancer. Read More
Researchers from Northeast Agricultural University and Jiangsu Kanion Pharmaceutical Co. Ltd. aimed to improve the oncolytic effect of existing Newcastle disease virus (NDV) vectors using engineering strategies to accelerate their clinical translation. Read More
Gluetacs Therapeutics (Shanghai) Co. Ltd. has synthesized molecular glue degraders comprising cereblon (CRBN) binding agents and protein degradation moieties reported to be useful for the treatment of cancer, infections, autoimmune diseases, anemia, transplant rejection, diabetes, cardiovascular disorders and inflammatory disorders, among others. Read More
In an effort to develop more effective estrogen receptor α (ERα) inhibitors, researchers at Nanjing University of Chinese Medicine and collaborators aimed to develop a proteolysis targeting chimera (PROTAC) against the receptor. Read More
Hansoh Bio LLC, Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have divulged transcriptional coactivator YAP1/transcriptional enhancer factor (TEAD) interaction inhibitors reported to be useful for the treatment of cancer. Read More
Brenig Therapeutics Inc. has described leucine-rich repeat kinase 2 (LRRK2; dardarin) and (LRRK2; dardarin) (G2019S mutant) inhibitors reported to be useful for the treatment of Parkinson’s disease, among others. Read More
In an effort to identify stronger and safer α-glucosidase inhibitors, researchers at the Chinese Academy of Sciences prepared a series of diarylpentane derivatives, the most promising of which, [I], reversibly inhibited α-glucosidase with an IC50 of 18 µM. Read More
