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.
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.
Samsung Life Public Welfare Foundation has described peptides acting as Toll-like receptor (TLR) signaling inhibitors and antioxidants reported to be useful for the treatment of cancer as well as immunological, dermatological and inflammatory disorders.
Insilico Medicine IP Ltd. has divulged serine/threonine-protein salt-inducible kinase (SIK) inhibitors reported to be useful for the treatment of autoimmune disease, cancer, metabolic diseases, transplant rejection, and cardiovascular, dermatological, inflammatory and respiratory disorders, among others.
Chengdu Chipscreen Pharmaceutical Ltd. has identified S-adenosylmethionine synthase isoform type-2 (Mat2A) inhibitors reported to be useful for the treatment of cancer.
Biohaven Therapeutics Ltd. has synthesized pyruvate kinase M2 (PKM2) activators reported to be useful for the treatment of age-related macular degeneration, amyotrophic lateral sclerosis, multiple sclerosis, retinitis pigmentosa and fibrosis.
Nikang Therapeutics Inc. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to a cyclin-dependent kinase 2 (CDK2)- and/or CDK4-targeting moiety through a linker reported to be useful for the treatment of cancer.
FGFR3 genomic alterations, including S249C as the most common, are recognized oncogenic drivers in 10%-60% of bladder cancers depending on the disease stage. Onco3r Therapeutics BV recently reported the identification of a novel series of highly potent, isoform-selective small-molecule FGFR3 inhibitors.
Triple-negative breast cancer (TNBC) is notoriously difficult to treat because it is quite aggressive and the tumors do not express the three major surface hormone receptors that can be targeted with available drugs. A potential target for treating this cancer may be glutathione peroxidase 4 (GPX4), which normally protects cells from ferroptosis, an iron-mediated form of cell death triggered by high oxidative stress.
Nectin-4 is a cell-adhesion molecule that is highly expressed in several malignancies, including bladder, colorectal, lung and breast cancers, while exhibiting minimal expression in most normal adult tissues.
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