A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided. Read More
Glucocorticoid replacement therapy is the current standard of care for congenital adrenal hyperplasia (CAH). However, new therapeutic strategies that can better recapitulate physiological requirements and reduce morbidity and mortality among CAH patients are urgently needed. Despite the promise of gene therapy for correcting monogenic disorders, the strategies investigated to date have not yielded satisfactory results. Read More
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH). Read More
A paper from Onchilles Pharma Inc. and collaborating institutions details the development of N-17350, a next-generation therapeutic elastase optimized for intratumoral delivery that targets the neutrophil elastase pathway. Read More
Researchers from Compass Therapeutics Inc. detailed the preclinical characterization of CTX-10726, a bispecific, tetravalent antibody that simultaneously targets VEGF-A and PD-1. Read More
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. Read More
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. Read More
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. Read More
Chengdu Chipscreen Pharmaceutical Ltd. has identified S-adenosylmethionine synthase isoform type-2 (Mat2A) inhibitors reported to be useful for the treatment of cancer. Read More
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. Read More
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. Read More
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. Read More
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. Read More
