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.
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).
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.
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.
Researchers at Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and Suzhou Vigonvita Life Sciences Co. Ltd. have described NMDA receptor antagonists and/or monoamine transporter inhibitors reported to be useful for the treatment of neurological disorders.
Shanghai Apeiron Biotechnology Co. Ltd. has divulged protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of brain metastatic cancer.
Université de Montréal has identified compounds acting as proto-oncogene tyrosine-protein kinase Src (SRC; SRC1) and/or tyrosine-protein kinase Yes (YES1) inhibitors reported to be useful for the treatment of cancer, fibrosis and liver diseases.
Nanjing Synnocare Pharmaceutical Technology Co. Ltd. has synthesized prodrugs of kinesin-like protein KIF18A inhibitors reported to be useful for the treatment of cancer.
Captor Therapeutics Inc. has disclosed molecular glue degraders comprising a cereblon (CRBN) binding moiety acting as serine/threonine-protein kinase Nek7 degradation inducers reported to be useful for the treatment of amyotrophic lateral sclerosis, Still’s disease, Behçet’s disease, familial Mediterranean fever, Alzheimer’s disease, pulmonary arterial hypertension and more.
Bakuchiol, a phenolic meroterpenoid from the seeds of Psoralea corylifolia L., has shown promise as an antitumor drug, so researchers at Bengbu Medical University synthesized novel derivatives of bakuchiol bearing modifications on the aromatic ring.
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