Entrada Therapeutics Inc. has received authorization from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) and research ethics committee to initiate a phase I/II study of ENTR-601-45 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 45 skipping.
Esophageal squamous cell carcinoma (ESCC) accounts for about 90% of esophageal cancer cases and is the sixth most common cause of cancer death worldwide. Targeting cancer stem cells offers a therapeutic approach for addressing cancer, concretely in those cases that are resistant to cisplatin.
Scientists at the Institute of Cancer Research (ICR) in the U.K. are developing a technology that analyzes, in vitro, how the 3D morphology of cancer cells changes when exposed to a compound, using AI to predict their response to new treatments. The researchers estimate that their methodology could accelerate drug development by 6 years, by ruling out unsuccessful drugs and thus reducing the number of preclinical trials.
Researchers from Anew Therapeutics Pte Ltd. recently detailed the discovery of novel oral IL-17A small-molecule inhibitors as candidates for the treatment of psoriasis.
Gasherbrum Bio Inc. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, schizophrenia, hypertension, obesity, stroke, myocardial infarction and gout, among others.
Eikon Therapeutics Inc. has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.
Agios Pharmaceuticals Inc. has synthesized phenylalanine hydroxylase (PAH) R408W mutant stabilizers reported to be useful for the treatment of phenylketonuria.
Anaplastic thyroid cancer (ATC) is a rare but aggressive form of thyroid cancer, accounting for nearly 50% of thyroid cancer-related deaths. Given its poor prognosis, the identification of new therapeutic strategies is a critical medical need. Molecular radiotherapy is an emerging field in oncology, and CD44v6, a cell surface antigen overexpressed in several cancers, has been proposed as a promising target for ATC treatment.
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