Tumors with high levels of microsatellite instability (MSI-H), such as some subsets of colorectal, endometrial and gastric cancers, are associated with a hypermutated phenotype and a specific dependency on the WRN gene. Pharmacologic inhibition of WRN has been shown to selectively impair the viability of MSI-H cancer cells, effectively blocking tumor growth while sparing microsatellite-stable (MSS) cells.
About 15% of all cancers have co-deletion of both the MTAP and CDKN2A genes, which results in sensitization to MAT2A inhibitors, thus opening a therapeutic window in these cancer types. MAT2A inhibitors have demonstrated efficacy in MTAD-deficient cancers. Shouyao Holdings (Beijing) Co. Ltd. has developed and released data for their MAT2A inhibitor SY-9453 for the treatment of MTAP-deficient cancers.
Interleukin-4 receptor α (IL-4Rα), a shared receptor subunit for both IL-4 and IL-13, along with thymic stromal lymphopoietin (TSLP), are key drivers of the type 2 inflammatory cascade involved in asthma and other inflammatory disorders.
Avenzo Therapeutics Inc. has announced IND clearance by the FDA for AVZO-1418 (DB-1418), an EGFR/HER3 bispecific antibody-drug conjugate (ADC). A first-in-human phase I/II study will begin later this year and will evaluate AVZO-1418 as a single agent and in combination therapy in patients with advanced solid tumors.
Nearly half of the world’s population suffers from allergic rhinitis, meaning that when they inhale allergens from the air, IgE becomes activated and induces paroxysmal sneezing, runny nose and nasal itching. Several types of drugs are currently used to manage the condition, including antihistamines, corticosteroids and antagonists of the leukotriene receptor; but some are associated with side effects.
The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.
Chengdu Easton Biopharmaceuticals Co. Ltd. has described menin (MEN1)/KMT2A (MLL) interaction inhibitors reported to be useful for the treatment of cancer and diabetes.
Chiesi Farmaceutici SpA has divulged sodium channel protein type 9 subunit α (Nav1.7) channel blockers reported to be useful for the treatment of chronic cough.
Scientists at Bayer AG and Dana Farber Cancer Institute Inc. have identified EGFR (exon 20 insertion [Ex20Ins] mutant) and/or HER2 (erbB2) (Ex20Ins mutant) inhibitors reported to be useful for the treatment of lung cancer.
Scientists at Ascentage Pharma (Suzhou) Co. Ltd. and China Pharmaceutical University have synthesized molecular glue degrader compounds acting as eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1)/CRBN interaction inducers for GSPT1 degradation reported to be useful for the treatment of cancer, viral infections, aging, immunological disorders and neurological disorders.
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