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. Read More
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. Read More
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. Read More
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. Read More
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. Read More
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. Read More
Tissue factor (TF), a transmembrane protein overexpressed in tumors such as cervical, head and neck, NSCLC, and pancreatic cancers, initiates the extrinsic coagulation pathway under normal subendothelial expression. TF-targeted vedotin antibody-drug conjugates (ADCs) are in clinical evaluation for solid tumors, but toxicities, including peripheral neuropathy, ocular effects and epistaxis, limit their use. Read More
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has synthesized poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors reported to be useful for the treatment of cancer. Read More
Arcus Biosciences Inc. has identified mast/stem cell growth factor receptor Kit (KIT; c-Kit; CD117) inhibitors reported to be useful for the treatment of cancer, allergy, autoimmune, inflammatory, cardiovascular, metabolic, dermatological and respiratory disorders, among others. Read More
Rheumatoid arthritis, atopic dermatitis, ulcerative colitis and several other inflammatory diseases involve hyperactivation of JAK-STAT signaling, in which the kinase JAK binds and phosphorylates the transcription factor STAT, which then turns on gene expression. Read More
Chengdu Zeling Biomedical Technology Co. Ltd. has divulged receptor-interacting serine/threonine-protein kinase 2 (RIPK2; RIP-2) inhibitors reported to be useful for the treatment of autoimmune diseases. Read More
Scientists at Jiangsu Hengrui Medicine Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have described GTPase KRAS (G12D mutant) and/or KRAS (G12V mutant) inhibitors reported to be useful for the treatment of cancer. Read More
Major histocompatibility complex class I-related protein A and B (MICA and MICB) are ligands for the natural killer group 2 member D (NKG2D) receptor and are widely expressed on tumor cells, with very limited expression on normal tissues. Read More
Incyte Corp. has disclosed GTPase KRAS (mutant) inhibitors reported to be useful for the treatment of cancer, as well as autoimmune and inflammatory disorders. Read More
Additional early-stage research and drug discovery news in brief, from: Connect Biopharma, Gri Bio, Marker Therapeutics, Pasithea Therapeutics, Rein Therapeutics. Read More
