The largest genetic analysis of abdominal aortic aneurysm (AAA) carried out to date has identified almost 100 new risk variants linked to the disorder. The study also highlighted a possible therapeutic target for this pathology that, at the moment, has no treatment. AAA affects 4% of people over 65 years of age in the U.S. and causes 41,000 deaths per year. The incidence is three to four times higher in men than in women.
Rapt Therapeutics Inc. has described chemokine CCR4 receptor antagonists reported to be useful for the treatment of cancer, allergic asthma, contact dermatitis, allergic rhinitis, immunological, inflammatory, cardiovascular and metabolic disorders.
Vertex Pharmaceuticals Inc. has identified cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
Blueprint Medicines Corp. has synthesized EGFR (HER1; erbB1) inhibitors reported to be useful for the treatment of cancer, particularly non-small-cell lung cancer.
Shenzhen Maxinovel Pharmaceuticals Co. Ltd. has disclosed proteolysis targeting chimeras (PROTACs) comprising a cereblon E3 ubiquitin ligase binding moiety covalently bonded to a Wee1-like protein kinase (Wee1)-targeting moiety through a linker.
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y, for the treatment of methyl-CpG binding protein 2 (MeCP2) duplication syndrome (MDS).
Ibio Inc. has expanded its artificial intelligence (AI)-powered technology offering with the launch of Shieldtx, an antibody masking technology designed to enable specific, highly targeted antibody delivery to diseased tissue without harming healthy tissue.
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