Artelo Biosciences Inc. has presented data on its fatty acid-binding protein 5 (FABP5) inhibitor ART-26.12 from testing of its efficacy in preclinical models of psoriasis.
The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.
Scientists from 4D Molecular Therapeutics Inc. disclosed the preclinical evaluation of 4D-710, an aerosolized gene therapy that consists of a lung-specific evolved A101 capsid vector, the promoter CMV173 and the transgene codon-optimized human CFTRΔR.
Italian researchers have presented a case study of pediatric advanced sleep-wake phase syndrome in a 4-year-old girl. Autosomal dominant forms of this syndrome due to loss-of-function mutations in the gene coding casein kinase I isoform delta (CSNK1D) have previously been described.
In the year’s fourth-largest deal, Prime Medicine Inc. will collaborate with Bristol Myers Squibb Co. in a research collaboration and license agreement totaling $3.61 billion. The two companies plan to develop reagents for ex vivo T-cell therapies. While the programs and targets have yet to be disclosed, BMS is expanding its CAR T development, begun more than five years ago, with this deal.
Petragen Inc. has described ectonucleotide pyrophosphatase/phosphodiesterase family member 1 (ENPP1) inhibitors reported to be useful for the treatment of cancer, gingivitis, musculoskeletal and connective disorders, hematological diseases, bone, cardiovascular, immunological and neurological disorders, among others.
Synnovation Therapeutics Inc. has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.
Uereka Biosciences Inc. has synthesized mitogen-activated protein kinase kinase kinase 13 (MAP3K13; LZK) inhibitors reported to be useful for the treatment of cancer.
Tohoku University has disclosed 3C-like proteinase (3CLpro; Mpro; nsp5) (SARS-CoV-2; COVID-19 virus) inhibitors reported to be useful for the treatment of viral infections and inflammatory disorders.
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