Satellos Bioscience Inc. has received orphan drug designation and rare pediatric disease designation from the FDA for SAT-3153 for the potential treatment of Duchenne muscular dystrophy (DMD). The first-in-class oral small-molecule therapeutic is designed to restore the innate muscle regeneration process independent of dystrophin and regardless of exon mutation status.
To identify candidate therapeutic targets for cancers with SF3B1 hotspot mutations, drug-sensitivity screening of an in-house library of 80 small-molecule inhibitors resulted in the identification of a series of candidate SF3B1 mutant (SF3B1[MUT]) synthetic lethal drugs that led to significant reduction of survival in SF3B1(K700E) cells.
One of the main causes of cervical cancer is persistent human papillomavirus (HPV) infection, with E6 and E7 being the main oncogenic genes of HPV3. For this reason, targeting HPV proteins E6 and E7 is a promising therapeutic strategy for the treatment of this disease.
Peptidream Inc. and Modulus Discovery Inc. have nominated the first clinical development candidate arising from the companies’ strategic drug discovery partnership.
Lawrence Livermore National Laboratory, Leidos Biomedical Research Inc. and Theras Inc. have jointly patented GTPase KRAS (G12C mutant) inhibitors reported to be useful for the treatment of cancer.
A Design Therapeutics Inc. patent details conjugates consisting of DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence linked to protein binding moiety through oligomeric backbone linker.
Research at Blossomhill Therapeutics Inc. has led to the discovery of new macrocyclic compounds acting as tyrosine kinase inhibitors and reported to be useful for the treatment of autoimmune disease.
Beijing Scitech-MQ Pharmaceuticals Ltd. described receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitors reported to be useful for the treatment of cancer.
China Pharmaceutical University and Shanghai Institute of Materia Medica of the Chinese Academy of Sciences have patented benzimidazole compounds acting as ectonucleotide pyrophosphatase/phosphodiesterase family member 1 (ENPP1) inhibitors reported to be useful for the treatment of cancer, infections, autoimmune disease and inflammatory disorders.
Researchers from Interius Biotherapeutics Inc. presented the development and preclinical evaluation of a novel gene therapy candidate, INT-2104, as potential candidate for the treatment of B-cell malignancies.
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