Scientists at Daegu-Gyeongbuk Medical Innovation Foundation and Korea Research Institute of Chemical Technology have synthesized phenaleno-isoquinolinium-based compounds reported to be useful for the treatment of cancer.
Epileptic encephalopathy due to mutations in the STXBP1 gene, also known as genetic epilepsy, is a rare disease characterized by intellectual disability, speech and motor impairment and behavioral issues, among others, that affects 1 in every 30,000 newborns and which has no approved therapies to date.
At the recently concluded American Society of Hematology meeting, F Hoffmann-La Roche Ltd. presented promising results for their next-generation coagulation factor VIIIa (FVIIIa) mimetic bispecific antibody, NXT-007 (RO-7589655), supporting the ongoing phase I/II studies.
Astria Therapeutics Inc. has gained IND clearance from the FDA for STAR-0310, a monoclonal antibody OX40 antagonist being developed for atopic dermatitis.
Work at the Institute of Materia Medica of Beijing has led to the discovery and preclinical characterization of novel fructose-1,6-bisphosphatase (FBPase) inhibitors as potential therapeutic candidates for the treatment of type 2 diabetes.
Nextcure Inc. has reported IND clearance from the FDA enabling initiation of a phase I trial of LNCB-74, a B7-H4-targeting antibody-drug conjugate (ADC) being developed for various cancers.
The effective targeting of RAS-mutant acute myeloid leukemia (AML) still remains a challenge; RAS mutations are tied to relapse to targeted therapy, such as resistance to FLT3 inhibitors due to the RAS/MAPK pathway, for example.