Tag: Biochemical Pharmacology

Regulation of Mutant Huntingtin Mitochondrial Toxicity by Phosphomimetic Mutations within Its N-Terminal Region

Huntington’s disease (HD), a neurodegenerative disease, affects approximately 30,000 people in the United States, with 200,000 more at risk. Mitochondrial dysfunction caused by mutant huntingtin (mHTT) drives early HD pathophysiology. mHTT binds the translocase of the mitochondrial inner membrane (TIM23) complex, inhibiting mitochondrial protein import and altering the mitochondrial proteome. The 17 aa HTT N-terminal sequence… Continue reading Regulation of Mutant Huntingtin Mitochondrial Toxicity by Phosphomimetic Mutations within Its N-Terminal Region

A phase 1 dose‐escalation study of LY3295668 erbumine as monotherapy and in combination with topotecan and cyclophosphamide in children with relapsed/refractory neuroblastoma

This phase 1 study demonstrated that LY3295668 erbumine, a highly selective, orally bioavailable, and reversible Aurora kinase A inhibitor, both as monotherapy and in combination with standard chemotherapy in children with relapsed/refractory neuroblastoma, had a manageable safety profile. Despite robust preclinical data, relatively modest clinical activity (two partial responses and two minor responses with combination… Continue reading A phase 1 dose‐escalation study of LY3295668 erbumine as monotherapy and in combination with topotecan and cyclophosphamide in children with relapsed/refractory neuroblastoma

A phase 1 dose‐escalation study of LY3295668 erbumine as monotherapy and in combination with topotecan and cyclophosphamide in children with relapsed/refractory neuroblastoma

This phase 1 study demonstrated that LY3295668 erbumine, a highly selective, orally bioavailable, and reversible Aurora kinase A inhibitor, both as monotherapy and in combination with standard chemotherapy in children with relapsed/refractory neuroblastoma, had a manageable safety profile. Despite robust preclinical data, relatively modest clinical activity (two partial responses and two minor responses with combination… Continue reading A phase 1 dose‐escalation study of LY3295668 erbumine as monotherapy and in combination with topotecan and cyclophosphamide in children with relapsed/refractory neuroblastoma

Intermediate-affinity CD19-directed CAR T cell product obecabtagene autoleucel demonstrates favourable safety and efficacy in R/R B-ALL

Recent data from the FELIX trial evaluating obecabtagene autoleucel in patients with relapsed and/or refractory B acute lymphoblastic leukaemia (R/R B-ALL) suggest that this novel intermediate-affinity CD19-directed chimeric antigen receptor (CAR) T cell therapy is associated with a reduced incidence of severe immune-mediated toxicities compared with other commercially available CAR T cell products. The increasing… Continue reading Intermediate-affinity CD19-directed CAR T cell product obecabtagene autoleucel demonstrates favourable safety and efficacy in R/R B-ALL