To address the existing challenges with CAR T-cell therapy, scientists at Umoja Biopharma Inc. developed the Vivovec platform, which is an off-the-shelf surface-engineered lentiviral vector (LVV) drug product designed to generate CAR T cells in vivo without lymphodepletion by selectively binding, activating and transducing T cells.
Researchers from State University of New Jersey (Rutgers) presented preclinical data for PW-507, a sigma-1 receptor antagonist being evaluated for the treatment of binge eating disorder.
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
Gynecological cancers with amplifications in the CCNE1 gene, which encodes cyclin E1, usually exhibit resistance to standard therapies. Since cyclin-dependent kinase 2 (CDK2) is the primary partner of cyclin E, CDK2 inhibitors represent a potentially effective treatment strategy for these malignancies.
In a presentation at the recent American Society of Hematology meeting in San Diego, Kite Pharma Inc. reported preclinical data for KITE-753, an autologous rapid manufactured anti-CD19/CD20 CAR T-cell therapy being developed for the treatment of B-cell malignancies.
Kura Oncology, Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor ziftomenib showed encouraging data across multiple studies, the most encouraging of which were in combination with other standard of care therapies in patients with NPM1-mutant and KMT2A-rearranged acute myeloid leukemia.
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.
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.
Researchers from Maplight Therapeutics Inc. presented preclinical data for the investigational muscarinic M1/M4 receptor agonist ML-007, being evaluated for the treatment of neuropsychiatric disorders, such as schizophrenia and Alzheimer’s disease (AD). The activity of ML-007 was compared to that of another muscarinic M1/M4 agonist, xanomeline.
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