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.
Four Chinese biopharmaceuticals unveiled early data on respective cancer therapies at the American Society of Hematology (ASH) 2024 meet in San Diego, including Cstone Pharmaceuticals Co. Ltd.’s receptor tyrosine kinase-like orphan receptor 1 (ROR1)-inhibiting antibody-drug conjugate (ADC) in phase I study for lymphomas.
At the 66th American Society of Hematology Annual Meeting, a plethora of companies presented clinical trial data highlighting their drugs targeting Bruton tyrosine kinase (BTK) in patients with blood cancers.
Researchers from Excellamol Inc. presented the design and preclinical characterization of XM161-SN38, a novel IL-13Rα2-targeting polypeptide-drug conjugate being evaluated for the treatment of gliomas.
With the aim of developing an effective and more tolerable conditioning for hematopoietic stem cell transplantation (HSCT), researchers from Molecular Partners AG applied the proprietary ankyrin repeat protein (DARPin) platform to generate a novel multi-specific Switch-DARPin candidate, MP-0621.
Researchers from Bright Minds Biosciences Inc. presented data on the mechanism of action of BMB-101, a novel scaffold 5-HT2C Gq-protein biased designed to treat drug-resistant neurological disorders.
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