Research led by St. Jude Children’s Research Hospital and Harvard University shows base-editing approaches could be more effective than CRISPR-Cas9 gene-editing approaches for treating conditions such as sickle cell disease and β-thalassemia. Writing in the July 3, 2023, issue of Nature Genetics, the researchers compared three base-editing approaches with two CRISPR-Cas9 approaches to increasing levels of fetal hemoglobin in CD34+ hematopoietic stem and progenitor cells, and found one of the base-editing approaches was the most potent.
Yingxi Intelligent Technology (Shanghai) Co. Ltd. has identified substituted monocyclic or bicyclic heterocyclic compounds acting as fibroblast growth factor receptor (FGFR) inhibitors and reported to be useful for the treatment of asthma, cancer, chronic heart failure, chronic obstructive pulmonary disease, rheumatoid arthritis, inflammation, autoimmune and Parkinson’s disease, among others.
Cannabidiol derivatives potentially useful for the treatment of Parkinson’s disease and epilepsy have been described in a Deyi Pharmaceutical Co. Ltd. patent.
Suzhou Puhe Biopharma Co. Ltd. researchers have prepared and tested substituted bicyclic heteroaryl compounds acting as GTPase KRAS (G12D mutant) inhibitors potentially useful for the treatment of cancer.
Research at Vrise Therapeutics Inc. has led to the development of 5,6,7,8-tetrahydro-2,6-naphthyridine derivatives acting as GTPase KRAS (G12D mutant) inhibitors and thus reported to be useful for the treatment of cancer.
Helmholtz Zentrum Dresden Rossendorf has patented 3-((3-([1,1’-biphenyl]-3-ylmethoxy)phenoxy)methyl)benzonitrile derivatives that are radiolabeled compounds targeting programmed cell death 1 ligand 1 (PD-L1; CD274). They are reported to be potentially useful for the diagnosis and/or radionuclide therapy treatment of cancer and SARS-CoV-2.
Researchers from the Mayo Clinic have reported findings from the preclinical evaluation of a novel cardiac targeting peptide (CTP)-based radiotracer – [68Ga]NOTA-CTP – being developed as a myocardial perfusion imaging PET probe.
B7-H4 is an immune-suppressive protein overexpressed in several tumors, including breast, endometrial and ovarian cancers, that has been successfully targeted with antibody-drug conjugate (ADC) strategies in preclinical testing.
Hillstream Biopharma Inc. has signed an exclusive agreement with Applied Biomedical Science Institute (ABSI) to license technology for human antibodies targeting novel HER2 and HER3 conformational epitopes. The goal is to develop proprietary multiformat biologics, including bispecific antibodies and antibody-drug conjugates (ADCs).
Aicuris Anti-infective Cures AG has announced its intention to focus on the development and commercialization of anti-infectives for immunocompromised individuals, and will discontinue research activities outside of this strategic scope.
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