Developing new gene therapies for autosomal dominant polycystic kidney disease (ADPKD) is still a challenge to date. A group of researchers from the Johns Hopkins Medicine in Baltimore has presented results from evaluation of a new gene therapy for treating ADPKD, AAV1-CBdelta27-264CFTR, where they focused on the surface receptors expressed in cystic epithelia.
Researchers from Tango Therapeutics Inc. presented preclinical efficacy data on TNG-260, an inhibitor of the co-repressor of repressor element-1 silencing transcription (CoREST) deacetylase complex, designed to treat immunotherapy-resistant STK11-mutant tumors.
During the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, scientists from Plexium Inc. presented the identification of a new class of potent, selective, cereblon (CRBN)-based molecular glue degraders of CDK2 using its proprietary monovalent degrader platform.
At the recent AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, researchers from Aurigene Oncology Ltd. presented the discovery and clinical characterization of novel macrocyclic KIF18A inhibitors.
Hutchmed (China) Ltd. has developed HMPL-A251, a novel HER2 antibody-drug conjugate (ADC) designed to deliver a potent PI3K/PIKK inhibitor directly to tumor cells.
Melanocortin-4-receptor MC4R is a crucial molecule involved in energy homeostasis, feeding behavior and metabolism, and loss-of-function mutations in this gene are the main monogenic cause of early-onset obesity. Congruence Therapeutics Inc. has developed a small molecule, CGX-926, that corrected MC4R deficiency and restored its cell-surface expression and function.
CIS Biopharma AG recently presented an antibody-drug conjugate (ADC) targeting the L1 cell adhesion molecule (L1CAM/CD171) as a potential candidate for the treatment of advanced tumors.
Pinotbio Inc. recently reported a novel antibody-drug conjugate (ADC), named PBX-004, consisting of an ITGB6-targeting antibody conjugated to a potent topoisomerase I (TOP1) inhibitor payload.
Researchers at 35pharma Inc. have recently presented data for the activin receptor inhibitor HS-235, which is under development for the treatment of obesity as it exerts body composition and metabolism control.
Gentibio Inc. has developed and presented data for GNTI-823, an allogeneic tissue Treg engineered from peripheral blood CD4+ T cells with CRISPR-based editing to induce FOXP3 expression, a rapamycin-inducible IL-2 signaling complex and an alarmin-responsive transgene.
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