Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.” Read More
Another brick in the ambitious Human Cell Atlas initiative has been put into place with the publication of the largest and most comprehensive cell map of the human lung. The open and freely available atlas catalogs the diversity of cells in the lung, including rare and previously undescribed cell types. Read More
Insilico Medicine IP Ltd. has identified small-molecule inhibitors of ubiquitin carboxyl-terminal hydrolase 1 (USP1) reported to be useful for the treatment of cancer. Read More
A group of scientists from the Center for Stem Cell and Translational Immunotherapy, Brigham and Women’s Hospital at Harvard Medical School have developed an antitumor immunotherapy that uses oncolytic viruses and stem cells for the treatment of metastatic brain melanoma. Read More
Son of sevenless 1 (SOS1) is a guanine exchange factor (GEF) primarily responsible for linking cell-surface receptors to RAS protein activation converting the inactive form of GDP-loaded RAS proteins into the active GTP-loaded RAS. This role together with its function in inhibiting MAPK pathway reactivation suggest that SOS1 may be a therapeutic target to treat KRAS-driven cancers. Read More
Shanghai Institute of Materia Medica of the Chinese Academy of Sciences has divulged aromatic heterocyclic compounds acting as lysine-specific histone demethylase 1A (KDM1A; LSD1) inhibitors reported to be useful for the treatment of cancer. Read More
Hangzhou Glubio Therapeutics Co. Ltd. has described proteolysis targeting chimera (PROTAC) compounds comprising E3 ubiquitin ligase protein binding moieties covalently linked to Wee1-like protein kinase (Wee1) binding moieties. Read More
Researchers from Eutilex Co. Ltd. have presented preclinical data for the novel V-set and immunoglobulin domain-containing 4 (VSIG4)-specific humanized monoclonal antibody, EU-103, being developed as a potential cancer immunotherapy candidate. Read More
