Editas Medicine Inc. has achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.
At the ongoing European Society of Gene & Cell Therapy meeting in Rome, Aviadobio Ltd. presented preclinical data for a novel ATXN2-targeting miRNA-containing vector, AVB-205, developed based on previous research that has shown the potential of ATXN2 silencing as a promising therapeutic strategy for amyotrophic lateral sclerosis (ALS) and tau-negative frontotemporal dementia (FTD).
Rectify Pharmaceuticals Inc. has nominated the positive functional modulator (PFM) RTY-694 as a development candidate for primary sclerosing cholangitis (PSC). As the lead PFM in Rectify’s hepatobiliary program, RTY-694 is now advancing to first-in-human trials for PSC.
Researchers from the University of Maryland in collaboration with the National Institutes of Health (NIH) and Duke University have identified angiopoietin-2 (Ang2) as a targetable protein to reverse cardiovascular dysfunction in Hutchinson-Gilford progeria syndrome (HGPS).
Evotec International GmbH and Nosopharm SAS have jointly described odilorhabdin analogues reported to be useful for the treatment of multidrug-resistant bacterial infections.
A patent from Nanjing Mingde New Drug Research Co. Ltd. and Zhangzhou Pientzehuang Pharmaceutical Co. Ltd. details the identification of salts of interleukin-1 receptor-associated kinase 4 (IRAK-4) degradation inducers reported to be useful for the treatment of diffuse large B-cell lymphoma.
Work at LG Chem Ltd. has led to the development of novel benzimidazolone derivatives acting as ectonucleotide pyrophosphatase/phosphodiesterase family member 2 (ENPP2; autotaxin) inhibitors potentially useful for the treatment of cancer and fibrosis.
Shanghai Fosun Pharmaceutical (Group) Co. Ltd. has patented new tricyclic compounds acting as serine/threonine-protein kinase ATR inhibitors and thus reported to be useful for the treatment of colon cancer.
RSS
