With CRISPR-Cas9 technology making its way toward clinical practice, laboratories are studying different gene-editing techniques, from base editors to prime editors, to correct mutations associated with various pathologies. Researchers at Tessera Therapeutics Inc. have been inspired by retrotransposons to develop a tool for editing DNA using RNA and reverse diseases such as phenylketonuria (PKU) or sickle cell disease (SCD). Read More
Several developmental biology and regenerative medicine laboratories that use cellular reprogramming techniques presented their latest results on the differences in the states of induced pluripotent stem cells (iPSCs) during a Plenary Session on “Epigenetic regulation of distinct cell states” at the Annual Meeting of the International Society for Stem Cell Research (ISSCR), in Boston from June 14 to 17, 2023. Read More
Identification of the mechanisms of innate immune sensors is fundamental to the understanding of health and disease. The pattern recognition receptor (PRR) subfamily of nucleotide-binding oligomerization domain (NOD)-like receptors (NLRs) work by recognizing either pathogen-associated molecular patterns (PAMPs), endogenous damage-associated molecular patterns (DAMPs) or associated molecular... Read More
Replay Holdings LLC and The University of Texas MD Anderson Cancer Center have announced that the FDA has cleared the IND application for NY-ESO-1 TCR/IL-15 NK, an engineered T-cell receptor natural killer (TCR-NK) cell therapy for advanced synovial sarcoma and myxoid/round cell liposarcoma. Read More
Jiangsu Hengrui Medicine Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have patented proteolysis targeting chimeras (PROTACs) comprising cereblon (CRBN) ligands coupled to an androgen receptor targeting moiety via linker. Read More
Oryzon Genomics SA has presented preclinical efficacy data on ORY-4001, a selective histone deacetylase 6 (HDAC6) inhibitor that was recently nominated as a clinical development candidate for the treatment of certain neurological diseases such as Charcot-Marie-Tooth (CMT) disease, amyotrophic lateral sclerosis and others. Read More
Regulus Therapeutics Inc. has provided an update on its preclinical studies evaluating a library of oligonucleotides designed to inhibit miR-155 for the treatment of amyotrophic lateral sclerosis (ALS). Read More
Kallyope Inc. patent details new AMP-activated protein kinase (AMPK) activators reported to be useful for the treatment of type 2 diabetes, allergy, cancer, depression, nutrition disorders, obesity, psoriasis and ulcerative colitis, among others. Read More
Mutations in the CEP290 (NPHP6) gene cause severe cilia formation defects and a wide range of ciliopathies, ranging from non-syndromic Leber congenital amaurosis (LCA10) to Meckel syndrome (MKS). Researchers from Medetia SAS and affiliated organizations recently presented the discovery and preclinical evaluation of a novel prostaglandin-E2 receptor agonist, MDT-110, as a potential treatment of NPHP6/CEP290-associated phenotypes. Read More
Several Lario Therapeutics Ltd. patents describe new voltage-dependent R-type calcium channel subunit α-1E (Cav2.3) antagonists reported to be useful for the treatment of neurodegeneration, epilepsy, Parkinson’s disease, cerebral vasospasm, pain, CDKL5 deficiency, endocrine and neurodevelopmental disorders, among others. Read More
Ectonucleotide pyrophosphatase/phosphodiesterase family member 2 (ENPP2; autotaxin) inhibitors have been reported by Shanghai Jemincare Pharmaceuticals Co. Ltd. to be potentially useful for the treatment of cancer, fibrosis, inflammation, pain, neurodegeneration, autoimmune, metabolic and cardiovascular disorders, among others. Read More
Researchers from the Norwegian University of Science and Technology (NTNU) reported the discovery of novel colony-stimulating factor 1 receptor (CSF-1R) inhibitors as potential candidates for the treatment of osteoporosis. Read More
Interius Biotherapeutics Inc. has reported preclinical data demonstrating the potential of its lead program to generate biologically active chimeric antigen receptor (CAR) cells directly in vivo for the treatment of B-cell malignancies. Read More
