The first complete DNA methylation atlas of 39 human cell types reveals which genes can talk or are silenced, depending on whether their sequence is linked to methyl group epigenetic modification that regulates their expression. This map of on-and-off switches shows differences between the alleles inherited from the father and those from the mother, providing a view of gene expression that can be explored in health and disease independently of the information contained in the DNA sequence. Read More
Mosaic variegated aneuploidy syndrome (MVAS) is an autosomal recessive disorder characterized by mosaic aneuploidy. Its clinical manifestations include growth and developmental delay, congenital malformations and increased cancer risk. Genetic variants involved in MVAS affect the chromosomal segregation during mitosis, where individuals often show mosaicism and chromosomal instability. Read More
Enigma Biomedical USA Inc. has selected two four-repeat tau (4R tau) protein PET imaging biomarkers to advance into phase I studies. These imaging biomarkers show promise as important new tools in advancing understanding of neurodegenerative diseases in which the misfolded 4R tau protein is implicated. Read More
CEPI is partnering with Astrazeneca plc to advance VHH-based multispecific antibodies targeting influenza, while Harbour Biomed Ltd. has entered into a global strategic collaboration with Astrazeneca to discover and develop next-generation multispecific antibodies for immunology, oncology and other diseases. Read More
Marengo Therapeutics Inc. has entered into a multiyear research collaboration in oncology and autoimmune diseases with University of Pennsylvania Perelman School of Medicine. The research collaboration will provide deeper insights into the mechanism of action of Marengo’s selective dual T-cell agonists in oncology. Read More
CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials with SYS-6040 for advanced solid tumors. Read More
Charcot-Marie-Tooth disease type 2E (CMT2E) is a slow and progressive neuropathy characterized by axonal dysfunction. Its clinical phenotype includes muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others. Read More
Researchers from Abl Bio Inc. have published preclinical data for ABL-112, an Fc-competent bispecific antibody (BsAb) targeting TIGIT and 4-1BB (TIGIT x 4-1BB), being developed for the treatment of cancer. Read More
Researchers from Anew Therapeutics Pte Ltd. recently detailed the discovery of novel oral IL-17A small-molecule inhibitors as candidates for the treatment of psoriasis. Read More
Agios Pharmaceuticals Inc. has synthesized phenylalanine hydroxylase (PAH) R408W mutant stabilizers reported to be useful for the treatment of phenylketonuria. Read More
Several lung conditions, such as acute lung injury, require the targeted delivery of pharmacological agents to the lower lung. However, the administration of complex biologics, such as anti-inflammatory cytokine mRNA, to the injury site in the lower lungs is particularly challenging, frequently leading to poor specificity and uneven distribution. Read More
Eikon Therapeutics Inc. has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer. Read More
Anaplastic thyroid cancer (ATC) is a rare but aggressive form of thyroid cancer, accounting for nearly 50% of thyroid cancer-related deaths. Given its poor prognosis, the identification of new therapeutic strategies is a critical medical need. Molecular radiotherapy is an emerging field in oncology, and CD44v6, a cell surface antigen overexpressed in several cancers, has been proposed as a promising target for ATC treatment. Read More
Gasherbrum Bio Inc. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, schizophrenia, hypertension, obesity, stroke, myocardial infarction and gout, among others. Read More
