Although the causes of polycystic ovary syndrome (PCOS) are unclear, researchers know this condition leads to endometrial dysfunction in women who have hormonal imbalances, and insulin resistance. Now, a study led by scientists at the Karolinska Institutet in Sweden has revealed the cellular and genetic differences that distinguish this disorder in the first atlas of the human endometrium during the proliferative phase of the menstrual cycle. Read More
Biomedical research seems like it should be the ultimate bipartisan issue. But under the Trump administration, unless and until Congress regains its will to make use of its constitutional powers, bipartisan support for research seems to be a thing of the past. On March 3, members of the National Academies of Science, Engineering and Medicine warned that the second Trump administration has been waging a “wholesale assault” on American research. Read More
Variant SAS and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) have received positive feedback from the EMA for VAR-002, a recombinant AAV vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations. Read More
Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) will award Arrepath Inc. $3.7 million to execute a lead optimization workplan for its first-in-class antibiotic targeting a clinically novel target for the treatment of complicated urinary tract infections caused by multidrug-resistant Enterobacterales. Read More
Dual-specificity tyrosine phosphorylation-regulated kinase 1A (DYRK1A) is an attractive therapeutic target due to its involvement in cancer and neurodegenerative diseases. Researchers from the National Health Research Institutes and their collaborators have presented a series of DYRK1A inhibitors for reducing neurofibrillary tangle formation in Alzheimer’s disease. Read More
Spark Therapeutics Inc. has presented a proprietary adeno-associated viral (AAV) vector expressing an artificial miRNA targeting human α-synuclein (α-Syn) mRNA. Accumulation of misfolded and insoluble α-Syn causes neuronal toxicity in preclinical models and has been identified as the underlying cause of synucleinopathies. Read More
Sangamo Therapeutics Inc. has entered into a license agreement with Eli Lilly and Co., allowing Lilly to use Sangamo’s novel proprietary neurotropic AAV capsid, STAC-BBB, to deliver intravenously administered genomic medicines to treat certain diseases of the central nervous system. Read More
Resvita Bio Inc.’s RVB-003 has been awarded orphan drug designation by the FDA for the skin disorder Netherton syndrome. RVB-003 was previously granted rare pediatric disease designation. Read More
Moma Therapeutics Inc. has disclosed tricyclic derivatives acting as Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer. Read More
Innovstone Therapeutics Ltd. has synthesized leucine-rich repeat kinase 2 (LRRK2; Dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease. Read More
The detection of metastases in Merkel cell carcinoma (MCC) requires additional testing, such as pancytokeratin (panCK) or CK20, with no guarantee of success. Recent findings have identified transcription factor SOX11 and insulinoma-associated protein 1 (INSM1) as sensitive nuclear neuroendocrine markers, but their usefulness in MCC has not been proven. Read More
Amenis Bioscience Inc. has identified anthranilic acid derivatives acting as peptidyl-prolyl cis-trans isomerase NIMA-interacting 1 (PIN1) and viral replication inhibitors reported to be useful for the treatment of parasitic and viral infections, obesity, cancer, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), inflammatory bowel disease and pulmonary fibrosis. Read More
AT-rich interactive domain-containing protein 1A (ARID1A) encodes a switch/sucrose nonfermentable (SWI/SNF) complex and is mutated in around 10% of colorectal cancers. ARID1A deficiency damages DNA damage repair increasing tumor mutation burden in ovarian and gastrointestinal cancers. Read More
NRG Therapeutics Ltd. has described mitochondrial permeability transition (MPT) inhibitors reported to be useful for the treatment of aging, ischemia-reperfusion injury, inflammation, neurodegeneration and autoimmune, metabolic and renal disorders. Read More
Additional early-stage research and drug discovery news in brief, from: Auron Therapeutics, Capricor Therapeutics, Ena Respiratory, Hoth Therapeutics, Neurogene. Read More
