Innovstone Therapeutics Ltd. has synthesized leucine-rich repeat kinase 2 (LRRK2; Dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
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.
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.
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.
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.
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.
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.
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.
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.
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.