Humans love to think of our species as unique. But on a genetic level, such uniqueness is surprisingly hard to find. And while that may be a blow to the ego, it also means that an evolutionary lens is one way to search for insights into human diseases. Animals are “adapted to use the same genes that you and I have, but in very different ways,” Ashley Zehnder told BioWorld. Zehnder is co-founder and CEO of Fauna Bio Inc., which uses comparative genomics to identify gene networks that underlie disease resistance in different animal species.
Kupffer cells are macrophages located in the liver that play a key role in liver pathology, concretely in metabolic dysfunction-associated steatotic liver disease (MASLD; formerly nonalcoholic fatty liver disease or NAFLD), but the mechanisms behind this are poorly understood.
Neuralace Medical Inc. tied up a second U.S. FDA clearance for its Axon therapy, adding painful diabetic neuropathy (PDN) to the chronic nerve pain indication it received in 2021. An ‘electroceutical’ device, Axon employs non-invasive magnetic peripheral nerve stimulation (mPNS) to provide relief without leads, injections or implants.
Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of MZ-101 as a potential candidate for the treatment of Pompe disease and other glycogen storage disorders.
Kissei Pharmaceutical Co. Ltd. has described 3,4-dihydroquinolin-2(1H)-one compounds acting as thyroid-stimulating hormone (thyrotropin) receptor (TSHR) antagonists reported to be useful for the treatment of hyperthyroidism, Graves’ disease and thyroid-associated ophthalmopathy.
Novel nicotinamide phosphoribosyltransferase (NAMPT) positive allosteric modulators (N-PAMs) have been discovered and evaluated by University of Illinois and University of Arizona investigators.
Many patients with cancer in advanced stages develop cachexia, a metabolic disorder characterized by systemic weight loss and muscle and adipose tissue wasting.
The EMA has awarded orphan drug designation to GC Biopharma Corp.’s intracerebroventricular enzyme replacement therapy (ERT) candidate, GC-1130A, for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A), developed in collaboration with Novel Pharma Inc.
Using interactions between viral peptides and human proteins as a starting point, researchers from Enyo Pharma Inc., the University of Lyon and other institutions were able to bootstrap themselves to a mitochondria-targeting small molecule that showed activity in a mouse model of nonalcoholic steatohepatitis (NASH) with chronic kidney disease.
Korro Bio Inc. has highlighted new data for KRRO-110, its first development candidate for the potential treatment of α-1 antitrypsin deficiency (AATD), an inherited genetic disorder caused by single nucleotide variants in the SERPINA1 gene.
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