The misassembly of transthyretin (TTR) into toxic amyloid aggregates leads to a variety of degenerative disorders known as TTR amyloidosis (ATTR). Researchers from the Universitat Autònoma de Barcelona and collaborators recently reported on the design and preclinical characterization of PITB, a TTR-selective kinetic stabilizer with potential for the treatment of ATTR.
REGN-4461 (mibavademab) is a monoclonal antibody targeting leptin receptor (LEP-R) under development by Regeneron Pharmaceuticals Inc. It acts as an allosteric agonist and does not compete with leptin binding to LEP-R.
A laboratory technique used to generate pluripotent stem cells from any tissue, cellular reprogramming, has led a group of researchers to the discovery of a process that could have an impact on natural tissue repair.
5’-AMP-activated protein kinase (AMPK) is a serine/threonine protein kinase that regulates energy levels by sensing cellular fuel status and it is highly expressed in metabolic tissues, such as adipose tissue, where it is activated upon energy deprivation or cellular stress stimuli, among others.
After an initial chemical screening, researchers at the University of Pennsylvania and Weill Cornell Medicine identified the Chk1/Chk2 dual inhibitor AZD-7762 among the strongest to induce insulin secretion in various assays.
Freeline Therapeutics plc is being taken private by its founding investor and majority shareholder Syncona Ltd, in an all-cash transaction that values the Nasdaq-listed gene therapy specialist at $28.3 million. Syncona also will provide up to $15 million cash to keep Freeline afloat as it awaits the next tranche of data on its lead program FLT-201, which is positioned as a one-off treatment for the lysosomal storage disorder, Gaucher’s disease.
Lysophosphatidylcholine acyltransferase 3 (LPCAT3), highly expressed in the liver, intestine, and adipose tissues, is an enzyme that preferentially incorporates polyunsaturated fatty acyl chain into lysophospholipids. Previous work showed that LPCAT3 and phospholipid remodeling play a crucial role in regulating glucose metabolism and contribute to the development of insulin resistance in type 2 diabetes.
The Swedish Medical Products Agency has authorized Uppsala University's clinical trial application to initiate an investigator-sponsored, first-in-human study of UP-421, an allogeneic, primary islet cell therapy engineered with Sana Biotechnology Inc.'s hypoimmune technology, in patients with type 1 diabetes.