Pseudoxanthoma elasticum (PXE) is an autosomal recessive connective tissue disorder caused by pathogenic variants in ATP-binding cassette subfamily C, member 6 (ABCC6). ABCC6 typically exports ATP, which is then converted by ENPP1 into AMP and pyrophosphate (PPi). Because PPi is a potent inhibitor of calcification, reduced systemic PPi production is a key driver of PXE. University of Pennsylvania investigators and collaborators proposed applying liver-targeted variant correction via genome editing as a single-intervention therapeutic approach for PXE, leading to subsequent restoration of systemic PPi.

Opioid use disorder (OUD) remains a significant public health crisis, with over 5 million people diagnosed in the U.S. in 2023 and more than 80,000 fatal overdoses involving opioids each year. Current FDA-approved medications for OUD target the micro-opioid receptor, but these treatments are still associated with high relapse rates during abstinence.

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.

Scientists at St. John’s University, The University of Pennsylvania and Université de Montréal have identified poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors reported to be useful for the treatment of cancer, Lewy body dementia, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease.