Passage Bio’s first attempt to develop a GM1 gangliosidosis gene therapy treatment ran into a roadblock in the form of a clinical hold.
Researchers at the University of Pennsylvania School of Medicine found that certain changes in DNA sequences can trigger the resulting genome to misfold in such a way that the person is at increased risk for Type 1 diabetes.
Researchers with the Abramson Cancer Center of the University of Pennsylvania, led by Carl June, published results from the first U.S. Phase I trial of CRISPR-Cas9-edited T-cells in humans with advanced cancer.
Eagle Pharmaceuticals Inc. and the University of Pennsylvania agreed on terms of a new exclusive worldwide license agreement for the development of dantrolene sodium for the potential treatment of people living with Alzheimer’s disease.
Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.