Dr. Jim Wilson, Director of the Gene Therapy Program at the University of Pennsylvania, recently delivered a virtual lecture as a part of the University of the Sciences in Philadelphia's Bicentennial Symposium. Dr. Wilson's presentation, “The Potential of Gene Therapy for Human Monogenic Diseases," is available to view on-demand.
iECURE, a mutation-agnostic in vivo gene editing company striving to cure devastating diseases with high unmet need, announced it has closed a $50 million Series A financing led by Versant Ventures and OrbiMed Advisors. The funds will be used to advance iECURE’s pipeline of up to 13 programs, all of which benefit from gene editing and in vivo delivery approaches being developed in the laboratory of James Wilson, M.D., Ph.D., of the University of Pennsylvania Gene Therapy Program (GTP).
Moderna, Inc. and the Institute for Life Changing Medicines announced a new collaboration to develop a new mRNA therapeutic for Crigler-Najjar Syndrome Type 1, an ultra-rare disease. The goal of the collaboration is to make an mRNA therapy for the treatment of CN-1 available at no cost to patients.
Dr. Jim Wilson and Alex Karnal announced the launch of The Institute for Life Changing Medicines, a nonprofit organization with a mission of identifying, developing and promoting access to therapies that have transformative potential but are not considered commercially viable because they are designed to treat small populations of patients suffering from rare diseases.
The SARS-CoV-2 pandemic has affected more than 185 million people worldwide resulting in over 4 million deaths. To contain the pandemic, there is a continued need for safe vaccines that provide durable protection at low and scalable doses and are easily delivered. Here, AAVCOVID-1, an adeno-associated viral (AAV), Spike gene-based vaccine candidate demonstrates potent immunogenicity in mouse and nonhuman primates following a single injection and confers complete protection from SARS-CoV-2 challenge in macaques.
The Gene Therapy Program at the University of Pennsylvania's expanded strategic collaboration and license agreement with Passage Bio establishes new research programs to discover and develop new genetic medicines for Alzheimer’s Disease and Temporal Lobe Epilepsy as well as additional large CNS indications upon mutual agreement. These new programs include a collaborative initial discovery phase focused on identifying relevant biological targets for these two diseases.
