University of Pennsylvania (Penn)

Passage Bio’s Gene Therapy for GM1 Gangliosidosis Hit with Clinical Hold

Adeno-Associated Virus (AAV), Clinical Hold, FDA, FDA/Regulatory, Gene Therapy, GM1 gangliosidosis (GM1), R&D, Rare Pediatric Disease Priority Review Voucher, University of Pennsylvania (Penn)

Passage Bio’s first attempt to develop a GM1 gangliosidosis gene therapy treatment ran into a roadblock in the form of a clinical hold.

August 13, 2020/by Andrew Humphreys

Research Roundup: Misfolding DNA and Type 1 Diabetes and More

DNA, Genes, Immunity, R&D, Research, Type 1 Diabetes, University of Pennsylvania School of Medicine

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.

February 21, 2020/by BioSpace

First Proof CRISPR Can Be Safe in Cancer Therapy

Abramson Cancer Center of the University of Pennsylvania, Advanced Cancer, Cancer Cells, Cancer Therapies, CAR-T Therapy, Clinical Trials, CRISPR/Cas9, Human Genome, R&D, Researchers, T-Cells, UC Berkeley, University of Pennsylvania (Penn)

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.

February 7, 2020/by BioSpace

Eagle Pharmaceuticals to Develop Dantrolene Sodium for Potential Treatment of Alzheimer’s Disease in Collaboration With University of Pennsylvania

Alzheimer's Association International Conference, Alzheimer’s, Business, Dementia, Licensing, Nervous System, University of Pennsylvania (Penn), World Health Organization

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.

January 13, 2020/by Business Wire

Amicus and Penn Broaden Gene Therapy Collaboration

Adeno-Associated Virus (AAV), Angelman Syndrome, Business, CDKL5 deficiency disorder (CDD), Collaborations, Fabry disease, Gene Therapy, Gene Transfer Technologies, Inherited Lysosomal Storage Disorders, Muscular Dystrophy, Mycopolysaccharidosis Type IIIA (MPS IIIA), Mycopolysaccharidosis Type IIIB (MPS IIIB), Myotonic Dystrophy, Niemann-Pick Type C (NPC), Pompe disease, Rett Syndrome, University of Pennsylvania (Penn)

Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.

May 29, 2019/by BioSpace