Welcome
We are a CHOP-based lab in Philadelphia working to develop therapies for Inborn Errors of Metabolism through the lens of genetics, biochemistry, and electrophysiology
Dr. Kiran Musunuru and Dr. Rebecca Ahrens-Nicklas with KJ, the patient featured in our recent publication!
In February, Vi Pham defended her PhD in the Cell and Molecular Biology Graduate Group (Gene Therapy and Vaccines Program) at the University of Pennsylvania, Perelman School of Medicine. For her thesis work, she generated novel iPSC-based models and ex vivo lentiviral gene strategies for the treatment of patients with an ultra-rare, neuronopathic lysosomal storage disorder called Multiple Sulfatase Deficiency.
Vi is currently a post-doctoral fellow in the labs of Kiran Musunuru (UPenn) and Rebecca Ahrens-Nicklas (CHOP) overseeing the preclinical development of their lead gene editing platforms for a variety of inborn of errors of metabolism (IEMs). She is screening novel editing strategies for target IEMs as well as completing the IND-enabling studies (i.e. dose-range finding, pharmacology, toxicology, and biodistribution) required to advance the leading programs to first-in-human trials.
Vi is currently seeking her next opportunity to continue developing novel gene therapies in a biotech/pharma industry setting. Please feel free to reach out if you have any relevant opportunities!