Lysosomal Disorders

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CGTLive®’s Weekly Rewind
May 16th 2025
CGTLive®’s Weekly Rewind – May 16, 2025

Review top news and interview highlights from the week ending May 16, 2025.

Kiran Musunuru, MD, PhD, a physician-scientist and Barry J. Gertz Professor for translational research in the Perelman School of Medicine at the University of Pennsylvania and CHOP and Rebecca Ahrens-Nicklas, MD, PhD, a physician-scientist and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP
May 16th 2025

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on the Next Steps for Personalized Gene Editing

May 15th 2025

REGENXBIO’s BLA for MPS II Gene Therapy RGX-121 Accepted for Priority Review by the FDA

Kiran Musunuru, MD, PhD, a physician-scientist and Barry J. Gertz Professor for translational research in the Perelman School of Medicine at the University of Pennsylvania and CHOP and Rebecca Ahrens-Nicklas, MD, PhD, a physician-scientist and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP
May 15th 2025

Rebecca Ahrens-Nicklas, MD, PhD; and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on Treating CPS1 Deficiency With Personalized Gene Editing

May 14th 2025

Around the Helix: Cell and Gene Therapy Company Updates – May 14, 2025

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