Lysosomal Disorders

The CGTLive Lysosomal Disorders specialty topic page contains video interviews with key opinion leaders discussing the most relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for Lysosomal storage diseases. It also houses the most up-to-date clinical news coverage in the field of gene and cell therapy for diseases related to lysosomal storage and dysfunction.

Latest News

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – September 6, 2024

September 6th 2024

Review top news and interview highlights from the week ending September 6, 2024.

Around the Helix: Cell and Gene Therapy Company Updates – September 4, 2024
Around the Helix: Cell and Gene Therapy Company Updates – September 4, 2024

September 4th 2024

Paul Harmatz, MD
REGENXBIO’s MPSII Gene Therapy RGX-121 Continues to Show Efficacy in Long-Term Trial Data

September 4th 2024

FDA Activity Recap: August 2024 Features TCR T-Cell Therapy Approval, REMS Modification, the Lifting of a Clinical Hold, and More
FDA Activity Recap: August 2024 Features TCR T-Cell Therapy Approval, REMS Modification, the Lifting of a Clinical Hold, and More

September 4th 2024

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – August 30, 2024

August 30th 2024

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6th Annual Precision Medicine Symposium: An Illustrated Tumor Board

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9th Annual Live Medical Crossfire®: Hematologic Malignancies

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