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 – February 14, 2025

February 14th 2025

Review top news and interview highlights from the week ending February 14, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – February 12, 2025
Around the Helix: Cell and Gene Therapy Company Updates – February 12, 2025

February 12th 2025

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – February 7, 2025

February 7th 2025

Robert Hopkin, MD
Updated Data Suggests Sangamo’s Gene Therapy ST-920 Improves Renal Function in Fabry Disease

February 6th 2025

Eric Crombez, MD, the chief medical officer at Ultragenyx
Ultragenyx’s Gene Therapy UX111 Improves Clinical Function in MPSIII

February 6th 2025

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February 27 - March 2, 2025

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