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

Heather Lau, MD, MS
Ultragenyx’s MPSIIIA Gene Therapy UX111 Met With CRL

July 14th 2025

According to Ultragenyx, the CRL relates to a need for additional CMC information and improvements and observations from inspections of manufacturing facilities.

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – July 11, 2025

July 11th 2025

Around the Helix: Cell and Gene Therapy Company Updates – July 9, 2025
Around the Helix: Cell and Gene Therapy Company Updates – July 9, 2025

July 9th 2025

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – July 4, 2025

July 4th 2025

Around the Helix: Cell and Gene Therapy Company Updates – July 2, 2025
Around the Helix: Cell and Gene Therapy Company Updates – July 2, 2025

July 2nd 2025

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