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

Around the Helix: Cell and Gene Therapy Company Updates – June 12, 2024
Around the Helix: Cell and Gene Therapy Company Updates – June 12, 2024

June 12th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Taysha’s Rett Syndrome Gene Therapy Gets RMAT Designation
Taysha’s Rett Syndrome Gene Therapy Gets RMAT Designation

June 11th 2024

Ineka Whiteman, PhD, head of research and medical affairs, BDSRA Australia, consultant to BDSRA Foundation (USA) and Beyond Batten Disease foundation
Batten Disease Gene Therapy Continues to Make Progress Despite Setbacks

June 11th 2024

Data Roundup: February 2024 Features Updates in Blood Cancers, Lysosomal Storage Disorders at WorldSymposium, Tandem Meetings
Data Roundup: May 2024 Features Updates from ASGCT in Rare Neurological, Retinal Diseases, and more.

June 8th 2024

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – June 7, 2024

June 7th 2024

Video Interviews
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