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 – August 14, 2025

By Noah Stansfield

August 15th 2025

Review top news and interview highlights from the week ending August 15, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – August 13, 2025

Around the Helix: Cell and Gene Therapy Company Updates – August 13, 2025

By Noah Stansfield

August 13th 2025

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – August 8, 2025

By Noah Stansfield

August 8th 2025

Around the Helix: Cell and Gene Therapy Company Updates – August 6, 2025

Around the Helix: Cell and Gene Therapy Company Updates – August 6, 2025

By Noah Stansfield

August 6th 2025

FDA Activity Recap: July 2025 Features Multiple CRLs, RMAT Designation, and More

FDA Activity Recap: July 2025 Features Multiple CRLs, RMAT Designation, and More

By Noah Stansfield

August 1st 2025

Video Interviews

Podcasts

ImmunoLogic Episode 5

ImmunoLogic Episode 4

ImmunoLogic

ImmunoLogic

ImmunoLogic

ImmunoLogic

Latest CME Events & Activities

More News

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – August 1, 2025

Noah Stansfield

August 1st 2025

Review top news and interview highlights from the week ending August 1, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – July 30, 2025

Around the Helix: Cell and Gene Therapy Company Updates – July 30, 2025

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – July 25, 2025

Noah Stansfield

Review top news and interview highlights from the week ending July 25, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – July 23, 2025

Around the Helix: Cell and Gene Therapy Company Updates – July 23, 2025

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – July 18, 2025

Noah Stansfield

Review top news and interview highlights from the week ending July 18, 2025.

Five Advanced Therapeutics to Watch in 2H 2025

Five Advanced Therapeutics to Watch in 2H 2025

Noah Stansfield

The CGTLive® team highlights 5 therapeutics that are nearing major decisions by the FDA.

Around the Helix: Cell and Gene Therapy Company Updates – July 16, 2025

Around the Helix: Cell and Gene Therapy Company Updates – July 16, 2025

Noah Stansfield

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

Heather Lau, MD, MS

Ultragenyx’s MPS IIIA Gene Therapy UX111 Met With CRL

Noah Stansfield

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

Noah Stansfield

Review top news and interview highlights from the week ending July 11, 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

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – July 4, 2025

Noah Stansfield

Review top news and interview highlights from the week ending July 4, 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

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – June 27, 2025

Noah Stansfield

Review top news and interview highlights from the week ending June 27, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – June 25, 2025

Around the Helix: Cell and Gene Therapy Company Updates – June 25, 2025

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – June 20, 2025

Noah Stansfield

Review top news and interview highlights from the week ending June 20, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – June 18, 2025

Around the Helix: Cell and Gene Therapy Company Updates – June 18, 2025

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – June 13, 2025

Noah Stansfield

Review top news and interview highlights from the week ending June 13, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – June 11, 2025

Around the Helix: Cell and Gene Therapy Company Updates – June 11, 2025

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – June 6, 2025

Noah Stansfield

Review top news and interview highlights from the week ending June 6, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – June 4, 2025

Around the Helix: Cell and Gene Therapy Company Updates – June 4, 2025

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – May 30, 2025

Noah Stansfield

Review top news and interview highlights from the week ending May 30, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – May 28, 2025

Around the Helix: Cell and Gene Therapy Company Updates – May 28, 2025

Noah Stansfield

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

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – May 23, 2025

Noah Stansfield

Review top news and interview highlights from the week ending May 23, 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

The Implications of the First Successful Personalized Gene Editing Therapy

Kiran Musunuru, MD, PhD, MPH, ML, MRA;Rebecca Ahrens-Nicklas, MD, PhD;Noah Stansfield

Kiran Musunuru, MD, PhD, and Rebecca Ahrens-Nicklas, MD, PhD, physician-scientists at CHOP, discussed long-term future expectations for personalized gene editing therapy.

Around the Helix: Cell and Gene Therapy Company Updates – May 21, 2025

Around the Helix: Cell and Gene Therapy Company Updates – May 21, 2025

Noah Stansfield

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

Rebecca Ahrens-Nicklas, MD, PhD, a physician-scientist and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP

The Next Steps for Personalized Gene Editing

Rebecca Ahrens-Nicklas, MD, PhD;Kiran Musunuru, MD, PhD, MPH, ML, MRA;Noah Stansfield

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, physician-scientists at CHOP, discussed what lies on the horizon after KJ’s treatment with a CRISPR-based gene editing therapy for CPS1 deficiency.

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

Treating CPS1 Deficiency With Personalized Gene Editing

Kiran Musunuru, MD, PhD, MPH, ML, MRA;Rebecca Ahrens-Nicklas, MD, PhD;Noah Stansfield

Kiran Musunuru, MD, PhD, and Rebecca Ahrens-Nicklas, MD, PhD, physician-scientists at CHOP, discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind – May 16, 2025

Noah Stansfield

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

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

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

Noah Stansfield

The PDUFA action date for the BLA has been set as November 9, 2025.

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

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

Noah Stansfield

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

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