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

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

By Kiran Musunuru, MD, PhD, MPH, ML, MRA
Rebecca Ahrens-Nicklas, MD, PhD

May 20th 2025

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 – May 16, 2025

By Noah Stansfield

May 16th 2025

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

By Noah Stansfield

May 15th 2025

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

By Noah Stansfield

May 14th 2025

Aneal Khan MD, MSc, FCCMG, FRCPC, president of MAGIC Clinic and CEO of Discovery DNA

Aneal Khan, MD, MSc, FCCMG, FRCPC, on Evaluating Gene Therapy for Fabry Disease

By Aneal Khan MD, MSc, FCCMG, FRCPC
Noah Stansfield

May 12th 2025

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April 26-27, 2025

Community Practice Connections™: Pre-Conference Workshop on Immune Cell-Based Therapy

28th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

Go To PER in Chicago

May 30, 2025 - June 3, 2025

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9th Annual Live Medical Crossfire®: Hematologic Malignancies

July 26, 2025

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET

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Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead

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More News

Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion

Victoria Johnson

May 23rd 2022

The positive opinion was based on findings from studies conducted in Taiwan.

Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene-Edited Cell Therapy in PKD

Ami J. Shah, MD

May 21st 2022

The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.

Gene Therapy for LAD-I Posts Impressive Survival Data

Alicia Bigica

May 20th 2022

In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.

CGTLive’s Weekly Rewind – May 20, 2022

CGTLive Staff

May 20th 2022

Review top news and interview highlights from the week ending May 20, 2022.

Florian Eichler, MD, on Promising Efficacy of AXO-AAV-GM2 in GM2 Gangliosidosis

Florian Eichler, MD

May 20th 2022

The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

Florian Eichler, MD, on How Novel Gene Therapy Delivery Improved Targeting in GM2 Gangliosidosis

Florian Eichler, MD

May 19th 2022

The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.

Lentiviral Gene Therapy for Infants With X-Linked SCID Helps Build ‘Functional’ Immune System

Alexandra Ward, MA

May 19th 2022

The interim study results presented at ASGCT 2022 likely represent the longest follow-up data to date for the largest cohort of infants with XSCID treated with lentiviral vector gene therapy.

Around the Helix at ASGCT: Cell and Gene Therapy Company Updates – May 18, 2022

CGTLive Staff

May 18th 2022

Catch up on the latest news and announcements in cell and gene therapies presented at the annual ASGCT meeting.

Analysis Elucidates Lentiviral Vector-Mediated Myelodysplastic Syndrome Cases

Victoria Johnson

May 18th 2022

Multiple insertions were observed in affected participants, including insertions in the MECOM and PRDM16 genes.

Improvements in Developmental Milestones Observed With GM1 Gangliosidosis Gene Therapy

Alicia Bigica

May 18th 2022

Data from 2 patients dosed in the Imagine-1 clinical trial were presented in a late breaking session at ASGCT 2022.

RP-A501 for Danon Disease: Gene Therapy Maintains Clinical Stability

Alexandra Ward, MA

May 16th 2022

Principal investigator Barry Greenberg, MD, FHFSA, shares extended results from the phase 1 trial and also preliminary pediatric safety results in a presentation at ASGCT 2022.

CGTLive’s Weekly Rewind – May 13, 2022

CGTLive Staff

May 13th 2022

Review top news and interview highlights from the week ending May 13, 2022.

Around the Helix: Cell and Gene Therapy Company Updates – May 11, 2022

CGTLive Staff

May 11th 2022

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

FDA Lifts Hold on LogicBio’s Phase 1/2 SUNRISE Trial of LB-001 for MMA

Alexandra Ward, MA

May 9th 2022

The hold was originally placed after reports of 2 serious adverse events in pediatric patients.

CGTLive’s Weekly Rewind – May 6, 2022

CGTLive Staff

May 6th 2022

Review top news and interview highlights from the week ending May 6, 2022.

Around the Helix: Cell and Gene Therapy Company Updates – May 4, 2022

CGTLive Staff

May 4th 2022

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

CGTLive’s Weekly Rewind – April 29, 2022

CGTLive Staff

April 29th 2022

Review top news and interview highlights from the week ending April 29, 2022.

Around the Helix: Cell and Gene Therapy Company Updates – April 28, 2022

CGTLive Staff

April 28th 2022

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

CGTLive’s Weekly Rewind – April 22, 2022

CGTLive Staff

April 22nd 2022

Review top news and interview highlights from the week ending April 15, 2022.

Around the Helix: Cell and Gene Therapy Company Updates – April 20, 2022

CGTLive Staff

April 20th 2022

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

"Low and Slow" vs "One and Done"? Unlocking the Potential of Gene Therapy

CGTLive Staff

April 19th 2022

Carsten Brunn, PhD, president and chief executive officer of Selecta Biosciences, joins CGTL to discuss ImmTOR, an immune tolerance platform that could ultimately enable gene therapy redosing in certain cases.