Lysosomal Disorders

Ultragenyx’s MPSIIIA Gene Therapy UX111 Met With CRL

July 16th 2025

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

CGTLive®’s Weekly Rewind – July 11, 2025

July 14th 2025

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

July 11th 2025

CGTLive®’s Weekly Rewind – July 4, 2025

July 9th 2025

Around the Helix: Cell and Gene Therapy Company Updates – January 3, 2024

July 4th 2025

Latest CME Events & Activities

Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma

Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights

Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches

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

Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?

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

Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios

More News

CGTLive's Clinical Trials to Watch in 2024

January 3rd 2024

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

Matt Hoffman

January 1st 2024

These, among many others, are some of the key clinical trials of gene, cell, and regenerative therapies that the CGTLive staff will be following throughout 2024.

CGTLive's Pillars of Progress 2023: Top FDA Approval News in Cell and Gene Therapy

Around the Helix: Cell and Gene Therapy Company Updates – December 27, 2023

December 31st 2023

Take a look at the stories that stood out as pillars of progress and success in endocrinology gene and cell therapy development in 2023.

CGTLive's Pillars of Progress 2023: Top News in Lysosomal Disorders

December 27th 2023

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

Around the Helix: Cell and Gene Therapy Company Updates – December 20, 2023

December 26th 2023

Take a look at the stories that stood out as pillars of progress and success in lysosomal storage disease gene and cell therapy development in 2023.

The Lupus Approach: CAR-T Begins the Jump from Blood Cancers to B-Cell Driven Autoimmune Disease

December 20th 2023

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

Around the Helix: Cell and Gene Therapy Company Updates – December 13, 2023

December 20th 2023

Throughout 2023, interest in the application of CAR-T and other cell therapies to B-cell-driven autoimmune disease skyrocketed, with a multitude of clinical trials being initiated by various companies.

Around the Helix: Cell and Gene Therapy Company Updates – December 6, 2023

December 13th 2023

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

uniQure Expanding Clinical Pipeline to Fabry Disease

December 6th 2023

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

Around the Helix: Cell and Gene Therapy Company Updates – November 29, 2023

November 30th 2023

The IND clearance sets AMT-191 to be evaluated in a clinical trial alongside the company's gene therapies for ALS and Huntington disease.

Around the Helix: Cell and Gene Therapy Company Updates – November 22, 2023

November 29th 2023

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

CGTLive’s Weekly Rewind – November 17, 2023

November 22nd 2023

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

Sanfilippo Awareness Day: A Peek Into the CGT Pipeline

November 17th 2023

Review top news and interview highlights from the week ending November 17, 2023.

Professor Robert Wynn, MB BChir, MD, MRCP, FRCPath

Around the Helix: Cell and Gene Therapy Company Updates – November 15, 2023

November 16th 2023

CGTLive takes a look at MPSIII therapies in the pipeline for World Sanfilippo Awareness Day on November 16.

First Clinical Trial for NGLY1 Deficiency Coming to Evaluate New Gene Therapy

November 15th 2023

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

CGTLive’s Weekly Rewind – November 10, 2023

November 12th 2023

The trial will follow a phase 1/2/3 design and will evaluate GS-100 in pediatric patients with NGLY1 deficiency.

Around the Helix: Cell and Gene Therapy Company Updates – November 8, 2023

November 10th 2023

Review top news and interview highlights from the week ending November 10, 2023.

CGTLive’s Weekly Rewind – November 3, 2023

November 8th 2023

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

Around the Helix: Cell and Gene Therapy Company Updates – November 1, 2023

November 3rd 2023

Review top news and interview highlights from the week ending Novmber 3, 2023.

CGTLive’s Weekly Rewind – October 27, 2023

November 1st 2023

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

Gaucher Disease Type 1 Gene Therapy Shows Promising Initial Data

October 27th 2023

Review top news and interview highlights from the week ending October 27, 2023.

Around the Helix: Cell and Gene Therapy Company Updates – October 25, 2023

October 26th 2023

The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.

CGTLive’s Weekly Rewind – October 20, 2023

October 25th 2023

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

Canavan Gene Therapy Improves Developmental Measures Compared to Natural History

October 20th 2023

Review top news and interview highlights from the week ending October 20, 2023.

Around the Helix: Cell and Gene Therapy Company Updates – October 18, 2023

October 19th 2023

MSEL 5-mean domain, receptive language, and expressive language scores were significantly improved from baseline in treated participants.

Intellia’s CRISPR Therapy for ATTR-CM First to Enter Late-Stage Trials

October 18th 2023

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

Around the Helix: Cell and Gene Therapy Company Updates – October 11, 2023

October 18th 2023

Intellia Therapeutics plans to initiate the phase 3 trial of NTLA-2001 by the end of 2023.

CGT Therapies More Likely to Gain Approval for Hematological Malignancies, Orphan Diseases

October 11th 2023

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

CGTLive’s Weekly Rewind – October 6, 2023

October 11th 2023

A NEWDIGS analysis compared clinical trial success rates from 1988 to 2020.