Lysosomal Disorders

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

University College London has initiated the single site trial at Great Ormond Street Hospital, London.

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

Altogether, there was a 91% reduction in median serum TTR at day 28.

The approval of exa-cel, the first gene editing therapy, may herald a new era in developing gene editing strategies.

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

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.

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

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

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.

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

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.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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