Lysosomal Disorders

At dose level 2, 3 of 4 participants had increases of β-Gal activity to normal levels for up to 12 months.

Review top news and interview highlights from the week ending August 4, 2023.

The gene-edited cell therapy OTL-200 was approved in Europe in 2020.

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

LEXEO also recently completed dosing in the first cohort of a trial for Friedreich’s ataxia cardiomyopathy.

The first-in-human MuST trial is being conducted in several centers in Germany.

Maria Escolar, MD, the chief medical officer of Forge Biologics, discussed the atypical approach being used in the company’s trial for AAV gene therapy FBX-101.

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

The chief medical officer of Forge Biologics discussed updated data from the phase 1/2 RESKUE clinical trial that she presented at ASGCT’s 2023 conference.

Review top news and interview highlights from the week ending July 21, 2023.

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

Rocket Pharma is working to soon initiate a phase 1 trial of RP-A601.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed biomarker findings from the phase 1/2 CAMPSIITE trial.

Data on cohort 2 of the AEROW trial are expected in November 2023.

Review top news and interview highlights from the week ending July 14, 2023.

The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.

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.

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.

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

Intellia noted that the first 3 patients who received treatment reached at least 1 year of follow-up without experiencing HAE attacks.

Follow-up data up to 3 years showed supraphysiologic levels of blood IDUA and decrease of GAGs.

For International Batten Awareness Day, held annually on June 9, CGTLive reached out to several experts to inquire about the potential impact of gene therapy on the Batten disease treatment landscape.

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.

Florian Eichler, MD, a neurologist at Massachusetts General Hospital, discussed efficacy data from the CANaspire clinical trial that he presented at ASGCT 2023 showing reductions in urine NAA levels.

Review top news and interview highlights from the week ending May 26, 2023.

All evaluable patients had improvements in natriuretic peptides, left ventricular volume, and NYHA class.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed updated data from the phase 1/2 CAMPSIITE trial presented at ASGCT 2023.