Lysosomal Disorders

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.

All evaluable patients had significant reductions in daily cornstarch intake at 1 year and at last visit during long-term follow-up.

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

The senior investigator at the National Cancer Institute Center for Cancer Research discussed updated data from a phase 1/2 trial presented at ASGCT 2023.

The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.

The neurologist at Massachusetts General Hospital discussed BBP-812, an investigational AAV vector-based gene therapy being evaluated for Canavan disease in a phase 1/2 trial.

Review top news and interview highlights from the week ending May 12, 2023.