Lysosomal Disorders

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

The company’s TSHA-201 AAV gene therapy for Rett syndrome is its current priority.

Review top news and interview highlights from the week ending April 26, 2024.

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

The FDA is allowing a small, single-arm study to support a BLA submission of ST-290, if Sangamo can find help to continue developing the therapy.

Robert Hopkin, MD, Cincinnati Children’s Hospital Medical Center, discussed unmet needs and patient-reported outcomes from the STAAR trial.

Review top news and interview highlights from the week ending April 19, 2024.

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

The more recent data were presented at the 2022 ASGCT Meeting.

Review top news and interview highlights from the week ending April 12, 2024.

Paul Harmatz, MD, professor, UCSF, discussed benefits in neurocognitive outcomes and ERT therapy usage after RGX-121 gene therapy for Hunter syndrome.

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.

A 2-year evaluation of a small cohort of patients with systemic lupus erythematosus, idiopathic inflammatory myositis, and systemic sclerosis showed safety and efficacy which were supportive of additional clinical trials.

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

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed emphasized topics at this year's ASGCT meeting.

Review top news and interview highlights from the week ending March 22, 2024.

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

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

The gene-edited cell therapy has been approved as Lenmeldy by the FDA.

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from a session on rare disease gene therapy at MDA.

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

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

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

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.