Lysosomal Disorders

Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza.

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

Updated data including a sixth patient and longer follow-up was presented at ASGCT’s 2024 Meeting.

Review top news and interview highlights from the week ending May 10, 2024.

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 May 3, 2024.

PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed ongoing initiatives.

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.