Lysosomal Disorders

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.

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.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges in investigating therapies for rare diseases.

In observance of Rare Disease Day, held this year on February 29, catch up on some of the latest data updates from clinical trials for rare diseases.

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

Investigators concluded that the data support the use of HS as a predictive biomarker in Sanfilippo syndrome.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed the dose response seen with RGX-121.

Review top news and interview highlights from the week ending February 23, 2024.

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

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed the statistical findings she presented at the 2024 WORLDSymposium.

Review top news and interview highlights from the week ending February 16, 2024.

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

Rocket submitted additional CMC data in response to FDA information requests.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.

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

Review top news and interview highlights from the week ending February 9, 2024.

The gene-edited cell therapy also showed a safety profile consistent with that previously seen in patients with early-onset MLD.

Data from defunct company Lysogene’s discontinued trial of LYS-GM101 were presented at WORLDSymposium.

Among the 5 patients treated in the study, sustained supraphysiological expression of the disease-targeted gene, SGSH, was rapidly achieved in the leukocytes and the plasma.

Hematological recovery was achieved and maintained in all 4 patients in the study.

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