Lysosomal Disorders

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 June 21, 2024.

The company anticipates that it will be able to submit a BLA in late 2024 or early 2025.

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

An investigator noted challenging side effects related to immunosuppressant treatment.

The scientist from Krembil Research Institute shared takeaways from the session she chaired.

There was a 98% (2.7% SD) mean reduction in monthly HAE attack rate through the patients’ most recent assessment.

Review top news and interview highlights from the week ending June 14, 2024.

The pediatric neurologist at Nemours Children’s Health discussed next steps in the field to fully enable the benefits of gene therapy.

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

The FDA granted the designation after reviewing safety and efficacy data from phase 1 of the trial.

Ineka Whiteman, PhD, head of research and medical affairs, BDSRA Australia, discussed what has occurred in the field of Batten disease gene therapy in the past year.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison shared highlights from the discussion.

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 June 7, 2024.

The associate professor of clinical pediatrics at Cincinnati Children's discussed further investigations into novel therapies and new research.

Other of the company’s RNAi therapies have recently shown promising data in hyperlipidemia and asthma.

According to recent studies and the latest insights provided by experts, early developments in gene and cell therapies show promise for patients living with Parkinson disease, but challenges remain.

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 31, 2024.

Ultragenyx is aiming to submit a marketing application for DTX401 next year.

Current handling guidelines do not align between the laboratory and clinical settings.

The deputy director, clinical research, San Raffaele Telethon Institute for gene therapy, discussed long-term follow-up data of up to 12 years.

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

An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.