Lysosomal Disorders

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.

The associate professor of otolaryngology from Harvard Medical School also shared his excitement for the positive data on AAV1-hOTOF.

Review top news and interview highlights from the week ending May 24, 2024.

The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.

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

The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.

The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.

Oncological trials of biologics also face particularly higher rates of clinical holds.

Review top news and interview highlights from the week ending May 17, 2024.

Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.

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.