Lysosomal Disorders

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.

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.