Neurology

Review top news and interview highlights from the week ending June 17, 2022.

The approval came earlier than the recently extended PDUFA date of July 24, 2022.

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

The trial was placed on hold after elevated liver function test scores.

Review top news and interview highlights from the week ending June 10, 2022.

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted in favor of the benefits of bluebird bio's eli-cel in patients with cerebral andrenoleukodystrophy despite substantial safety concerns.

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 3, 2022.

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

Stefan Braam, PhD, chief executive officer of Ncardia and Cellistic discussed the companies’ work in the cell therapy space.

Review top news and interview highlights from the week ending May 27, 2022.

The chief executive officer of Ring Therapeutics, Tuyen Ong, MD, discussed the potential of anellovirus vectors for use in gene therapy.

The vice president of neuroscience at Sangamo Therapeutics discussed identifying novel capsids with improved neuronal transduction to the CNS.

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

All study participants met criteria to remain off enzyme replacement therapy through 104 weeks after treatment.

The senior investigator from NINDS discussed findings investigating serious AEs across different trials.

The positive opinion was based on findings from studies conducted in Taiwan.

Review top news and interview highlights from the week ending May 20, 2022.

Motor and pulmonary function have stabilized or improved in participants treated with SGT-001.

Catch up on the latest news and announcements in cell and gene therapies presented at the annual ASGCT meeting.

Multiple insertions were observed in affected participants, including insertions in the MECOM and PRDM16 genes.

Data from 2 patients dosed in the Imagine-1 clinical trial were presented in a late breaking session at ASGCT 2022.

Trial sponsors Pfizer, Sarepta, Genethon, and Solid Biosciences organized a group of experts to investigate shared serious AEs.

Study sponsor Pfizer recently announced the launch of a phase 3 trial in patients with Duchenne muscular dystrophy.

Review top news and interview highlights from the week ending May 13, 2022.