Neurology

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.

Late-breaking interim data were presented at the 14th Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders.

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

The trial was set to begin patient dosing in the first half of this year.

Review top news and interview highlights from the week ending May 6, 2022.

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

The AAV9 gene therapy is currently being evaluated in a clinical trial conducted by NINDS.

Review top news and interview highlights from the week ending April 29, 2022.

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

The CIFFREO trial is underway in 11 countries but was halted in December 2021 amid safety concerns, including the death of a participant in a phase 1b study in the non-ambulatory cohort.

Lexeo recently announced FDA clearance of its investigational new drug application for LX2006 for Friedreich's ataxia cardiomyopathy.

The one-time therapy is meant to address familial early-onset dementia linked to the GRN gene.

Review top news and interview highlights from the week ending April 15, 2022.

As patients who undergo CAR T-cell therapy survive longer, more research is required into the long-term neurocognitive effects of this treatment.

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 April 15, 2022.

Shephard Mpofu, MD, part of the Novartis Gene Therapies leadership, shared his thoughts on the latest phase 3 data on onasemnogene abeparvovec (Zolgensma) from the SPR1NT trial.

Erandi De Silva, PhD, co-founder and senior vice president of product development at Forge Biologics, discusses the dual approach of her company in manufacturing and also developing adeno-associated viral (AAV) -based gene therapies, including FBX-101 for Krabbe disease.