Neurology

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.

Peter J. McAllister, MD, FAAN, presented promising data from the phase 2 STEMTRA trial at AAN 2022.

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 8, 2022.

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

The biotechnology company previously pulled out of Europe after encountering payor challenges for their gene therapies.

The same therapy by Capricor Therapeutics has demonstrated efficacy in Duchenne muscular dystrophy.

The latest data build on previous research that showed on-target effects analyzed across multiple measurements and a favorable safety profile.

Review top news and interview highlights from the week ending April 1, 2022.

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 March 25, 2022.

The professors and researchers from The University of Texas at Austin discussed new findings of Cas9 structure and mechanisms.

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

The director of the Powell Gene Therapy Center at the University of Florida discussed unmet needs that remain in Pompe disease.

A sister study of AMT-130 in Europe dosed its first patients in February.

The company recently received IND clearance for a solid tumor cell therapy.

Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.

The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.