Neurology

bluebird bio is also planning to withdraw the marketing authorization for their β-thalassemia therapy, beti-cel, from the EU and UK.

Review top news and interview highlights from the week ending October 22, 2021.

The professor from National Taiwan University Hospital discussed 3 studies of PTC-AADC for AADC deficiency.

AVROBIO shared interim safety data from ongoing phase 1 and 2 trials.

Tofersen failed to meet its primary end point in change from baseline in ALSFRS-R but differences in total cerebrospinal fluid SOD1 protein and neurofilament light chain were observed.

The director of the Powell Gene Therapy Center at the University of Florida discussed improving access to gene therapies for patients with rare diseases worldwide.

Review top news and interview highlights from the week ending October 15, 2021.

New human gene editing therapies, drug discoveries, targets, and CRISPR technology holds the potential to usher in a new age in medicine.

Sarepta Therapeutics has also initiated the pivotal phase 3 EMBARK study in pediatric patients with DMD.

The chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.

Review top news and interview highlights from the week ending October 8, 2021.

The director of the Powell Gene Therapy Center at the University of Florida discussed adverse events in gene therapies.

Forge Biologics and Solid Biosciences have also partnered to help develop a gene therapy for Duchenne muscular dystrophy.

Paul Wotton, PhD, chief executive officer, Obsidian Therapeutics, discussed the company’s pipeline and technologies.

The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed safety concerns with CAR T-cell therapies.

Improvements in peak expiratory flow, forced expiratory volume, and forced vital capacity were observed in all evaluable treated patients in the IGNITE-DMD trial.

A clinical hold was previously placed after patients treated with the highest dose developed lower extremity weakness.

Improvements persisted through up to 10 years of follow-up.

The director of the Powell Gene Therapy Center at the University of Florida discussed the recent meeting of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee.

Review top news and interview highlights from the week ending October 1, 2021.

The decision comes after 3 participants experienced serious adverse events during the phase 3 trial.

CAP-1002 met primary and secondary end points in the HOPE-2 trial.

Patient-reported outcomes, 6-minute walk test, and NSAA scores all showed improvements over natural history data.

The director of the Powell Gene Therapy Center at the University of Florida discussed gene therapy programs being conducted for Pompe disease.

Review top news and interview highlights from the week ending September 24, 2021.