Neurology

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.

The recent FDA Advisory Committee meeting follows a turbulent year for gene therapy studies.

Following a voluntary pause, a patient experiencing a liver AE has died and the trial is now on clinical hold.

Samarendra Mohanty, PhD, cofounder and president, Nanoscope Therapeutics, discussed the company’s technological platforms and RESTORE trial data.

NeuExcell and Spark Therapeutics each add to their gene therapy pipelines with the collaborative program.

Eric Crombez, PhD, senior vice president and chief medical officer of Ultragenyx Gene Therapy, discussed indications the company is targeting.

Magali Taiel, MD, chief medical officer, GenSight Biologics, discussed future research the company plans to pursue.

With numerous targets in CNS diseases and a unique partnership with gene therapy experts at UT Southwestern, Taysha is hoping to fill significant unmet needs.

The first US-based patient has been dosed in Lysogene’s clinical trial of LYS-GM101, following 2 other competing trials.

The 2 companies will focus their strategic partnership on the development of gene therapies for rare diseases, as well.

Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, discussed the different initiatives the company is conducting in Dravet syndrome.