Neurology

Review top news and interview highlights from the week ending May 12, 2023.

Bloomsbury Genetic Therapies’ BGT-DTDS, a neuron-targeted and AAV2-mediated therapy, previously received orphan drug designation from the FDA and EC.

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

Biogen and Novartis terminated collaborations with the company that first initiated in 2020.

The cell therapy field applications staff scientist at ThermoFisher discussed advantages of the company’s DynaCellect system.

The single-administration allogeneic cell therapy has shown positive safety—and signs of efficacy—in preliminary data from the first 2 patients dosed with the Neurona Therapeutics’ product.

CK0803 is being evaluated for safety and tolerability in the REGALS study.

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

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

The investigators from Children’s Hospital of Philadelphia discussed the follow-up studies they are conducting in light of their recent findings regarding DNA virus replication.

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

The investigators from Children’s Hospital of Philadelphia discussed the potential implications of their research into human adenoviruses for the development of new gene delivery tools.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the rapidly changing landscape of diagnosing and treating genetic disorders.

In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to gene therapies in development for this rare disease.

Review top news and interview highlights from the week ending April 14, 2023.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed past, current, and future approaches to diagnosing and treating neuromuscular diseases.

Matthew Gornet, MD, spine surgeon from The Orthopedic Center of St. Louis discussed data seen with IDCT cell therapy.

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

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the trend towards gene-targeted approaches in the neuromuscular disease space.

REGENXBIO’s RGX-202 previously received orphan drug and rare pediatric disease designations for DMD in late 2021 and early 2022, respectively.

The executive vice president and chief research officer of MDA discussed the barriers to FDA approval currently facing those developing treatments for ultra-rare diseases.

The director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.

Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, discussed his keynote address at the 2023 MDA Conference. c

Review top news and interview highlights from the week ending April 7, 2023.

Study authors Matthew Charman, PhD, and Matthew D. Weitzman, PhD, elaborated on the findings and implications of their research.