Neurology

The head of neuroscience at Sangamo Therapeutics discussed advances in understanding disease biology that allow for more specific gene targeting.

The head of neuroscience at Sangamo Therapeutics discussed the potential of ZFPs in central nervous system disorders.

Interim data from a phase 1 study from Intellia Therapeutics and Regeneron Pharmaceuticals is the first to support in vivo CRISPR genome editing in humans.

The head of global research and executive vice president of Atara Biotherapeutics discussed the development of ATA188.

Corlieve Therapeutics’ lead program, AMT-260, previously demonstrated proof-of-concept in preclinical studies of temporal lobe epilepsy.

Newly published data continues to demonstrate Zolgensma's efficacy in presymptomatic and symptomatic SMA Type 1.

LogicBio's Mariana Nacht, PhD, shares details of the company's novel capsid discovery platform and gene editing technology.

The news comes after CRISPR’s favorable data readouts in its sickle cell and beta thalassemia studies.

Cynata Therapeutics' Ross Macdonald, PhD, discusses the company's pipeline and clinical-phase trials.

Bayer provided an update on trials currently underway for the treatment of PD by its subsidiaries.

Locanabio CEO James Burns, PhD, discusses the firm's targeted approach to treating genetic disorders of the central nervous system and the eye.

Asklepios BioPharmaceuticals noted that it plans to initiate dosing for the LION-101 phase 1/2 clinical trial in the first half of 2022.

Ross Macdonald, PhD, managing director and CEO of Cynata Therapeutics, discusses the biotech company's approach to overcoming reproducibility challenges with mesenchymal stem cells.

An independent board has advised that a phase 1/2 study assessing AMT-130 for adults with early manifest Huntington disease should continue as planned.

Maria A. Croyle, PhD, discusses the development of a novel film matrix that can safely and effectively store and transport AAV-based gene therapies.

SRP-9001 manufactured with commercial materials demonstrated robust transduction efficiency and high gene expression levels for DMD.

Maria A. Croyle, PhD, discusses the manufacturing challenges facing the gene therapy space and her inspiration for developing a novel preservation method for viral vector-based therapies.

Locanabio CEO James Burns, PhD, shares his outlook on the quickly changing landscape of gene therapy research.

Participants in both the low- and high-dose cohorts showed improved functional outcomes at 1-year post-dose.

No adverse effects on weight, general activity, or survival in knockout mice and wild type littermates were observed throughout the study.

The CEO of Locanabio shares details of 2 presentations the company is making this week at the 24th Annual Meeting of the American Society of Gene & Cell Therapy.

The partnership will allow for the development of disease-specific AAV vectors that will serve Biogen's current gene therapy pipeline.

The FDA has lifted a clinical hold placed on an IND for VY-HTT01 in Huntington disease, allowing Voyager Therapeutics to initiate a phase 1/2 study for the agent later this year.

In an AAN plenary talk, Mark H. Tuszynski, MD, PhD, detailed the work he and colleagues have done to push stem cell therapy from the lab to the clinic to improve care for spinal cord injury.

The FDA has granted a fast track designation to LX1001 as a potential treatment for adult patients with APOE4-associated Alzheimer disease.