Neurology

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.

The first patient has been enrolled and treated with RGX-121 in the third cohort of a phase 1/2 study for patients up to 5 years old with severe MPS II.

AAV2 TrkB-2A-mBDNF resulted in early signs of efficacy in preclinical studies of glaucoma and humanized tauopathy, which could be translatable to other neurodegenerative polygenic disorders.

In 2021, Abeona Therapeutics is anticipating to complete enrollment in its phase 3 VIITAL study and additional findings for ABO-102 and ABO-101.

NurOwn was safe and led to improvements in function and cognition at 28 weeks compared with baseline for patients with progressive multiple sclerosis.

SGT-001 elicited promising improvements in functional and biomarker end points for patients with Duchenne muscular dystrophy.

SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.

Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.

Ninety percent of patients with cerebral adrenoleukodystrophy who were treated with elivaldogene autotemcel were alive and free of major functional disabilities at 2-years.

The FDA has granted a fast track designation to PBFT02, PBKR03, and PBGM01, which are intended as treatments for patients with various rare CNS disorders.

Orchard Therapeutics released its plans for 2021, which included the launch of OTL-200 (Libmeldy) in Europe and development of an FDA submission strategy in the United States.

Rocket Pharmaceuticals has summarized multiple major milestones for its 5 gene therapies from 2020 and provided a forecast and outlook for what is expected to be a banner year for the organization.