STANFORD UNIVERSITY

UC San Francisco and Stanford University teams, funded by NIH, will study cardiotoxicities caused by covalent kinase inhibitors in cancer treatments, aiming to identify genetic causes and develop drug discovery pipelines.

13 participants, median age 15, received CAR-T cell therapy for DIPG/spinal cord diffuse midline glioma; 9 experienced benefits, including tumor volume reduction and improved neurologic function. Researchers determined lower CAR-T cell doses were safer. Drew, a participant, remains alive four years post-diagnosis, suggesting CAR-T therapy's potential. The study aims to refine the therapy for future patients.

The FDA has cleared GE HealthCare's Signa Magnus 3T MRI system for head-only scanning, featuring HyperG gradient technology with 300 mT/m and 750 T/m/s performance, enabling faster scans, advanced diffusion imaging, and high SNR. The system's asymmetrical design enhances gradient amplitude and slew rate compared to whole-body MRI platforms.

FDA accepts BLA resubmission for pz-cel to treat RDEB, with a target action date of April 25, 2025. Pz-cel, an autologous COL7A1 gene-corrected epidermal sheet, demonstrated wound healing and pain reduction in phase 3 and phase 1/2a studies. The therapy aims to provide VII expression at wound sites via a stably integrated copy of the COL7A1 gene.

£78 million funding for UCL scientists developing a potential treatment for motor neurone disease, targeting the restoration of the UNC13A protein essential for brain and spinal cord communication. Trace Neuroscience, a UCL spinout, aims to slow disease progression by re-establishing healthy nerve cell communication.

GE HealthCare's SIGNA™ MAGNUS, a 3.0T high-performance head-only MRI scanner, received FDA 510(k) clearance, offering advanced neuroimaging capabilities for detecting neurological, oncological, and psychiatric conditions. The system features an asymmetrical gradient coil design, HyperG technology, and aims to support advanced imaging and biomarker research.

Trace Neuroscience, with $101M Series A funding, aims to restore UNC13A protein communication in nerves and muscles to treat ALS, frontotemporal dementia, and Alzheimer’s. Their lead program uses an antisense oligonucleotide (ASO) to address sporadic ALS, the most common form, affecting 90% of cases.

Third Rock Ventures leads $101M funding for Trace Neuroscience, aiming to treat ALS by replacing broken genetic machinery. Trace focuses on TDP-43, a molecule essential for RNA processing, which, when dysfunctional, impairs protein production linked to nerve-destroying disorders. The startup uses antisense therapy to correct UNC13A instructions, with a drug program targeting ALS and potentially other neurodegenerative diseases, set to enter human testing in 2026.