CTx1000

CTx1000 is a novel AAV9-based gene therapy developed by Celosia Therapeutics, a Macquarie University spin-out, for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The therapy utilizes a single-dose genetic approach to deliver a gene encoding a short peptide derived from the 14-3-3 protein. This peptide specifically binds to pathological, misfolded accumulations of the TDP-43 protein, tagging them for cellular degradation while preserving the function of normal TDP-43. Developed based on 15 years of research at Macquarie University's Dementia Research Centre, CTx1000 aims to be a disease-modifying treatment by addressing the underlying proteinopathy associated with motor neuron degeneration. Preclinical studies in mouse models demonstrated the therapy's ability to halt disease progression and significantly increase survival. It is currently being evaluated in the Phase 1b Koanewa clinical trial for patients with ALS.

Other names

AAV9 Gene therapyAAV-9 Gene therapyAAV 9 Gene therapy