Celosia Therapeutics announced on 23 March 2026, that it has dosed the first participant in KOANEWA, a first-in-human, open-label Phase 1b trial evaluating CTx1000 in people with amyotrophic lateral sclerosis.
The study is being conducted at the Neurology Department of Macquarie University Hospital in Sydney and is designed to assess the safety and tolerability of a single administration of CTx1000, with biomarker profiling and clinical measures included as secondary exploratory efficacy endpoints.
CTx1000 is an investigational genetic medicine intended to target pathological forms of the TDP-43 protein implicated in ALS; according to the company, the therapy was developed from a February 2024 Neuron discovery that has been exclusively licensed to Celosia. The company claims preclinical studies showed CTx1000 halted disease progression in multiple ALS models and partially reversed manifestations in some cases.
