24 March 2026
CTx1000 designed to clear pathological TDP-43 protein, a central driver of ALS
Celosia Therapeutics, an Australian biotech company developing advanced gene therapies for neurodegenerative diseases, announced dosing of the first participant in its Phase 1b KOANEWA trial evaluating CTx1000, an investigational genetic medicine targeting pathological forms of the TDP-43 protein in people with amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND). The study is being conducted at the Neurology Department of Macquarie University Hospital in Sydney, Australia.
Dr Kathryn Sunn, Chief Executive Officer at Celosia Therapeutics, said: “ALS is a progressive and ultimately fatal neurodegenerative disease with limited therapeutic options. The initiation of dosing in the KOANEWA study marks an important milestone for Celosia and, most importantly, for the ALS community. Treating the first patient begins the clinical evaluation of this genetic medicine and represents a meaningful step toward a potential disease‑modifying therapy for people living with ALS“
KOANEWA is a first-in-human, open-label Phase 1b clinical trial, designed to evaluate the safety and tolerability of a single administration of CTx1000 in participants with ALS. In addition to safety outcomes, the study will profile biomarkers and clinical measures as seconday exploratoty efficacy endpoints.
Prof Lars Ittner, Chief Medical Officer at Celosia and Director of the Macquarie University Dementia Research Centre, said: “This study is a major milestone for our research program, advancing a novel disease-modifying therapeutic strategy into the clinic that, for the first time, directly targets a fundamental disease mechanism in ALS—the pathological accumulation of TDP-43. Evaluating CTx1000 in people living with ALS will enable us to assess the safety of this approach while also exploring its potential to therapeutically address one of the key drivers of disease.”
About CTx1000
In February 2024, Professors Lars Ittner and Professor Yazi Ke reported a groundbreaking discovery in the prestigious journal Neuron, which has been exclusively licensed to Celosia. Their work identified a unique binder to TDP-43, the key pathological protein implicated in ALS. Building on this discovery, Ittner and Ke developed the genetic medicine CTx1000, designed to selectively bind and clear toxic forms of TDP-43. In multiple preclinical ALS models, CTx1000 halted disease progression, including at advanced stages, and in some cases partially reversed disease manifestations.
Source: Celosia Therapeutics