Celosia Therapeutics has taken a significant step forward in the fight against amyotrophic lateral sclerosis, announcing that the first patient has been dosed in its Phase 1b clinical trial of CTx1000, an experimental genetic medicine targeting a key driver of the disease.
The milestone marks the beginning of human evaluation for a therapy aimed at addressing the underlying biology of ALS rather than only managing symptoms.
The trial, known as KOANEWA, is being conducted at the Neurology Department of Macquarie University Hospital in Sydney. It represents a first in human, open-label study focused primarily on assessing the safety and tolerability of a single administration of CTx1000 in people living with ALS. Researchers will also explore biomarkers and clinical indicators to better understand whether the therapy shows early signs of effectiveness.
At the centre of this approach is TDP 43, a protein widely recognised as a critical contributor to neurodegeneration in the vast majority of ALS cases.
CTx1000 has been engineered to selectively bind to and clear pathological forms of this protein, offering a novel strategy that directly targets disease biology. For patients facing a condition with limited treatment options and a typically short survival window, this approach signals a meaningful shift toward disease-modifying therapies.
Celosia Chief Executive Officer Dr Kathryn Sunn described the dosing of the first patient as an important moment not only for the company but for the broader ALS community. She emphasised that initiating clinical evaluation brings the field closer to therapies that may alter the course of a currently progressive and fatal disease.
Chief Medical Officer Professor Lars Ittner highlighted the scientific significance of the trial, noting that it is the first time a therapy targeting TDP43 pathology has entered clinical testing in ALS patients. He said the study will provide crucial insights into both safety and the potential for this strategy to address one of the central mechanisms driving neurodegeneration.
The origins of CTx1000 trace back to a 2024 discovery that identified a unique binder for TDP 43. Building on this work, researchers developed the therapy to clear toxic forms of the protein. In preclinical models, the treatment halted disease progression and, in some cases, partially reversed symptoms, even at advanced stages. These findings underpin the optimism surrounding its clinical development.
ALS, also known as motor neuron disease, progressively damages nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis and eventually respiratory failure. With most patients surviving only two to five years after diagnosis, the need for therapies that can slow or stop disease progression remains urgent.