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Celosia Therapeutics starts Phase 1b trial of CTx1000, a gene therapy targeting ALS protein TDP-43.

Others
23 Mar 2026
PRNewsWire
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Bullish
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Celosia Therapeutics has begun dosing the first participant in its Phase 1b KOANEWA trial to test CTx1000, a gene therapy designed to clear pathological TDP-43 protein, a key driver of amyotrophic lateral sclerosis (ALS). This first-in-human study at Macquarie University Hospital in Sydney aims to evaluate the safety and tolerability of a single dose of CTx1000 in ALS patients, with secondary measures assessing biomarkers and clinical effects. The therapy builds on a 2024 discovery of a unique binder to TDP-43, showing promise in preclinical models for halting and partially reversing ALS progression. This trial marks a significant step toward a potential disease-modifying treatment for ALS, which currently has limited options.

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