Celosia Therapeutics Initiates First-in-Human Trial of CTx1000 Gene Therapy for ALS
Key Insights
Celosia Therapeutics (search) has dosed the first patient in its Phase 1b KOANEWA trial evaluating CTx1000, a genetic medicine targeting pathological TDP-43 protein in amyotrophic lateral sclerosis (search) patients.
CTx1000 represents the first therapeutic approach to directly target the pathological accumulation of TDP-43, a fundamental disease mechanism in ALS (search).
The open-label trial will evaluate safety and tolerability of a single administration while profiling biomarkers and clinical measures as secondary exploratory endpoints.
Celosia Therapeutics (search), an Australian biotech company developing advanced gene therapies for neurodegenerative diseases, has 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 (search) (ALS (search)). The study is being conducted at the Neurology Department of Macquarie University Hospital (search) in Sydney, Australia.
Novel Therapeutic Approach Targets Key Disease Driver
CTx1000 is designed to selectively bind and clear toxic forms of TDP-43, a central driver of ALS (search). According to Prof Lars Ittner, Chief Medical Officer at Celosia and Director of the Macquarie University Dementia Research Centre (search), this represents "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."
Dr Kathryn Sunn, Chief Executive Officer at Celosia Therapeutics (search), emphasized the significance of this milestone: "ALS (search) 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."
Trial Design and Objectives
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 (search). In addition to safety outcomes, the study will profile biomarkers and clinical measures as secondary exploratory efficacy endpoints.
The trial represents the clinical translation of research that will enable assessment of the safety of this approach while exploring its potential to therapeutically address one of the key drivers of disease.
Preclinical Foundation
The development of CTx1000 is based on groundbreaking research published in February 2024 by Professors Lars Ittner and Professor Yazi Ke in the 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 (search).
Building on this discovery, the researchers developed CTx1000 as a genetic medicine designed to selectively bind and clear toxic forms of TDP-43. In multiple preclinical ALS (search) models, CTx1000 demonstrated the ability to halt disease progression, including at advanced stages, and in some cases partially reversed disease manifestations.