CTx1000 / Celosia Therapeutics - LARVOL DELTA

Targeting TDP-43 in ALS: Regulatory hurdles, trial design deficiencies, and the causal evidence gap for CTx1000. (PubMed, Drug Discov Today) - "This review critically evaluates the regulatory hurdles and trial design deficiencies that have historically undermined ALS clinical development, and incorporates the dual sequestration hypothesis as a framework to interpret the convergence of TDP-43 pathology across neurodegenerative diseases. It concludes that it is imperative that the field adopts more rigorous biomarker-led methodologies, and that although target-specific degraders offer a sophisticated technological leap, their success depends on addressing fundamental knowledge gaps in target engagement, age-dependent vector tropism, and trial design architecture."

Journal • Review • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • Targeted Protein Degradation • TARDBP

Celosia Therapeutics…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)… (PRNewswire) - "The study is being conducted at the Neurology Department of Macquarie University Hospital in Sydney, Australia....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."

Trial status • Amyotrophic Lateral Sclerosis