Early pipeline
CTx1000
Also known as DD-θFx, KOANEWA trial drug
Bottom line
Very early: a first safety test in a handful of people in Sydney, with no results yet. It targets a process seen in almost all ALS and worked well in mice, but most drugs at this stage do not reach approval.
Very early. This drug is in its first safety trial in people and has not been tested for whether it helps in ALS. Most drugs at this stage never reach approval. More about early drugs
CTx1000 is a one-time gene therapy meant to clear the clumps of a protein called TDP-43 that are found in the nerve cells of almost everyone with ALS, while leaving the healthy form alone. Its first trial in people, KOANEWA, dosed its first participant in Sydney in March 2026 and plans to treat up to 15 people. There are no results yet.
Information checked 5 Nov 2026
Evidence: Results pending
Studies: 1 running
Trials are running, but there are no efficacy results yet.
KOANEWA (Phase 1b, NCT07401121)Pending
Up to 15 people with ALS get a single dose of CTx1000, with no placebo. Main goal is safety over 52 weeks; first participant dosed in March 2026 in Sydney.
At a glance
CompanyCelosia TherapeuticsExplanation and sources
Celosia is a small private Sydney company founded in 2022 by Professor Lars Ittner, whose university lab discovered the approach. CTx1000 is its lead drug. In March 2026 it said it was raising US$80 million to take CTx1000 into later trials and that it plans to file a US trial application with the FDA.
Sources
- Biotech Daily, March 24, 2026 (Celosia Therapeutics)Biotech Daily · 24 Mar 2026 · News
- Celosia Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of CTx1000 in Amyotrophic Lateral SclerosisCelosia Therapeutics (via PR Newswire) · 23 Mar 2026 · Company press release
How it worksOne-time gene therapy meant to clear toxic clumps of TDP-43AAV9-delivered TDP-43 degron (14-3-3θ-based binder)Explanation and sources
In ALS the protein TDP-43 leaves the cell nucleus and builds up in toxic clumps. Researchers found that a helper protein, 14-3-3θ, sticks mainly to this faulty TDP-43. CTx1000 uses an emptied-out virus (AAV9) to deliver a small piece of 14-3-3θ joined to a "destroy me" tag, so faulty TDP-43 is sent to the cell's recycling system. In mice it stopped and sometimes partly reversed ALS-like disease, even when given late, but most treatments that work in mice do not work in people.
Sources
- Targeting 14-3-3θ-mediated TDP-43 pathology in amyotrophic lateral sclerosis and frontotemporal dementia miceNeuron · 15 Feb 2024 · Peer-reviewed paper
- Celosia Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of CTx1000 in Amyotrophic Lateral SclerosisCelosia Therapeutics (via PR Newswire) · 23 Mar 2026 · Company press release
StagePhase 1Phase 1b in people with ALS, up to 15 people (Australia) · Dosing: people with alsExplanation and sources
KOANEWA is the first test of CTx1000 in people. It is open-label, so everyone gets the drug, and each person gets a single dose. The first participant was dosed at Macquarie University Hospital in Sydney, announced on March 23, 2026. Celosia has not said how many people have been treated since.
Sources
- Celosia Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of CTx1000 in Amyotrophic Lateral SclerosisCelosia Therapeutics (via PR Newswire) · 23 Mar 2026 · Company press release
- KOANEWA: Safety and Tolerability Study of CTx1000 In Participants With ALS (NCT07401121)ClinicalTrials.gov · Trial registry
Best reported effectSafety study; no effect data yetSafety; NfL in spinal fluid and ALSFRS-R exploredExplanation and sources
No results have been reported. The main goal is safety over 52 weeks. The trial will also track the nerve-damage marker NfL and the ALSFRS-R score for up to 3 years, but with no placebo group and so few people it cannot show whether the therapy slows ALS.
Sources
- KOANEWA: Safety and Tolerability Study of CTx1000 In Participants With ALS (NCT07401121)ClinicalTrials.gov · Trial registry
- Celosia Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of CTx1000 in Amyotrophic Lateral SclerosisCelosia Therapeutics (via PR Newswire) · 23 Mar 2026 · Company press release
Who it's forALS within 2 years of first symptoms; most genetic forms excludedSporadic · Single dose, one timeExplanation and sources
The trial takes people whose first ALS symptoms began no more than 2 years ago and who have no or low antibodies against the AAV9 virus shell. People with genetic forms of ALS, including C9orf72, are excluded, except those with changes in the TDP-43 gene (TARDBP) itself. The registry does not say how the single dose is given.
Sources
- KOANEWA: Safety and Tolerability Study of CTx1000 In Participants With ALS (NCT07401121)ClinicalTrials.gov · Trial registry
What's nextFirst safety results from KOANEWAExpected December 2027Explanation and sources
Celosia has not given a date for results. The trial registry estimates the main 52-week safety data will be collected by December 2027, with long-term follow-up running to 2030.
Sources
- KOANEWA: Safety and Tolerability Study of CTx1000 In Participants With ALS (NCT07401121)ClinicalTrials.gov · Trial registry
Can I join?Recruiting at one site in Sydney, AustraliaRecruiting nowExplanation and sources
The registry lists one recruiting site, Macquarie University Hospital in Sydney. Participants must be on stable ALS medicines for 30 days before screening and test low for antibodies to the AAV9 virus. We found no expanded access programme.
- KOANEWA (Phase 1b) (NCT07401121) recruiting · Macquarie University Hospital, Sydney, Australia
Sources
- KOANEWA: Safety and Tolerability Study of CTx1000 In Participants With ALS (NCT07401121)ClinicalTrials.gov · Trial registry
- Celosia Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of CTx1000 in Amyotrophic Lateral SclerosisCelosia Therapeutics (via PR Newswire) · 23 Mar 2026 · Company press release
Signal: Quiet
Signal measures how often, and how reliably, Celosia Therapeutics tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.
| Last company update | 23 Mar 2026 (7 months ago) | 4 / 40 |
|---|---|---|
| Company updates in the last 12 months | 1 | 6 / 35 |
| Promises kept | Not enough history yet (neutral score) | 12.5 / 25 |
| Signal score | 23 / 100 · Quiet | |
70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.
Timeline
4 events, newest first. Company updates are marked; independent news and regulator decisions are shown too.
2026
Celosia seeks US$80 million
Biotech Daily reported that Celosia is raising US$80 million to take CTx1000 toward later trials and plans to apply to the FDA to run trials in the US.
First participant dosed
Celosia announced the first person had received CTx1000 at Macquarie University Hospital in Sydney, in a single-dose safety trial.
KOANEWA trial registered
The first trial of CTx1000 in people was posted on ClinicalTrials.gov, with a start date of January 12, 2026 and up to 15 participants.
2024
Mouse study published in Neuron
Macquarie University researchers described the TDP-43-clearing approach behind CTx1000, which reduced ALS-like disease in several mouse models, including in mice that already had symptoms.
All sources for this page
- Targeting 14-3-3θ-mediated TDP-43 pathology in amyotrophic lateral sclerosis and frontotemporal dementia miceNeuron · 15 Feb 2024 · Peer-reviewed paper
- Celosia Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of CTx1000 in Amyotrophic Lateral SclerosisCelosia Therapeutics (via PR Newswire) · 23 Mar 2026 · Company press release
- Biotech Daily, March 24, 2026 (Celosia Therapeutics)Biotech Daily · 24 Mar 2026 · News
- First Patient Dosed in Phase 1b Trial of AAV9 Gene Therapy CTx1000 for ALSPackGene Biotech · 27 Mar 2026 · News
- KOANEWA: Safety and Tolerability Study of CTx1000 In Participants With ALS (NCT07401121)ClinicalTrials.gov · Trial registry